Submitted by: Pritikanta Paul, MD
Edited by: Elliot Bodofsky, MD
Datta, et al.
Retrospective analysis of eculizumab in patients with acetylcholine receptor antibody-negative myasthenia gravis: A case series. Journal of Neuromuscular Diseases. 2020;7:269-277.
Summary: Eculizumab has been approved and used for AChR antibody positive myasthenia gravis but little is known about its efficacy for AChR seronegative cases. In this small retrospective study (N=6), treatment outcome with eculizumab for AChR negative patients was assessed. Clinically meaningful reductions (≥2 points) in total MG-ADL scores were observed before or at five months and were maintained to month twelve in all patients. There was also a reduction in the mean number of exacerbations per patient, before and after eculizumab initiation, respectively and no treatment related adverse effects.
Comments: There is preliminary evidence of a potential beneficial role of eculizumab in treatment refractory seronegative myasthenia gravis warranting larger randomized trials.