Haematopoietic stem cell transplantation for treatment of relapsing-remitting multiple sclerosis in Sweden: an observational cohort study.

Autor:	Silfverberg T; Medical Sciences, Uppsala universitet Medicinska och farmaceutiska vetenskapsomradet, Uppsala, Sweden.; Center for Clinical Research Dalarna, Falun, Sweden., Zjukovskaja C; Department of Medical Sciences, Uppsala University, Uppsala, Sweden., Ljungman P; Department of Medicine Huddinge, Karolinska Institutet, Stockholm, Sweden.; Department of Cellular Therapy and Allogeneic Stem Cell Transplantation, Karolinska Comprehensive Cancer Center, Karolinska University Hospital Huddinge, Stockholm, Sweden., Nahimi A; Department of Neurology, Rehabilitation Medicine, Memory Disorders, and Geriatrics, Skåne University Hospital Lund, Lund, Sweden., Ahlstrand E; Department of Medicine, Faculty of Medicine and Health, Örebro University, Örebro, Sweden., Dreimane A; Department of Hematology, Linköping University Hospital, Linkoping, Sweden., Einarsdottir S; Institute of Medicine, University of Gothenburg, Gothenburg, Sweden.; Department of Hematology and Coagulation, Sahlgrenska Sjukhuset, Gothenburg, Sweden., Fagius J; Department of Medical Sciences, Uppsala University, Uppsala, Sweden., Iacobaeus E; Department of Clinical Neuroscience, Karolinska Institutet, Stockholm, Sweden.; Department of Neurology, Karolinska University Hospital, Stockholm, Sweden., Hägglund H; Department of Medical Sciences, Uppsala University, Uppsala, Sweden.; Department of Cellular Therapy and Allogeneic Stem Cell Transplantation, Karolinska Comprehensive Cancer Center, Karolinska University Hospital Huddinge, Stockholm, Sweden., Lange N; Department of Medicine, Faculty of Medicine and Health, Örebro University, Örebro, Sweden., Lenhoff S; Department of Hematology, Oncology & Radiophysics, Skåne University Hospital Lund, Lund, Sweden., Lycke J; Department of Clinical Neuroscience, Institute of Neuroscience and Physiology, Sahlgrenska Academy, Gothenburg, Sweden., Mellergård J; Department of Neurology, Linköping University, Linköping, Sweden.; Department of Biomedical and Clinical Sciences, Linköping University, Linköping, Sweden., Piehl F; Department of Clinical Neuroscience, Karolinska Institutet, Stockholm, Sweden.; Department of Neurology, Karolinska University Hospital, Stockholm, Sweden., Svenningsson A; Department of Clinical Sciences, Karolinska Institutet Institutionen för kliniska vetenskaper Danderyds sjukhus, Stockholm, Sweden.; Department of Neurology, Karolinska Institutet Institutionen för kliniska vetenskaper Danderyds sjukhus, Stockholm, Sweden., Tolf A; Department of Medical Sciences, Uppsala University, Uppsala, Sweden., Cherif H; Department of Medical Sciences, Uppsala University, Uppsala, Sweden., Carlson K; Department of Medical Sciences, Uppsala University, Uppsala, Sweden., Burman J; Department of Medical Sciences, Uppsala University, Uppsala, Sweden joachim.burman@uu.se.
Jazyk:	angličtina
Zdroj:	Journal of neurology, neurosurgery, and psychiatry [J Neurol Neurosurg Psychiatry] 2024 Jan 11; Vol. 95 (2), pp. 125-133. Date of Electronic Publication: 2024 Jan 11.
DOI:	10.1136/jnnp-2023-331864
Abstrakt:	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. Competing Interests: Competing interests: EI has received speakers fee from Merck and honoraria from advisory boards for Sanofi-Aventis, Biogen and Merck. FP previously received research grants from Merck KGaA, Janssen and UCB outside this study. FP has received payment for expert testimony from Novartis. FP has participated in Data Monitoring Committee for clinical trials from Chugai, Lundbeck and Roche. JM has received lecture honorarium from Merck. NL has received honoraria from Sanofi. All other individual authors declare that there is no conflict of interest. (© Author(s) (or their employer(s)) 2024. Re-use permitted under CC BY-NC. No commercial re-use. See rights and permissions. Published by BMJ.)
Databáze:	MEDLINE
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