ANCA-associated vasculitis anti-GBM disease and cryoglobulinemic vasculitis (vasculitis = small vessel inflammatory disease) Myasthenia Gravis
Conditions
Interventions
None listed
Sponsors
Universiteit Maastricht
Eligibility
Age
2 Years to 99 Years
Inclusion criteria
Inclusion criteria: In order to be eligible to participate in this study, a subject must meet the following criteria: #Patients with a new or previous diagnosis of: * Anti-AChR or anti-MuSK Myasthenia Gravis * MPO- or PR3-ANCA associated vasculitis * Anti-GBM glomerulonephritis * Cryoglobulinemia vasculitis with severe organ involvement * Refractory and severe antibody-mediated diseases* # Patients with an indication for plasma exchange or IA based on: 1. AChR or MuSK-positive MG with at least one of the following features: - Insufficient clinical response to immunosuppressive therapy of severe ocu-lar, bulbar or generalized muscle weakness. - Exacerbation with severe bulbar or respiratory dysfunction. 2. MPO- or PR3-ANCA vasculitis defined at least by one of the following features: - Renal involvement indicated by renal biopsy and/or glomerular erythrocy-turia WITH active glomerulonephritis and newly reported eGFR 10 IU/ml). - Serological evidence of circulating anti-GBM autoantibodies (> 10 IU/ml). 4. Cryoglobulinemia vasculitis with at least one of the following features: - Symptomatic hyperviscosity syndrome. - (Life-threatening) organ involvement (e.g. renal involvement (proven by renal biopsy and newly reported eGFR
Exclusion criteria
Exclusion criteria: - Pregnancy at time of study entry. - Previously reported allergic reactions to the immunosuppressive therapies or IA. - Plasmapheresis or IA within 3 months before inclusion. - Intravenous immunoglobulin within 3 months before inclusion. - Patients aged 7 days of oral cyclophosphamide or >1 IV dose of cyclophos-phamide within 3 months prior to inclusion and/or >7 days of predni-sone/prednisolone (>30 mg/day or > 1 mg/kg/day in paediatric patients) within 1 month prior to inclusion expect for patients with MG and/or >1 dose of rituximab within 9 months prior to inclusion.
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| The main study parameter is the change in immune phenotype over time in patients with B-cell mediated autoimmune disease, prior, and in response to immune suppression and IA as part of their standard treatment. The course of plasma levels of disease specific autoantibodies (e.g. anti-GBM, PR3- or MPO-ANCA) will be monitored for 12 months on a regular basis. Moreover, autoantibody typing (IgG isotypes, IgA, IgM) and characteristics (e.g. glycosylation/galactosylation profiles) as well as characteristics of B- and T-cells and plasma cell subsets, in-vitro profiles (e.g. PBMC activation and Ig release) and antibody effector functions will be analysed before and during the follow-up of this study. Clinical parameters and questionnaires will be collected to correlate the clinical course with differences in the immune phenotype. The results of antibody tests and cell subset data will be compared between patients with the same disease and between different diseases. If feasible such as in AAV, these data will be also compared to a matched disease control group with a similar immunosuppressive therapy but without an indication for add-on IA. | — |
Secondary
| Measure | Time frame |
|---|---|
| . | — |
Countries
Netherlands
Outcome results
None listed