AUTOLOGOUS HEMATOPOIETIC STEM CELL TRANSPLANTATION TO TREAT MYASTHENIA GRAVIS: EXPERIENCE OF A DEVELOPING COUNTRY

Abstract

Introduction. Patients with refractory myasthenia gravis (MG) have been successfully treated with stem cell transplantation in all reported cases worldwide. The objective of the study was to describe our first experience with autologous hematopoietic stem cell transplantation (aHSCT) to treat patients with refractory MG. Material and methods. Autologous HSCT was used to treat 4 MG patients. MG disease activity, intervention status along with method-related adverse events following aHSCT were evaluated. MG disease activity was accessed using the Myasthenia Gravis Composite (MGC) score. Results. 4 MG patients underwent aHSCT. The ratio of females to males was 3:1. The median ages at the time of diagnosis and transplantation were 32.5 and 43.3 years, respectively. The mean disease duration before aHSCT was 10.75 years, with a poor response to conventional treatment. After aHSCT, the clinical symptoms of all four patients have remarkably improved. The average MGC score of patients before aHSCT was 24, reduced significantly after transplantation. Anti-acetylcholine receptor antibodies concentration wasn't changed significantly following aHSCT. Three patients encountered temporary acute side effects. The oldest patient had infectious complications with Klebsiella pneumonia, Cytomegalovirus, and BK infection reactivation. Three patients had completely discontinued MG medications, while one patient with shorter follow-up durations was still using low-dose anticholinesterase medications. Conclusions. Our initial findings regarding the efficacy and safety of aHSCT in MG patients are highly encouraging and comparable to previous reports.