Efficacy and Safety of Low-dose Rituximab in the Treatment of Refractory Myasthenia Gravis
Status:
Recruiting
Trial end date:
2022-07-01
Target enrollment:
Participant gender:
Summary
This study is designed to evaluate the effectiveness and safety of low-dose rituximab in
patients with refractory myasthenia gravis.
The traditional treatment of MG is immunosuppressive therapy, usually beginning with
corticosteroids. However, up to 70% of treated patients show an incomplete response,
including 10 - 30% who are unresponsive. Corticosteroids and other immunosuppressive
therapies presented also many side effects. The investigators propose to evaluate in a pilot,
open, prospective, single central study, the interest of rituximab (RTX) in the treatment of
patients with refractory MG. Fifty patients with refractory MG will be included in the study
and divided into two stages: 14 patients in the first stage were followed up after using RTX
according to the study protocol. The study will move into the second stage on if the number
of effective cases is greater than 3, otherwise, the study will be discontinued (based on
Simon's Optimal Two-stage Design). The remaining 36 patients were enrolled in the second
stage. In the first and second stages, the treatment plan and follow-up plan were consistent.
The therapeutical schema is rituximab 100-200 mg/week for a total of 2-4 times, followed by
adequate organ function, laboratory parameters and assessment of MG after each injection and
end of follow up for 24 weeks.