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.
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.
There is preliminary evidence of a potential beneficial role of eculizumab in treatment refractory seronegative myasthenia gravis warranting larger randomized trials.