muscle inflammation myositis
Conditions
Interventions
Standaard regimen
- Week 0: IVIg 2g/kg
- Week 3: IVIg 1g/kg
- Week 6: IVIg 1g/kg
- Week 9: IVIg 1g/kg + start standaarbehandeling
Optiona regimen: in case of insufficient respons on evaluation at wee
Idiopathic inflammatory myopathies
Sponsors
Academisch Medisch Centrum
Eligibility
Age
18 Years to 64 Years
Inclusion criteria
Inclusion criteria: * Adult patients (age * 18 years);* Subacute-onset of disease (disease duration of * 9 months) of muscle symptoms;* Biopsy proven IIMs (see for diagnostic criteria Hoogendijk et al. 2004, note: ASS is considered a separate entity, but new criteria in which it has been included, has yet to be published). o Dermatomyositis o Non-specific myositis/antisynthetase syndrome o Immune-mediated necrotizing myopathy;* Minimal disability of: o MMT score reduction of 10% or lower and, o 2 other of the core set measures score abnormalities defined as: - Physician Global Activity 2 cm or more - Patient Global Activity 2 cm or more - Health Assessment Questionnaire 0.25 or more - Muscle enzymes more than 1.5 times upper limit of normal - Extra-Muscular Activity 2 cm or more
Exclusion criteria
Exclusion criteria: * IVIg treatment related: o Subjects who have received clinical relevant immunosuppressive medication (e.g. plasmapheresis, biologicals, immune therapy etc.) within the last 6 months with the exception of prednisone dosed as follows: * daily dose of 20mg or lower * used for 2 weeks or less * no evident clinical response;o history of thrombotic episodes within the 2 years prior to enrolment o known allergic reactions or other severe reactions to any blood-derived product o known IgA deficiency and anti-IgA serum antibodies o pregnancy (wish).;* Conditions that are likely to interfere with: o compliance (legal incompetent and/or incapacitated patients are excluded) or, o evaluation of efficacy (e.g. due to severe pre-existing disability as result of any other disease than IIM or language barrier).;* Lack of informed consent (IC)
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| The number of patients with clinical significant improvement, defined as *40 points improvement on a continuous, weighted score of 6 core set measures (a internationally validated and combined outcome measure of the International Myositis Assessment and Clinical Studies group) after 9 weeks of treatment. 1. Physician Global Activity 2. Patient Global Activity 3. Manual Muscle Testing 4. Health Assessment Questionnaire 5. Muscle enzymes 6. Extra-Muscular Activity | — |
Secondary
| Measure | Time frame |
|---|---|
| Efficacy of IVIg (measured 9 weeks after treatment) 1. Time to at least moderate improvement on the CIS 2. Minimal improvement (20-40 points) on each of the 6 IMACS CSMs a. Physician Global Activity b. Patient Global Activity c. Manual Muscle Testing d. Health Assessment Questionnaire e. Muscle enzymes f. Extra-Muscular Activity 3. Moderate improvement or more (*40 points) on each of the 6 IMACS CSMs a. Physician Global Activity b. Patient Global Activity c. Manual Muscle Testing d. Health Assessment Questionnaire e. Muscle enzymes f. Extra-Muscular Activity 4. The number of deterioration patients needing rescue therapy 5. Significant improvement in Academic Medical Center Linear Disability Scale (ALDS) 6. Significant improvement in Modified Rankin Scale (MRS) 7. Significant improvement of dysphagia (if present) on the Amyotrofic Lateral Sclerosis Severity Scale Swallowing (ALSSS-SW) 8. Significant improvement in dynamometric muscle strength 9. Significant improvement on the Rasch modified MRC Sum Score (Rasch-MRC) 10. Significant improvement on the EuroQol Group Health Questionnaire (EQ-5D-5L) 11. The number of participants with significant decrease of T2 weighted (T2)/short tau inversion recovery (STIR) hyperintensity of muscles and fascia on MRI 12. The number of patients with significant change of size and echo intensity of muscles and fascia on ultrasound (US) 13. The number of patients with change of highly expanded B-cell clones 14. RNA and RBM20 expression before and after IVIg treatment 15. galectin-9 and CXCL10 levels before and after IVIg treatment 16. relation between treatment respons and serum IgG Safety of IVIg (measured during the total duration of the study) The number of serious adverse events (SAEs) Feasibility of a future trial (assessed at end report) 1. Process a. recruitment rate b. retention rate 2. Resources a. Estimation of total time investment b. Estimation of total financial investment 3. Manage | — |
Countries
The Netherlands
Outcome results
None listed