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Long-term outcomes following stem cell transplant for multiple sclerosis

February 20, 2017

A new study published online by JAMA Neurology examines the long-term outcomes of patients with aggressive forms of multiple sclerosis (MS) who failed to respond to standard therapies and who underwent autologous hematopoietic stem cell transplantation using their own stem cells.

More than 2.3 million people in the world are affected by MS, which can cause severe neurological disability. Autologous hematopoietic stem cell transplantation (AHSCT) has been investigated as a treatment for aggressive MS, the rationale for which is immune reconstitution. It is important to examine the course of MS after AHSCT over the long term.

The current study by Paolo A. Muraro, M.D., of Imperial College London, and coauthors included data from 13 countries on 281 patients who underwent AHSCT between 1995 and 2006. Primary outcomes examined by the study were MS progression-free survival and overall survival.

Eight deaths (2.8 percent) were reported within 100 days of transplant and were considered transplant-related. Transplant-related death is a major concern for a disease, such as MS, which is not life threatening. The authors suggest the 2.8 percent death rate in the current study likely reflects the early experience with AHSCT because only transplants performed through 2006 were included.

Additionally, MS progression-free survival was 46 percent at five years after AHSCT, with younger age, a relapsing form of MS, use of fewer prior immunotherapies and lower neurological disability scores associated with better outcomes, according to the report.

The authors note some study limitations.

"In this large observational study of patients with MS treated with AHSCT, almost half of them remained free from neurological progression for five years after transplant. ... The results support the rationale for further randomized clinical trials of AHSCT for the treatment of MS," the article concludes.
-end-
(JAMA Neurol. Published online February 20, 2017. doi:10.1001/jamaneurol.2016.5867; available pre-embargo at the For The Media website.)

Editor's Note: The article contains conflict of interest and funding/support disclosures. Please see the article for additional information, including other authors, author contributions and affiliations, financial disclosures, funding and support, etc.

The JAMA Network Journals

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