This protocol addresses patients with generalized myasthenia gravis who test positive for antibodies to acetylcholine receptors (AChR-Ab+). This antibody status is the defining feature of the patient population and is central to treatment decisions, particularly in the refractory setting.
Prior steroid-sparing immunosuppressive therapy — including methotrexate — did not achieve or maintain minimal manifestation (MM) status and did not achieve the required reduction in prednisone dose. Failure to reach these targets is the trigger for escalation to the next treatment line described here.
In this refractory setting, rituximab is among the options considered for patients who have not responded to prior immunosuppressive agents.
The clinical target for this protocol is to achieve minimal manifestation (MM) status or better.
DOI: 10.1212/WNL.0000000000011124
In nonthymomatous, generalized MG patients with AChR-Ab, aged 18–50 years, thymectomy should be considered early in the disease to improve clinical outcomes and to minimize immunotherapy requirements and the need for hospitalizations for disease exacerbations.
Eculizumab should be considered in the treatment of severe, refractory, AChR-Ab+ generalized MG.
The efficacy of RTX in refractory AChR-Ab+ MG is uncertain. It is an option if patients fail or do not tolerate other IS agents.
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