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Haematopoietic stem cell transplantation for treatment of relapsing-remitting multiple sclerosis in Sweden: an observational cohort study
Uppsala University, Disciplinary Domain of Medicine and Pharmacy, Disciplinary Domain of Medicine and Pharmacy, research centers etc., Center for Clinical Research Dalarna. Uppsala University, Disciplinary Domain of Medicine and Pharmacy, Faculty of Medicine, Department of Medical Sciences, Haematology.ORCID iD: 0000-0002-3829-285X
Uppsala University, Disciplinary Domain of Medicine and Pharmacy, Faculty of Medicine, Department of Medical Sciences, Experimental neurology.ORCID iD: 0000-0003-0934-4478
Karolinska Inst, Dept Med Huddinge, Stockholm, Sweden.;Karolinska Univ, Karolinska Comprehens Canc Ctr, Dept Cellular Therapy & Allogene Stem Cell Transpl, Hosp Huddinge, Stockholm, Sweden..
Skane Univ Hosp, Dept Neurol Rehabil Med Memory Disorders & Geriatr, Lund, Sweden..
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2024 (English)In: Journal of Neurology, Neurosurgery and Psychiatry, ISSN 0022-3050, E-ISSN 1468-330X, Vol. 95, no 2, p. 125-133Article in journal (Refereed) Published
Abstract [en]

Background A growing evidence base supports the use of autologous haematopoietic stem cell transplantation (aHSCT) for treatment of relapsing-remitting multiple sclerosis (RRMS), but it has not yet been integrated into most national clinical guidelines. The objective of this study was to assess efficacy and safety when aHSCT is implemented in routine healthcare.

Methods We assessed 231 patients and the final analysis included 174 RRMS patients who were treated with aHSCT in Sweden before 1 January 2020. Efficacy was evaluated by performing a retrospective analysis of prospectively collected data from the Swedish MS registry. Procedure-related safety was assessed by analysing data from electronic patient records covering a period of 100 days following aHSCT.

Results With a median follow-up time of 5.5 (IQR: 3.4–7.5) years, the Kaplan-Meier estimate for no evidence of disease activity was 73% (95% CI 66% to 81%) at 5 years and 65% (95% CI 57% to 75%) at 10 years. Out of the 149 patients with baseline disability, 80 (54%) improved, 55 (37%) were stable and 14 (9%) deteriorated. The mean number of adverse events per patient was 1.7 (±SD: 1.5) for grade 3 events and 0.06 (±SD: 0.3) for grade 4 events. Febrile neutropenia was the most common adverse event, affecting 68% of patients. There was no treatment-related mortality.

Conclusions Treatment with aHSCT for RRMS is associated with freedom from disease activity in a majority of patients, with acceptable adverse events. This procedure should be considered a standard of care for patients with highly active RRMS.

Place, publisher, year, edition, pages
BMJ Publishing Group Ltd, 2024. Vol. 95, no 2, p. 125-133
Keywords [en]
multiple sclerosis, haematology, clinical neurology
National Category
Neurology
Identifiers
URN: urn:nbn:se:uu:diva-528598DOI: 10.1136/jnnp-2023-331864ISI: 001072638600001PubMedID: 37748927OAI: oai:DiVA.org:uu-528598DiVA, id: diva2:1860705
Funder
Marianne and Marcus Wallenberg FoundationRegion StockholmSwedish Research CouncilSwedish Society for Medical Research (SSMF), 2020-02700Swedish Society of MedicineAvailable from: 2024-05-24 Created: 2024-05-24 Last updated: 2024-05-24Bibliographically approved

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Silfverberg, ThomasZjukovskaja, ChristinaEinarsdottir, SigrunFagius, JanHägglund, HansTolf, AndreasCherif, HonarCarlson, KristinaBurman, Joachim
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