Long-term Clinical Outcomes of Hematopoietic Stem Cell Transplantation in Multiple Sclerosis

Objective To determine whether autologous hematopoietic stem cell transplantation (aHSCT) is able to induce durable disease remission in people with multiple sclerosis (MS), we analyzed the long-term outcomes after a large cohort of patients MS. Methods be included, minimum dataset (consisting age, MS phenotype, Expanded Disability Status Scale [EDSS] score at baseline, information on technology, and least 1 follow-up visit transplantation) was required. Results Two hundred ten were included (relapsing-remitting [RR] 122 [58%]). Median baseline EDSS 6 (1–9); mean 6.2 (±5.0) years. Among RRMS, disability worsening–free survival (95% confidence interval [CI]) 85.5% (76.9%–94.1%) 5 years 71.3% (57.8%–84.8%) 10 In progressive MS, 71.0% (59.4%–82.6%) 57.2% (41.8%–72.7%) years, respectively. significantly reduced aHSCT ( p = 0.001; change per year ?0.09 [95% CI ?0.15% ?0.04%]). use BCNU+Etoposide+Ara-C+Melphalan (BEAM) + anti-thymocyte globulin (ATG) conditioning protocol independently associated risk no evidence activity 3 failure (hazard ratio 0.27 0.14–0.50], < 0.001). Three died within 100 days from (1.4%); deaths occurred transplanted 2007. Conclusions prevents worsening majority induces improvement RRMS. The BEAM ATG more pronounced suppression clinical relapses MRI inflammatory activity. Classification Evidence This study provides Class IV that for most patients.