No abstract

Rituximab is a chimeric mouse/human anti-CD20 monoclonal immunoglobulin. We reviewed the efficacy and safety of rituximab in 169 myasthenia gravis (MG) patients from case reports and series. Antibodies to the acetylcholine receptor (AChR) were present in 59% and muscle-specific tyrosine kinase (MuSK) in 34%. Modified Myasthenia Gravis Foundation of America postintervention scale of minimal manifestations (MM) or better occurred in 44%, and combined pharmacologic and chronic stable remission in 27% overall; MM or better was achieved in 72% of MuSK MG and 30% of AChR MG (P < 0.001). Posttreatment relapses decreased more in MuSK MG (P = 0.05). Response predictors were MuSK MG, less severe disease, and younger age at treatment. Among a responder subset, 26% of AChR and 82% of MuSK MG patients showed decreased posttreatment antibody titers. Rituximab was generally well tolerated. Detectable serum rituximab and depleted CD20 B-cells were observed up to 20 and 16 weeks, respectively, after 4 weekly infusions. Muscle Nerve 56: 185-196, 2017.

This study provides Class IV evidence that for patients with anti-MuSK MG, rituximab increased the probability of a favorable outcome.

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Objective We evaluated the safety, tolerability, and effectiveness of prednisone in patients with ocular myasthenia gravis (OMG) concurrently treated with pyridostigmine. Methods Randomized, double-blind, placebo-controlled trial. Participants whose symptoms failed to remit on pyridostigmine were randomized to receive placebo or prednisone, initiated at 10mg every other day and titrated to a maximum of 40mg/day over 16-weeks. Primary outcome measure was treatment failure. Results Fewer subjects were randomized than the 88 planned. Of the 11 randomized, 9 completed 16-weeks of double-blind therapy. Treatment failure incidence was 100% (95% CI 48–100%) in the placebo group (n=5) vs. 17% (95% CI 0–64%) in the prednisone group, P = 0.02 (n=6). Median time to sustained minimal manifestation status (MMS) was 14-weeks, requiring an average prednisone dosage of 15mg/day. Adverse events were infrequent and generally mild in both groups. Conclusions A strategy of low-dose prednisone with gradual escalation appears to be safe, well tolerated, and effective in treating OMG.