Safety and efficacy of autologous hematopoietic stem cell transplantation for multiple sclerosis in Norway. (December 2020)

• Record Type: Journal Article
• Authors: Kvistad, Silje Agnethe Stokke; Lehmann, Anne Kristine; Trovik, Linn Hereide; Kristoffersen, Einar Klæbo; Bø, Lars; Myhr, Kjell-Morten; Torkildsen, Øivind
• Is Part Of: Multiple sclerosis. Volume 26:Number 14(2020)
• Page Start: 1889
• Page End: 1897
• Publication Date: 2020-12
• Subjects: Multiple sclerosis -- relapsing remitting -- no evidence of disease activity status -- hematopoietic stem cell treatment; Central nervous system -- Diseases -- Periodicals; Myelin sheath -- Diseases -- Periodicals; Inflammation -- Periodicals; Multiple sclerosis -- Periodicals; Central Nervous System Diseases -- Periodicals; Demyelinating Diseases -- Periodicals; Inflammation -- Periodicals; Multiple Sclerosis -- Periodicals; Système nerveux central -- Maladies -- Périodiques; Gaine de myéline -- Maladies -- Périodiques; Inflammation (Pathologie) -- Périodiques; Sclérose en plaques -- Périodiques; Electronic journals; 616.834005
• DOI: 10.1177/1352458519893926
• Languages: English
• ISSNs: 1352-4585

Abstract

Background: Hematopoietic stem cell treatment (HSCT) is a promising treatment option for multiple sclerosis (MS), but detailed safety and efficacy measures are still scarce. Objective: To evaluate the efficacy and safety of HSCT in MS. Methods: Retrospective single-center observational study of all MS patients that underwent HSCT in Norway during January 2015 to January 2018. The primary outcome was no evidence of disease activity (NEDA-3) status. Results: A total of 30 patients with a median follow-up time of 26 months (range: 11–48) were evaluated. In total, 25 (83%) achieved NEDA-3 status, and none received disease-modifying treatment after HSCT. For 13 (43%) of the patients, there were sustained improvement in Expanded Disability Status Scale (EDSS) score, and 10 (33%) were working full time after the treatment, compared to only 1 (3%) before treatment. There were no serious treatment-related complications and was no mortality. Five patients (17%) were diagnosed with an autoimmune thyroid disease after the procedure, and 10 (43%) of the women had amenorrhea lasting >12 months and symptoms of ovarian failure. Conclusion: HSCT in MS is an effective and relatively safe treatment option, with few serious complications and no mortality in Norway, so far. However, long-term adverse event with amenorrhea is a common problem.