Results from a preliminary study indicate that among patients with relapsing-remitting multiple sclerosis (RRMS), treatment with non-myeloablative hematopoietic stem cell transplantation (HSCT) is associated with improvement in measures of disability and quality of life.
The findings are published in the January 20 issue of JAMA.
Richard K. Burt, MD, Northwestern University Feinberg School of Medicine, Chicago, Illinois, and colleagues studied the association of non-myeloablative HSCT with neurological disability and other clinical outcomes in patients with RRMS (n = 123) or secondary-progressive MS (n = 28) treated between 2003 and 2014.
Outcome analysis was available for 145 patients with an average follow-up of 2.5 years. On a measure of disability (Expanded Disability Status Scale [EDSS] score), there was significant improvement in 41 patients (50% of patients tested at 2 years) and in 23 patients (64% of patients tested at 4 years).
“To our knowledge, this is the first report of significant and sustained improvement in the EDSS score following any treatment for MS,” the authors wrote.
Receipt of HSCT was also associated with improvement in physical function, cognitive function and quality of life. There was also improvement in the number of brain lesions associated with MS seen on magnetic resonance imaging (MRI). Four-year relapse-free survival was 80 percent and progression-free survival was 87 percent.
Patient selection is important in determining outcome, the researchers noted. “In the post hoc analysis, the EDSS score did not improve in patients with secondary-progressive MS or in those with disease duration longer than 10 years,” they wrote.
The authors acknowledged that the results of their study are limited because it was an observational study without a control group. “Definitive conclusions will require a randomized trial; however, this analysis provides the rationale, appropriate patient selection, and therapeutic approach for a randomized study,” the authors wrote.
In an accompanying editorial, Stephen L. Hauser, MD, University of California at San Francisco, San Francisco, California, wrote: “First, autologous HSCT does not appear to be effective against established progressive forms of MS and, absent new data, additional trials of these protocols are probably not indicated for patients with progressive MS. Second, immunosuppressive regimens that include HSCT appear to be effective against the relapsing-remitting form of MS, at least over several years of observation. However, it is by no means clear that the beneficial effects result from the infusion of stem cells rather than from the conditioning regimen. Given the availability of highly effective FDA [US Food and Drug Administration]-approved therapies against relapsing-remitting MS, it would seem reasonable to use these proven monotherapies in the clinical setting before considering complex HSCT regimens. Third, the mechanism of action of autologous HSCT in MS needs to be clarified. Fourth, it is important to remember that MS is a chronic disease, usually arising in young adults and lasting throughout the lifespan. Many important disability-related outcomes take many years or decades to develop. To understand the role of any therapy for MS, and especially an intensive regimen with uncertain long-term risk, very long follow-up periods are required to meaningfully assess if the disease has indeed been rebooted over the long-term, and also to increase confidence that these therapies have not caused undue harm.”</p>
