Cerebrale parese bij kinderen

Initiatief: VRA Aantal modules: 70

Dyskinesie

Uitgangsvraag

Welke meetinstrumenten zijn geschikt om vast te stellen in welke mate er sprake is van gestoorde lichaamsfuncties die frequent voorkomen bij CP?

Aanbeveling

Overweeg het gebruik van DIS of de BADS voor het vaststellen van (de mate) van dyskinesie (dystonie en hyperkinesie).

 

Overweeg het gebruik van de BADS indien het van belang is dat er een geringe tijdsinvestering gevraagd wordt.

 

Overweeg het gebruik van de DIS indien er noodzaak is om te kunnen differentiëren tussen (de mate van) dystonie en (de mate van) hyperkinesie.

 

Wees terughoudend met het gebruik van de onderzochte meetinstrumenten (BFMDRS, BADS, UDRS, MD-CRS, MD-CRS 0-3, DIS) bij het evalueren van een behandeling voor dyskinesie.

Overwegingen

Voor- en nadelen van het meetinstrument en de kwaliteit van het bewijs

In de literatuursamenvatting zijn alle zes beschikbare instrumenten voor het meten van dyskinesie bij kinderen met CP meegenomen. Voor deze instrumenten zijn een systematische review met daarin 18 relevante studies (Stewart, 2017) en drie originele studies (Haberfehlner, 2020; Vanmechelen, 2020; Claassen, 2023) die rapporteren over validiteit, betrouwbaarheid en/of responsiviteit samengevat en is de bewijskracht beoordeeld. De resultaten zijn samengevat in Tabel 8. In de literatuursamenvatting is zoveel mogelijk de werkwijze van de COSMIN groep gevolgd. Deze groep geeft ook aanwijzingen hoe te bepalen welk instrument kan worden aanbevolen voor gebruik in de klinische praktijk (Mokkink, 2018). Hiervoor kunnen de beschikbare instrumenten in drie categorieën worden ingedeeld:

  1. Instrumenten met bewijs (elk niveau) voor voldoende inhoudsvaliditeit EN tenminste lage bewijskracht voor voldoende interne consistentie. Deze instrumenten kunnen worden aanbevolen voor gebruik in de klinische praktijk.
  2. Instrumenten die niet in categorie A of C kunnen worden ingedeeld. Deze instrumenten hebben wel potentieel om te worden aanbevolen maar verder onderzoek naar de meeteigenschappen is noodzakelijk.
  3. Instrumenten met hoge bewijskracht dat een of meerdere meeteigenschappen onvoldoende zijn gebleken. Deze instrumenten zouden niet aanbevolen moeten worden voor gebruik in de klinische praktijk.

Indien alleen instrumenten in categorie ‘B’ beschikbaar zijn kan het instrument met het beste bewijs voor inhoudsvaliditeit voorlopig worden aanbevolen totdat meer bewijs beschikbaar komt.

 

Hieronder worden de resultaten uit de literatuursamenvatting langs deze criteria voor inhoudsvaliditeit en interne consistentie gelegd.

 

Wat betreft inhoudsvaliditeit, in de review van Stewart (2017) zijn alleen studies geïncludeerd waarin de inhoudsvaliditeit is geëvalueerd door het raadplegen van experts. Voor de BFMDRS, BADS en UDRS gaven experts aan dat de vragenlijst niet goed aansluit bij de vernieuwde definitie en classificatie van CP (Bax, 2005). Voor de MD-CRS 0-3 werd in de geïncludeerde studies geen informatie over inhoudsvaliditeit gerapporteerd. Voor de DIS werd in één van de geïncludeerde studies beschreven dat bij de ontwikkeling van dit instrument negen experts werden betrokken om de inhoudsvaliditeit te waarborgen.

In één van de originele studies (Claassen, 2023) werden acht kinderen en adolescenten met dyskinetische CP en 12 verzorgers geïnterviewd om de inhoudsvaliditeit van de MD-CRS te evalueren. De resultaten van deze interviews lijken op voldoende inhoudsvaliditeit van de MD-CRS te wijzen.

Op basis van het criterium ‘bewijs van voldoende inhoudsvaliditeit’ valt de MD-CRS 0-3 af en zijn er kanttekeningen te plaatsen bij de BFMDRS, BADS, UDRS en DIS. De MD-CRS zou wat betreft inhoudsvaliditeit in aanmerking kunnen komen voor gebruik in de klinische praktijk.

 

Wat betreft interne consistentie, in de review van Stewart (2017) werd voor zowel de BFMDRS, BADS, UDRS als DIS een acceptabele tot goede of uitstekende interne consistentie gevonden. Stewart (2017) beoordeelde de bewijskracht voor de interne consistentie van deze vier instrumenten als redelijk. Voor de MD-CRS en MD-CRS 0-3 was geen informatie over interne consistentie beschikbaar. Op basis van het criterium ‘lage bewijskracht voor voldoende interne consistentie’ komen dus vier instrumenten in aanmerking: de BFMDRS, BADS, UDRS en DIS.

Bij het combineren van beide criteria komt geen duidelijke beste optie naar voren. Echter,

aanvullend op de selectie op basis van de methodologische criteria is van belang dat alleen de BADS en de DIS specifiek zijn ontworpen om (secundaire) dystonie als gevolg van CP te meten. De overige meetinstrumenten die in de praktijk weliswaar ook voor de diagnosegroep CP worden ingezet, zijn ontworpen om primaire dystonie te meten of dystone bewegingsstoornissen met diverse etiologie. De DIS is zo ontworpen dat het kan differentiëren tussen dystonie als hyperkinesiezowel in rust en tijdens activiteiten.

 

Voor de MD-CRS en MD-CRS 0-3 is geen informatie bekend over de betrouwbaarheid. Hier ligt een kennislacune.

 

Wat betreft responsiviteit is voor alle 6 meetinstrumenten slechts beperkt bewijs dat veranderingen in dystonie en/of hyperkinesie bijvoorbeeld ten gevolge van behandeling zullen worden gedetecteerd.  Daarbij is de vraag of dat aan de instrumenten ligt of het geringe effect op dystonie bij kinderen met CP. Immers, bij medicamenteus geïnduceerde vormen van dystonie (als bijwerking) worden wel klinisch relevante verschillen gemeten op diverse dystonie schalen waaronder de UDRS (Anand, 2023).

 

Vooralsnog lijken de verschillende meetinstrumenten vooral ingezet te kunnen worden om vast te leggen óf er sprake is van dyskinesie om de vorm van CP te kunnen classificeren en de ernst ervan op stoornisniveau in te schatten.

 

Waarden en voorkeuren van patiënten (en evt. hun verzorgers)

Het kunnen onderscheiden van de verschillende stoornissen in bewegingssturing bij CP, heeft consequenties voor het toekomstig functioneren en is daarmee een belangrijk onderdeel van informatieverstrekking aan ouders.

Met name de mate waarin de spiertonus gestoord is zoals bij de spastische en dyskinetische vorm van CP, heeft enige voorspellende waarde voor de voortgang van de motorische ontwikkeling en het ontstaan van contracturen en daarmee gepaard gaande ossale en arthrogene deformiteiten. Deze aspecten kunnen onderdeel zijn van gesprekken met kinderen met CP en/of hun ouders als er behoefte is meer te willen weten over het verdere beloop van het CP-beeld, en zijn tevens het vertrekpunt van gesprekken over in te zetten behandelingen.

 

Kosten (middelenbeslag)

De kosten van het scoren van dyskinesie zijn voor klinisch gebruik zeer gering. Vastleggen met video heeft wel de voorkeur zonodig in de thuissituatie als in het ziekenhuis. Belangrijkste is het vastleggen van het persoonlijke doel van de patiënt. De kosten zitten in de tijd om gestructureerd de doelen vast te stellen.

 

Aanvaardbaarheid, haalbaarheid en implementatie

De toepasbaarheid van de verschillende schalen in de praktijk varieert, training is niet nodig en er zijn handleidingen en videoprotocollen beschikbaar in wetenschappelijke tijdschriften hoe de testen af te nemen en te scoren. Alleen voor afname van de DIS wordt enige ervaring vereist en begrip van het onderscheid tussen dystonie en choreaathetose. Voor de meest schalen geldt dat de betrouwbaarheid toeneemt met meer klinische ervaring.

 

De huidige beschikbare behandelingen om dystonie en chorea athetose bij CP te verminderen en het functioneren te verbeteren behoeven een nauwkeurige monitoring om een goede kwantitatieve voor- en nameting te doen en fine-tuning in behandeling te kunnen bieden. Alle 6 meetinstrumenten zijn gebaseerd op het scoren van het voorkomen en de frequentie van onwillekeurige bewegingen van hoofd, romp en ledematen en vragen niet meer dan een interview en/of video-opname in rust en/of tijdens een activiteit. Hoewel de tijdsinvestering van met name de DIS groot is voor degene die de scoring doet is de belasting voor de patiënt minimaal. Dit maakt de DIS minder geschikt voor de dagelijkse klinische praktijk. De BADS is compacter en minder tijdsintensief en daardoor beter geschikt om in de kliniek af te nemen. De BADS meet echter geen aspecten van hyperkinesie.

 

Tabel 8: Overzicht van de validiteit, betrouwbaarheid en responsiviteit van de zes meetinstrumenten voor dyskinesie

 

Validiteit

Betrouwbaarheid

Responsiviteit

Construct-validiteit

Inhouds-validiteit

Criterium-validiteit (concurrent)

Criterium-validiteit (predictief)

Intra-rater

Test-hertest

Inter-rater

Interne consistentie

Burke–Fahn–Marsden Dystonia Rating Scale (BFMDRS)

Geen informatie

 

 

 

Redelijk bewijs (+)

 

Bewijs alleen gebaseerd op expert opinion, meerdere kritische kanttekeningen (?)

 

Redelijk bewijs

Beperkt bewijs

Geen informatie

 

 

Geen informatie

Redelijk bewijs

Redelijk bewijs (+)

 

Goede tot uitstekende interne consistentie (+)

Beperkt bewijs

 

Barry–Albright Dystonia Scale (BADS)

Redelijk bewijs

Beperkt bewijs (+)

 

Bewijs alleen gebaseerd op expert opinion, instrument sluit niet goed aan bij definitie dystonie, alleen gericht op dystonie (?)

 

Redelijk bewijs

Beperkt bewijs

Geen informatie

Geen informatie

Redelijk bewijs

Redelijk bewijs (+)

 

Goede tot uitstekende interne consistentie (+)

Beperkt bewijs

 

Unified Dystonia Rating Scale (UDRS)

Geen informatie

Redelijk bewijs (+)

 

Bewijs alleen gebaseerd op expert opinion, instrument sluit niet goed aan bij definitie dystonie, alleen gericht op dystonie (?)

 

Redelijk bewijs

Geen informatie

Geen informatie

Geen informatie

Redelijk bewijs

Redelijk bewijs (+)

 

Uitstekende interne consistentie (+)

Beperkt bewijs

Movement Disorder-Childhood Rating Scale (MD-CRS)

Geen informatie

Redelijk bewijs (+)

 

Inhoudsvaliditeit lijkt voldoende op basis van interviews met kinderen/adolescenten en hun verzorgers (+)

Geen informatie

Geen informatie

Geen informatie

Geen informatie

Geen informatie

Geen informatie (-)

Beperkt bewijs

Movement Disorder-Childhood Rating Scale 0-3 years (MD-CRS 0-3)

Geen informatie

Geen informatie (-)

Geen informatie

Geen informatie

Geen informatie

Geen informatie

Geen informatie

Geen informatie (-)

Beperkt bewijs

Dyskinesia Impairment Scale (DIS)

Geen informatie

Beperkt bewijs (+)

 

Bij ontwikkeling van de schaal zijn negen experts betrokken geweest om de inhoudsvaliditeit te waarborgen (?)

Redelijk bewijs

Beperkt bewijs

Geen informatie

Beperkt bewijs

Redelijk bewijs

Redelijk bewijs (+)

 

Adequate tot goede of uitstekende interne consistentie (+)

Beperkt bewijs

+: voldoet aan criteria voor categorie A

- : voldoet niet aan criteria voor categorie A

? : twijfelachtig of de resultaten voldoen aan de criteria voor categorie A

 

Rationale van de aanbeveling: weging van argumenten voor en tegen de diagnostische procedure

De werkgroep van de richtlijn CP is van mening dat voor de keuze van meetinstrumenten om (de mate van) dyskinesie vast te leggen, de psychometrische eigenschappen en de praktische toepasbaarheid dienen mee te wegen. Daarbij is het van belang of het meetinstrument specifiek is ontworpen voor de doelgroep CP. De BADS en de DIS komen hiervoor het meest in aanmerking. Indien het van belang is dat onderscheid wordt gemaakt tussen de component dystonie en hyperkinesie is het gebruik van de DIS het meest van toepassing. Indien de overweging is om een minder tijdsintensief meetinstrument in te zetten is de BADS het meest van toepassing.

 

Onderbouwing

Een van de gevolgen van de centraal motorische parese (equivalent voor de Engelse term upper motor neuron syndrome) is de ontregeling in spiertonus, wat zich bij kinderen met cerebrale parese (CP) kan uiten in dyskinesie. Voor het indiceren van een behandelvorm gericht op het verminderen van de (gevolgen van) ontregelde spiertonus zijn meetinstrumenten nodig die kunnen vastleggen óf er sprake is van dyskinesie en in welke mate.

 

De dyskinetische vorm van CP betreft een mengbeeld met kenmerken van dystonie en hyperkinesie (voorheen chorea-athetose genoemd). De bijdrage van beide vormen kan wisselen en ook veranderen in de tijd. Bij dystonie is er net als bij spasticiteit sprake van aanhoudende onwillekeurige spieractiviteit, maar is deze nooit snelheidsafhankelijk en vaak toenemend bij (poging tot) redressie van de standsafwijking in het gewricht. Hyperkinesie betreft een overvloed aan niet te onderdrukken onwillekeurige aritmische grofslagige (choreatische/ athetotische) bewegingen met verschillende snelheid. De diverse schalen die zijn ontwikkeld om dystonie en/of hyperkinesie bij CP te meten zijn bedoeld om een indruk te krijgen van de ernst van de symptomatologie. Deze testen berusten op visuele of videobased beoordeling en zijn vaak tijdrovend en daardoor minder toepasbaar in de kliniek (Stewart, 2017).

1. Burke–Fahn–Marsden Dystonia Rating Scale (BFMDRS)

Burke–Fahn–Marsden Dystonia Rating Scale (BFMDRS) - validity

Moderate level of evidence*

 

 

There is moderate confidence in the content validity and concurrent criterion validity of the BFMDRS for measuring dystonia/hyperkinesia in children with CP.

 

Source: Stewart (2017)

Low level of evidence*

 

There is limited confidence in the predictive criterion validity of the BFMDRS for measuring dystonia/hyperkinesia in children with CP.

 

Source: Stewart (2017)

-

No evidence was found regarding the construct validity of the BFMDRS for measuring dystonia/hyperkinesia in children with CP.

 

Source: -

 

Burke–Fahn–Marsden Dystonia Rating Scale (BFMDRS) - reliability

Moderate level of evidence*

There is moderate confidence in the inter-rater reliability and internal consistency of the BFMDRS for measuring dystonia/hyperkinesia in children with CP.

 

Source: Stewart (2017)

-

No evidence was found regarding the intra-rater reliability and test-retest reliability of the BFMDRS for measuring dystonia/hyperkinesia in children with CP.

 

Source: -

 

Burke–Fahn–Marsden Dystonia Rating Scale (BFMDRS) - responsiveness

(Very) low level of evidence*

There is (very) limited confidence in the responsiveness of the BFMDRS for measuring dystonia/hyperkinesia in children with CP.

 

Source: Stewart (2017)

 

2. Barry–Albright Dystonia Scale (BADS)

Barry–Albright Dystonia Scale (BADS) - validity

 

Low level of evidence*

 

There is limited confidence in the content validity and predictive criterion validity of the BADS for measuring dystonia/hyperkinesia in children with CP.

 

Source: Stewart (2017)

Moderate level of evidence*

 

There is moderate confidence in the concurrent criterion validity and construct validity of the BADS for measuring dystonia/hyperkinesia in children with CP.

 

Source: Stewart (2017)

 

Barry–Albright Dystonia Scale (BADS) - reliability

Moderate level of evidence*

There is moderate confidence in the inter-rater reliability and internal consistency of the BADS for measuring dystonia/hyperkinesia in children with CP.

 

Source: Stewart (2017)

-

No evidence was found regarding the intra-rater reliability and test-retest reliability of the BADS for measuring dystonia/hyperkinesia in children with CP.

 

Source: -

 

Barry–Albright Dystonia Scale (BADS) - responsiveness

(Very) low level of evidence*

There is (very) limited confidence in the responsiveness of the BADS for measuring dystonia/hyperkinesia in children with CP.

 

Source: Stewart (2017)

 

3. Unified Dystonia Rating Scale (UDRS)

Unified Dystonia Rating Scale (UDRS) - validity

 

Moderate level of evidence*

 

There is moderate confidence in the content validity and concurrent criterion validity of the UDRS for measuring dystonia/hyperkinesia in children with CP.

 

Source: Stewart (2017)

-

 

No evidence was found regarding the predictive criterion validity and construct validity of the UDRS for measuring dystonia/hyperkinesia in children with CP.

 

Source: -

 

Unified Dystonia Rating Scale (UDRS) - reliability

Moderate level of evidence*

There is moderate confidence in the inter-rater reliability and internal consistency of the UDRS for measuring dystonia/hyperkinesia in children with CP.

 

Source: Stewart (2017)

-

No evidence was found regarding the intra-rater reliability and test-retest reliability of the UDRS for measuring dystonia/hyperkinesia in children with CP.

 

Source: -

 

Unified Dystonia Rating Scale (UDRS) - responsiveness

Low level of evidence*

There is very limited confidence in the responsiveness of the UDRS for measuring dystonia/hyperkinesia in children with CP.

 

Source: Stewart (2017)

 

4. Movement Disorder-Childhood Rating Scale (MD-CRS)

Movement Disorder-Childhood Rating Scale (MD-CRS) - validity

 

Moderate level of evidence*

 

There is moderate confidence in the content validity of the MD-CRS for measuring dystonia/hyperkinesia in children with CP.

 

Source: Claassen (2023)

-

 

No evidence was found regarding the concurrent criterion validity, predictive criterion validity and construct validity of the MD-CRS for measuring dystonia/hyperkinesia in children with CP.

 

Source: -

 

Movement Disorder-Childhood Rating Scale (MD-CRS) - reliability

-

No evidence was found regarding the intra-rater reliability, test-retest reliability, inter-rater reliability and internal consistency of the MD-CRS for measuring dystonia/hyperkinesia in children with CP.

 

Source: -

 

Movement Disorder-Childhood Rating Scale (MD-CRS) - responsiveness

(Very) low level of evidence*

There is (very) limited confidence in the responsiveness of the MD-CRS for measuring dystonia/hyperkinesia in children with CP.

 

Source: Stewart (2017)

 

5. Movement Disorder-Childhood Rating Scale 0-3 years (MD-CRS 0-3)

Movement Disorder-Childhood Rating Scale 0-3 years (MD-CRS 0-3) - validity

-

 

No evidence was found regarding the content validity, concurrent criterion validity, predictive criterion validity and construct validity of the MD-CRS 0-3 for measuring dystonia/hyperkinesia in children with CP.

 

Source: -

 

Movement Disorder-Childhood Rating Scale 0-3 years (MD-CRS 0-3) - reliability

-

No evidence was found regarding the intra-rater reliability, test-retest reliability, inter-rater reliability and internal consistency of the MD-CRS 0-3 for measuring dystonia/hyperkinesia in children with CP.

 

Source: -

 

Movement Disorder-Childhood Rating Scale 0-3 years (MD-CRS 0-3) - responsiveness

(Very) low level of evidence*

There is (very) limited confidence in the responsiveness of the MD-CRS for measuring dystonia/hyperkinesia in children with CP.

 

Source: Stewart (2017)

 

 

6. Dyskinesia Impairment Scale (DIS)

Dyskinesia Impairment Scale (DIS) - validity

 

Low level of evidence*

 

There is limited confidence in the content validity and predictive criterion validity of the DIS for measuring dystonia/hyperkinesia in children with CP.

 

Source: Stewart (2017)

Low level of evidence*

There is limited confidence in the concurrent criterion validity of the DIS for measuring dystonia/hyperkinesia in children with CP.

 

Source: Stewart (2017)

Low level of evidence*

There is limited confidence in the concurrent criterion validity of the DIS for measuring dystonia/hyperkinesia in non-ambulatory children with CP.

 

Source: Haberfehlner (2020)

-

 

No evidence was found regarding the construct validity of the DIS for measuring dystonia/hyperkinesia in children with CP.

 

Source: -

 

Dyskinesia Impairment Scale (DIS) - reliability

Moderate level of evidence*

There is moderate confidence in the inter-rater reliability and internal consistency of the DIS for measuring dystonia/hyperkinesia in children with CP.

 

Source: Stewart (2017)

Very low level of evidence*

The evidence is very uncertain about the test-retest reliability of the DIS for measuring dystonia/hyperkinesia in children with CP.

 

Source: Vanmechelen (2020)

-

No evidence was found regarding the intra-rater reliability of the DIS for measuring dystonia/hyperkinesia in children with CP.

 

Source: -

 

Dyskinesia Impairment Scale (DIS) - responsiveness

Very low level of evidence*

The evidence is very uncertain about the test-retest reliability of the DIS for measuring dystonia/hyperkinesia in non-ambulatory children with CP.

 

Source: Haberfehlner (2020)

 

* The level of evidence was mostly performed in accordance with COSMIN guidance for systematic reviews of PROMS (Mokkink, 2018). See Table 6 in the attachment for the definitions of these quality levels.

Description of studies

Stewart (2017) performed a systematic review on the psychometric properties and clinical utility of instruments to measure dystonia and choreoathetosis in children with dyskinetic CP. The electronic databases PubMed, Medline, Embase, Proquest, CINAHL, and Web of Science were searched from inception up to April 2016. Studies were included in the review if they were reported in English, included children with CP aged 0 to 18 years, evaluated a clinical measure of dystonia and/or choreoathetosis according to the definition of Himmelmann (2007), and reported original psychometric data for children with CP.

Relevant outcome measures included validity, reliability, and responsiveness.

Stewart (2017) used the Consensus-based Standards for selection of health Measurement Instruments (COSMIN) checklist to assess the level of evidence (strong, moderate, limited, conflicting, or unknown). This assessment included the number of studies available, the methodological quality of each study, the strength of psychometric data, and the results (see Table 3.1 in the attachment for more detailed information).

Thirty-four studies were included in the review, reporting on six instruments. Several of these studies reported results for mixed cohorts of children and/or adults with CP and other conditions. In this literature summary, results are presented only for the 18 studies that reported data specifically for children with CP. Where evidence was available, Stewart (2017) judged the level of evidence to be limited (defined as one study of fair methodological quality) or moderate (defined as consistent findings in multiple studies of fair methodological quality OR in one study of good methodological quality) (see Table 4 in the attachment). The authors of the systematic review reported no conflicts of interests.

 

After publication of the systematic review by Stewart (2017), three additional original studies were published that matched the predefined PICO (Haberfehlner, 2020; Vanmechelen, 2020; Claassen, 2023). These studies are summarized below.

 

Haberfehlner (2020) performed a secondary analysis of data collected in the IDYS trial, a Dutch randomized controlled trial evaluating the effects of intrathecal baclofen (ITB) in children and young adults with dyskinetic CP. The aim of Haberfehlner’s secondary analysis was to assess the concurrent validity, responsiveness, and feasibility of the Dyskinesia Impairment Scale (DIS) in non-ambulatory children and young adults with dyskinetic CP. Inclusion criteria for the IDYS trial were: (1) children and young adults with dyskinetic CP aged 4 to 25 years; (2) classified in GMFCS levels IV and V; (3) lesions on magnetic resonance imaging; and (4) eligible for ITB treatment using commonly applied criteria. All participants who met these criteria and completed the Dyskinesia Impairment Scale (DIS) and the Barry-Albright Dystonia Scale (BADS) at baseline and three months after pump implantation were included in the analysis by Haberfehlner (n=33). Relevant outcomes included concurrent validity and responsiveness. Concurrent validity was evaluated by assessing the correlation between the DIS dystonia subscale and the BADS at baseline and follow-up. Responsiveness was evaluated by comparing the effect sizes and correlation of change after treatment between the DIS dystonia subscale and the BADS. More information on study characteristics is provided in Table 3.2 in the attachment. This study also included young adults aged 18-25, however most study participants were below the age of 18 and this is not expected to have a substantial impact on the outcomes of the study.

The methodological quality for evaluating concurrent validity was assessed to be very good (see Table 5.1 in the attachment), and the methodological quality for evaluating responsiveness was assessed to be doubtful (see Table 5.2-5.4 in the attachment).

 

Vanmechelen (2020) assessed the test-retest reliability of the DIS using a subset of participants included in a previous study by Monbaliu (2012) which was included in the systematic review by Stewart (2017). Children and young adults were included in the study if they had predominant dyskinetic CP, as diagnosed by a paediatric neurologist within a reference centre for CP, and were born at term. Children or young adults were excluded if there had been changes in muscle tone-changing medication within the last three months, if they had had any orthopaedic or neurosurgical interventions within the previous year, or if they had spine fusion. Fifteen children and young adults were included and were videotaped twice, in two separate sessions within seven days. Intraclass correlation coefficients, standard errors of measurement and the minimal detectable difference were assessed. More information on study characteristics is provided in Table 3.2 in the attachment. The methodological quality for evaluating reliability was assessed to be doubtful (see Table 5.5 in the attachment). 

 

Claassen (2023) conducted an interview study among children and adolescents with dyskinetic CP and caregivers of children with dyskinetic CP to assess the content validity of the Movement Disorder-Childhood Rating Scale. Children (6-11 years) or adolescents (12-18 years) living in the United States were eligible if they had self-reported dyskinetic CP and predominant choreiform movement or dystonia, if they had been treated for CP, had a caregiver willing to participate in the study themselves, and were able to communicate verbally and were willing and able to participate in the interview. Caregivers were eligible if they acted as a caregiver for a child that met the eligibility criteria, lived in the United States and spoke and read English fluently. Eight children or adolescents and 12 caregivers were interviewed. Children and adolescents were asked which body regions and daily functions were affected by dyskinetic CP. Caregivers were also asked to evaluate the relevance of the items and response options in part I of the instrument. More information on study characteristics is provided in Table 3.2 in the attachment. The methodological quality for evaluating content validity was assessed to be adequate (see Table 5.6 in the attachment).

 

Results

Psychometric properties of the following six scales were assessed:

  1. Burke–Fahn–Marsden Dystonia Rating Scale (BFMDRS) (12 studies in review Stewart [2017]);
  2. Barry–Albright Dystonia Scale (BADS) (5 studies in review Stewart [2017]);
  3. Unified Dystonia Rating Scale (UDRS) (2 studies in review Stewart [2017]);
  4. Movement Disorder-Childhood Rating Scale (MD-CRS) (1 study in review Stewart, 2017; Claassen, 2023);
  5. Movement Disorder-Childhood Rating Scale 0-3 years (MD-CRS 0-3) (1 study in review Stewart [2017]);
  6. Dyskinesia Impairment Scale (DIS) (3 studies in review Stewart, 2017; Haberfehlner, 2020; Vanmechelen, 2020).

The review by Stewart (2017) provides an overview of these six instruments, including details of each instrument, scoring criteria, scales, subscales, and information on administration/clinical utility. See Table 7 in the attachment for an adapted version of this overview.

 

For each instrument, results regarding validity, reliability, and responsiveness are summarised below. In the review by Stewart (2017), results were presented in tables which included a description of the results reported in the included studies as well as the level of evidence as assessed by the review authors (for validity and reliability). Table 4 in the attachment shows an adapted version of this table, including results for the three original studies.

 

1. Burke–Fahn–Marsden Dystonia Rating Scale (BFMDRS)

Twelve studies in the systematic review by Stewart (2017) reported on the measurement properties of the BFMDRS. Results are shown in Table 4 in the attachment.

 

1.1   Validity

None of the studies reported on construct validity. Three studies reported on content validity; in these studies expert opinion was sought, detailed results are presented in Table 4 in the attachment. Experts made several critical remarks, including the remarks that the BFMDRS was not aligned with the current definition of dystonia, and involves dystonia only. Two studies reported on concurrent validity. One study reported a moderate correlation of r=0.59 (p=0.05) between the BFMDRS and BADS. The other study reported high correlations between the BFMDRS and the BADS for three raters (rater 1: r=0.93, 95% CI 0.73–0.98, p<0.001; rater 2: r=0.95, 95% CI 0.79–0.99, p<0.001; rater 3: r=0.95, 95% CI 0.69–0.98, p<0.001). One study reported on predictive ability, there was no correlation between changes in BFMDRS score and changes in Canadian Occupational Performance Measure after deep brain stimulation (r=0.201, p=0.511 at 6 months and r=0.176, p=0.565 at 12 months).

 

1.2   Reliability

None of the studies reported on intra-rater or test-retest reliability. One study reported on inter-rater reliability. For total scores, inter-rater reliability between three raters ranged from excellent (intraclass correlation coefficient (ICC)=0.89, ICC=0.86) to adequate (ICC=0.64). For item scores, inter-rater reliability between three raters ranged from poor to excellent (ICC=0.34-0.77, ICC=0.32-0.93, ICC=0.45-0.86). The standard error of the measurement was high (9.9%), and the smallest detectable change was large (27.4%). The same study also reported on internal consistency. Excellent internal consistency was found (Cronbach α = 0.83-0.94).

 

1.3   Responsiveness

Eleven intervention studies reported on responsiveness of the BFMDRS. The movement scale was found to have excellent responsiveness to change, however for the disability scale responsiveness to change was lower. Detailed results are presented in Table 4 in the attachment.

 

2. Barry–Albright Dystonia Scale (BADS)

Five studies in the systematic review by Stewart (2017) reported on the measurement properties of the BADS. Results are shown in Table 4 in the attachment.

 

2.1 Validity

Three studies reported on construct validity. In clinical studies, a higher level of dystonia was not correlated with greater spasticity as assessed with the Ashworth scale (r=0.36, p=0.22). In laboratory studies, higher BADS score was correlated with longer reach paths (r=0.75, p=0.005) and higher total BADS score was correlated with poorer hand-tapping (r=0.79, p=0.003) but not slower eye-blinking (r=0.41, p=0.24). Affected arm BADS score was correlated with poorer hand-tapping (r=0.76, p=0.007) and slower eye-blinking (r=0.72, p=0.02). One study reported on content validity; in this study expert opinion was sought, experts mentioned that the BADS was not aligned with the current definition of dystonia, and involves dystonia only. Four studies reported on concurrent criterion validity. One study reported a moderate correlation between the BADS and BFMDRS (r=0.59, p=0.05) and one study reported high correlations between the BADS and BFMDRS (rater 1: r=0.93, 95% CI 0.73–0.98, p<0.001; rater 2: r=0.95, 95% CI 0.79–0.99, p<0.001; rater 3: r=0.95, 95% CI 0.69–0.98, p<0.001). A good correlation was found between the BADS and the DIS (r=0.84, 95%CI 0.66-0.92, p<0.001), and moderate to high correlations were found between the BADS and the UDRS (rater 1: r=0.89, 95% CI 0.60–0.97, p<0.001; rater 2: r=0.84, 95% CI 0.6–0.96, p=0.002; rater 3: r=0.86, 95% CI 0.51–0.97, p<0.001). One study reported on predictive criterion validity. BADS scores for 10 children with CP before and after a trial were compared with global rating of change, raters agreed on ‘same’ (n=1), ‘worse’ (n=1), and ‘better’ (n=8).

 

2.2 Reliability

None of the studies reported intra-rater reliability or test-retest reliability specifically for children with CP. One study reported on inter-rater reliability. For total score, excellent reliability was found (ICC=0.87). For item scores, adequate reliability was found for limbs and trunk (ICC=0.61-0.76) while poor reliability was found for eyes, mouth, and neck (ICC=0.39-0.57). The standard error of the measurement was high (6.4%) and the smallest detectable change was large (17.7%). The same study also reported on internal consistency. Excellent internal consistency was found (Cronbach α = 0.87-0.91).

 

2.3 Responsiveness

Eleven intervention studies reported on responsiveness to change of the BADS. Responsiveness was excellent; however, data are predominantly descriptive and more in-depth analysis is required. Detailed results are presented in Table 4 in the attachment.

 

3. Unified Dystonia Rating Scale (UDRS)

Two studies in the systematic review by Stewart (2017) reported on the measurement properties of the UDRS. Results are shown in Table 4 in the attachment.

 

3.1 Validity

Neither of the studies reported on construct validity or predictive criterion validity. One study reported on content validity; in this study expert opinion was sought, experts mentioned that the BADS was not aligned with the current definition of dystonia, and involves dystonia only. The same study also reported on concurrent criterion validity. A moderate to high correlation was found between total UDRS and BADS scores of three raters (rater 1: r=0.89, 95% CI 0.60–0.97, p<0.001; rater 2: r=0.84, 95% CI 0.6–0.96, p=0.002; rater 3: r=0.86, 95% CI 0.51–0.97, p<0.001). In addition, high correlations were found between total scores (rater 1: r=0.87, 95%CI 0.53-0.97, p<0.001; rater 2: r=0.88, 95%CI 0.56-0.97, p=0.002; rater 3: r=0.93, 95%CI 0.73-0.98, p<0.001).

 

3.2 Reliability

Neither of the studies reported on intra-rater reliability or test-retest reliability. One study reported on inter-rater reliability. For total scores, adequate reliability was found (ICC=0.74-0.79). For item scores, poor to excellent reliability was found (ICC=0.17-0.71, ICC=0.26-0.83, ICC=0.37-0.81). The standard error of the measurement was high (8.9%) and the smallest detectable change was large (24.6%). The same study also reported on internal consistency. Excellent internal consistency was found (Cronbach α = 0.90-0.95).

 

3.3 Responsiveness

One intervention study reported on responsiveness to change. One month after injections with Botulinum Toxin-A, proximal upper extremity change was not statistically significant (p=0.12) while distal change was statistically significant (p=0.0088).

 

4. Movement Disorder-Childhood Rating Scale (MD-CRS)

One study in the systematic review by Stewart (2017) and the original study by Claassen (2023) reported on the measurement properties of the MD-CRS. Results are shown in Table 4 in the attachment.

 

4.1   Validity

The study by Claassen (2023) provided evidence on content validity. Most body regions and activities included in the MD-CRS were relevant to the experience of children/adolescents with dyskinetic CP and caregivers. Most items and response options in part I of the tool were deemed to be appropriate by the caregivers. Neither of the studies reported on construct validity or concurrent or predictive criterion validity.

 

4.2   Reliability

Neither of the studies reported on the reliability of the MD-CRS.

 

4.3 Responsiveness

One intervention study in the review by Stewart (2017) suggested that the MD-CRS is appropriate for detecting change 6 and 12 months after anticholinergic treatment in a population of children with CP. The results of an ANOVA showed that the main effect of time was statistically significant for part I, part II, and the total score (p<0.0001). Post-hoc analysis showed that change scores were statistically significant between all time points apart from the change in part II between 6 and 12 months. There was no significant correlation between observed changes in scores (part I, part II, and total score) at 12 months and children’s age.

 

5. Movement Disorder-Childhood Rating Scale 0-3 years (MD-CRS 0-3)

One study in the systematic review by Stewart (2017) reported on the measurement properties of the MD-CRS 0-3. Results are shown in Table 4 in the attachment.

 

5.1 Validity

No information was reported on the validity of the MD-CRS 0-3.

 

5.2 Reliability

No information was reported on the validity of the MD-CRS 0-3.

 

5.3 Responsiveness

One intervention study in the review by Stewart (2017) suggested that the MD-CRS 0-3 is appropriate for detecting change six and 12 months after anticholinergic treatment in a population of children with CP. The results of an ANOVA showed that the main effect of time was statistically significant for part I, part II, and the total score (p<0.0001). Post-hoc analysis showed that change scores were statistically significant between all time points apart from the change in part II between 6 and 12 months. There was no significant correlation between observed changes in scores (part I, part II, and total score) at 12 months and children’s age.

 

6. Dyskinesia Impairment Scale (DIS)

Three studies in the systematic review by Stewart (2017) and the original studies by Haberfehlner (2020) and Vanmechelen (2020) reported on the measurement properties of the DIS. Results are shown in Table 4 in the attachment.

 

6.1 Validity

None of the studies reported on construct validity. One study in the review by Stewart (2017) reported on content validity; in this study expert opinion was sought on content and available scales. The same study in the review by Stewart (2017) also reported on concurrent criterion validity. A good correlation was found between the DIS and the BADS (r=0.84, 95%CI 0.66-0.92, p<0.001). In addition, the original study by Haberfehlner (2020) in a population of non-ambulatory children and young adults with dyskinetic CP also reported on concurrent criterion validity. Sufficient concurrent validity (r > 0.70) was found between the DIS dystonia subscale and the BADS (r=0.78, 95% CI 0.59-0.88, p<0.001 at baseline and r=0.79, 95% CI 0.60-0.89, p<0.001 at follow-up). One study in the review by Stewart (2017) reported on predictive criterion validity. Good correlations were found between a higher Gross Motor Function Classification System (GMFCS) and higher total dystonia score (r=0.70, 95%CI 0.53-0.81, p<0.001) and dystonia in the leg region (r=0.65, 95%CI 0.47-0.78, p<0.01). In addition, good correlations were found between higher Manual Ability Classification System (MACS) level and total dystonia scores (r=0.68, 95%CI 0.47-0.81, p<0.001) and dystonia in the arm region (r=0.74, 95%CI 0.59-0.84, p<0.001). A fair correlation was found between higher Communication Function Classification System (CFCS) levels and total DIS scores (r=0.36, 95%CI 0.11-0.57, p=0.002) and dystonia in the mouth region (r=0.36, 95%CI 0.11-0.57, p=0.002). No significant correlations were found between the choreoathetosis subscale and GMFCS scores (total p=0.18, leg region p=0.86), or MACS (total p=0.09, arm region p=0.08), or CFCS (total p=0.06, mouth region p=0.30).

 

6.2 Reliability

None of the studies reported on intra-rater reliability. The study by Vanmechelen (2020) on test-retest reliability reported that the ICC of the DIS was 0.98 (95%CI 0.94 to 0.99), the ICC of the dystonia subscale was 0.97 (95%CI 0.92 to 0.99), and the ICC of the choreoathetosis subscale was 0.96 (95%CI 0.90 to 0.99). These ICCs meet the criteria for good measurement properties (ICC ≥ 0.70). The standard error of measurement and minimal detectable difference values for the total DIS were 2.6% and 7.2% respectively. The standard error of measurement and minimal detectable difference values were 3.7% and 10% for the DIS dystonia subscale and 3.6% and 9.9% for the DIS choreoathetosis subscale. Two studies in the review by Stewart (2017) reported on inter-rater reliability. One study reported that for total scores, excellent reliability was found overall (ICC=0.96), as well as for the dystonia subscale (ICC=0.91) and the choreoathetosis subscale (ICC=0.98). For regional items, poor to excellent reliability was found for the dystonia (ICC=0.46-1.00) and choreoathetosis (ICC=0.48-0.96) items. The standard error of the measurement was low overall (3%), as well as for the dystonia subscale (5%) and the choreoathetosis subscale (3%). The smallest detectable change was moderate overall (9%) and for the choreoathetosis subscale (7%), and large for the dystonia subscale (15%). The other study reported that for junior physical therapists, excellent reliability was found for total scores for the total DIS score and for both subscales (ICC=0.88, ICC=0.82, ICC=0.88). The standard error of measurement was moderate for the total DIS score and for both subscales (5.5%, 6.9%, 6.4%). The smallest detectable change was large for the DIS overall and for both subscales (15.3%, 18,4%, and 17.6%). For senior physical therapists, excellent reliability was found for total scores for the total DIS score and for the choreoathetosis subscale (ICC=0.88, ICC=0.91), but only adequate reliability was found for the dystonia subscale (ICC=0.71). The standard error of measurement was low for the total DIS score (4.4%) and for the choreoathetosis subscale (5%), and moderate for the dystonia subscale (7.7%). The smallest detectable change was large for the DIS overall and for both subscales (12.1%, 21.3%, and 13.9%). One study in the review by Stewart (2017) reported on internal consistency. Excellent internal consistency was found among two experienced raters for the dystonia subscale (Cronbach α = 0.90-0.93) as well as the choreoathetosis subscale (Cronbach α = 0.89-0.94). Furthermore, excellent internal consistency was found among five inexperienced raters for the total DIS and within the subscales for junior physical therapists (Cronbach α = 0.87-0.95) and senior physical therapists (Cronbach α = 0.76-0.93).

 

6.3 Responsiveness

The study by Haberfehlner (2020) reported that responsiveness was insufficient when comparing the DIS dystonia subscale to the BADS, responsiveness was sufficient when comparing the intervention and placebo subgroups, and responsiveness was insufficient when comparing scores before and after the intervention.

 

Level of evidence of the literature

1. Burke–Fahn–Marsden Dystonia Rating Scale (BFMDRS)

Validity

Stewart (2017) assessed the level of evidence for content validity (three fair studies) and concurrent criterion validity (two fair studies) to be moderate, and for predictive criterion validity to be limited (one fair study). The level of evidence for construct validity could not be assessed as none of the included studies reported on this measurement property.

 

Reliability

Stewart (2017) assessed the level of evidence for inter-rater reliability and internal consistency to be moderate (one good study). The level of evidence for intra-rater reliability and test-retest reliability could not be assessed as none of the included studies reported on this measurement property.

 

Responsiveness

Stewart (2017) did not report the level of evidence for responsiveness. However, the authors of the review described that the results of responsiveness should be interpreted with caution as most studies did not specifically aim to evaluate responsiveness and did not include a criterion standard, therefore it can be assumed that the level of evidence would be downgraded by at least one level from high to moderate because of serious risk of bias. Furthermore, the total number of participants included was lower than 100, therefore the level of evidence would be downgraded by one level from moderate to low because of serious imprecision. The level of evidence can be assumed to be (very) low.

 

2. Barry–Albright Dystonia Scale (BADS)

Validity

Stewart (2017) assessed the level of evidence for content validity and predictive criterion validity to be limited (one fair study), and for construct validity (three fair studies) and concurrent criterion validity (four fair-good studies) to be moderate.

 

Reliability

Stewart (2017) assessed the level of evidence for inter-rater reliability and internal consistency to be moderate (one good study). The level of evidence for intra-rater reliability and test-retest reliability could not be assessed as none of the included studies reported on this measurement property.

 

Responsiveness

Stewart (2017) did not report the level of evidence for responsiveness. However, the authors of the review described that the results of responsiveness should be interpreted with caution as most studies did not specifically aim to evaluate responsiveness and did not include a criterion standard, therefore it can be assumed that the level of evidence would be downgraded by at least one level from high to moderate because of serious risk of bias. Furthermore, the total number of participants included was lower than 100, therefore the level of evidence would be downgraded by one level from moderate to low because of serious imprecision. The level of evidence can be assumed to be (very) low.

 

3. Unified Dystonia Rating Scale (UDRS)

Validity

Stewart (2017) assessed the level of evidence for content validity and concurrent criterion validity to be moderate (one good study). The level of evidence for predictive criterion validity and construct validity could not be assessed as none of the included studies reported on these measurement properties.

 

Reliability

Stewart (2017) assessed the level of evidence for inter-rater reliability and internal consistency to be moderate (one good study). The level of evidence for intra-rater reliability and test-retest reliability could not be assessed as none of the included studies reported on this measurement property.

 

Responsiveness

Stewart (2017) did not report the level of evidence for responsiveness. However, the authors of the review described that the results of responsiveness should be interpreted with caution as most studies did not specifically aim to evaluate responsiveness and did not include a criterion standard, therefore it can be assumed that the level of evidence would be downgraded by at least one level from high to moderate because of serious risk of bias. Furthermore, the total number of participants included was lower than 50, therefore the level of evidence would be downgraded by two levels from moderate to very low because of serious imprecision. The level of evidence can be assumed to be very low.

 

4. Movement Disorder-Childhood Rating Scale (MD-CRS)

Validity

The level of evidence for content validity as evaluated in the study by Claassen (2023) was downgraded by one level from high to moderate because of serious risk of bias (only one study of adequate quality available). The level of evidence for concurrent criterion validity, predictive criterion validity and construct validity could not be assessed as none of the included studies reported on these measurement properties.

 

Reliability

The level of evidence for intra-rater reliability, test-retest reliability, inter-rater reliability and internal consistency could not be assessed as none of the included studies reported on these measurement properties.

 

Responsiveness

Stewart (2017) did not report the level of evidence for responsiveness, however only one study with a sample size lower than 50 was included, therefore the level of evidence would be downgraded by at least two levels from high to low because of very serious imprecision. The level of evidence can be assumed to be (very) low.

 

5. Movement Disorder-Childhood Rating Scale 0-3 years (MD-CRS 0-3)

Validity

The level of evidence for content validity, concurrent criterion validity, predictive criterion validity and construct validity could not be assessed as none of the included studies reported on these measurement properties.

 

Reliability

The level of evidence for intra-rater reliability, test-retest reliability, inter-rater reliability and internal consistency could not be assessed as none of the included studies reported on these measurement properties.

 

Responsiveness

Stewart (2017) did not report the level of evidence for responsiveness, however only one study with a sample size lower than 50 was included, therefore the level of evidence would be downgraded by at least two levels from high to low because of very serious imprecision. The level of evidence can be assumed to be (very) low.

 

6. Dyskinesia Impairment Scale (DIS)

Validity

Stewart (2017) assessed the level of evidence for content validity and predictive criterion validity to be limited (one fair study). For concurrent criterion validity, Stewart (2017) assessed the level of evidence to be limited (one fair study). The study of Haberfehlner (2020) provided evidence on concurrent criterion validity for the DIS in a population of non-ambulatory children with dyskinetic CP. The level of evidence for concurrent criterion validity as evaluated in the study by Haberfehlner (2020) was downgraded by two levels from high to low because of very serious imprecision (sample size < 50). The level of evidence for construct validity could not be assessed as none of the included studies reported on this measurement property.

 

Reliability

Stewart (2017) assessed the level of evidence for inter-rater reliability (two good studies) and internal consistency (one good study) to be moderate. The level of evidence for test-retest reliability as evaluated in the study by Vanmechelen (2020) was downgraded by four levels from high to very low because of very serious risk of bias (only one study of doubtful quality available) and very serious imprecision (sample size < 50). The level of evidence for intra-rater reliability could not be assessed as none of the included studies reported on this measurement property.

 

Responsiveness

The level of evidence for responsiveness as evaluated in the study by Haberfehlner (2020) was downgraded by four levels from high to very low because of very serious risk of bias (only one study of doubtful quality available) and very serious imprecision (sample size < 50).

A systematic review of the literature was performed to answer the following question: ‘What is the validity, reliability and responsiveness of instruments for measuring dyskinesia in children with cerebral palsy?’

 

P: Children with cerebral palsy (CP) and dyskinesia
I:

Burke–Fahn–Marsden Dystonia Rating Scale (BFMDRS)

Barry–Albright Dystonia Scale (BADS)

Unified Dystonia Rating Scale (UDRS)

Movement Disorder-Childhood Rating Scale (MD-CRS)

Movement Disorder-Childhood Rating Scale 0-3 years (MD-CRS 0-3)

Dyskinesia Impairment Scale (DIS)

C: see “I” (comparison of instruments if applicable)
O: validity, reliability, responsiveness

Timing and setting: at first diagnosis and evaluation of therapy

 

Relevant instruments

All available instruments for measuring dyskinesia in children with CP were included in the review; no preselection was performed.

 

Relevant outcome measures

The working group defined the outcome measures according to the Consensus-based Standards for the selection of health Measurement INstruments (COSMIN) taxonomy (Mokkink, 2010). Validity refers to ‘the degree to which a health-related patient reported outcome instrument measures the construct(s) it purports to measure’. Reliability refers to ‘the degree to which the measurement is free from measurement error’. Responsiveness refers to ‘the ability of a health-related patient reported outcome instrument to detect change over time in the construct to be measured’. See Table 1 in the attachment for definitions of the measurement properties for each of these three domains (e.g. content validity). Following COSMIN guidance, the working group considered content validity and internal consistency as critical outcome measures and all other outcomes regarding validity, reliability and responsiveness as important outcome measures. The outcomes were assessed against the criteria for good measurement outcomes as shown in Table 2 in the attachment (Prinsen, 2016).

 

Search and select (Methods)

The databases Medline (via OVID) and Embase (via Embase.com) were searched with relevant search terms until 11 May 2023. The detailed search strategy is depicted under the tab Methods. The systematic literature search resulted in 311 hits. Studies were selected based on the following criteria:

  • Systematic reviews (searched in at least two databases, and detailed search strategy, risk of bias assessment and results of individual studies available) or original studies evaluating measurement properties;
  • Children and adolescents aged <18 years;
  • Reporting about at least one of the prespecified instruments;
  • Full-text English language publication; and
  • Studies according to the PICO.

Eighteen studies were initially selected based on title and abstract screening. After reading the full text, fourteen studies were excluded (see the table with reasons for exclusion under the tab Methods), and four studies were included.

 

Results

Four studies were included in the analysis of the literature, one systematic review (Stewart, 2017) and three original studies (Haberfehlner, 2020; Vanmechelen, 2020; Claassen, 2023).

Important study characteristics are shown in Tables 3.1 and 3.2 in the attachment, and results are summarized in Table 4 in the attachment.

 

The systematic review by Stewart (2017) included an assessment of the level of evidence for each instrument, mostly following COSMIN guidance. More details on the methods used are included in Table 3.1 in the attachment.

 

For the three original studies, the level of evidence was mostly performed in accordance with COSMIN guidance for systematic reviews of PROMS (Mokkink, 2018). This assessment was performed per measurement property per instrument. Four factors were taken into account: (1) risk of bias; (2) inconsistency; (3) imprecision; and (4) indirectness. Following COSMIN guidance, publication bias was not assessed because of a lack of registries for these types of studies. To assess the methodological quality of the three original studies, i.e. assessing the risk of bias for each measurement property, the corresponding COSMIN Risk of Bias criteria were completed. To determine the overall quality of a study, the lowest rating of any standard in the box was taken (i.e. “the worst score counts” principle). The risk of bias assessment for the three original studies is reported in Tables 5.1-5.6 in the attachment.

For these types of studies, the level of evidence starts at ‘high’. The quality of evidence can be downgraded by one, two, or three levels per factor to moderate, low or very low level of evidence. See Table 6 in the attachment for the definitions of these quality levels (Mokkink, 2018).

 

For risk of bias, the level of evidence could be downgraded by three levels: one level in case of a serious risk of bias (multiple studies of doubtful quality or only one study of adequate quality), two levels in case of a very serious risk of bias (multiple studies of inadequate quality or only one study of doubtful quality), or three levels in case of an extremely serious risk of bias (only one study of inadequate quality).

 

For inconsistency, the level of evidence could be downgraded by one or two levels in case of unexplained inconsistency.

 

For imprecision, the level of evidence could be downgraded by one or two levels: one level if the total sample size was 50-100, or two levels if the total sample size was below 50. Downgrading was not performed if the sample size requirement was already included in the risk of bias assessment (for the properties content validity, structural validity, and cross-cultural validity).

 

For indirectness, the level of evidence could be downgraded by one or two levels if studies were performed in another population or another context of use compared with the population and context as defined in the PICO.

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  13. Monbaliu E, Ortibus E, De Cat J, Dan B, Heyrman L, Prinzie P, De Cock P, Feys H. The Dyskinesia Impairment Scale: a new instrument to measure dystonia and choreoathetosis in dyskinetic cerebral palsy. Dev Med Child Neurol. 2012 Mar;54(3):278-83. doi: 10.1111/j.1469-8749.2011.04209.x. PMID: 22428172.
  14. Prinsen CA, Vohra S, Rose MR, Boers M, Tugwell P, Clarke M, Williamson PR, Terwee CB. How to select outcome measurement instruments for outcomes included in a "Core Outcome Set" - a practical guideline. Trials. 2016 Sep 13;17(1):449. doi: 10.1186/s13063-016-1555-2. PMID: 27618914; PMCID: PMC5020549.
  15. Prinsen CAC, Mokkink LB, Bouter LM, Alonso J, Patrick DL, de Vet HCW, Terwee CB. COSMIN guideline for systematic reviews of patient-reported outcome measures. Qual Life Res. 2018 May;27(5):1147-1157. doi: 10.1007/s11136-018-1798-3. Epub 2018 Feb 12. PMID: 29435801; PMCID: PMC5891568.
  16. Stewart K, Harvey A, Johnston LM. A systematic review of scales to measure dystonia and choreoathetosis in children with dyskinetic cerebral palsy. Dev Med Child Neurol. 2017 Aug;59(8):786-795. doi: 10.1111/dmcn.13452. Epub 2017 May 9. PMID: 28485494.
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  18. Terwee CB, Bot SD, de Boer MR, van der Windt DA, Knol DL, Dekker J, Bouter LM, de Vet HC. Quality criteria were proposed for measurement properties of health status questionnaires. J Clin Epidemiol. 2007 Jan;60(1):34-42. doi: 10.1016/j.jclinepi.2006.03.012. Epub 2006 Aug 24. PMID: 17161752.
  19. Vanmechelen I, Dan B, Feys H, Monbaliu E. Test-retest reliability of the Dyskinesia Impairment Scale: measuring dystonia and choreoathetosis in dyskinetic cerebral palsy. Dev Med Child Neurol. 2020 Apr;62(4):489-493. doi: 10.1111/dmcn.14424. Epub 2019 Dec 13. PMID: 31833574.

Table 1: COSMIN definitions of domains, measurement properties, and aspects of measurement properties (Mokkink, 2010)

Term

Definition

Domain

Measurement property

Aspect of a measurement property

Reliability

 

 

The degree to which the measurement is free from measurement error

Reliability (extended definition)

 

 

The extent to which scores for patients who have not changed are the same for repeated measurement under several conditions; e.g. using different sets of items from the same health related-patient reported outcomes (HR-PRO) (internal consistency); over time (test-retest); by different persons on the same occasion (inter-rater; or by the same persons (i.e. raters or responders) on different occasions (intra-rater)

 

Internal consistency

 

The degree of the interrelatedness among the items

 

Reliability

 

The proportion of the total variance in the measurements which is due to ‘true’† differences between patients

 

Measurement

 

The systematic and random error of a patient’s score that is not attributed to true changes in the construct to be measured

Validity

 

 

The degree to which an HR-PRO instrument measures the construct(s) it purports to measure

 

Content validity

 

The degree to which the content of an HR-PRO instrument is an adequate reflection of the construct to be measured

 

 

Face validity

The degree to which (the items of) an HR-PRO instrument indeed looks as though they are an adequate reflection of the construct to be measured

 

Construct validity

 

The degree to which the scores of an HR-PRO instrument are consistent with hypotheses (for instance with regard to internal relationships, relationships to scores of other instruments, or differences between relevant groups) based on the assumption that the HR-PRO instrument validly measures the construct to be measured

 

 

Structural validity

The degree to which the scores of an HR-PRO instrument are an adequate reflection of the dimensionality of the construct to be measured

 

 

Hypotheses testing

Idem construct validity

 

 

Cross-cultural validity

The degree to which the performance of the items on a translated or culturally adapted HR-PRO instrument are an adequate reflection of the performance of the items of the original version of the HR-PRO instrument

 

Content validity

 

The degree to which the content of an HR-PRO instrument is an adequate reflection of the construct to be measured

 

 

Face validity

The degree to which (the items of) an HR-PRO instrument indeed looks as though they are an adequate reflection of the construct to be measured

 

Criterion validity

 

The degree to which the scores of an HR-PRO instrument are an adequate reflection of a ‘gold standard’

Responsiveness

 

 

The ability of an HR-PRO instrument to detect change over time in the construct to be measured

 

Responsiveness

 

Idem responsiveness

Interpretability*

 

 

Interpretability is the degree to which one can assign qualitative meaning – that is, clinical or commonly understood connotations – to an instrument’s quantitative scores or change in scores.

† The word ‘true’ must be seen in the context of the CTT, which states that any observation is composed of two components – a true score and error associated with the observation. ‘True’ is the average score what would be obtained if the scale were given an infinite number of times. It refers only to the consistency of the score, and not to its accuracy (Streiner, 2008).

* Interpretability is not considered a measurement property, but an important characteristic of a measurement instrument.

 

Abbreviations

HR-PRO: health related-patient reported outcomes

 

Table 2: Criteria for Good MEasurement Properties (adapted from Prinsen, 2018; based on Terwee, 2007 and Prinsen, 2016)

Measurement Property

Rating

Criteria

Structural validity

+

 

CTT: CFI or TLI or comparable measure > 0.95 OR RMSEA < 0.06 OR SRMR < 0.082

IRT/Rasch: No violation or unidimensionality: CFI or TLI or comparable measure > 0.95 OR RMSEA < 0.06 OR SRMR < 0.08

AND

no violation of local independence: residual correlations among the items after controlling for the dominant factor < 0.20 OR Q3’s < 0.37

AND

no violation of monotonicity: adequate looking graphs OR item scalability > 0.30

AND

adequate model fit IRT: X2 > 0.01.

Rasch: infit and outfit means squares ≥ 0.5 and ≤ 1.5 OR Z-standardised values > -2 and < 2

?

CTT: not all information for ‘+’ reported

IRT/Rasch: model fit not reported

-

Criteria for ‘+’ not met

Internal consistency

+

 

At least low evidence for sufficient structural validity

AND

Cronbach’s alpha(s) ≥ 0.70 for each unidimensional scale or subscale

?

Criteria for “At least low evidence for sufficient structural validity” not met

-

At least low evidence for sufficient structural validity

AND

Cronbach’s alpha(s) < 0.70 for each unidimensional scale or subscale

Reliability

+

ICC or weighted Kappa ≥ 0.70

?

ICC or weighted Kappa not reported

-

ICC or weighted Kappa < 0.70

Measurement error

+

SDC or LoA < MIC

?

MIC not defined

-

SDC or LoA >MIC

Hypotheses testing for construct validity

+

The result is in accordance with the hypothesis

?

No hypothesis defined (by the review team)

-

The result is not in accordance with the hypothesis

Cross-cultural validity/measurement invariance

+

 

No important differences found between group factors (such as age, gender, language) in multiple group factor analysis OR no important DIF for group factors (McFadden’s R2 <0.02)

?

No multiple group factor analysis OR DIF analysis performed

-

Important differences between group factors OR DIF was found

Criterion validity

+

Correlation with gold standard ≥ 0.70 or AUC ≥ 0.70

?

Not all information for ‘+’ reported

-

Correlation with gold standard < 0.70 or AUC < 0.70

Responsiveness

+

The result is in accordance with the hypothesis OR AUC ≥ 0.70

?

No hypothesis defined (by the review team)

-

The result is not in accordance with the hypothesis or AUC < 0.70

AUC: Area under the curve; CFA: confirmatory factor analysis; CFI: comparative fit index; CTT: classical test theory; DIF: differential item functioning; ICC: intraclass correlation coefficient; IRT: item response theory; LoA: limits of agreement; MIC: minimal important change; RMSEA: root mean square error of approximation; SEM: standard error of measurement; SDC: smallest detectable change; SRMR: standardised root mean residuals; TLR: Tucker-Lewis Index

“+” = sufficient
“?” = indeterminate
“-“ = insufficient

 

Table 3.1: Description of systematic review of studies on measurement properties of health status measurement instruments

Research question: ‘What is the validity, reliability and responsiveness of instruments for measuring dystonia/hyperkinesia in children with CP?’

Study reference

Study characteristics

Participant characteristics

Measurement properties

 

Comments

Stewart, 2017

 

 

 

Systematic review, literature search up to April 2016

 

34 studies were included, of which 18 presented results specifically for children with CP

 

Burke–Fahn–Marsden Dystonia Rating Scale (BFMDRS)

A: Monbaliu, 2010

B: Marks, 2011

C: Keen, 2014

D: Motta, 2008

E: Gimeno, 2012

F: Air, 2011

G: Lumsden, 2013

H: Marks, 2013

I: Olaya, 2013

J: Romito, 2015

K: Sanger, 2007

L: Trabacca, 2011

 

Barry–Albright Dystonia Scale (BADS)

A: Monbaliu, 2010

D: Motta, 2008

M: Gordon, 2006

N: Kawamura, 2012

O: Monbaliu, 2012

 

Unified Dystonia Rating Scale (UDRS)

A: Monbaliu, 2010

K: Sanger, 2007

 

Movement Disorder-Childhood Rating Scale (MD-CRS)

P: Battini, 2014

 

MD-CRS (0-3 years)

P: Battini, 2014

 

Dyskinesia Impairment Scale (DIS)

O: Monbaliu, 2012

Q: Monbaliu, 2015

R: Monbaliu, 2013

 

Source of funding and conflicts of interest:

No funding reported. No conflicts of interest reported.

 

Inclusion criteria SR:

- include a clinical measure of dystonia and/or choreoathetosis according to the definition of Himmelmann (2007);

- include children with CP aged 0 to 18 years;

- report original psychometric data that could be isolated for children with CP;

- reported in English.

 

Exclusion criteria SR:

- reported only laboratory-based measures.

 

 

 

Psychometric and clinical utility data were extracted for the selected instruments reported in the included papers using the CanChild Outcome Measures Rating Form for validity, reliability, responsiveness, and clinical utility.

 

Measurement properties considered in the review by Stewart (2017) were:

 

Validity

- construct validity (structural validity, hypothesis testing, cross-cultural validity)

- content validity

- criterion validity (concurrent and predictive)

Reliability

- intra-rater

- test-retest

- inter-rater

- internal consistency

Responsiveness

- responsiveness

Reliability was evaluated as the degree to which an assessment yielded the same score when administered by the same examiner on different occasions (intrarater or test-retest), or by a different examiner on the same occasion (interrater). Reliability data presented using intra- class correlation coefficients and Cronbach’s alpha were rated using the following criteria: greater than or equal to 0.8, excellent; 0.6 to 0.79, adequate; less than or equal to 0.6, poor.

Validity was evaluated as the extent to which the tools measured what they purported to measure and included construct, content, and criterion validity.

 

Responsiveness, the ability of the tool to detect change over time in the construct being measured, was reported as the mean change in scale scores after an intervention and where available statistical significance of this change was noted.

 

Rating of level of evidence:

Stewart (2017) used the Consensus-based Standards for selection of health Measurement Instruments (COSMIN) checklist used to rate the evidence for each scale using the levels strong, moderate, limited, conflicting, or unknown.

This rating was based on:

(1) The number of studies available involving the scale;

(2) the methodological quality of each study (excellent, good, fair, poor);

(3) the strength of psychometric data (good, intermediate, poor, unknown);

(4) the finding of each study (positive or negative.

 

The overall quality score for each measurement property was determined by the lowest rating of any of the items as recommended by the COSMIN manual.

 

Author’s conclusion

Each of the six tools identified in this review provides valuable information about dystonia and/ or choreoathetosis severity in children with CP; however, only the BADS and DIS have been developed specifically for CP and only the DIS addresses both dystonia and choreoathetosis. Further studies are required to examine the validity, reliability, responsiveness, and clinical utility of each scale for use with children with CP before clear recommendations can be made about tool choice for research and clinical purposes. In the meantime, the BADS can be considered the most clinically useful tool for measuring dystonia in children with CP. For research purposes or CP register settings, the DIS presents as the most com- prehensive tool, enabling experienced users to distinguish between and measure both dystonia and choreoathetosis.

 

 

 

 

Table 3.2: Description of studies on measurement properties of health status measurement instruments

Research question: ‘What is the validity, reliability and responsiveness of instruments for measuring dystonia/hyperkinesia in children with CP?’

Study reference

Study characteristics

Participant characteristics

Measurement properties and procedures

Comments

Haberfehlner (2020)

Instrument assessed:

Dyskinesia Impairment Scale

 

Type of study: secondary analysis of an RCT on the effects of intrathecal baclofen (ITB)

 

Setting and country: two university medical centres in the Netherlands

 

Funding and conflicts of interest: No funding was reported. The authors reported no conflicts of interest.

Inclusion criteria:

- presenting with dyskinetic CP;

- classified in Gross Motor Function Classification System levels IV and V;

- aged 4 to 25 years;

- lesions on magnetic resonance imaging;

- eligible for ITB treatment using commonly applied criteria;

- completed the DIS and BADS at baseline and 3 months after pump implantation.

 

Exclusion criteria:

- contraindication for general anaesthesia or baclofen;

- oral pharmacological treatment was sufficient;

- deep brain stimulation;

- ventriculoperitoneal shunt;

- other disorders interfering with treatment.

 

N=33

 

Mean age ± SD:

ITB-treated (n=17): 14y1mo ± 4y 1mo

Placebo-treated (n=16): 14y7mo ± 4y

 

Sex: 73% M / 27% F

 

Brief video sequences of all participants were made, according to the DIS video protocol. Two experienced paediatric therapists were trained to score the DIS and BADS and scored all videos. They were blinded to the measurement time points (baseline and follow-up) and group allocation and scored the videos in random order within a year’s time. The same rater assessed both time points (baseline and follow-up) and both scales (DIS and BADS) in an individual child. Scoring of the BADS was performed on a selection of videos recorded for the DIS: sitting at rest; speaking (if performed); grasping/reaching for a pen from a lying position (left and right), and rolling over left and right.

 

Concurrent validity

The score of the DIS and the score for the criterion standard (i.e. BADS) were considered at the same time point (at baseline and follow-up). At both time points, the correlation between the DIS dystonia subscale and the BADS was assessed.

 

Responsiveness

The effect sizes of the DIS were interpreted using Cohen’s criteria. Effect sizes of the DIS dystonia subscale were compared to the effect size of the BADS.

 

Mean follow-up time was 3 months (SD 0.2 months) in the ITB-treated group and 2.8 months (SD 0.6 months) in the placebo-treated group.

 

Authors’ conclusion

The DIS dystonia subscale shows similar responsiveness in comparison to the BADS in non-ambulatory children and young adults with dyskinetic CP. Concurrent validity of the DIS is adequate as reported previously. Feasibility of the DIS activity items is restricted in patients with dyskinetic CP in GMFCS levels IV and V. Reducing the activity items of the DIS should be investigated.

Vanmechelen (2020)

Instrument assessed:

Dyskinesia Impairment Scale

 

Type of study: subset of participants included in the study by Monbaliu (2012) who were available for a retest recording.

 

Setting and country: special education schools for children with motor disability in Belgium

 

Funding and conflicts of interest: This work was supported by a grant from the Marguerite-Marie Delacroix Foundation. The authors have stated that they had no interest that could be perceived as posing a conflict or bias.

 

Inclusion criteria:

-predominant dyskinetic CP diagnosed by a paediatric neurologist within a reference centre for CP and birth at term

 

Exclusion criteria:

-changes in muscle tone-changing medication within the last 3 months;

-orthopaedic or neurosurgical interventions within the previous year;

-spine fusion.

 

N=15

 

Mean age ± SD: 14y ± 4y, range 5-22y

 

Sex: 87% M / 13% F

 

 

Participants were videotaped according to the DIS video protocol, which contains three rest postures and 24 activities. During filming, male participants wore shorts and female participants wore shorts and a bra to maximize visibility of all body parts for scoring. To assess reliability over time, participants were videotaped twice, the second time within 7 days after the first recording to minimize environmental or therapy effects. Test and retest videos were filmed by the same assessor (EM). Scoring of both test and retest videos was done by the same rater (EM) within 15 days, with 3 months in between the scoring of the test and retest video to minimize recall bias. The DIS film protocol varies between 40 and 60 minutes. Scoring was done on a 5-point scale from 0 to 4 for each body part, during both action and rest. Each score was summed for a total dystonia score, a total choreoathetosis score, and a total DIS score. To assess test–retest reliability for the region scores, all scores for each region both in activity and rest, were summed.

Test-retest reliability (7 days)

Intraclass correlation coefficients and 95% confidence intervals, standard error of measurement and the minimal detectable difference were assessed.

 

Authors’ conclusion

The good results of the assessment of dystonia and choreoathetosis over time show that the DIS can be used in clinical practice and research to assess the effect of various interventions on the presence and severity of dystonia and choreoathetosis.

 

Claassen (2023)

Instrument assessed: Movement Disorder-Childhood Rating Scale

 

Type of study: qualitative cross-sectional interview study

 

Setting and country: children and adolescents were recruited from patient panels, through physician outreach, and social media. The study was conducted in the United States of America

 

Funding and conflicts of interest:

This study was funded by Teva Branded Pharmaceutical Products R&D, Inc., West Chester, PA, USA. The sponsor was involved in the study design, data collection and analysis, writing the report, and the decision to submit the article. R.B. thanks Current Research (RC) of the Italian Ministry of Health, IRCCS Stella Maris Foundation (FSM), entitled “Development of a Genetic and Acquired Movement Disorders Register in Children: From Creating Italian Network to Providing New Outcome Measures” and 51000 IRCCS FSM. The authors thank William J. Kelley, PhD (Cello Health Communications/ MedErgy, with funding from Teva Pharmaceuticals), for editorial assistance in the preparation of this manuscript.

 

Several authors reported various potential conflicts of interest.

 

Inclusion criteria for children and adolescents:

- children aged six to 11 years (inclusive) or adolescent aged 12 to 18 years (inclusive);

- living in the United States;

- having self-reported DCP and predominant choreiform movement or dystonia;

- have been treated for DCP;

- have a caregiver willing to provide permission for the child to participate and who is willing to participate in the study themselves as part of a child-caregiver dyad;

- able to communicate verbally;

- willing and able to participate in a 60-minute interview.

 

Inclusion criteria for caregivers:

- acting as a caregiver to a child or adolescent aged six to 18 years with DCP who experiences choreiform movements or dystonia;

- living in the United States;

- speaking and reading English fluently.

 

Caregivers were defined as anyone, paid or unpaid, who spends at least four hours per day providing care (e.g., helping child with bathing, dressing, eating, taking medication, or transportation assistance) for a child or adolescent with DCP.

 

N=8 children/adolescents and 12 caregivers

 

Children and adolescents mean age ± SD: 14y ± 3.83y

 

Sex: 63% M / 38% F

 

 

60-minute interviews were conducted with children and adolescents with DCP and caregivers of children with DCP.

 

As formative work, the study team closely reviewed all published literature specific to the MD- CRS and conducted interviews with key opinion leaders, including the developers of the MD-CRS and three neurologists who treat children with DCP, to gather insight on the instrument.

Two semi-structured interview guides were developed (version 1: ages six to 11 years, version 2: ages 12 to 18 years, version 3: caregivers).

 

 All interviews were conducted by the study team's qualitative researchers, who were familiar with the specific objectives and requirements of the study and trained to ensure proper interview preparation. Interviews were recorded using digital audio-recording equipment. All interview recordings were transcribed verbatim.

Content validity

Participants were asked to identify body regions affected by DCP. The interviewer probed for any regions included in the MD-CRS that were not mentioned spontaneously by participants.

Participants were asked how DCP affected their daily functioning. The interviewer probed for any functions or activities included in the MD-CRS that were not mentioned spontaneously by participants.

 

Caregivers were asked to reflect on the relevance and usefulness of each item and response option in Part I of the MD-CRS.

 

 

Authors’ conclusion

The information gathered from this qualitative study provides evidence in support of the content validity of the MD-CRS for children/adolescents with DCP. The children/adolescents and caregivers interviewed for this study confirmed that all body regions and activities listed in the MD-CRS were relevant to their experiences with DCP. This study suggests that the MD-CRS is fit for purpose as a clinical trial end point to assess the effect of treatment for children and adolescents with DCP.

 

 

Table 4: Evidence table for studies on measurement properties of health status measurement instruments

Research question: ‘What is the validity, reliability and responsiveness of instruments for measuring dystonia/hyperkinesia in children with CP?’

Instrument

Psychometric property

1st author (year of publication)

Results

For studies included in the review by Stewart (2017): Stewart’s rating of evidence using the COSMIN checklist

For the three original studies:

 

COSMIN Risk of bias assessment

 

Assessment of study findings

Burke–Fahn–Marsden Dystonia Rating Scale (BFMDRS)

Validity

Construct

-

-

-

-

Content

Monbaliu (2010)*

Expert opinion: independent authors judged the ability of BFMDRS to measure six features of the dystonic movement disorder. (1) Body regions: yes (nine regions); (2) proximal and distal limb segments: no; (3) during rest vs activity: partial; (4) duration: partial; (5) amplitude: partial; (6) influence on functional activities: yes. Overall: tool not aligned with recent dystonia definitions and considers dystonia only.

Moderate: three fair studies

n/a

Marks (2011)*

Expert opinion: scale most sensitive to change. Scales not necessarily suitable for children with CP with mixed motor type.

n/a

Keen (2014)*

Expert opinion: not appropriately tailored for children with dystonic CP with mixed motor type. Does not reflect on subtle quality of life or caretaking changes.

n/a

Criterion (concurrent)

Motta (2008)*

BADS vs BFMDRS (n=19 CP, 2y 5mo–16y 6mo): moderate correlation between total scores (r=0.59, p=0.05)

Moderate: two fair studies

n/a

Monbaliu (2010)*

BADS vs BFMDRS (n=10 CP, 5–22y): high correlation between total scores of three raters (rater 1: Pearson’s r=0.93, 95% CI 0.73–0.98; rater 2: r=0.95, 95% CI 0.79–0.99; rater 3: r=0.95, 95% CI 0.69–0.98; all p<0.001).

n/a

Criterion (predictive)

Gimeno (2012)*

BFMDRS vs COPM (n=14 CP, 3y 6mo–18y): no correlation between changes in BFMDRS score and changes in COPM after deep brain stimulation at 6mo (r=0.201, p=0.511) or 12mo (r=0.176, p=0.565).

Limited: one fair study

n/a

Reliability

Intra-rater

-

-

-

-

Test-retest

-

-

-

-

Inter-rater

Monbaliu (2010)*

Video-based assessment: (CP n=10, age 5-22 years, 3 experienced raters)
Total scores - excellent reliability for the ‘Severity factor’ (ICC2,1=0.89) and the ‘Provoking factor x severity factor’ (ICC2,1=0.86) and adequate reliability for the ‘Provoking factor’ (ICC2,1=0.64)
Item scores - poor to adequate reliability for ‘Severity factor’ items (ICC2,1=0.34-0.77); and poor to excellent reliability for ‘Provoking factor’ (ICC2,1 = 0.32-0.93) and ‘Severity factor x Provoking factor’ items (ICC2,1=0.45-0.86).
Measurement error - high (SEM = 9.88%)
Smallest detectable change - large (SDD = 27.39%)

Moderate: one good study

n/a

Internal consistency

Monbaliu (2010)*

Video-based assessment: (CP n=10, 5-22 years, 3 experienced raters) Excellent internal consistency (Cronbach α = 0.83-0.94)  

Moderate: one good study

n/a

Responsiveness

 

Air (2011)*

Intervention: Deep Brain Stimulation

Sample: CP=6 (data for CP=4 only, 6.9 to 17.8 years)
Follow-up: range 3-9m

Responsiveness:

movement scale: 9.67% mean decrease(ref*)

disability scale: 14% mean decrease

-

-

Gimeno (2012)*

Intervention: Deep Brain Stimulation

Sample: CP=6

Follow-up: 6 & 12m

Responsiveness:

movement scale: no clinical/significant change, mean decrease 5.9% (range 1.5% to 9%)

-

-

Keen (2014)*

Intervention: Deep Brain Stimulation

Sample: CP=5 (8 to 17 years)

Follow-up: mean 26.6m

Responsiveness:

movement scale: mean decrease 28.5% (p=0.1)

-

-

Lumsden (2013)*

Intervention: Deep Brain Stimulation

Sample: CP=28 (3.3 to 20 years)

Follow-up: 6 & 12 m

Responsiveness:

movement scale: median (% improvement) Pre: 99, 6m: 97 (5.3%); 12m 95.5 (7.3%)

-

-

Marks (2011)*

Intervention: Deep Brain Stimulation

Sample: CP=8 (7 to 15 years)

Follow-up: 6 m

Responsiveness:

movement scale: mean decrease 37.84%, p=0.012

disability scale: mean decrease 14.44%, p=0.02

-

-

Marks (2013)*

Intervention: Deep Brain Stimulation

Sample: CP=9 (mean 11.02 years)

Follow-up: 6, 12 & 18 m

Responsiveness:

movement scale: clinically significant 24.3% mean decrease (p=0.90)

disability scale: 3.62% mean decrease (p=0.70)

-

-

Olaya (2013)*

Intervention: Deep Brain Stimulation

Sample: CP=2 (6 and 16 years)

Follow-up: 3 m

Responsiveness:

movement scale: 0% change

-

-

Romito (2015)*

Intervention: Deep Brain Stimulation

Sample: CP=4 (15 to 18 years)

Follow-up: range 3-6.2 years

Responsiveness:

movement scale: 37.34% mean decrease

disability scale: 17.89% mean decrease

-

-

Motta (2008)*

Intervention: Intrathecal Baclofen

Sample: CP=19 (2y5m to 16y6m)

Follow-up: 12 m

Responsiveness:

movement scale: significant decrease, p<0.001

disability scale: no change

-

-

Sanger (2007)*

Intervention: Botulinum Toxin-A

Sample: CP=7 (2.1 to 15.7 years)

Follow-up: 1m post injections

Responsiveness:

movement scale: upper extremity subscore: change p=0.0069

-

-

Trabacca (2011)*

Intervention: Tetrabenazine

Sample: CP=1 (12 years)

Follow-up: 6 m

Responsiveness:

movement scale: 18% decrease in dystonia score

-

-

Barry–Albright Dystonia Scale (BADS)

Validity

Construct

Gordon (2006)*

Clinical studies

Spasticity: Ashworth scale (n=13 CP). Higher dystonia not correlated with greater spasticity (r=0.36, p=0.22).

Moderate: three fair studies

n/a

Gordon (2006)*

Laboratory studies
Motor control: kinematic analysis of reaching (n=13 CP). Higher BADS score correlated with longer reach paths (r=0.75, p=0.005).

n/a

Kawamura (2012)*

Motor control: Kinematic Dystonia Measure (n=8 CP). Poorer hand- tapping correlated with higher total BADS score (r=0.79, p=0.003) and affected arm BADS score (r=0.76, p=0.007). Slower eye-blinking correlated with adjusted affected arm BADS scores (r=0.72, p=0.02) but not total BADS score (r=0.41, p=0.24).

n/a

Content

Monbaliu (2010)*

Expert opinion: ability to measure (1) body regions: yes (eight regions); (2) proximal and distal limb segments: no; (3) during rest vs activity: no; (4) duration: yes; (5) amplitude: yes; (6) influence on functional activities: yes. Overall: tool not aligned with recent dystonia definitions and considers dystonia only.

Limited: one fair study

n/a

Criterion (concurrent)

Motta (2008)*

BFMDRS vs BADS (n=19 CP, 2y 5mo–16y 6mo): moderate correlation between total scores (r=0.59, p=0.05).

Moderate: four fair-good studies

n/a

Monbaliu (2010)*

BFMDRS vs BADS (n=10 CP, 5–22y): high correlation between total scores of three raters (rater 1: Pearson’s r=0.93, 95% CI 0.73–0.98; rater 2: r=0.95, 95% CI 0.79–0.99; rater 3: r=0.95, 95% CI 0.69–0.98; all p<0.001).

n/a

Monbaliu (2012)*

DIS vs BADS (n=25 CP, 5–22y): good correlation between scales (r=0.84; 95% CI 0.66–0.92; p<0.001).

n/a

Monbaliu (2010)*

UDRS vs BADS (n=10 CP, 5–22y): moderate–high correlation between total scores of three raters (rater 1: r=0.89, 95% CI 0.60–0.97, p<0.001; rater 2: r=0.84, 95% CI 0.6–0.96, p=0.002; rater 3: r=0.86, 95% CI 0.51–0.97, p<0.001).

n/a

 

Criterion (predictive)

Battini (2008)*

BADS vs ‘global rating of change’ (before and after trial) (n=10 CP, 3–15y): assessed by doctor, physical therapist, and family after intrathecal baclofen therapy trial; agreement same (n=1), worse (n=1), better (n=8).

Limited: one fair study

n/a

Reliability

Intra-rater

-

No data specifically for children with CP

-

-

Test-retest

-

No data specifically for children with CP

-

-

Inter-rater

Monbaliu (2010)*

Video-based assessment: (CP n=10, age 5-22 years, 3 experienced raters)
Total score - excellent reliability (ICC2,1=0.87).
Item scores - adequate reliability for limbs and trunk (ICC2,1=0.61-0.76); but poor for eyes, mouth & neck (ICC2,1=0.39-0.57).
Measurement error - high (SEM = 6.36%)
Smallest detectable change - large (SDD = 17.72%)

Moderate: one good study

n/a

Internal consistency

Monbaliu (2010)*

Video-based assessment: (CP n=10, 5-22 years, 3 experienced raters) Excellent internal consistency (Cronbach α = 0.87-0.91)

Moderate: one good study

n/a

Responsiveness

 

Air (2011)*

Intervention: Deep Brain Stimulation

Sample: CP=1 (8 years)

Follow-up: 9 m

Responsiveness: mean decrease 21.7% (no p-value)

-

-

Bhanpuri (2014)*

Intervention: Deep Brain Stimulation

Sample: CP=3 (8 to 17 years)

Follow-up: varied, >5 m

Responsiveness: no mean change (no p-value)

-

-

Marks (2011)*

Intervention: Deep Brain Stimulation

Sample: CP=8 (7 to 15 years)

Follow-up: 6 m

Responsiveness: mean decrease 19.48%, p=0.024

-

-

Albright (2009)*

Intervention: Intra-ventricular Baclofen

Sample: CP=4 (2 to 10 years)

Follow-up: range 3-21 months

Responsiveness: mean decrease 54.87% (no p-value)

-

-

Barry (1999)*

Intervention: Intrathecal Baclofen

Sample: CP=10 (3 to 15 years)

Follow-up: range 3-9 days

Responsiveness: mean decrease 47.3% (no p-value)

-

-

Bollo (2012)*

Intervention: Intrathecal Baclofen

Sample: CP=2 (3.7 and 17 years)

Follow-up: 6 m

Responsiveness: mean decrease 62.15% (no p-value)

-

-

Motta (2008)*

Intervention: Intrathecal Baclofen

Sample: CP=19 (2y5m to 16y6m)

Follow-up: 12 m

Responsiveness: significant decrease, p<0.001; mean baseline=23.84, mean 12 months post=17.79

-

-

Motta (2011)*

Intervention: Intrathecal Baclofen

Sample: CP=7 (mean 15y11m, SD 10y8m)

Follow-up: 12 m

Responsiveness: median decrease 21 (IQR 10) to 15 (IQR 4) (p=0.018)

-

-

Ward (2009)*

Intervention: Intrathecal Baclofen

Sample: CP=4 (3-16 years)

Follow-up: 6 m

Responsiveness: mean decrease 44.82% (no p-value)

-

-

Dachy (2004)*

Intervention: Baclofen test bolus

Sample: CP=5 (2 to 16 years)

Follow-up: immediate

Responsiveness: mean baseline=16.6, mean post test dose=14.8

-

-

Rice (2009)*

Intervention: Artane

Sample: CP=16 (2 to 17 years)

Follow-up: 12 and 28 weeks

Responsiveness: mean baseline=18.4, mean end of drug phase=18.3, mean end of placebo phase=16.9

-

-

Unified Dystonia Rating Scale (UDRS)

Validity

Construct

-

-

-

-

Content

Monbaliu (2010)*

Expert opinion: ability to measure (1) body regions: yes (14 regions); (2) proximal and distal limb segments: yes; (3) during rest vs activity: no; (4) duration: partial; (5) amplitude: yes; (6) influence on functional activities. Overall: tool not aligned with recent dystonia definitions and considers dystonia only.

Moderate: one good study

n/a

Criterion (concurrent)

Monbaliu (2010)*

BADS vs UDRS (n=10 CP, 5–22y): moderate–high correlation between total scores of three raters (rater 1: r=0.89, 95% CI 0.60–0.97, p<0.001; rater 2: r=0.84, 95% CI 0.6–0.96, p=0.002; rater 3: r=0.86, 95% CI 0.51–0.97, p<0.001)

BFMDRS vs UDRS (n=10 CP, 5–22y): high correlation between total scores (rater 1: Pearson’s r=0.87, 95% CI 0.53– 0.97, p<0.001; rater 2: r=0.88, 95% CI 0.56–0.97, p=0.002; rater 3: r=0.93, 95% CI 0.73–0.98, p<0.001).

Moderate: one good study, two comparisons

n/a

Reliability

Intra-rater

-

-

-

-

Test-retest

-

-

-

-

Inter-rater

Monbaliu (2010)*

Video-based assessment: (CP n=10, age 5-22 years, 3 experienced raters)
Total scores - adequate reliability for all totals (‘Duration Factor’ ICC2,1=0.74; ‘Motor Severity’ ICC2,1=0.79; and ‘Duration Factor x Motor Severity Factor’ ICC2,1=0.79).
Item scores - poor to adequate reliability for ‘Duration Factor’ items (ICC2,1=0.17-0.71); and poor to excellent reliability for ‘Motor Severity Factor’ items (ICC2,1=0.26-0.83) and Duration Factor x Motor Severity Factor’ items (ICC2,1=0.37-0.81).
Measurement error - high (SEM = 8.89%)
Smallest detectable difference - large (SDD = 24.63%)

Moderate: one good study

n/a

Internal consistency

Monbaliu (2010)*

Video-based assessment: (CP n=10, 5-22 years, 3 experienced raters) Excellent internal consistency (Cronbach α = 0.90-0.95)

Moderate: one good study

n/a

Responsiveness

 

Sanger (2007)*

Study: Botulinum Toxin-A

Sample: CP=7 (2.1 to 15.7 years)

Follow-up: 1 month post injections

Responsiveness: proximal upper extremity change p=0.12, distal change p=0.00088

-

-

Movement Disorder-Childhood Rating Scale (MD-CRS)

Validity

Construct

-

-

-

-

Content

Claassen (2023)

Interviews with children/adolescents and caregivers: 

Participants confirmed that the body regions and activities listed in the MD-CRS were affected by DCP and that involuntary movements interfered with all motor, oral/verbal, self-care, and video protocol activities. Caregivers endorsed the response options for 12 of 15 items in MD-CRS Part I and suggested clarifications for others.

n/a

Methodological quality:

adequate

 

sufficient content validity

Criterion (concurrent or predictive)

-

-

-

-

Reliability

Intra-rater

-

-

-

-

Test-retest

-

No data specifically for children with CP

-

-

Inter-rater

-

No data specifically for children with CP

-

-

Internal consistency

-

No data specifically for children with CP

-

-

Responsiveness

 

Battini (2014)*

Study: Trihexyphenidyl trial

Sample: CP=27 (4 to 18.3 years)

Follow-up: 6 and 12 months

Responsiveness:

ANOVA main effect of time for all 3 Indices significant, p<0.0001

Bonferroni post-hoc test on change scores significant between all time points for all indices (expect Index II at 12m vs 6m)

Regression analysis: not significant (p>0.05) for changes at 12m & age for all Indices.

-

-

Movement Disorder-Childhood Rating Scale 0-3 years (MD-CRS 0-3)

Validity

Construct

-

-

-

-

Content

-

-

-

-

Criterion (concurrent or predictive)

-

-

-

-

Reliability

Intra-rater

-

-

-

-

Test-retest

-

No data specifically for children with CP

-

-

Inter-rater

-

No data specifically for children with CP

-

-

Internal consistency

-

No data specifically for children with CP

-

-

Responsiveness

 

Battini (2014)*

Study: Trihexyphenidyl trial

Sample: CP=20 (0.7 to 3.3 years)

Follow-up: 6 and 12 months

Responsiveness:

ANOVA main effect of time for all 3 Indices significant, p<0.0001

Bonferroni post-hoc test on change scores significant between all time points for all indices (expect Index II at 12m vs 6m)

Regression analysis: not significant (p>0.05) for changes ta 12m & age for all Indices.

-

-

Dyskinesia Impairment Scale (DIS)

Validity

Construct

-

-

-

-

Content

Monbaliu (2012)*

Expert opinion sought on content and available scales

Limited: one fair study

n/a

Criterion (concurrent)

Monbaliu (2012)*

DIS vs BADS (n=25 CP, 5–22y): good correlation between scales (r=0.84; 95% CI 0.66–0.92; p<0.001.

Limited: one fair study

n/a

Haberfehlner (2020)

The Pearson’s correlation coefficients between the DIS dystonia subscale and BADS at baseline and follow-up were 0.78 (95% CI 0.59–0.88; p<0.001) and 0.79 (95% CI 0.60–0.89; p<0.001) respectively.

n/a

Methodological quality: very good

 

Sufficient validity (correlations ≥ 0.70)

 

Criterion (predictive)

Monbaliu (2015)*

DIS vs GMFCS, MACS, and CFCS (n=55 CP, 6–22y). Higher GMFCS showed good correlation with higher total dystonia score (Spearman’s q=0.70, 95% CI 0.53–0.81, p<0.001) and dystonia in the leg region (q=0.65, 95% CI 0.47– 0.78, p<0.01). Higher MACS showed good correlations with total dystonia (q=0.68, 95% CI 0.47–0.81, p<0.001) and dystonia in the arm region (q=0.74, 95% CI 0.59–0.84, p<0.001). Higher CFCS showed fair correlation with total DIS (q=0.36, 95% CI 0.11–0.57, p=0.002) and dystonia in the mouth region (q=0.36, 95% CI 0.11–0.57, p=0.002). No significant correlations were found between choreoathetosis and GMFCS (total p=0.18, leg region p=0.86); or MACS (total p=0.09, arm region p=0.08); or CFCS (total p=0.06; mouth region p=0.30).

Limited: one fair study

n/a

Reliability

Intra-rater

-

-

-

-

Test-retest

Vanmechelen (2020)

Intraclass correlation coefficients of the DIS, the dystonia subscale of the DIS, and the choreoathetosis subscale of the DIS were 0.98 (95% CI 0.94–0.99), 0.97 (95% CI 0.92–0.99), and 0.96 (95% CI 0.90–0.99). The standard error of measurement and minimal detectable difference were 2.6% and 7.2%.

n/a

Methodological quality:

doubtful

 

sufficient test-retest reliability (ICC ≥ 0.70)

Inter-rater

Monbaliu (2012)*

Video-based assessment: (CP n=25, age 5-22 years, 2 experienced raters)
Total scores - excellent reliability overall (ICC=0.96) and for the subscales of ‘Dystonia’ (ICC=0.91) and ‘Choreoathetosis’ (ICC=0.98).
Regional items - poor to excellent reliability for ‘Dystonia’ (ICC=0.46-1.00) and ‘Choreoathetosis’ (ICC=0.48-0.96) items.
Measurement error - low for the Total DIS (SEM=3%) and both the ‘Dystonia’ (SEM=5%) and ‘Choreoathetosis’ (SEM=3%) subscales.
Minimal detectable difference - moderate for the Total DIS (MDD=9%) and ‘Choreoathetosis’ subscale (MDD=7%); and large for the ‘Dystonia’ subscale (MDD=15%)

Moderate: two good studies

n/a

Monbaliu (2013)*

Video-based assessment: (CP n=25, age 5-22 years, 5 inexperienced raters - 3 senior and 2 junior PTs)
Junior PTs
Total scores - excellent reliability for the Total DIS (ICC=0.88) and both subscales (Dystonia: ICC=0.82, Choreoathetosis: ICC=0.88).
Measurement error – moderate for the Total DIS (SEM=5.5%) and both subscales (Dystonia SEM=6.9%; Choreoathetosis SEM=6.4%).
Minimal detectable difference - large for the Total DIS (MDD=15.3%) and both subscales (Dystonia MDD=18.4%; Choreoathetosis MDD=17.6%).
Senior PTs
Total scores - excellent reliability for the Total DIS (ICC=0.88) and the ‘Choreoathetosis’ subscale (ICC=0.91), but only adequate reliability for the ‘Dystonia’ subscale (ICC=0.71).
Measurement error - low for the Total DIS (SEM=4.4%) and ‘Choreoathetosis’ subscale (SEM=5%) and moderate for the ‘Dystonia’ subscale (SEM=7.7%);
Minimal detectable difference - large for the Total DIS (SEM=12.1%) and both subscales (Dystonia MDD=21.3%; Choreoathetosis SEM=13.9%)

n/a

Internal consistency

Monbaliu (2012)*

Video-based assessment: (CP n=25, age 5-22 years, 2 experienced raters).
Internal consistency was excellent for the ‘Dystonia’ subscale (Cronbach α=0.90-0.93). ; and excellent for the ‘Choreoathetosis’ subscale (α=0.89 0.94)

Moderate: one good study

n/a

Monbaliu (2013)*

Video-based assessment: (CP n=25, age 5-22 years, 5 inexperienced raters). Excellent internal consistency for items across the total DIS and within Dystonia and Choreoathetosis subsections for Junior PTs (α=0.87-0.95) and Senior PTs (α=0.76 -0.93)

n/a

Responsiveness

-

Haberfehlner (2020)

Dystonia subscale

In the ITB-treated group, the effect size for the DIS dystonia subscale was 0.22 (small favourable effect), while in the placebo-treated group the effect size was +0.34 (small unfavourable effect).

The effect size of the BADS in the ITB-treated group was comparable to the effect size for the DIS dystonia subscale. However, for the placebo-treated group the effect size on the DIS dystonia subscale showed a small unfavourable effect, which was not measured on the BADS. The correlation of change scores between the DIS dystonia subscale and BADS was 0.64 (95% confidence interval (CI) 0.37–0.80; p<0.001)

 

Choreoathetosis subscale

The effect size in the ITB-treated group for the total DIS choreoathetosis subscale was 0.25 (small favourable effect). In the placebo-treated group, the effect size was +0.10 (no effect)

 

Both subscales showed no floor or ceiling effects.

n/a

 

 

 

Methodological quality: doubtful

 

Comparison with BADS:

insufficient responsiveness

 

Comparison between ITB group and placebo group:

sufficient responsiveness

 

Before and after the intervention:

insufficient responsiveness

 

* included in the systematic review of Stewart (2017)

 

Scales are presented in order of first publication date.

Results are presented only for studies where CP-specific data are available.

 

Abbreviations

BFMDRS, Burke–Fahn–Marsden Dystonia Rating Scale; BADS, Barry–Albright Dystonia Scale; 95% CI, 95% confidence interval; COPM, Canadian Occupational Performance Measure; DIS, Dyskinesia Impairment Scale; UDRS, Unified Dystonia Rating Scale; MD-CRS, Movement Disorder-Childhood Rating Scale; MD-CRS 0–3, Movement Disorder-Childhood Rating Scale 0–3 Years; GMFCS, Gross Motor Function Classification System; MACS, Manual Ability Classification System; CFCS, Communication Function Classification System.

 

Table 5.1: Risk of bias table for the original study by Haberfehlner (2020) – Dyskinesia Impairment Scale - Criterion validity

Criterion validity (concurrent)

Author: Haberfehlner (2020)

Instrument: Dyskinesia Impairment Scale

 

very good

adequate

doubtful

inadequate

NA

Statistical methods

 

 

 

 

 

For continuous outcomes: were correlations, or the area under the receiver operating curve calculated?

Correlations or AUC calculated

 

 

Correlations or AUC NOT calculated

Not applicable

For dichotomous outcomes: were sensitivity and specificity determined?

Sensitivity and specificity calculated

 

 

Sensitivity and specificity NOT calculated

Not applicable

Other

 

 

 

 

 

Were there any other important flaws in the design or statistical methods of the study?

No other important methodological flaws

 

Other minor methodological flaws

Other important methodological flaws

 

 

Table 5.2: Risk of bias table for the original study by Haberfehlner (2020) – Dyskinesia Impairment Scale – responsiveness

Responsiveness - construct approach (i.e. hypothesis testing: comparison with other outcome measurement instruments [BADS])

Author: Haberfehlner (2020)

Instrument: Dyskinesia Impairment Scale

 

very good

adequate

doubtful

inadequate

NA

Design requirements

 

 

 

 

 

Is it clear what the comparator instrument(s) measure(s)?

Constructs measured by the comparator instrument(s) is clear

 

 

Constructs measured by the comparator instrument(s) is not clear

 

Were the measurement properties of the comparator instrument(s) sufficient?

Sufficient measurement properties of the comparator instrument(s) in a population similar to the study population

Sufficient measurement properties of the comparator instrument(s) but not sure if these apply to the study population

Some information on measurement properties of the comparator instrument(s) in any study population

NO information on the measurement properties of the comparator instrument(s) OR evidence of poor quality of comparator instrument(s)

 

Statistical methods

 

 

 

 

 

Was the statistical method appropriate for the hypotheses to be tested?

Statistical method was appropriate

Assumable that statistical method was appropriate

Statistical method applied NOT optimal

Statistical method applied NOT appropriate

 

Other

 

 

 

 

 

Were there any other important flaws in the design or statistical methods of the study?

No other important methodological flaws

 

Other minor methodological flaws

Other important methodological flaws

 

 

Table 5.3: Risk of bias table for the original study by Haberfehlner (2020) –Dyskinesia Impairment Scale – responsiveness

Responsiveness - construct approach (i.e. hypotheses testing: comparison between subgroups [ITB group and placebo group])

Author: Haberfehlner (2020)

Instrument: Dyskinesia Impairment Scale

 

very good

adequate

doubtful

inadequate

NA

Design requirements

 

 

 

 

 

Was an adequate description provided of important characteristics of the subgroups?

Adequate description of the important characteristics of the subgroups

Adequate description of most of the important characteristics of the subgroups

Poor or no description of the important characteristics of the subgroups

 

 

Statistical methods

 

 

 

 

 

Was the statistical method appropriate for the hypotheses to be tested?

Statistical method was appropriate

Assumable that statistical method was appropriate

Statistical method applied NOT optimal

Statistical method applied NOT appropriate

 

Other

 

 

 

 

 

Were there any other important flaws in the design or statistical methods of the study?

No other important methodological flaws

 

Other minor methodological flaws

Other important methodological flaws

 

 

Table 5.4: Risk of bias table for the original study by Haberfehlner (2020) – Dyskinesia Impairment Scale – responsiveness

Responsiveness - construct approach [i.e. hypotheses testing: before and after the intervention]

Author: Haberfehlner (2020)

Instrument: Dyskinesia Impairment Scale

 

very good

adequate

doubtful

inadequate

NA

Design requirements

 

 

 

 

 

Was an adequate description provided of the intervention given?

Adequate description of the intervention

 

Poor description of the intervention

NO description of the intervention

 

Statistical methods

 

 

 

 

 

Was the statistical method appropriate for the hypotheses to be tested?

Statistical method was appropriate

Assumable that statistical method was appropriate

Statistical method applied NOT optimal

Statistical method applied NOT appropriate

 

Other

 

 

 

 

 

Were there any other important flaws in the design or statistical methods of the study?

No other important methodological flaws

 

Other minor methodological flaws

Other important methodological flaws

 

 

Table 5.5: Risk of bias table for the original study by Vanmechelen (2020) – Dyskinesia Impairment Scale - reliability

Reliability

Author: Vanmechelen (2020)

Instrument: Dyskinesia Impairment Scale

 

very good

adequate

doubtful

inadequate

NA

Design requirements

 

 

 

 

 

Were patients stable in the interim period on the construct to be measured?

Evidence provided that patients were stable

Assumable that patients were stable

Unclear if patients were stable

Patients were NOT stable

 

Was the time interval appropriate?

Time interval appropriate

 

Doubtful whether time interval was appropriate or time interval was not stated

Time interval NOT appropriate

 

Were the test conditions similar for the measurements? E.g. type of administration, environment, instructions

Test conditions were similar (evidence provided)

Assumable that test conditions were similar

Unclear if test conditions were similar

Test conditions were NOT similar

 

Statistical methods

 

 

 

 

 

For continuous scores: was an intraclass correlation coefficient (ICC) calculated?

ICC calculated and model or formula of the ICC is described

ICC calculated but model or formula of the ICC not described or not optimal. Pearson or Spearman correlation coefficient calculated with evidence provided that no systematic change has occurred.

Pearson or Spearman correlation coefficient calculated WITHOUT evidence provided that no systematic change has occurred or WITH evidence that systematic change has occurred

No ICC or Pearson or Spearman correlations calculated

Not applicable

For dichotomous/nominal/ordinal scores: was kappa calculated?

Kappa calculated

 

 

No kappa calculated

Not applicable

For ordinal scores: was a weighted kappa calculated?

Weighted kappa calculated

 

Unweighted Kappa calculated or not described

 

Not applicable

For ordinal scores: was the weighting scheme described? E.g. linear, quadratic

Weighting scheme described

Weighting scheme NOT described

 

 

Not applicable

Other

 

 

 

 

 

Were there any other important flaws in the design or statistical methods of the study?

No other important methodological flaws

 

Other minor methodological flaws

 

Other important methodological flaws

 

 

Table 5.6: Risk of bias table for the original study by Claassen (2023) –Movement Disorder-Childhood Rating Scale – content validity

Content validity

Author: Claassen (2023)

Instrument: Movement Disorder-Childhood Rating Scale

 

very good

adequate

doubtful

inadequate

NA

Asking patients about relevance

 

 

 

 

 

Design requirements

 

 

 

 

 

Was an appropriate method used to ask patients whether each item is relevant for their experience with the condition?

Widely recognized or well justified method used

Only quantitative (survey) method(s) used or assumable that the method was appropriate but not clearly described

Not clear if patients were asked whether each item is relevant or doubtful whether the method was appropriate

Methods used not appropriate or patients not asked about the relevance of all items

 

Was each item tested in an appropriate number of patients?

    For qualitative studies

    For quantitative (survey)

    studies

 

 

 

≥ 7

≥ 50

 

 

 

4-6

≥30

 

 

 

<4 or not clear

<30 or not clear

 

 

Were skilled group moderators/interviewers used?

Skilled group moderators/interviewers used

Group moderators/interviewers had limited experience or were trained specifically for the study

Not clear if group moderators/interviewers were trained or group moderators/interviewers not trained and no experience

 

Not applicable

Were the group meetings or interviews based on an appropriate topic or interview guide?

Appropriate topic or interview guide

Assumable that the topic or interview guide was appropriate, but not clearly described

Not clear if a topic guide was used or doubtful if topic or interview guide was appropriate or no guide

 

Not applicable

Were the group meetings or interviews recorded and transcribed verbatim?

All group meetings or interviews were recorded and transcribed verbatim

Assumable that all group meetings or interviews were recorded and transcribed verbatim, but not clearly described

Not clear if all group meetings or interviews were recorded and transcribed verbatim or recordings not transcribed verbatim or only notes were made during the group meetings/interviews

No recordings and no notes

Not applicable

Analyses

 

 

 

 

 

Was an appropriate approach used to analyse the data?

A widely recognized or well justified approach was used

Assumable that the approach was appropriate, but not clearly described

Not clear what approach was used or doubtful whether the approach was appropriate

Approach not appropriate

 

Were at least two researchers involved in the analysis?

At least two researchers involved in the analysis

Assumable that at least two researchers were involved in the analysis, but not clearly described

Not clear if two researchers were included in the analysis or only one researcher involved in the analysis

 

 

 

Table 6 Definitions of quality levels (Mokkink, 2018)

Quality level

Definition

High

We are very confident that the true measurement property lies close to that of the estimate* of the measurement property

Moderate

We are moderately confident in the measurement property estimate: the true measurement property is likely to be close to the estimate of the measurement property, but there is a possibility that it is substantially different

Low

Our confidence in the measurement property estimate is limited: the true measurement property may be substantially different from the estimate of the measurement property

Very low

We have very little confidence in the measurement property estimate: the true measurement property is likely to be substantially different from the estimate of the measurement property

* Estimate of the measurement property refers to the pooled or summarized result of the measurement property of a PROM

The COSMIN working group adapted these definitions from the GRADE approach.

 

Table 7: Description of selected instruments including references to the original papers (overview adapted from Stewart [2017])

Instrument and reference to original paper

Intended population (age range)

Body parts

Scale

Administration/clinical utility

Burke–Fahn–Marsden Dystonia Rating Scale (BFMDRS)

 

Burke (1985)

Primary dystonia (adults, age not specified)

 

n=9: eyes, mouth, speech/ swallow, neck, trunk, four limbs

Score: each body part; scores halved for mouth, eyes, neck

 

Movement scale (120 points)

For each body part: severity rating, provoking factor(s), severity x provoking factor

 

Disability scale (30 points)

Rates the patient’s view of the impact of dystonia on activities of daily including: speech, handwriting, feeding, eating/swallowing, hygiene, dressing, and walking.

 

Manual: nil

Administration instructions: adequatea

Administration time: NS (video recording time for items 4min 15s)

Scoring mode: face-to-face or video (protocol in journal)

Scoring criteria: in journal

Scoring time: NS

Training requirements: NS

Interpretability: higher score means greater severity

 

Barry–Albright Dystonia Scale (BADS)

 

Barry (1999)

Secondary dystonia
CP and ABI
(children and
adults)

 

n=8: eyes, mouth, neck, trunk, four limbs

Score: each body part

Total BADS (32 points)

 

Manual: nil

Administration instructions: adequate

Administration time: NS

Scoring mode: face-to-face or video (protocol in journal)

Scoring criteria: in journal

Scoring time: NS

Training requirements: training improves reliability, details not provided

Interpretability: higher score means greater severity

 

Unified Dystonia Rating Scale (UDRS)

 

Comella (2003)

Primary dystonia
(adults 18–65y)

 

n=14: eyes/
upper face,
lower face,
jaw/tongue, larynx,
neck, trunk,
four limbs,
proximal, and distal

 

Score: each body part

Total UDRS (112 points)

Duration scale (56 points)

Severity scale (56 points)

 

Manual: nil

Administration instructions: adequate

Administration time: NS (video

recording time for items about 5min)

Scoring mode: recommended video (protocol in journal)

Scoring criteria: in journal

Scoring time: NS

Training requirements: NS; experience improves reliability

Interpretability: higher score means greater severity

 

Movement Disorder-Childhood Rating Scale (MD-CRS)

 

Battini (2008)

CP and other
disorders Hypokinetis–rigid syndrome; chorea/ ballism; dystonia/ athetosis; myoclonus; tic; tremor (children 4– 18y)

 

n=7: eye and
periorbital
region, face, tongue and perioral region, neck, trunk, four limbs

 

 

Score: global index calculated (0–1): 0–0.2, healthy;

0.2–0.4, mildly affected;

0.4–0.6, moderately affected;

0.6–0.8, severely affected;

0.8–1, profoundly affected

 

 

Manual: nil

Administration instructions: adequate

Administration time: NS (video recording time for items about 20min)

Scoring mode: recommended video (protocol in journal)

Scoring criteria: in journal, statistical analysis required

Scoring time: NS

Training requirements: NS

Interpretability: higher score means greater severity

 

Movement Disorder-Childhood Rating Scale 0-3 years (MD-CRS 0-3)

 

Battini (2009)

CP and other
disorders Hypokinetis–rigid
syndrome; chorea/
ballism; dystonia/
athetosis; myoclonus; tic;
tremor (children 0–
3y)

 

n=7: eye and periorbital
region, face, tongue and perioral region,
neck, trunk,
four limbs

 

Score: global index calculated (0–1): 0–0.2, healthy;

0.2–0.4, mildly affected;

0.4–0.6, moderately affected;

0.6–0.8, severely affected;

0.8–1, profoundly affected

 

Manual: nil

Administration instructions: adequate Administration time: NS

Scoring mode: recommended video (protocol in journal)

Scoring criteria: in journal, statistical analysis required

Scoring time: NS

Training requirements: NS

Interpretability: higher score means greater severity

 

Dyskinesia Impairment Scale (DIS)

 

Monbaliu (2012)

Dyskinetic CP
Dystonia and choreoathetosis
subscales (children
and adults 5–22y)

 

n=12: eyes,
mouth,
neck, trunk,
four limbs, proximal,
and distal

 

Score: each body part

Total DIS (288 points)

On action (192 points)

At rest (96 points)

Dystonia Choreoathetosis

 

Manual: nil

Administration instructions: adequate Administration time: video recording

time maximum 30min; ROM also measurement required

Scoring mode: recommended video (protocol in journal)

Scoring criteria: in journal

Scoring time: 30–45min per subscale Training requirements: clinical

experience with dyskinesia

Interpretability: higher score means greater severity

 

aScale instructions were rated using the CanChild Outcome Measures Rating Form with the criteria good, adequate, or inadequate.

 

Abbreviations

NS, not stated; ABI, acquired brain injury; ROM, range of movement.

 

Tabel 8: Overzicht van de validiteit, betrouwbaarheid en responsiviteit van de zes meetinstrumenten voor dyskinesie

 

Validiteit

Betrouwbaarheid

Responsiviteit

Construct-validiteit

Inhouds-validiteit

Criterium-validiteit (concurrent)

Criterium-validiteit (predictief)

Intra-rater

Test-hertest

Inter-rater

Interne consistentie

Burke–Fahn–Marsden Dystonia Rating Scale (BFMDRS)

Geen informatie

 

 

 

Redelijk bewijs (+)

 

Bewijs alleen gebaseerd op expert opinion, meerdere kritische kanttekeningen (?)

 

Redelijk bewijs

Beperkt bewijs

Geen informatie

 

 

Geen informatie

Redelijk bewijs

Redelijk bewijs (+)

 

Goede tot uitstekende interne consistentie (+)

Beperkt bewijs

 

Barry–Albright Dystonia Scale (BADS)

Redelijk bewijs

Beperkt bewijs (+)

 

Bewijs alleen gebaseerd op expert opinion, instrument sluit niet goed aan bij definitie dystonie, alleen gericht op dystonie (?)

 

Redelijk bewijs

Beperkt bewijs

Geen informatie

Geen informatie

Redelijk bewijs

Redelijk bewijs (+)

 

Goede tot uitstekende interne consistentie (+)

Beperkt bewijs

 

Unified Dystonia Rating Scale (UDRS)

Geen informatie

Redelijk bewijs (+)

 

Bewijs alleen gebaseerd op expert opinion, instrument sluit niet goed aan bij definitie dystonie, alleen gericht op dystonie (?)

 

Redelijk bewijs

Geen informatie

Geen informatie

Geen informatie

Redelijk bewijs

Redelijk bewijs (+)

 

Uitstekende interne consistentie (+)

Beperkt bewijs

Movement Disorder-Childhood Rating Scale (MD-CRS)

Geen informatie

Redelijk bewijs (+)

 

Inhoudsvaliditeit lijkt voldoende op basis van interviews met kinderen/adolescenten en hun verzorgers (+)

Geen informatie

Geen informatie

Geen informatie

Geen informatie

Geen informatie

Geen informatie (-)

Beperkt bewijs

Movement Disorder-Childhood Rating Scale 0-3 years (MD-CRS 0-3)

Geen informatie

Geen informatie (-)

Geen informatie

Geen informatie

Geen informatie

Geen informatie

Geen informatie

Geen informatie (-)

Beperkt bewijs

Dyskinesia Impairment Scale (DIS)

Geen informatie

Beperkt bewijs (+)

 

Bij ontwikkeling van de schaal zijn negen experts betrokken geweest om de inhoudsvaliditeit te waarborgen (?)

Redelijk bewijs

Beperkt bewijs

Geen informatie

Beperkt bewijs

Redelijk bewijs

Redelijk bewijs (+)

 

Adequate tot goede of uitstekende interne consistentie (+)

Beperkt bewijs

+: voldoet aan criteria voor categorie A

- : voldoet niet aan criteria voor categorie A

? : twijfelachtig of de resultaten voldoen aan de criteria voor categorie A

 

Table of excluded studies

Reference

Reason for exclusion

Pavone L, Burton J, Gaebler-Spira D. Dystonia in childhood: clinical and objective measures and functional implications. J Child Neurol. 2013 Mar;28(3):340-50. doi: 10.1177/0883073812444312. Epub 2012 Jun 29. PMID: 22752485.

Wrong publication type: narrative review (older than the included systematic review by Stewart (2017))

Vanmechelen I, Danielsson A, Lidbeck C, Tedroff K, Monbaliu E, Krumlinde-Sundholm L. The Dyskinesia Impairment Scale, Second Edition: Development, construct validity, and reliability. Dev Med Child Neurol. 2023 May;65(5):683-690. doi: 10.1111/dmcn.15444. Epub 2022 Oct 31. PMID: 36310446.

Wrong index test: second version of the Dyskinesia Impairment scale, more research needed before this scale can be used in clinical practice

Kuiper MJ, Meiners LC, Chandler ES, Brandsma R, Bos AF, Horst HT, Sival DA; Neonatal neurologic Assessment Group. Dyskinesia Impairment Scale scores in Dutch pre-school children after neonatal therapeutic hypothermia. Eur J Paediatr Neurol. 2020 Sep;28:70-76. doi: 10.1016/j.ejpn.2020.07.013. Epub 2020 Aug 5. PMID: 32950367.

Wrong population: children at risk of developing dyskinetic CP

Rice J, Skuza P, Baker F, Russo R, Fehlings D. Identification and measurement of dystonia in cerebral palsy. Dev Med Child Neurol. 2017 Dec;59(12):1249-1255. doi: 10.1111/dmcn.13502. Epub 2017 Aug 8. PMID: 28786476.

Wrong population: children with CP but not all children have dystonia

Danielsson A, Vanmechelen I, Lidbeck C, Krumlinde-Sundholm L, Ortibus E, Monbaliu E, Tedroff K. Reliability and Validity of the Dyskinesia Impairment Scale in Children and Young Adults with Inherited or Idiopathic Dystonia. J Clin Med. 2020 Aug 11;9(8):2597. doi: 10.3390/jcm9082597. PMID: 32796550; PMCID: PMC7463647.

Wrong population: children and youth with inherited or idiopathic dystonia

Stewart K, Lewis J, Wallen M, Bear N, Harvey A. The Dyskinetic Cerebral Palsy Functional Impact Scale: development and validation of a new tool. Dev Med Child Neurol. 2021 Dec;63(12):1469-1475. doi: 10.1111/dmcn.14960. Epub 2021 Jun 19. PMID: 34145577.

Wrong index test: Dyskinetic Cerebral Palsy Functional Impact Scale

Battini R, Guzzetta A, Sgandurra G, Di Pietro R, Petacchi E, Mercuri E, Giannini MT, Leuzzi V, Cioni G. Scale for evaluation of movement disorders in the first three years of life. Pediatr Neurol. 2009 Apr;40(4):258-64. doi: 10.1016/j.pediatrneurol.2008.11.003. PMID: 19302937.

Included in the systematic review by Stewart (2017)

Battini R, Casarano M, Sgandurra G, Olivieri I, Di Pietro R, Romeo DM, Mercuri E, Cioni G. Responsiveness of the MD-childhood rating scale in dyskinetic cerebral palsy patients undergoing anticholinergic treatment. Eur J Paediatr Neurol. 2014 Nov;18(6):698-703. doi: 10.1016/j.ejpn.2014.06.004. Epub 2014 Jun 19. PMID: 25022341.

Included in the systematic review by Stewart (2017)

Battini R, Olivieri I, Di Pietro R, Casarano M, Sgandurra G, Romeo DM, Cioni G. Movement Disorder-Childhood Rating Scale: A Sensitive Tool to Evaluate Movement Disorders. Pediatr Neurol. 2015 Jul;53(1):73-7. doi: 10.1016/j.pediatrneurol.2015.02.014. Epub 2015 Mar 27. PMID: 26092416.

Included in the systematic review by Stewart (2017)

Elze MC, Gimeno H, Tustin K, Baker L, Lumsden DE, Hutton JL, Lin JP. Burke-Fahn-Marsden dystonia severity, Gross Motor, Manual Ability, and Communication Function Classification scales in childhood hyperkinetic movement disorders including cerebral palsy: a 'Rosetta Stone' study. Dev Med Child Neurol. 2016 Feb;58(2):145-53. doi: 10.1111/dmcn.12965. Epub 2015 Nov 30. PMID: 26616635.

Included in the systematic review by Stewart (2017)

Kawamura A, Klejman S, Fehlings D. Reliability and validity of the kinematic dystonia measure for children with upper extremity dystonia. J Child Neurol. 2012 Jul;27(7):907-13. doi: 10.1177/0883073812443086. Epub 2012 Apr 25. PMID: 22535705.

Included in the systematic review by Stewart (2017)

Monbaliu E, Ortibus E, Roelens F, Desloovere K, Deklerck J, Prinzie P, de Cock P, Feys H. Rating scales for dystonia in cerebral palsy: reliability and validity. Dev Med Child Neurol. 2010 Jun;52(6):570-5. doi: 10.1111/j.1469-8749.2009.03581.x. Epub 2010 Jan 28. PMID: 20132143.

Included in the systematic review by Stewart (2017)

Monbaliu E, Ortibus E, De Cat J, Dan B, Heyrman L, Prinzie P, De Cock P, Feys H. The Dyskinesia Impairment Scale: a new instrument to measure dystonia and choreoathetosis in dyskinetic cerebral palsy. Dev Med Child Neurol. 2012 Mar;54(3):278-83. doi: 10.1111/j.1469-8749.2011.04209.x. PMID: 22428172.

Included in the systematic review by Stewart (2017)

Monbaliu E, Ortibus E, Prinzie P, Dan B, De Cat J, De Cock P, Feys H. Can the Dyskinesia Impairment Scale be used by inexperienced raters? A reliability study. Eur J Paediatr Neurol. 2013 May;17(3):238-47. doi: 10.1016/j.ejpn.2012.10.004. Epub 2012 Nov 22. PMID: 23177615.

Included in the systematic review by Stewart (2017)

Autorisatiedatum en geldigheid

Laatst beoordeeld  : 09-08-2024

Laatst geautoriseerd  : 09-08-2024

Geplande herbeoordeling  : 01-05-2025

Autorisatie Nederlandse Vereniging van Revalidatieartsen onder voorbehoud van goedkeuring door de ALV.

Initiatief en autorisatie

Initiatief:
  • Nederlandse Vereniging van Revalidatieartsen
Geautoriseerd door:
  • Koninklijk Nederlands Genootschap voor Fysiotherapie
  • Nederlandse Orthopaedische Vereniging
  • Nederlandse Vereniging van Revalidatieartsen
  • Nederlandse Vereniging voor Neurochirurgie
  • Nederlandse Vereniging voor Neurologie
  • Nederlandse Vereniging voor Plastische Chirurgie
  • Ergotherapie Nederland
  • Nederlandse Vereniging voor Kinderfysiotherapie
  • CP-Net
  • CP Nederland

Algemene gegevens

De ontwikkeling/herziening van deze richtlijnmodule werd ondersteund door het Kennisinstituut van de Federatie Medisch Specialisten (www.demedischspecialist.nl/kennisinstituut) en werd gefinancierd uit de Kwaliteitsgelden Medisch Specialisten (SKMS). Patiëntenparticipatie bij deze richtlijn werd medegefinancierd uit de Kwaliteitsgelden Patiënten Consumenten (SKPC) binnen het programma KIDZ.

De financier heeft geen enkele invloed gehad op de inhoud van de richtlijnmodule.

Samenstelling werkgroep

Voor het ontwikkelen van de richtlijnmodule is in 2022 een multidisciplinaire werkgroep ingesteld, bestaande uit vertegenwoordigers en ervaringsdeskundigen van alle relevante specialismen (zie hiervoor de Samenstelling van de werkgroep) die betrokken zijn bij de zorg voor kinderen met cerebrale parese.

 

Werkgroep

  • Prof. dr. A.I. Buizer, (kinder)revalidatiearts, VRA
  • Dr. M.W. Alsem, (kinder)revalidatiearts, VRA
  • Dr. M.J. Nederhand, (kinder)revalidatiearts , VRA
  • Drs. R.A. van Stralen, orthopedisch chirurg, NOV
  • Prof. dr. R.J. Vermeulen, (kinder)neuroloog, NVN
  • Dr. K.M. Slot, (kinder)neurochirurg, NVvN
  • Dr. M.C. Obdeijn, plastisch chirurg, NVPC
  • Dr. E.A.A. Rameckers, onderzoeker en kinderfysiotherapeut, KNGF/NVFK
  • Dr. P.B.M. Aarts, onderzoeker en ergotherapeut, EN
  • Dr. M. Ketelaar, senior onderzoeker, persoonlijke titel
  • Drs. M.G. van Driel-Boerrigter, voorzitter, CP Nederland
  • Ing. E.P.E. Beije, bestuurslid en penningmeester, CP Nederland

Klankbordgroep

  • Dr. C.J.I. Raats, CP-Net
  • Dr. C.A. van Nieuwenhoven, plastisch chirurg
  • Dr. J. Verhof, plastisch chirurg en handchirurg
  • Drs. T. Tempelman, plastisch chirurg

Met ondersteuning van

  • Dr. M. den Ouden – Vierwind, adviseur, Kennisinstituut van de Federatie Medisch Specialisten
  • Drs. F. Ham, adviseur, Kennisinstituut van de Federatie Medisch Specialisten
  • Drs. L. van Wijngaarden, junior adviseur, Kennisinstituut van de Federatie Medisch Specialisten
  • Dr. L. Oostendorp, adviseur, Kennisinstituut van de Federatie Medisch Specialisten

Belangenverklaringen

De Code ter voorkoming van oneigenlijke beïnvloeding door belangenverstrengeling is gevolgd. Alle werkgroepleden hebben schriftelijk verklaard of zij in de laatste drie jaar directe financiële belangen (betrekking bij een commercieel bedrijf, persoonlijke financiële belangen, onderzoeksfinanciering) of indirecte belangen (persoonlijke relaties, reputatiemanagement) hebben gehad. Gedurende de ontwikkeling of herziening van een module worden wijzigingen in belangen aan de voorzitter doorgegeven. De belangenverklaring wordt opnieuw bevestigd tijdens de commentaarfase.

Een overzicht van de belangen van werkgroepleden en het oordeel over het omgaan met eventuele belangen vindt u in onderstaande tabel. De ondertekende belangenverklaringen zijn op te vragen bij het secretariaat van het Kennisinstituut van de Federatie Medisch Specialisten.

 

Lid

Functie

Nevenfuncties

Gemelde belangen

Ondernomen actie

Werkgroep

Prof. dr. A.I. Buizer

 

(Kinder)revalidatiearts
Amsterdam UMC

Voorzitter bestuur Dutch Academy of Childhood Disability (DACD)

* MegaMuscle: observationeel onderzoek naar effecten van interventies op spiereigenschappen bij cerebrale parese. Deels gefinancierd door onderzoeksinstituut Amsterdam Movement Sciences, deels door liefdadigheids-fondsen: Johanna Kinderfonds, en Phelps Stichting voo Spasticy (niet-commercieel). Betrokken als Projectleider.

* Co-auteur van diverse wetenschappelijke artikelen over cerebrale parese, die mogelijk in de richtlijn zullen worden opgenomen.

* Financiering aangevraagd bij BeNeFIT voor een studie naar SDR vs ITB bij niet-ambulante kinderen met CP (nog niet toegekend).

* Betrokkenheid Power2Walk studie.

Geen restricties.

Dr. M.W. Alsem

 

(Kinder)revalidaitearts UMC Utrecht

* Co-editor tijdschrift Child: Care, Health and Development (betaald)
* Lid onderzoeksconsortium TCU: Bestendig op weg naar (t)huis (onbetaald)

* ZonMW: Bestendig op weg naar (t)huis

* Betrokken geweest bij ontwikkeling Kwaliteitsstandaard Psychosociale zorg in de kinderrevalidatie

Geen restricties.

Dr. M.J. Nederhand

 

* (Kinder)revalidatiearts 0.8: Roessingh, Centrum voor Revalidatie
* Senior onderzoeker 0.1: Roessingh. Research and Development

Geen.

Geen.

Geen restricties.

Drs. R.A. van Stralen

 

Orthopedisch chirurg in het ErasmusMC.

Geen.

* Financiering toegekend van de for Wishdom foundation (restricted grant) voor 2 studies, getiteld 'Guided growth van het proximale femur bij kinderen met CP'. Betrokken als projectleider samen met Jaap Tolk.

* Toename van eigen expertise op (deel)gebied waar het advies/richtlijn zich op richt.

* Vernieuwde aanpak van eigen organisatie.

* Boegbeeldfunctie bij een patiënten- of beroepsorganisatie.

Geen restricties.

Prof. dr. R.J. Vermeulen

 

* (Kinder)neuroloog bij Academisch ziekenhuis Maastricht.
* Hoogleraar kinderneurologie, Universiteit Maastricht.

Geen.

* Revalidatie NL - Iunilaterale cerebrale parese, functionele electrische stimulatie, verbeteren van lopen. Betrokken als projectleider..

* Stchting Vooruit -Behandeling van dystonie bij kinderen. Betrokken als projectleider.

* Stichting Janivo - Behandeling van dystonie bij kinderen. Geen projectleider.

* Lid van de "general management committee" van de European academy of child hood disability (EACD). Wij hebben richtlijn ontwikkeling maar classificatie van visuele stoornissen gesponsort.  * Aanwezig bij gebruikersdag voor professionals die werken met baclofen pompen (2-12-2022), gefinancierd door NVN (zonder invloed van MEdtronic op de presentaties), vergoeding voor tijd aan het ziekenhuis.

* Betrokkenheid Power2Walk studie.

Geen restricties.

Dr. K.M. Slot

 

(Kinder)neurochirurg Amsterdam UMC.

Geen.

* Financiering aangevraagd bij BeNeFIT voor een studie naar SDR vs ITB bij niet-ambulante kinderen met CP (nog niet toegekend).

Geen restricties.

Dr. M.C. Obdeijn

 

Plastisch chirurg in het Amsterdam UMC.

* Opleider

* Lid van het Concillium Plastico Chirurgicum (onbetaald).

*Lid van Raad Opleidingen (onbetaald).

Door deel te nemen aan de richtlijn commissie kan mijn reputatie en bekendheid als ervaren CP chirurg toenemen.

Geen restricties.

Dr. E.A.A. Rameckers

 

Senior onderzoeker en kinderfysiotherapeut Adelante revalidatie, Hasselt universiteit 25%.

* Werkzaam Universiteit Maastricht (betaald).

*  Senior onderzoeker Adelante kenniscentrum (betaald).

* Projectleider bij veel kinderrevalidatieprojecten:

Wij wheelen mee, Power2walk, klaar om te eten, Promis studie.

* Ik ben co-auteur van diverse wetenschappelijke artikelen over cerebrale parese, die mogelijk in de richtlijn zullen worden opgenomen.

Geen restricties.

Dr. P.B.M. Aarts

 

 (Pre-pensioen) als hoofd unit Kinderrevalidatie van de Sint Maartenskliniek.

* Voorzitter bestuur stichting CP net (onbetaald).

* Senior onderzoeker bij 3 nog lopende promotieonderzoeken (onbetaald).

*Scholing (betaald vanuit VOF EDUTIVEAA).

Co-auteur van diverse wetenschappelijke artikelen over arm-hand diagnostiek en behandeling bij kinderen met cerebrale parese, waarvan er mogelijk iets in de richtlijn genoemd wordt.

Geen restricties.

Dr. M. Ketelaar

 

Senior onderzoeker
Kenniscentrum Revalidatiegeneeskunde, UMC Utrecht en De Hoogstraat Revalidatie.

Bestuurslid CP-Net (onbetaald).

* Projectleider diverse studies bij kinderen en jongeren met CP.

* Co-auteur van diverse wetenschappelijke artikelen over CP, die mogelijk in de richtlijn zullen worden opgenomen (gefinancierd door ‘neutrale’ subsidiegevers, zoals ZonMw).

* Lid van Committee Education and Training van de European Academy of Childhood Disability (EACD).

Geen restricties.

Drs. M.G. van Driel-Boerrigter

* Voorzitter van CP Nederland (onbezoldigd)
* Bestuurslid CP-Net (onbezoldigd)

Geen.

Geen.

Geen restricties.

Ing. E.P.E. Beije

 

–Penningmeester bij CP-Nederland (onbetaald).

Geen.

* Zoon 10 jaar heeft CP.

 

Geen restricties.

Klankbordgroep

Dr. C.J.I. Raats

Projectcoördinator, Stichting CP-Net.

Zelfstandig adviseur/ trainer/ projectleider (ZZP) op het gebied van kwaliteit van zorg en patiëntgerichte zorg voor diverse opdrachtgevers, zoals zorginstellingen, patiëntenorganisaties, beroepsorganisaties, brancheorganisaties, kennisinstituten.

Stichting CP-Net houdt zich o.a. bezig met de implementatie van de richtlijn CP.

Geen restricties.

Inbreng patiëntenperspectief

Er werd aandacht besteed aan het patiëntenperspectief door een afgevaardigde van de patiëntenvereniging CP Nederland in de werkgroep uit te nodigen. De verkregen input is meegenomen bij het opstellen van de uitgangsvragen, de keuze voor de uitkomstmaten en bij het opstellen van de overwegingen (zie kop Waarden en voorkeuren van patiënten). De conceptrichtlijn is tevens voor commentaar voorgelegd aan deelnemers van de patiëntenverenigingen en de eventueel aangeleverde commentaren zijn bekeken en verwerkt.

 

Kwalitatieve raming van mogelijke financiële gevolgen in het kader van de Wkkgz

Bij de richtlijnmodule is conform de Wet kwaliteit, klachten en geschillen zorg (Wkkgz) een kwalitatieve raming uitgevoerd om te beoordelen of de aanbevelingen mogelijk leiden tot substantiële financiële gevolgen. Bij het uitvoeren van deze beoordeling is de richtlijnmodule op verschillende domeinen getoetst (zie het stroomschema op de Richtlijnendatabase).

 

Module

Uitkomst raming

Toelichting

Module  Dyskinesie

Geen financiële gevolgen

Uit de toetsing volgt dat de aanbevelingen niet breed toepasbaar zijn (<5.000 patiënten) en daarom naar verwachting geen substantiële financiële gevolgen zullen hebben voor de collectieve uitgaven.

 

Werkwijze

AGREE

Deze richtlijnmodule is opgesteld conform de eisen vermeld in het rapport Medisch Specialistische Richtlijnen 2.0 van de adviescommissie Richtlijnen van de Raad Kwaliteit. Dit rapport is gebaseerd op het AGREE II instrument (Appraisal of Guidelines for Research & Evaluation II; Brouwers, 2010).

 

Knelpuntenanalyse en uitgangsvragen

Tijdens de voorbereidende fase inventariseerde de werkgroep de knelpunten in de zorg voor kinderen met cerebrale parese. De werkgroep beoordeelde de aanbevelingen uit de eerdere richtlijn (VRA, 2018) op noodzaak tot revisie aan de hand van een onderhoudsplan die in 2021 is opgesteld voor deze richtlijn. Tevens zijn er knelpunten aangedragen door CP-Nederland, CP-Net, EN, KNGF/NVFK, NOV, NVD, NVK, NVLF, NVPC, RN, en VRA via een invitational conference. Een verslag hiervan is opgenomen onder aanverwante producten.

 

Op basis van de uitkomsten van de knelpuntenanalyse zijn door de werkgroep concept-uitgangsvragen opgesteld en definitief vastgesteld.

 

Uitkomstmaten

Na het opstellen van de zoekvraag behorende bij de uitgangsvraag inventariseerde de werkgroep welke uitkomstmaten voor de patiënt relevant zijn, waarbij zowel naar gewenste als ongewenste effecten werd gekeken. Hierbij werd een maximum van acht uitkomstmaten gehanteerd. De werkgroep waardeerde deze uitkomstmaten volgens hun relatieve belang bij de besluitvorming rondom aanbevelingen, als cruciaal (kritiek voor de besluitvorming), belangrijk (maar niet cruciaal) en onbelangrijk. Tevens definieerde de werkgroep tenminste voor de cruciale uitkomstmaten welke verschillen zij klinisch (patiënt) relevant vonden.

 

Methode literatuursamenvatting

Een uitgebreide beschrijving van de strategie voor zoeken en selecteren van literatuur is te vinden onder ‘Zoeken en selecteren’ onder Onderbouwing. Indien mogelijk werd de data uit verschillende studies gepoold in een random-effects model. Review Manager 5.4 werd gebruikt voor de statistische analyses. De beoordeling van de kracht van het wetenschappelijke bewijs wordt hieronder toegelicht.

 

Beoordelen van de kracht van het wetenschappelijke bewijs

De kracht van het wetenschappelijke bewijs werd bepaald volgens de GRADE-methode. GRADE staat voor ‘Grading Recommendations Assessment, Development and Evaluation’ (zie http://www.gradeworkinggroup.org/). De basisprincipes van de GRADE-methodiek zijn: het benoemen en prioriteren van de klinisch (patiënt) relevante uitkomstmaten, een systematische review per uitkomstmaat, en een beoordeling van de bewijskracht per uitkomstmaat op basis van de acht GRADE-domeinen (domeinen voor downgraden: risk of bias, inconsistentie, indirectheid, imprecisie, en publicatiebias; domeinen voor upgraden: dosis-effect relatie, groot effect, en residuele plausibele confounding).

GRADE onderscheidt vier gradaties voor de kwaliteit van het wetenschappelijk bewijs: hoog, redelijk, laag en zeer laag. Deze gradaties verwijzen naar de mate van zekerheid die er bestaat over de literatuurconclusie, in het bijzonder de mate van zekerheid dat de literatuurconclusie de aanbeveling adequaat ondersteunt (Schünemann, 2013; Hultcrantz, 2017).

 

Aangezien deze richtlijn een niet veelvoorkomende heterogene aandoening betreft, en tevens behandelingen beschrijft die voor een selecte groep van deze populatie van toepassing is, betreffen de geïncludeerde studies vaak onvoldoende power. Bij de beoordeling van de bewijskracht is er daarom in veel gevallen gedowngraded voor imprecisie (tot GRADE low of very low). Hiermee kunnen literatuurconclusies geen duidelijke richting geven aan de besluitvorming. In het kader van passend bewijs is de werkgroep van mening dat dit GRADE low in veel gevallen de sterkste evidentie is in deze richtlijn.

 

GRADE

Definitie

Hoog

  • er is hoge zekerheid dat het ware effect van behandeling dichtbij het geschatte effect van behandeling ligt;
  • het is zeer onwaarschijnlijk dat de literatuurconclusie klinisch relevant verandert wanneer er resultaten van nieuw grootschalig onderzoek aan de literatuuranalyse worden toegevoegd.

Redelijk

  • er is redelijke zekerheid dat het ware effect van behandeling dichtbij het geschatte effect van behandeling ligt;
  • het is mogelijk dat de conclusie klinisch relevant verandert wanneer er resultaten van nieuw grootschalig onderzoek aan de literatuuranalyse worden toegevoegd.

Laag

  • er is lage zekerheid dat het ware effect van behandeling dichtbij het geschatte effect van behandeling ligt;
  • er is een reële kans dat de conclusie klinisch relevant verandert wanneer er resultaten van nieuw grootschalig onderzoek aan de literatuuranalyse worden toegevoegd.

Zeer laag

  • er is zeer lage zekerheid dat het ware effect van behandeling dichtbij het geschatte effect van behandeling ligt;
  • de literatuurconclusie is zeer onzeker.

 

Bij het beoordelen (graderen) van de kracht van het wetenschappelijk bewijs in richtlijnen volgens de GRADE-methodiek spelen grenzen voor klinische besluitvorming een belangrijke rol (Hultcrantz, 2017). Dit zijn de grenzen die bij overschrijding aanleiding zouden geven tot een aanpassing van de aanbeveling. Om de grenzen voor klinische besluitvorming te bepalen moeten alle relevante uitkomstmaten en overwegingen worden meegewogen. De grenzen voor klinische besluitvorming zijn daarmee niet één op één vergelijkbaar met het minimaal klinisch relevant verschil (Minimal Clinically Important Difference, MCID). Met name in situaties waarin een interventie geen belangrijke nadelen heeft en de kosten relatief laag zijn, kan de grens voor klinische besluitvorming met betrekking tot de effectiviteit van de interventie bij een lagere waarde (dichter bij het nuleffect) liggen dan de MCID (Hultcrantz, 2017).

 

Overwegingen (van bewijs naar aanbeveling)

Om te komen tot een aanbeveling zijn naast (de kwaliteit van) het wetenschappelijke bewijs ook andere aspecten belangrijk en worden meegewogen, zoals aanvullende argumenten uit bijvoorbeeld de biomechanica of fysiologie, waarden en voorkeuren van patiënten, kosten (middelenbeslag), aanvaardbaarheid, haalbaarheid en implementatie. Deze aspecten zijn systematisch vermeld en beoordeeld (gewogen) onder het kopje ‘Overwegingen’ en kunnen (mede) gebaseerd zijn op expert opinion. Hierbij is gebruik gemaakt van een gestructureerd format gebaseerd op het evidence-to-decision framework van de internationale GRADE Working Group (Alonso-Coello, 2016a; Alonso-Coello 2016b). Dit evidence-to-decision framework is een integraal onderdeel van de GRADE methodiek.

 

Formuleren van aanbevelingen

De aanbevelingen geven antwoord op de uitgangsvraag en zijn gebaseerd op het beschikbare wetenschappelijke bewijs en de belangrijkste overwegingen, en een weging van de gunstige en ongunstige effecten van de relevante interventies. De kracht van het wetenschappelijk bewijs en het gewicht dat door de werkgroep wordt toegekend aan de overwegingen, bepalen samen de sterkte van de aanbeveling. Conform de GRADE-methodiek sluit een lage bewijskracht van conclusies in de systematische literatuuranalyse een sterke aanbeveling niet a priori uit, en zijn bij een hoge bewijskracht ook zwakke aanbevelingen mogelijk (Agoritsas, 2017; Neumann, 2016). De sterkte van de aanbeveling wordt altijd bepaald door weging van alle relevante argumenten tezamen. De werkgroep heeft bij elke aanbeveling opgenomen hoe zij tot de richting en sterkte van de aanbeveling zijn gekomen.

In de GRADE-methodiek wordt onderscheid gemaakt tussen sterke en zwakke (of conditionele) aanbevelingen. De sterkte van een aanbeveling verwijst naar de mate van zekerheid dat de voordelen van de interventie opwegen tegen de nadelen (of vice versa), gezien over het hele spectrum van patiënten waarvoor de aanbeveling is bedoeld. De sterkte van een aanbeveling heeft duidelijke implicaties voor patiënten, behandelaars en beleidsmakers (zie onderstaande tabel). Een aanbeveling is geen dictaat, zelfs een sterke aanbeveling gebaseerd op bewijs van hoge kwaliteit (GRADE gradering HOOG) zal niet altijd van toepassing zijn, onder alle mogelijke omstandigheden en voor elke individuele patiënt.

 

Implicaties van sterke en zwakke aanbevelingen voor verschillende richtlijngebruikers

 

 

Sterke aanbeveling

Zwakke (conditionele) aanbeveling

Voor patiënten

De meeste patiënten zouden de aanbevolen interventie of aanpak kiezen en slechts een klein aantal niet.

Een aanzienlijk deel van de patiënten zouden de aanbevolen interventie of aanpak kiezen, maar veel patiënten ook niet. 

Voor behandelaars

De meeste patiënten zouden de aanbevolen interventie of aanpak moeten ontvangen.

Er zijn meerdere geschikte interventies of aanpakken. De patiënt moet worden ondersteund bij de keuze voor de interventie of aanpak die het beste aansluit bij zijn of haar waarden en voorkeuren.

Voor beleidsmakers

De aanbevolen interventie of aanpak kan worden gezien als standaardbeleid.

Beleidsbepaling vereist uitvoerige discussie met betrokkenheid van veel stakeholders. Er is een grotere kans op lokale beleidsverschillen. 

 

Organisatie van zorg

In de knelpuntenanalyse en bij de ontwikkeling van de richtlijnmodule is expliciet aandacht geweest voor de organisatie van zorg: alle aspecten die randvoorwaardelijk zijn voor het verlenen van zorg (zoals coördinatie, communicatie, (financiële) middelen, mankracht en infrastructuur). Randvoorwaarden die relevant zijn voor het beantwoorden van deze specifieke uitgangsvraag zijn genoemd bij de overwegingen. Meer algemene, overkoepelende, of bijkomende aspecten van de organisatie van zorg worden behandeld in de module Organisatie van zorg.

 

Commentaar- en autorisatiefase

De conceptrichtlijnmodule werd aan de betrokken (wetenschappelijke) verenigingen en (patiënt) organisaties voorgelegd ter commentaar. De commentaren werden verzameld en besproken met de werkgroep. Naar aanleiding van de commentaren werd de conceptrichtlijnmodule aangepast en definitief vastgesteld door de werkgroep. De definitieve richtlijnmodule werd aan de deelnemende (wetenschappelijke) verenigingen en (patiënt) organisaties voorgelegd voor autorisatie en door hen geautoriseerd dan wel geaccordeerd.

 

Literatuur

Agoritsas T, Merglen A, Heen AF, Kristiansen A, Neumann I, Brito JP, Brignardello-Petersen R, Alexander PE, Rind DM, Vandvik PO, Guyatt GH. UpToDate adherence to GRADE criteria for strong recommendations: an analytical survey. BMJ Open. 2017 Nov 16;7(11):e018593. doi: 10.1136/bmjopen-2017-018593. PubMed PMID: 29150475; PubMed Central PMCID: PMC5701989.

 

Alonso-Coello P, Schünemann HJ, Moberg J, Brignardello-Petersen R, Akl EA, Davoli M, Treweek S, Mustafa RA, Rada G, Rosenbaum S, Morelli A, Guyatt GH, Oxman AD; GRADE Working Group. GRADE Evidence to Decision (EtD) frameworks: a systematic and transparent approach to making well informed healthcare choices. 1: Introduction. BMJ. 2016 Jun 28;353:i2016. doi: 10.1136/bmj.i2016. PubMed PMID: 27353417.

 

Alonso-Coello P, Oxman AD, Moberg J, Brignardello-Petersen R, Akl EA, Davoli M, Treweek S, Mustafa RA, Vandvik PO, Meerpohl J, Guyatt GH, Schünemann HJ; GRADE Working Group. GRADE Evidence to Decision (EtD) frameworks: a systematic and transparent approach to making well informed healthcare choices. 2: Clinical practice guidelines. BMJ. 2016 Jun 30;353:i2089. doi: 10.1136/bmj.i2089. PubMed PMID: 27365494.

 

Brouwers MC, Kho ME, Browman GP, Burgers JS, Cluzeau F, Feder G, Fervers B, Graham ID, Grimshaw J, Hanna SE, Littlejohns P, Makarski J, Zitzelsberger L; AGREE Next Steps Consortium. AGREE II: advancing guideline development, reporting and evaluation in health care. CMAJ. 2010 Dec 14;182(18):E839-42. doi: 10.1503/cmaj.090449. Epub 2010 Jul 5. Review. PubMed PMID: 20603348; PubMed Central PMCID: PMC3001530.

 

Hultcrantz M, Rind D, Akl EA, Treweek S, Mustafa RA, Iorio A, Alper BS, Meerpohl JJ, Murad MH, Ansari MT, Katikireddi SV, Östlund P, Tranæus S, Christensen R, Gartlehner G, Brozek J, Izcovich A, Schünemann H, Guyatt G. The GRADE Working Group clarifies the construct of certainty of evidence. J Clin Epidemiol. 2017 Jul;87:4-13. doi: 10.1016/j.jclinepi.2017.05.006. Epub 2017 May 18. PubMed PMID: 28529184; PubMed Central PMCID: PMC6542664.

 

Medisch Specialistische Richtlijnen 2.0 (2012). Adviescommissie Richtlijnen van de Raad Kwalitieit. http://richtlijnendatabase.nl/over_deze_site/over_richtlijnontwikkeling.html

 

Neumann I, Santesso N, Akl EA, Rind DM, Vandvik PO, Alonso-Coello P, Agoritsas T, Mustafa RA, Alexander PE, Schünemann H, Guyatt GH. A guide for health professionals to interpret and use recommendations in guidelines developed with the GRADE approach. J Clin Epidemiol. 2016 Apr;72:45-55. doi: 10.1016/j.jclinepi.2015.11.017. Epub 2016 Jan 6. Review. PubMed PMID: 26772609.

 

Schünemann H, Brożek J, Guyatt G, et al. GRADE handbook for grading quality of evidence and strength of recommendations. Updated October 2013. The GRADE Working Group, 2013. Available from http://gdt.guidelinedevelopment.org/central_prod/_design/client/handbook/handbook.html.

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