Topical therapy
Uitgangsvraag
What does symptomatic therapy with topical therapy look like in patients with dermatomyositis or juvenile dermatomyositis?
Hoe ziet de symptomatische therapie middels topicale therapie bij myositis eruit?
Aanbeveling
Adviseer zonbeschermende/vermijdende maatregelen (aangepast aan huidtype, seizoensgebonden zonintensiteit en verwachte algemene blootstelling aan de zon):
- Vermijden van de middagzon
- UV-straling beschermende kleding
- Dagelijkse toepassing van breedspectrum zonnebrand met hoge zonbeschermingsfactor (bijv. SPF 30 of hoger)
Overweeg verzachtende middelen, vochtinbrengende crèmes of levomenthogel wanneer jeuk een hoofdklacht is van de dermatomyositis-geassocieerde dermatitis
Gebruik topicale corticosteroïden op dermatomyositis-geassocieerde huidlaesies die klachten geven, ongeacht andere systemische therapie. Krachtige tot zeer krachtige preparaten worden aanbevolen voor laesies op het lichaam, hoofdhuid en ledematen, en preparaten met een lage tot matige werking voor laesies op het gezicht.
Gebruik topicale calcineurineremmers op dermatomyositis-geassocieerde huidlaesies die klachten geven, met name voor corticosteroïden gevoelige gebieden (dunne huid) zoals het gezicht en intertrigineuze regio, ongeacht andere systemische therapie.
Overwegingen
Considerations – from evidence to recommendation
Pros and cons of the intervention and the quality of the evidence
A systematic literature search was conducted to assess whether topical therapies such as corticosteroids can be used to treat dermatitis in patients with dermatomyositis, juvenile dermatomyositis or amyopathic dermatomyositis. Outcome measures related to the skin of the patients were defined as crucial outcome measures. Dermatological quality of life and cosmetic outcome measures were defined as important outcome measures.
No studies were found that could be used to answer the PICO-question of this module; therefore no systematic literature analyses could be performed. However, a number of (narrative) reviews of literature were found as well as consensus documents.
Sun
Photosensitivity likely occurs in >50% of dermatomyositis patients, either aggravating or provoking new lesions (Cheong, 1994). Therefore, it is called a photo-exacerbated condition, and though the pathogenesis for ultraviolet (UV) exacerbation is unknown, both UV-A and UV-B likely play a role (Sontheimer, 2004; Callen, 1999; Sontheimer, 1996). Phototesting has however not been able to reliably reproduce the skin lesions; thus, the wavelength of light that is responsible for the clinical manifestations (action spectrum) is not known (Callen & Wortman, 2006). Its photo-exacerbation shows in its clinical appearances, with many of the cutaneous findings located on sunexposed areas of the body. However, clinical observations suggest that not only is the skin disease exacerbated by light, but muscle disease may be worsened after sun exposure (Woo, 1985; Callen, 1993; Zuber, 1996; Callen, 1999).
Therefore, general advice concerning sunprotection is applicable: avoidence of midday sun, photoprotective clothing, and daily application of broad-spectrum with high sun protection factor (e.g. SPF 30-50 or higher) (Callen, 2000 amongst others), along with regular reapplication of sunscreen are generally recommended (Femia, 2013).
One could debate on the advice to avoid potentially photosensitizing drugs.
In addition, assessment of vitamin D levels and proper supplementation should be considered given the need for extensive photoprotection (Femia, 2013; Vleugels & Callen, 2009).
In the case of JDM, Enders (2017) recommended “Sun protection, including the routine use of sunblock on sun-exposed areas should be encouraged for patients with JDM” based on expert opinion by members of the Single Hub and Access point for pediatric Rheumatology in Europe (SHARE) initiative, as did Walling (2010) amongst others). Since the disease-mechanism that is proposed (generally unclear), we propose the same advice as for DM for patients with JDM.
Topical antipruritics
Emollients, moisturizers and levomenthogel can be useful in shortly relieving itch in pruritic skin diseases. In JDM, this is also recommended to be tried (Quain & Werth, 2006 amongst others).
Topical medicinal treatment
Topical calcineurin inhibitors
Currently, in The Netherlands, three topical calcineurin inhibitors have been registered; tacrolimus 0,1% ointment and tacrolimus 0,03% ointment (Protopic®, Takrozem®) and pimecrolimus 1% cream (Elidel®).
In 2002, a cohort study with 4 DM patients showed slight to very high effect on facial lesions when tacrolimus 0,1% ointment was applied once daily during 4 weeks (Yoshimasu, 2002). Hollar and Jorrizo (2004) showed that three out of six patients with recalcitrant DM had at least some (to dramatic) improvement in their skin disease with the twice-daily application of topical tacrolimus for 6–8 weeks. However, another small study (Garcia-Doval & Cruses, 2004) was less encouraging; none of the five patients experienced any effect of topical tacrolimus. Areas treated were the chest, hands, arms, and back, and the authors hypothesize that impaired penetration due to the large size of the tacrolimus molecule may be responsible for the lack of effect, suggesting that topical tacrolimus may be more effective for areas of thinner skin. Enders (2017) also recommend topical tacrolimus 0,1% ointment in localized skin disease in JDM, particularly for symptomatic redness or itching. They do not apply an agelimit for application; however tacrolimus 0,1% ointment is officially registered for eczema in patients of 16 years and older, and the 0,03% variant is registered for patients of 2-16 years. Kim (2011) showed 2 cases of DM with effect of pimecrolimus 1% cream in patients with systemic therapy resistant cutaneous lesions. After application of pimecrolimus twice daily during a couple of months the treated skin lesions showed mild to good response. Vleugels & Callen (2009) reported some effect of pimecrolimus 1% cream in their patients.
Tacrolimus ointment and pimecrolimus cream are known not to be atrophogenic and are associated with minimal systemic absorption. Common side effects are transient local skin burning, pruritus and erythema (Soter, 2001; Langley, 2007).
Topical corticosteroids
Systemic corticosteroids are widely prescribed in DM/JDM, however, these can also have an effect in topical formulation for the dermatitis that comes with the myositis. In the systematic review of Callander (2016), 43 out of 153 included patients with DM used topical corticosteroids, generally as addition to a diverse array of systemic drugs. Four of them used it as single treatment, of which 2 patients noticed no improvement (and no worsening) of skin complaints, one noticed improved pruritus and one experienced skin clearence. The authors did not state any data on the class of the topical corticosteroid used.
Other studies specifically assessing the outcome of treatment with topical corticosteroids are lacking until now. The American group of Femia (2013) and Vleugels & Callen (2009) recommend very potent (for example clobetasol) or potent topical corticosteroids (example mometason) for affected areas, based on expert opinion. Very potent topical corticosteroids are often used to treat the hands, extensor surfaces and scalp, while lower potency agents are often employed for the facial erythema and periorbital eruption. For dosing advice (finger tip unit, maximal dosing per age per class) we refer to the “Leidraad dermatocorticoïden” of the Dutch dermatology Society (2019). Using topical corticosteroids under occlusion can be helpful for maximal penetration in the case of persisting or hyperkeratotic lesions on the body or extremities (Sontheimer, 2004). Chosen vehicles are somewhat dependent upon patient preference, though ointments (and creams) are generally recommended for the body, whereas lotions, shampoos and foams can be helpful for scalp involvement. The SHARE initiative on JDM (Enders, 2016) did not include the use of topical corticosteroids in their consensus based recommendations. Walling (2010) did mention the use of topical corticosteroids in JDM in their overview; based on expert opinion consensus they agree with the recommendations of Femia (2013) and Vleugels & Callen (2009)
Possible cutaneous adverse effects include skin atrophy, telangiectasia, and striae formation (especially with extensive use of very potent topical corticosteroids). Opposed to systemic glucocorticoid use, there is a much lower chance of systemic side effects. However, one should be aware that systemic absorption from widespread application, with resulting suppression of the hypothalamic-pituitary axis (HPA), is possible, though rare (Ellison, 2000).
Immune mediated necrotizing myopathy (IMNM)
Skin complaints are rarely found in other forms of idiopathic inflammatory myopathy (IIM) (see module diagnostic value of disease signs). However, anti-HMGCR necrotizing myopathy is sometimes reported together with dermatomyositis-like dermatitis (Watanabe, 2016; Kadoya, 2016; Hou, 2022).
Currently, there is no literature available on topical treatment of skin complaints in patients with necrotizing myopathy.
The anti-HMGCR necrotizing myopathy-associated dermatitis is not considered the same or a variant of JDM, but since the skin complaints clinically overlap with the ones of JDM, the Guideline Working Group would advice to extrapolate the data and expert opinion on topical treatment in (J)DM to skin complaints associated with necrotizing myopathy.
Patient values and preferences
Daily application of broad-spectrum sunscreen with high sun protection factor can be burdensome, but it outweighs the negative consequences of frequent sunburns and disease exacerbations. Based on expert opinion of the Guideline Working Group, the application of sunscreen can be adjusted to the seasonal sun intensity and expected general sun exposure.
Costs
Sunscreens and other photoprotective measures, although preventive of nature, are currently not covered by common Dutch healthcare insurance. Prescribed emollients are insured for but over the counter preparations are not. Topical calcineurine inhibitors and topical corticosteroids are currently paid for by healthcare insurance, when prescribed by a medical doctor.
Acceptability, feasibility and implementation
There are no problems expected with regard to acceptability, feasibility and implementation. However, when the physician treating the patient does not feel comfortable with prescribing topical calcineuring inhibitors or topical corticosteroids, it is advisable to refer to a dermatologist for evaluation of the skin complaints, for topical prescribtions and monitoring of the cutaneous complaints.
Recommendations
Although there is little and not very strong evidence in favor of topical treatment of dermatomyositis associated dermatitis, the burden and side effects are relatively low, with expected positive effect. It should be advised, solely or most often, concomitant with systemic therapy.
Onderbouwing
Achtergrond
Patients with DM of JDM always show dermatitis, sometimes even without myositis (in the case of amyopathic DM of JDM). There are also cases in which the myositis is treated adequately, but the dermatitis persists. Sometimes patients experience little to no complaints from the dermatitis, however generally patients do experience pruritus, a burning sensation, erythema (redness) or desquamation. Furthermore, the dermatitis can be cosmetically unacceptable for a patient.
This drives the search to gain more insight in the question what the best topical (sole or combined with systemic therapy) treatment is to serve the patients. Since recently, we know that necrotizing autoimmune myopathy also can come with skin complaints, the working group would like to formulate recommendations on topical therapy for this category of patients as well.
Zoeken en selecteren
Search and select
A systematic review of the literature was performed to answer the following question:
What is the effect of topical therapy, such as corticosteroids or calcineurin inhibitors, on dermatomyositis or juvenile dermatomyositis?
P: patients with dermatomyositis, juvenile dermatomyositis or amyopathic dermatomyositis.
I: topical therapy, such as corticosteroids or calcineurin inhibitors.
C: placebo or usual care.
O: outcomes concerning the skin, such as clinical improvement (PGA or other measurement tools), itching, pain, burning (NRS/VAS score or otherwise), redness of the skin, scaling, dermatological quality of life, cosmetic outcomes.
Relevant outcome measures
The guideline development group considered outcomes concerning the skin as a critical outcome measure for decision making; and dermatological quality of life and cosmetic outcomes as important outcome measures for decision making.
A priori, the working group did not define the outcome measures listed above but used the definitions used in the studies.
The working group defined a threshold of 10% for continuous outcome measures and 25% threshold in relative risk (RR) for dichotomous outcomes as a minimal clinically (patient) important difference.
Search and select (Methods)
The databases Medline (via OVID) and Embase (via Embase.com) were searched with relevant search terms until 13-09-2021. The detailed search strategy is depicted under the tab Methods. The systematic literature search resulted in 643 hits. Studies were selected based on the following criteria:
- Article full text available in Engels or Dutch
- Primary comparative research
- Study design Systematic Review, RCT or observational research
- Published between 2000 and September 2021
- Study population are patients with dermatomyositis, juvenile dermatomyositis or amyopathic dermatomyositis.
Twenty-eight studies were initially selected based on title and abstract screening. After reading the full text, all twenty-eight studies were excluded (see the table with reasons for exclusion under the tab Methods), and none of the studies were included.
Results
Due to the absence of studies meeting the search criteria, no studies were included in the literature analysis.
Referenties
- Callen JP. Photosensitivity in collagen vascular diseases. Semin Cutan Med Surg. 1999 Dec;18(4):293-6.
- Callen JP. Photodermatitis in a 6-year-old child. Arthritis Rheum. 1993 Oct;36(10):1483-5.
- Callen JP, Wortmann RL. Dermatomyositis. Clin Dermatol. 2006 Sep-Oct;24(5):363-73.
- Cheong WK, Hughes GR, Norris PG, Hawk JL. Cutaneous photosensitivity in dermatomyositis. Br J Dermatol. 1994 Aug;131(2):205-8.
- Ellison JA, Patel L, Ray DW, David TJ, Clayton PE. Hypothalamic-pituitary-adrenal function and glucocorticoid sensitivity in atopic dermatitis. Pediatrics. 2000 Apr;105(4 Pt 1):794-9.
- Bellutti Enders F, Bader-Meunier B, Baildam E, Constantin T, Dolezalova P, Feldman BM, Lahdenne P, Magnusson B, Nistala K, Ozen S, Pilkington C, Ravelli A, Russo R, Uziel Y, van Brussel M, van der Net J, Vastert S, Wedderburn LR, Wulffraat N, McCann LJ, van Royen-Kerkhof A. Consensus-based recommendations for the management of juvenile dermatomyositis. Ann Rheum Dis. 2017 Feb;76(2):329-340.
- Femia AN, Vleugels RA, Callen JP. Cutaneous dermatomyositis: an updated review of treatment options and internal associations. Am J Clin Dermatol. 2013 Aug;14(4):291-313.
- García-Doval I, Cruces M. Topical tacrolimus in cutaneous lesions of dermatomyositis: lack of effect in side-by-side comparison in five patients. Dermatology. 2004;209(3):247-8. doi: 10.1159/000079903. PMID: 15459546.
- Hollar CB, Jorizzo JL. Topical tacrolimus 0.1% ointment for refractory skin disease in dermatomyositis: a pilot study. J Dermatolog Treat. 2004 Jan;15(1):35-9.
- Hou Y, Shao K, Yan Y, Dai T, Li W, Zhao Y, Li D, Lu JQ, Norman GL, Yan C. Anti-HMGCR myopathy overlaps with dermatomyositis-like rash: a distinct subtype of idiopathic inflammatory myopathy. J Neurol. 2022 Jan;269(1):280-293
- Kadoya M, Hida A, Hashimoto Maeda M, Taira K, Ikenaga C, Uchio N, Kubota A, Kaida K, Miwa Y, Kurasawa K, Shimada H, Sonoo M, Chiba A, Shiio Y, Uesaka Y, Sakurai Y, Izumi T, Inoue M, Kwak S, Tsuji S, Shimizu J. Cancer association as a risk factor for anti-HMGCR antibody-positive myopathy. Neurol Neuroimmunol Neuroinflamm. 2016 Oct 7;3(6):e290.
- Kim JE, Jeong MG, Lee HE, Ko JY, Ro YS. Successful treatment of cutaneous lesions of dermatomyositis with topical pimecrolimus. Ann Dermatol. 2011 Aug;23(3):348-51.
- Langley RG, Luger TA, Cork MJ, Schneider D, Paul C. An update on the safety and tolerability of pimecrolimus cream 1%: evidence from clinical trials and post-marketing surveillance. Dermatology. 2007;215 Suppl 1:27-44. doi: 10.1159/000102118. Epub 2007 Dec 18. PMID: 18174691.
- Middelkamp Hup MA, Arents BWM, De Bruin-Weller MS, Van Everdingen JJE, Galimont AFS, Geleedst-de Vooght MMM, Van Lümig PPM, Rustemeijer T, Van der Sande AAJ, Teligui L, Van der Schoot LS. Leidraad Dermatocorticosteroïden. 2019, Nederlandse Vereniging voor Dermatologie en Venereologie (NVDV)
- Sontheimer RD. The management of dermatomyositis: current treatment options. Expert Opin Pharmacother. 2004 May;5(5):1083-99.
- Sontheimer RD. Photoimmunology of lupus erythematosus and dermatomyositis: a speculative review. Photochem Photobiol. 1996 May;63(5):583-94.
- Soter NA, Fleischer AB Jr, Webster GF, Monroe E, Lawrence I. Tacrolimus ointment for the treatment of atopic dermatitis in adult patients: part II, safety. J Am Acad Dermatol. 2001 Jan;44(1 Suppl):S39-46.
- Vleugels RA & Callen JP. Dermatomyositis: current and future treatments, Expert Review of Dermatology. 2009;4:6, 581-594.
- Walling HW, Gerami P, Sontheimer RD. Juvenile-onset clinically amyopathic dermatomyositis: an overview of recent progress in diagnosis and management. Paediatr Drugs. 2010;12(1):23-34.
- Watanabe Y, Uruha A, Suzuki S, Nakahara J, Hamanaka K, Takayama K, Suzuki N, Nishino I. Clinical features and prognosis in anti-SRP and anti-HMGCR necrotising myopathy. J Neurol Neurosurg Psychiatry. 2016 Oct;87(10):1038-44.
- Woo TR, Rasmussen J, Callen JP. Recurrent photosensitive dermatitis preceding juvenile dermatomyositis. Pediatr Dermatol. 1985 Mar;2(3):207-12. doi: 10.1111/j.1525-1470.1985.tb01054.x. PMID: 3991376.
- Yoshimasu T, Ohtani T, Sakamoto T, Oshima A, Furukawa F. Topical FK506 (tacrolimus) therapy for facial erythematous lesions of cutaneous lupus erythematosus and dermatomyositis. Eur J Dermatol. 2002 Jan-Feb;12(1):50-2. PMID: 11809595.
- Zuber M, John S, Pfreundschuh M, Gause A. A young woman with a photosensitive pruritic rash on her face and upper trunk. Arthritis Rheum. 1996 Aug;39(8):1419-22. doi: 10.1002/art.1780390822. PMID: 8702453.
Evidence tabellen
Table of excluded studies
|
Author and year |
Reason for exclusion |
|
Chung (2019) |
Wrong study design: review of the literature |
|
Hollar (2004) |
Non-comparative study |
|
Quain (2006) |
Wrong study design: review of the literature |
|
Vleugels (2009) |
Wrong study design: review of the literature |
|
Femia (2013) |
Wrong study design: review of the literature |
|
Gordon Spratt (2015) |
Wrong intervention |
|
Griger (2017) |
Wrong study design: overview of the literature |
|
Yoshimasu (2002) |
Wrong study design: case report (n=1) |
|
Wang (2021) |
Wrong study design: review of the literature |
|
Bogdanov (2018) |
Wrong study design: review of the literature |
|
Callen (2000) |
Wrong study design: review of the literature |
|
Choy (2002) |
Wrong study design: review of the literature |
|
Clarke (2010 |
Wrong study design: review of the literature |
|
Kuhn (2016) |
Wrong study design: overview of the literature |
|
Mann (2021) |
Wrong study design: review of the literature |
|
Papadopoulou (2017) |
Wrong study design: overview of the literature |
|
Walling (2010) |
Wrong study design: overview of the literature |
|
Zaba (2012) |
Wrong study design: review of the literature |
|
Bellutti (2017) |
Wrong study design: review of the literature |
|
Fujimoto (2016) |
Non-comparative study |
|
Gerami (2007) |
Non-comparative study |
|
Kul Cinar (2021) |
Wrong study design: review of the literature |
|
Fernandez (2019) |
Wrong study design: review of the literature |
|
Brauer (2014) |
Wrong intervention |
|
Braunstein (2013) |
Wrong study design: review of the literature |
|
Callander (2018) |
Non-comparative study |
|
Pipitone (2020) |
Wrong study design: review of the literature |
|
Shinjo (2020) |
Wrong study design: review of the literature |
Verantwoording
Autorisatiedatum en geldigheid
Laatst beoordeeld : 07-02-2024
Laatst geautoriseerd : 07-02-2024
Geplande herbeoordeling :
Algemene gegevens
The development of this guideline module was supported by the Knowledge Institute of the Federation of Medical Specialists (www.demedischspecialist.nl/ kennisinstituut) and was financed from the Quality Funds for Medical Specialists (SKMS). The financier has had no influence whatsoever on the content of the guideline module.
Samenstelling werkgroep
A multidisciplinary working group was set up in 2020 for the development of the guideline module, consisting of representatives of all relevant specialisms and patient organisations (see the Composition of the working group) involved in the care of patients with IIM/myositis.
Working group
- Dr. A.J. van der Kooi, neurologist, Amsterdam UMC, location AMC. Nederlandse Vereniging voor Neurologie (chair)
- Dr. U.A. Badrising, neurologist, LUMC. Nederlandse Vereniging voor Neurologie
- Dr. C.G.J. Saris, neurologist, Radboudumc. Nederlandse Vereniging voor Neurologie
- Dr. S. Lassche, neurologist, Zuyderland MC. Nederlandse Vereniging voor Neurologie
- Dr. J. Raaphorst, neurologist, Amsterdam UMC, locatie AMC. Nederlandse Vereniging voor Neurologie
- Dr. J.E. Hoogendijk, neurologist, UMC Utrecht. Nederlandse Vereniging voor Neurologie
- Drs. T.B.G. Olde Dubbelink, neurologist, Rijnstate, Nederlandse Vereniging voor Neurologie
- Dr. I.L. Meek, rheumatologist, Radboudumc. Nederlandse Vereniging voor Reumatologie
- Dr. R.C. Padmos, rheumatologist, Erasmus MC. Nederlandse Vereniging voor Reumatologie
- Prof. dr. E.M.G.J. de Jong, dermatologist, werkzaam in het Radboudumc. Nederlandse Vereniging voor Dermatologie en Venereologie
- Drs. W.R. Veldkamp, dermatologist, Ziekenhuis Gelderse Vallei. Nederlandse Vereniging voor Dermatologie en Venereologie
- Dr. J.M. van den Berg, pediatrician, Amsterdam UMC, locatie AMC. Nederlandse Vereniging voor Kindergeneeskunde
- Dr. M.H.A. Jansen, pediatrician, UMC Utrecht. Nederlandse Vereniging voor Kindergeneeskunde
- Dr. A.C. van Groenestijn, rehabilitation physician, Amsterdam UMC, locatie AMC. Nederlandse Vereniging van Revalidatieartsen
- Dr. B. Küsters, pathologist, Radboudumc. Nederlandse Vereniging voor Pathologie
- Dr. V.A.S.H. Dalm, internist, Erasmus MC. Nederlandse Internisten Vereniging
- Drs. J.R. Miedema, pulmonologist, Erasmus MC. Nederlandse Vereniging van Artsen voor Longziekten en Tuberculose
- I. de Groot, patient representatieve. Spierziekten Nederland
Advisory board
- Prof. dr. E. Aronica, pathologist, Amsterdam UMC, locatie AMC. External expert.
- Prof. dr. D. Hamann, Laboratory specialist medical immunology, UMC Utrecht. External expert.
- Drs. R.N.P.M. Rinkel, ENT physician, Amsterdam UMC, locatie VUmc. Vereniging voor Keel-Neus-Oorheelkunde en Heelkunde van het Hoofd-Halsgebied
- dr. A.S. Amin, cardiologist, werkzaam in werkzaam in het Amsterdam UMC, locatie AMC. Nederlandse Vereniging voor Cardiologie
- dr. A. van Royen-Kerkhof, pediatrician, UMC Utrecht. External expert.
- dr. L.W.J. Baijens, ENT physician, Maastricht UMC+. External expert.
- Em. Prof. Dr. M. de Visser, neurologist, Amsterdam UMC. External expert.
Methodological support
- Drs. T. Lamberts, senior advisor, Knowledge institute of the Federation of Medical Specialists
- Drs. M. Griekspoor, advisor, Knowledge institute of the Federation of Medical Specialists
- Dr. M. M. J. van Rooijen, advisor, Knowledge institute of the Federation of Medical Specialists
Belangenverklaringen
The ‘Code ter voorkoming van oneigenlijke beïnvloeding door belangenverstrengeling’ has been followed. All working group members have declared in writing whether they have had direct financial interests (attribution with a commercial company, personal financial interests, research funding) or indirect interests (personal relationships, reputation management) in the past three years. During the development or revision of a module, changes in interests are communicated to the chairperson. The declaration of interest is reconfirmed during the comment phase.
An overview of the interests of working group members and the opinion on how to deal with any interests can be found in the table below. The signed declarations of interest can be requested from the secretariat of the Knowledge Institute of the Federation of Medical Specialists.
|
Werkgroeplid |
Functie |
Nevenfuncties |
Gemelde belangen |
Ondernomen actie |
|
van der Kooi |
Neuroloog, Amsterdam UMC |
|
Immediate studie (investigator initiated, IVIg behandeling bij therapie naive patienten). --> Financiering via Behring. Studie januari 2019 afgerond |
Geen restricties (middel bij advisory board is geen onderdeel van rcihtlijn) |
|
Miedema |
Longarts, Erasmus MC |
Geen. |
|
Geen restricties |
|
Meek |
Afdelingshoofd a.i. afdeling reumatische ziekten, Radboudumc |
Commissaris kwaliteit bestuur Nederlandse Vereniging voor Reumatologie (onkostenvergoeding) |
Medisch adviseur myositis werkgroep spierziekten Nederland |
Geen restricties |
|
Veldkamp |
AIOS dermatologie Radboudumc Nijmegen |
|
Geen. |
Geen restricties |
|
Padmos |
Reumatoloog, Erasmus MC |
Docent Breederode Hogeschool (afdeling reumatologie EMC wordt hiervoor betaald) |
Geen. |
Geen restricties |
|
Dalm |
Internist-klinisch immunoloog Erasmus MC |
Geen. |
Geen. |
Geen restricties |
|
Olde Dubbelink |
Neuroloog in opleiding Canisius-Wilhelmina Ziekenhuis, Nijmegen |
Promotie onderzoek naar diagnostiek en outcome van het carpaletunnelsyndroom (onbetaald) |
Geen. |
Geen restricties |
|
van Groenestijn |
Revalidatiearts AmsterdamUMC, locatie AMC |
Geen. |
Lokale onderzoeker voor de I'M FINE studie (multicentre, leiding door afdeling Revalidatie Amsterdam UMC, samen met UMC Utrecht, Sint Maartenskliniek, Klimmendaal en Merem. Evaluatie van geïndividualiseerd beweegprogramma o.b.v. combinatie van aerobe training en coaching bij mensen met neuromusculaire aandoeningen, NMA). Activiteiten: screening NMA-patiënten die willen participeren aan deze studie. Subsidie van het Prinses Beatrix Spierfonds. |
Geen restricties |
|
Lassche |
Neuroloog, Zuyderland Medisch Centrum, Heerlen en Sittard-Geleen |
Geen. |
Geen. |
Geen restricties |
|
de Jong |
Dermatoloog, afdelingshoofd Dermatologie Radboudumc Nijmegen |
Geen. |
All funding is not personal but goes to the independent research fund of the department of dermatology of Radboud university medical centre Nijmegen, the Netherlands |
Geen restricties |
|
Hoogendijk |
Neuroloog Universitair Medisch Centrum Utrecht (0,4) Neuroloog Sionsberg, Dokkum (0,6) |
beide onbetaald |
Geen. |
Geen restricties |
|
Badrising |
Neuroloog Leids Universitair Medisch Centrum |
(U.A.Badrising Neuroloog b.v.: hoofdbestuurder; betreft een vrijwel slapende b.v. als overblijfsel van mijn eerdere praktijk in de maatschap neurologie Dirksland, Het van Weel-Bethesda Ziekenhuis) |
Medisch adviseur myositis werkgroep spierziekten Nederland |
Geen restricties |
|
van den Berg |
Kinderarts-reumatoloog/-immunoloog Emma kinderziekenhuis/ Amsterdam UMC |
Geen. |
Geen. |
Geen restricties |
|
de Groot |
Patiënt vertegenwoordiger/ ervaringsdeskundige: voorzitter diagnosewerkgroep myositis bij Spierziekten Nederland in deze commissie patiënt(vertegenwoordiger) |
|
Geen |
Geen restricties |
|
Küsters |
Patholoog, Radboud UMC |
Geen. |
Geen. |
Geen restricties |
|
Saris |
Neuroloog/ klinisch neurofysioloog, Radboudumc |
Geen. |
Geen. |
Geen restricties |
|
Raaphorst |
Neuroloog, Amsterdam UMC |
Geen. |
|
Restricties m.b.t. opstellen aanbevelingen IvIg behandeling. |
|
Jansen |
Kinderarts-immunoloog-reumatoloog, WKZ UMC Utrecht |
Docent bij Mijs-instituut (betaald) |
Onderzoek biomakers in juveniele dermatomyositis. Geen belang bij uitkomst richtlijn. |
Geen restricties |
Inbreng patiëntenperspectief
Attention was paid to the patient's perspective by offering the Vereniging Spierziekten Nederland to take part in the working group. Vereniging Spierziekten Nederland has made use of this offer, the Dutch Artritis Society has waived it. In addition, an invitational conference was held to which the Vereniging Spierziekten Nederland, the Dutch Artritis Society nd Patiëntenfederatie Nederland were invited and the patient's perspective was discussed. The report of this meeting was discussed in the working group. The input obtained was included in the formulation of the clinical questions, the choice of outcome measures and the considerations. The draft guideline was also submitted for comment to the Vereniging Spierziekten Nederland, the Dutch Artritis Society and Patiëntenfederatie Nederland, and any comments submitted were reviewed and processed.
Qualitative estimate of possible financial consequences in the context of the Wkkgz
In accordance with the Healthcare Quality, Complaints and Disputes Act (Wet Kwaliteit, klachten en geschillen Zorg, Wkkgz), a qualitative estimate has been made for the guideline as to whether the recommendations may lead to substantial financial consequences. In conducting this assessment, guideline modules were tested in various domains (see the flowchart on the Guideline Database).
The qualitative estimate shows that there are probably no substantial financial consequences, see table below.
|
Module |
Estimate |
Explanation |
|
Module diagnostische waarde ziekteverschijnselen |
No substantial financial consequences |
Outcome 1 No financial consequences. The recommendations are not widely applicable (<5,000 patients) and are therefore not expected to have any substantial financial consequences on collective expenditures. |
|
Module Optimale strategie aanvullende diagnostiek myositis |
No substantial financial consequences |
Outcome 1 No financial consequences. The recommendations are not widely applicable (<5,000 patients) and are therefore not expected to have any substantial financial consequences on collective expenditures. |
|
Module Autoantibody testing in myositis |
No substantial financial consequences |
Outcome 1 No financial consequences. The recommendations are not widely applicable (<5,000 patients) and are therefore not expected to have any substantial financial consequences on collective expenditures. |
|
Module Screening op maligniteiten |
No substantial financial consequences |
Outcome 1 No financial consequences. The recommendations are not widely applicable (<5,000 patients) and are therefore not expected to have any substantial financial consequences on collective expenditures. |
|
Module Screening op comorbiditeiten |
No substantial financial consequences |
Outcome 1 No financial consequences. The recommendations are not widely applicable (<5,000 patients) and are therefore not expected to have any substantial financial consequences on collective expenditures. |
|
Module Immunosuppressie en -modulatie bij IBM |
No substantial financial consequences |
Outcome 1 No financial consequences. The recommendations are not widely applicable (<5,000 patients) and are therefore not expected to have any substantial financial consequences on collective expenditures. |
|
Module Treatment with Physical training |
No substantial financial consequences |
Outcome 1 No financial consequences. The recommendations are not widely applicable (<5,000 patients) and are therefore not expected to have any substantial financial consequences on collective expenditures. |
|
Module Treatment of dysphagia in myositis |
No substantial financial consequences |
Outcome 1 No financial consequences. The recommendations are not widely applicable (<5,000 patients) and are therefore not expected to have any substantial financial consequences on collective expenditures. |
|
Module Treatment of dysphagia in IBM |
No substantial financial consequences |
Outcome 1 No financial consequences. The recommendations are not widely applicable (<5,000 patients) and are therefore not expected to have any substantial financial consequences on collective expenditures. |
|
Module Topical therapy |
No substantial financial consequences |
Outcome 1 No financial consequences. The recommendations are not widely applicable (<5,000 patients) and are therefore not expected to have any substantial financial consequences on collective expenditures. |
|
Module Treatment of calcinosis |
No substantial financial consequences |
Outcome 1 No financial consequences. The recommendations are not widely applicable (<5,000 patients) and are therefore not expected to have any substantial financial consequences on collective expenditures. |
|
Module Organization of care |
No substantial financial consequences |
Outcome 1 No financial consequences. The recommendations are not widely applicable (<5,000 patients) and are therefore not expected to have any substantial financial consequences on collective expenditures. |
Werkwijze
Methods
AGREE
This guideline module has been drawn up in accordance with the requirements stated in the Medisch Specialistische Richtlijnen 2.0 report of the Advisory Committee on Guidelines of the Quality Council. This report is based on the AGREE II instrument (Appraisal of Guidelines for Research & Evaluation II; Brouwers, 2010).
Clinical questions
During the preparatory phase, the working group inventoried the bottlenecks in the care of patients with IIM. Bottlenecks were also put forward by the parties involved via an invitational conference. A report of this is included under related products.
Based on the results of the bottleneck analysis, the working group drew up and finalized draft basic questions.
Outcome measures
After formulating the search question associated with the clinical question, the working group inventoried which outcome measures are relevant to the patient, looking at both desired and undesired effects. A maximum of eight outcome measures were used. The working group rated these outcome measures according to their relative importance in decision-making regarding recommendations, as critical (critical to decision-making), important (but not critical), and unimportant. The working group also defined at least for the crucial outcome measures which differences they considered clinically (patient) relevant.
Methods used in the literature analyses
A detailed description of the literature search and selection strategy and the assessment of the risk-of-bias of the individual studies can be found under 'Search and selection' under Substantiation. The assessment of the strength of the scientific evidence is explained below.
Assessment of the level of scientific evidence
The strength of the scientific evidence was determined according to the GRADE method. GRADE stands for Grading Recommendations Assessment, Development and Evaluation (see http://www.gradeworkinggroup.org/). The basic principles of the GRADE methodology are: naming and prioritizing the clinically (patient) relevant outcome measures, a systematic review per outcome measure, and an assessment of the strength of evidence per outcome measure based on the eight GRADE domains (downgrading domains: risk of bias, inconsistency, indirectness, imprecision, and publication bias; domains for upgrading: dose-effect relationship, large effect, and residual plausible confounding).
GRADE distinguishes four grades for the quality of scientific evidence: high, fair, low and very low. These degrees refer to the degree of certainty that exists about the literature conclusion, in particular the degree of certainty that the literature conclusion adequately supports the recommendation (Schünemann, 2013; Hultcrantz, 2017).
|
Definitie |
|
|
High |
|
|
Moderate |
|
|
Low |
|
|
Very low |
|
When assessing (grading) the strength of the scientific evidence in guidelines according to the GRADE methodology, limits for clinical decision-making play an important role (Hultcrantz, 2017). These are the limits that, if exceeded, would lead to an adjustment of the recommendation. To set limits for clinical decision-making, all relevant outcome measures and considerations should be considered. The boundaries for clinical decision-making are therefore not directly comparable with the minimal clinically important difference (MCID). Particularly in situations where an intervention has no significant drawbacks and the costs are relatively low, the threshold for clinical decision-making regarding the effectiveness of the intervention may lie at a lower value (closer to zero effect) than the MCID (Hultcrantz, 2017).
Considerations
In addition to (the quality of) the scientific evidence, other aspects are also important in arriving at a recommendation and are taken into account, such as additional arguments from, for example, biomechanics or physiology, values and preferences of patients, costs (resource requirements), acceptability, feasibility and implementation. These aspects are systematically listed and assessed (weighted) under the heading 'Considerations' and may be (partly) based on expert opinion. A structured format based on the evidence-to-decision framework of the international GRADE Working Group was used (Alonso-Coello, 2016a; Alonso-Coello 2016b). This evidence-to-decision framework is an integral part of the GRADE methodology.
Formulation of conclusions
The recommendations answer the clinical question and are based on the available scientific evidence, the most important considerations, and a weighting of the favorable and unfavorable effects of the relevant interventions. The strength of the scientific evidence and the weight assigned to the considerations by the working group together determine the strength of the recommendation. In accordance with the GRADE method, a low evidential value of conclusions in the systematic literature analysis does not preclude a strong recommendation a priori, and weak recommendations are also possible with a high evidential value (Agoritsas, 2017; Neumann, 2016). The strength of the recommendation is always determined by weighing all relevant arguments together. The working group has included with each recommendation how they arrived at the direction and strength of the recommendation.
The GRADE methodology distinguishes between strong and weak (or conditional) recommendations. The strength of a recommendation refers to the degree of certainty that the benefits of the intervention outweigh the harms (or vice versa) across the spectrum of patients targeted by the recommendation. The strength of a recommendation has clear implications for patients, practitioners and policy makers (see table below). A recommendation is not a dictate, even a strong recommendation based on high quality evidence (GRADE grading HIGH) will not always apply, under all possible circumstances and for each individual patient.
|
Implications of strong and weak recommendations for guideline users |
||
|
|
||
|
|
Strong recommendation |
Weak recommendations |
|
For patients |
Most patients would choose the recommended intervention or approach and only a small number would not. |
A significant proportion of patients would choose the recommended intervention or approach, but many patients would not. |
|
For practitioners |
Most patients should receive the recommended intervention or approach. |
There are several suitable interventions or approaches. The patient should be supported in choosing the intervention or approach that best reflects his or her values and preferences. |
|
For policy makers |
The recommended intervention or approach can be seen as standard policy. |
Policy-making requires extensive discussion involving many stakeholders. There is a greater likelihood of local policy differences. |
Organization of care
In the bottleneck analysis and in the development of the guideline module, explicit attention was paid to the organization of care: all aspects that are preconditions for providing care (such as coordination, communication, (financial) resources, manpower and infrastructure). Preconditions that are relevant for answering this specific initial question are mentioned in the considerations. More general, overarching or additional aspects of the organization of care are dealt with in the module Organization of care.
Commentary and authtorisation phase
The draft guideline module was submitted to the involved (scientific) associations and (patient) organizations for comment. The comments were collected and discussed with the working group. In response to the comments, the draft guideline module was modified and finalized by the working group. The final guideline module was submitted to the participating (scientific) associations and (patient) organizations for authorization and authorized or approved by them.
References
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 Kwaliteit. 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, . 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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