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Effect of nonmyeloablative hematopoietic stem cell transplantation vs continued disease-modifying therapy on disease progression in patients with relapsing-remitting multiple sclerosis: A randomized clinical trial

JAMA Jan 18, 2019

Burt RK, et al. - In this study, 110 patients with highly active relapsing-remitting multiple sclerosis were assessed between September 20, 2005 and July 7, 2016 for the comparative impact of nonmyeloablative hematopoietic stem cell transplantation (HSCT) vs disease-modifying therapy (DMT) on disease progression. They observed mean Expanded Disability Status Scale (EDSS) scores decreased (improved) from 3.38 to 2.36 in the HSCT group and increased (worsened) from 3.31 to 3.98 in the DMT group during the first year. They recorded no development of nonhematopoietic grade 4 toxicities (myocardial infarction, sepsis, or other disabling or potential life-threatening events) in candidates who received HSCT. No death was noted among subjects who received HSCT during that period too. In this preliminary study, nonmyeloablative HSCT vs DMT resulted in prolonged time to disease progression.

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