Researchers recently published the 5-year results of the HALT-MS study, reporting that 69% of patients who received high-dose immunosuppressive therapy after autologous hematopoietic stem cell transplant (HDIT/HCT) experienced no disability progression, relapse, or new brain lesions associated with relapsing-remitting multiple sclerosis (RRMS).1
“These extended findings suggest that one-time treatment with HDIT/HCT may be substantially more effective than long-term treatment with the best available medications for people with a certain type of MS,” said National Institute of Allergy and Infectious Diseases (NIAID) director Anthony S. Fauci, MD, in an NIH news release.2 “These encouraging results support the development of a large randomized trial to directly compare HDIT/HCT to standard of care for this often-debilitating disease.”
The trial included 24 participants with RRMS who underwent HDIT/HCT. The primary end point was “event-free survival,” which the investigators defined as “survival without death or disease activity from any one of: disability progression, relapse, or new lesions on MRI.” After a median follow-up of 62 months, the rate of event-free survival was 69.2%, with progression-free survival, clinical relapse-free survival, and MRI activity-free survival at 91.3%, 86.9%, and 86.3%, respectively. Improvements of -0.5 in Expanded Disability Status Scale score were also noted in patients who survived and completed the study.
Adverse events included cytopenias and infections related to the toxic effects of the high-dose chemotherapy used for immunosuppression. Of note, no acute neurologic adverse effects occurred. Three patients who experienced disease progression over the course of follow-up died, although the deaths were not attributed to treatment.
- Nash RA, Hutton GJ, Racke MK, et al. High-dose immunosuppressive therapy and autologous HCT for relapsing-remitting MS [published online February 1, 2017]. Neurology. doi: 10.1212/WNL.0000000000003660
- National Institutes of Health. Stem cell transplants may induce long-term remission of multiple sclerosis [news release]. National Institutes of Health newsroom; February 1, 2017. www.nih.gov/news-events/news-releases/stem-cell-transplants-may-induce-long-term-remission-multiple-sclerosis. Accessed February 8, 2017.