A report on seven cases of severe myasthenia gravis suggests that autologous hematopoietic stem cell transplantation (HSCT) may result in long-term remission. The report was published online April 4 in JAMA Neurology.
In this retrospective study, each patient who underwent autologous HSCT from January 1, 2001, through December 31, 2014, in the Ottawa Hospital Bone Marrow Transplant Programme Database was observed. Lead author Adam Bryant, MD, of the Division of Hematology at the University of Ottawa, Canada, and colleagues reported that every patient who received HSCT experienced relief of symptoms and a prolonged period of treatment-free remission. Prior to treatment, the condition of each patient in the study was labeled as moderate (grade III) to life threatening (grade V) by the Myasthenia Gravis Foundation of America's classification system.
Treatment consisted of high-dose chemotherapy and antilymphocyte antibodies to eliminate the autoreactive immune system, followed by an infusion of stem cells previously harvested from the patient. No unexpected acute toxic effects were observed, no patient was admitted to the ICU for treatment-related effects, and there were no treatment-related deaths. Viral reactivation occurred in three patients (43%) as a result of the initial immune dysregulation.
This study suggests that autologous HSCT is rigorous enough to eliminate autoreactivity and that it re-establishes a long-lasting immune system that is functional and self-tolerant, thus eliminating the need for maintenance immunosuppression or additional treatment, the researchers said. HSCT has effectively treated other severe autoimmune neurologic conditions and maybe an option for patients with myasthenia gravis who do not respond to conventional treatment.
In an accompanying editorial, Daniel Drachman, MD, of the Johns Hopkins School of Medicine in Baltimore, explained that HSCT "is a major undertaking, involving skilled and experienced management, hospitalization, and important risks."
Current therapies used for myasthenia gravis, including cyclophosphamide and monoclonal antibodies like rituximab, have led to clinical improvement, but complete remission, as was seen with HSCT, is rare.
—Adaeze Stephanie Onyechi