The aim of this pilot study is to investigate the application of proteasome inhibitor Bortezomib (Velcade®, approved for therapy of multiple myeloma) in patients with therapy-refractory antibody-mediated autoimmune diseases. The investigators hypothesis is that the proteasome inhibition will lead to reduced antibody titers and improved clinical outcome.
Study Type
INTERVENTIONAL
Allocation
NA
Purpose
TREATMENT
Masking
NONE
Enrollment
11
Bortezomib will be subcutaneously applicated in 2 treatment cycles with 4 injections of 1.3 mg Bortezomib /m2 body surface per cycle.
Charite - Universitätsmedizin Berlin, NeuroCure Clinical Research Center
Berlin, Germany
Charité - Universitätsmedizin Berlin, Internal Medicine / Rheumathology
Berlin, Germany
change in disease specific antibody titers after application of Bortezomib
Change in disease specific antibody titers (anti-ACh for myasthenia gravis, anti-dsDNA for systemic lupus erythematosus, anti-ACPA for rheumatoid arthritis) 6 months after end of Bortezomib therapy (duration 6 weeks) compared to baseline (before therapy).
Time frame: 6 months after end of therapy (6 weeks) compared to baseline (before therapy)
Change in disease specific antibody titer after Bortezomib application
Change in disease specific antibody titer after Bortezomib application (except at time point 6 months after end of therapy = primary outcome measure)
Time frame: at regular intervals up to 30 weeks compared to baseline
Change in quality of life (Qol score)
Time frame: at regular intervals up to 30 weeks compared to baseline
Change in Activities of Daily Living (Adl score)
Time frame: at regular intervals up to 30 weeks compared to baseline
change in dose of immunosuppressive co-medication
Time frame: at regular intervals up to 30 weeks compared to baseline
Change in titers of protective antibodies (e.g. measles)
Change in titers of protective antibodies against measles virus, rubella virus, varicella zoster virus, pneumococcus, cytomegalovirus
Time frame: at regular intervals up to 30 weeks compared to baseline
Change in number of antibody producing plasmablasts/cells
Change in number of antibody producing plasmablasts/cells in peripheral blood
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Time frame: at regular intervals up to 30 weeks compared to baseline
Change in concentration of soluble mediators (e.g. IL-6)
Change in concentration of soluble mediators (e.g. IL-6) in peripheral blood
Time frame: at regular intervals up to 30 weeks compared to baseline
need for hospitalisation
Time frame: at regular intervals up to 30 weeks