How should the care for patients with idiopathic inflammatory myopathy (IIM) be organised?

Hoe moet de zorg voor patiënten met idiopathische inflammatoire myopathie (IIM) worden georganiseerd?

Considerations

Shared decision making

To determine which medical policy best suits the patient, a process is necessary in which the doctor and the patient decide together, the so-called shared decision making. In this process, the patient should be well informed about the situation and the diagnosis. Shared decision making is especially important when there are several treatment options and the choice depends in particular on what the patient finds most important in terms of side effects, 'benefits' to be achieved, etc. If there is limited health literacy, the decision-making process requires more attention (Nivel, 2019). When providing information, the practitioner must point out (digital) aids and tools to the patient that have been made available through patient associations. Information about this can be found on the website of Spierziekten Nederland (via: https://www.spierziekten.nl/themas/medische-zorg and https://www.spierziekten.nl/themas/revalidatie-en-spierziekten). Both websites contain hyperlinks to informative videos, consultation cards, digital decision aids and an accessible knowledge module.

The optimal choice is highly dependent on the values and preferences of the patient: what is the life of the patient with IIM like, what is important to him/her and what is the goal of the treatment, are important starting questions (e.g. advance care planning). In addition, explicit attention should be paid to the wishes of the patient in terms of quality of life (such as pain, fatigue, muscle complaints and physical functioning) (Esfandiary, 2020). Decisions are also largely based on the degree of disease activity, progression of structural damage, co-morbidity and safety considerations.

Several studies show that shared decision-making increases patients' knowledge and risk perception and improves the quality of the conversation and decision-making (Stacey, 2017). One of the most important ways to involve a patient in his/her care process is to facilitate shared decision making. Shared decision making is especially important when there are several treatment options and the choice mainly depends on what the patient considers most important in terms of side effects, the 'benefits' to be achieved, Etcetera.

The OMERACT Shared Decision Making Working Group describe in a white paper (Toupin-April, 2021) the steps in the process, which also correspond to the steps in the Nivel report:

1. Clinician and patient identify the decision to be made. It is explicitly stated that a decision must be made, and that the opinion of the patient is important in this.
2. The practitioner informs the patient about the various options and their characteristics (advantages and disadvantages, impact on daily life in both the short and long term, possible risks, etcetera). This may also include (temporarily) withholding disease-oriented treatment.
3. The patient understands this information and discusses with the practitioner which characteristics are most important to him, in line with his personal values, interests, preferences, etc. The practitioner supports the patient in formulating his preferences.
4. The patient and practitioner 'weigh' the different options
5. The patient and practitioner decide together or explicitly postpone it.
6. The patient and practitioner together discuss the possible obstacles and incentives surrounding the decision taken.
7. In the event of a wait-and-see policy or a changed situation, the decision taken should be revisited.

When explaining the options, a pitfall can be to provide too little information or information which is too complex. Make sure that the explanation is tailored and verify whether the information has been received properly. Indicate that there is a possibility to bring a family member or acquaintance to the consultation and ask in advance to write down any questions that arise. There are also decision aids or consultation cards that can be used as aids. The Patientenfederatie Nederland developed several of these decision aids, such as:

www.3goedevragen.nl of www.begineengoedgesprek.nl

When discussing preferences, it is important to explicitly include preferences or the daily life situation of patients. Pitfalls when making a decision can be that it is not made clear which healthcare provider(s) is (are) responsible for the decision-making process or that it is not made clear to what extent the patient is responsible for the shared decision-making process. Sometimes mistrust of the proposed treatment plan (or wait-and-see policy) leads participants to experiment on their own initiative or stop taking medication or look for alternative treatments.

If the decision is left to the practioner, the patient's values and norms are still important to increase the chances of treatment success. The practitioner then has the challenging task of deciding what is best for the patient; the treatment that best matches the personal values and lifestyle of the patient (Voshaar, 2015).

Points of attention for primary care

For adults as well as children, good communication with primary care professionals is especially important in the (suspicion of the) diagnosis of IIM, because systemic complications and side effects of drug treatment can occur. This concerns communication with the general practitioner (refer to the NHG information brochure “Dermatomyositis and polymyositis” (NHG, 2010)), and communication with the pharmacist and paramedics such as physiotherapists (refer to the information brochure “Fysiotherapie bij volwassenen met een langzaam progressieve spierziekte" (Spierziekten Nederland/KNGF, 2020)).

When the diagnosis IIM is made, the main physician (regiebehandelaar) informs the general practitioner as soon as possible, but at least within 2 weeks, including the treatment instituted. In the stable phase, patients remain under the supervision and treatment of the main physician or a combination of physicians depending on disease manifestations.

If the general practitioner or pharmacist is confronted with side effects, complications, changes in the clinical picture, intercurrent diseases or other questions from patients, they must be able to contact the main physician or his/her replacement in an accessible manner and at all times.

Preconception care and pregnancy

IIM is not a contraindication for pregnancy. However, certain immunosuppressive drugs are teratogenic and higher disease activity results in higher maternal and fetal complications. Female patients should be informed about the risks of getting pregant during active disease (the physician can even consider to advice against getting pregnant during active disease) and pregnancy can best be planned during stable and low disease activity. If there is a wish to have children, patients (both women and men) are advised to discuss this with the main physician. Therapy with glucocorticoids (ideally below 10mg/day), hydroxychloroquin, sulfasalazin, azathioprine, cyclofosfamide (if no other options and only in 2nd or 3rd semester), colchicine, IVIg is possible during pregnancy. Rituximab is to consider when all other therapy options have failed. Therapy with mycophenolate mofetil methotrexate, abatacept and cyclofosfamide (1^st semester) should be avoided.

Vaccinations

All important recommendations on vaccinations can be found in the ‘Landelijke adviezen voor vaccinatie bij chronisch inflammatoire aandoeningen’ of the RIVM (RIVM, 2018).

All patients, including children, with IIM have an indication for flu and Covid-19 vaccination (NHG, 2021). When a patient is being treated with immunosuppressive medication, a pneumococcal vaccination may be considered. In that case, the general practitioner can be asked to check whether the patient has had a pneumococcal vaccination in the past 5 years. If this is not the case and there is no contraindication, the physician can ask the general practitioner to administer pneumovax (NVR, 2011).

For questions about traveler vaccinations when using DMARDs, the patient is referred by the physician or general practitioner to the GGD, traveler vaccination department or a center with expertise. The use of vaccination with live microorganisms (such as yellow fever vaccine and the mumps-measles-rubella vaccine) is contraindicated during treatment with immunosuppressive drugs because a generalized infection may occur.

Vaccinations with killed causative agents or derived antigen during treatment with immunosuppressive drugs are less effective due to a reduced immune response. In some cases the vaccine can be repeated, or a titer can be assessed.

Care pathway

Treatment should ideally be delivered according to the principles of stepped care: most effective and appropriate form of treatment possible according to the nature and severity of problems. Severity and disease course should be carefully monitored, treatment effects should be evaluated and if necessary, treatment must be intensified to reach treatment goals.

Aerobic capacity of muscles is diminished in IIM, even in muscles without weakness. In the early stages of the disease, patients are advised to keep moving, guided by symptoms/muscle weakness and within the limits of his/her ability. After starting medical treatment and/or at signs of recovery, strength and endurance training can be considered. In clinically stable disease activity, intensification of strength and endurance training should be tried, as this seems to accelerate recovery. In case of weight loss, underweight or increase energy expenditure, referral to a dietician is to be considered.

Juvenile dermatomyositis and idiopathic inflammatory myopathy

JDM and IIM are characterized by rapidly progressive proximal muscle weakness and potential severe cardiopulmonary comorbidity. Mortality is increased compared to the general population, with an increased relative risk of 2-4 in IIM (Li, 2020). JDM and IIM can be treated with immunosuppressive and immunomodulatory medications. Longer diagnostic delay and delayed start of treatment are associated with worse outcomes (Bronner, 2006). Hence, rapid diagnosis is essential when there is a suspicion of IIM. It is preferred to complete the diagnostic process before initiating treatment, because corticosteroids can affect the results of muscle biopsy and other diagnostic investigations.

A patient with JDM or IIM can present with several different complaints to several different specialists. Diagnostic procedures in a patient with suspected IIM can be performed by rheumatologists, neurologists, dermatologists, ENT-physician, internal medicine specialists and/or clinical immunologists. It is recommended to develop a local multidisciplinary diagnostic protocol. Specialized neuromuscular centers and myositis expertise centers offer fast-track diagnostic pathways that provide all necessary diagnostic testing and a recommendation for treatment within 1-2 days.

It is recommended that patients with JDM or IIM are treated by an experienced specialist, preferably within a multidisciplinary team in which cases are discussed at diagnosis and at predetermined moments during treatment. If the required expertise is not available locally, patients can be referred to a myositis expertise center.

If there are limitations in activities of daily life such as self care or mobility, or participation in society on any level due to the IIM, referral to an in NMA specialised rehabilitation physician/team or myositis expertise centre is recommended. If indicated, the rehabilitation physician can then start an interdisciplinary rehabilitation treatment in a neuromuscular specialised rehabilitation team or refer to relevant first- and second-line paramedics with experience in IIM, such as physical therapists, occupational therapists, speech-language therapists, social work and/or psychologists.

Inclusion body myositis

IBM is characterized by slow progressive muscle weakness and dysphagia, with severe disability at later disease stages (Cox, 2011). It is recommended to refer patients with suspected IBM to a center with expertise on IBM, as this can reduce diagnostic delay and lead to earlier diagnosis and initiation of supportive treatment. Patients with established IBM can also benefit from a multidisciplinary evaluation in a center with expertise in IBM or specialized rehabilitation center, for specific evaluation and treatment of dysphagia or any other limitations in functioning due to muscle weakness. Annual or semi- annual routine follow-up by an experienced neurologist and rehabilitation physician is recommended.

If there are limitations in activities of daily life such as self care or mobility, or participation in society on any level due to the IIM, we recommend referral to an in NMA specialised rehabilitation physician/team or myositis/center with expertise on IBM.

If indicated, the rehabilitation physician can then start an interdisciplinary rehabilitation treatment in a neuromuscular specialised rehabilitation team or refer to relevant first- and second-line paramedics with experience in IIM or IBM such as physical therapists, occupational therapists, speech-language therapists, social work and/or psychologists. In local networks, it is generally well known where physicians are active and proficient in the diagnosis and treatment of myositis. Otherwise, you can find which centers in the Netherlands specialize in myositis on myositisexpertisecentrum.nl.

Transition of care from pediatrics to adulthood

The leading physician is responsible for a complete and comprehensive transfer and guidance of parents and child during transition and functions as transition coordinator. After transition the adult specialists are responsible; they will actively involve other treating physicians if necessary. If possible, the patient will be seen together by both the peadiatrician and the physician whom will be the leading physician during the adult life of the patient during the last visit at the child department or the first visit at the adult outpatient department (warm transfer). During transition attention will be paid to the associated responsibilities, such as taking responsibility for appointments, making own choices, etc.

At some point young people with IIM have to take the step towards adult care. This step should be taken with attention of patients, parents and (professional) care-givers. Adulthood requires certain skills, knowledge, development of independence and autonomy. In case of insufficient guidance patient might not feel involved in the treatment; problems during daily life and other areas of life might occur. Except for the role of the patient itself, the role of parents/care givers changes as well.

An important starting point is that the patient is central: it is an unique individual with its own feelings, needs and desires. Topics such as school/education, work, relations, friendships, sports/leisure time, self-sustainability and sexuality deserve attention.

Main item during transition is strengthening self-management; it is necessary that the patient is in control over his life, taking into account all aspects of the IIM. Care should be future-oriented, continuity of care is essential, preferably on the basis of shared decision making. For more information, see the Quality standard ‘Jongeren in transitie van kinderzorg naar volwassenenzorg’ (NVK, 2022).

Advance care planning

Advance care planning

Patients with IIM can develop several complications, in part as a direct consequence of their condition but also – in patients with JDM and IIM – as side effects or treatment with immunosuppressive treatment. Dysphagia, pain, fatigue, impaired mobility and activities of daily living, exercise intolerance, use of aids and (nocturnal) ventilation can negatively impact quality of life. Screen for these potential complications at routine follow-up and perform additional investigations and/or refer to relevant subspecialties.

In frail and/or elderly patients, or in those with severe refractory disease, it is recommended to discuss treatment wishes at the time of diagnosis, initiation and intensification of treatment. Topics can include medication, supportive treatment, hospital admission, resuscitation and ventilation. Evaluate these wishes when significant changes in the clinical condition occur, for example with the onset of dysphagia, loss of ambulation, development of progression of ILD, start of ventilation, refractory symptoms, increase in immunosuppressant therapy or with improvement in the clinical condition.

Juvenile dermatomyositis and idiopathic inflammatory myopathy

Potential severe complications in JDM and IIM include rapidly progressive interstitial lung disease (RP-ILD), respiratory failure, heart failure and pneumonia. Pneumonia can be related to aspiration in patients with dysphagia, and to increased susceptibility to (opportunistic) infections due to immunosuppressants. Infection, exacerbation of myositis, or a combination of both are the most common reasons for Intensive Care admission (Peng, 2016).

Inclusion body myositis

Inclusion body myositis is characterized by an eventual severe impairment of mobility and frequent dysphagia due to progressive muscle weakness, often developing over decades (Cox, 2011). Average life expectancy in IBM is similar to the average in the Dutch population. Compared to the general population, patients with IBM more often died as the result of pneumonia (41% vs. 12%) and cachexia (6.5% vs. 0%). A recent study in the United States of America showed a slight decrease in life expectancy compared to the general population (84.1 years vs. 87.5 years) (Shelly, 2021).

Palliation and end-of-life care

Few studies have addressed palliative care in IIM and other neuromuscular disorders. It can be difficult to recognize the last phase of life in patients with neuromuscular diseases, especially because clear definitions are lacking (Tripodoro, 2015; de Visser, 2017). In general, unfavorable prognostic factors are severe diminishment of pulmonary function (in particular vital capacity and peak cough flow), substantial weight loss, recurrent pulmonary infections and severe dysphagia (Carter, 2012).

Comorbid disease can influence prognosis, in particular in case of paraneoplastic dermatomyositis or in case of cardiovascular disease and sleep apnea which are often encountered in IBM.

In patients with JDM and IIM who are treated with immunosuppressive and/or immunomodulatory medication it can sometimes be difficult to assess whether a further treatment response can be expected, or whether symptoms are refractory.

In patients with end stage disease, palliative sedation or euthanasia can be considered if the appropriate conditions apply. In Dutch IBM patients, palliative sedation or euthanasia were applied in 13% of cases (Cox, 2011).