Haematopoietic stem cell transplantation for treatment of relapsing-remitting multiple sclerosis in Sweden : An observational cohort study
(2023) In Journal of Neurology, Neurosurgery and Psychiatry- Abstract
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... (More)
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.
(Less)
- author
- organization
- publishing date
- 2023
- type
- Contribution to journal
- publication status
- in press
- subject
- keywords
- CLINICAL NEUROLOGY, HAEMATOLOGY, MULTIPLE SCLEROSIS
- in
- Journal of Neurology, Neurosurgery and Psychiatry
- article number
- jnnp-2023-331864
- publisher
- BMJ Publishing Group
- external identifiers
-
- pmid:37748927
- scopus:85174506660
- ISSN
- 0022-3050
- DOI
- 10.1136/jnnp-2023-331864
- language
- English
- LU publication?
- yes
- additional info
- Publisher Copyright: © Author(s) (or their employer(s)) 2023. Re-use permitted under CC BY-NC. No commercial re-use. See rights and permissions. Published by BMJ.
- id
- 48351675-aed2-43af-ac24-7553fcf3ae26
- date added to LUP
- 2023-12-20 14:19:52
- date last changed
- 2024-04-19 00:55:41
@article{48351675-aed2-43af-ac24-7553fcf3ae26,
abstract = {{<p>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.</p>}},
author = {{Silfverberg, Thomas and Zjukovskaja, Christina and Ljungman, Per and Nahimi, Adjmal and Ahlstrand, Erik and Dreimane, Arta and Einarsdottir, Sigrun and Fagius, Jan and Iacobaeus, Ellen and Hägglund, Hans and Lange, Niclas and Lenhoff, Stig and Lycke, Jan and Mellergård, Johan and Piehl, Fredrik and Svenningsson, Anders and Tolf, Andreas and Cherif, Honar and Carlson, Kristina and Burman, Joachim}},
issn = {{0022-3050}},
keywords = {{CLINICAL NEUROLOGY; HAEMATOLOGY; MULTIPLE SCLEROSIS}},
language = {{eng}},
publisher = {{BMJ Publishing Group}},
series = {{Journal of Neurology, Neurosurgery and Psychiatry}},
title = {{Haematopoietic stem cell transplantation for treatment of relapsing-remitting multiple sclerosis in Sweden : An observational cohort study}},
url = {{http://dx.doi.org/10.1136/jnnp-2023-331864}},
doi = {{10.1136/jnnp-2023-331864}},
year = {{2023}},
}