An updated review on the utility of hematopoietic stem cell transplant in the treatment of refractory myasthenia gravis

INTRODUCTION: Hematopoietic stem cell transplantation (HSCT) has been shown to be an effective treatment in many severe autoimmune diseases. There have been a number of reported cases of successful HSCT in severe, refractory myasthenia gravis (MG). OBJECTIVE: To review and summarize the current literature on the role of HSCT in the management of refractory MG. METHODS: Databases including PubMed, clinical trials.gov, and Cochrane Reviews were searched for research articles containing “hematopoietic stem cell transplantation,” “stem cell,” and “myasthenia gravis.” Articles were excluded if they were not written in English. RESULTS: A total of 16 patients across 8 publications were identified as having undergone HSCT in the setting of MG. Following HSCT, all patients had significant improvement in their MG status, with 11 achieving complete stable remission. Common adverse effects were mostly infection-related and included neutropenic fever, cytomegalovirus viremia or reactivation, mucositis, and upper respiratory tract infection. CONCLUSION: HSCT has the potential to provide sustained benefit for patients with MG who are refractory to conventional treatment. A large-scale prospective study is warranted to better define its role in the treatment of MG.