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Effectiveness of autologous haematopoietic stem cell transplantation versus natalizumab in progressive multiple sclerosis
T. Kalincik, S. Sharmin, I. Roos, J. Massey, I. Sutton, B. Withers, MS. Freedman, H. Atkins, E. Krasulova, E. Kubala Havrdova, M. Trneny, T. Kozak, J. Burman, R. Macdonell, Ø. Torkildsen, L. Bø, AK. Lehmann, B. Sharrack, J. Snowden
Language English Country England, Great Britain
Document type Journal Article, Observational Study, Comparative Study, Multicenter Study
NLK
ProQuest Central
from 1944-07-01 to 6 months ago
Nursing & Allied Health Database (ProQuest)
from 1944-07-01 to 6 months ago
Health & Medicine (ProQuest)
from 1944-07-01 to 6 months ago
Psychology Database (ProQuest)
from 1944-07-01 to 6 months ago
- MeSH
- Transplantation, Autologous * MeSH
- Multiple Sclerosis, Chronic Progressive * drug therapy therapy MeSH
- Adult MeSH
- Immunologic Factors therapeutic use MeSH
- Middle Aged MeSH
- Humans MeSH
- Natalizumab * therapeutic use MeSH
- Disability Evaluation MeSH
- Disease Progression MeSH
- Hematopoietic Stem Cell Transplantation * MeSH
- Treatment Outcome MeSH
- Check Tag
- Adult MeSH
- Middle Aged MeSH
- Humans MeSH
- Male MeSH
- Female MeSH
- Publication type
- Journal Article MeSH
- Multicenter Study MeSH
- Observational Study MeSH
- Comparative Study MeSH
BACKGROUND: Natalizumab was not shown to modify disability in progressive multiple sclerosis (MS). This matched observational study compared the effectiveness of autologous haematopoietic stem cell transplantation (AHSCT) with natalizumab in progressive MS. METHODS: Patients with primary/secondary progressive MS from seven AHSCT MS centres and the MSBase registry, treated with AHSCT or natalizumab, were matched on a propensity score derived from sex, age, Expanded Disability Status Scale (EDSS), number of relapses 12/24 months before baseline, time from MS onset, the most effective prior therapy and country. The pairwise-censored groups were compared on hazards of 6-month confirmed EDSS worsening and improvement, relapses and annualised relapse rates (ARRs), using Andersen-Gill proportional hazards models and conditional negative binomial model. RESULTS: 39 patients treated with AHSCT (37 with secondary progressive MS, mean age 37 years, EDSS 5.7, 28% with recent disability progression, ARR 0.54 during the preceding year) were matched with 65 patients treated with natalizumab. The study found no evidence for difference in hazards of confirmed EDSS worsening (HR 1.49, 95% CI 0.70 to 3.14) and improvement (HR 1.50, 95% CI 0.22 to 10.29) between AHSCT and natalizumab over up to 4 years. The relapse activity was also similar while treated with AHSCT and natalizumab (ARR: mean±SD 0.08±0.28 vs 0.08±0.25; HR 1.05, 95% CI 0.39 to 2.82). In the AHSCT group, 3 patients experienced febrile neutropenia during mobilisation, 9 patients experienced serum sickness, 6 patients required intensive care unit admission and 36 patients experienced complications after discharge. No treatment-related deaths were reported. CONCLUSION: This study does not support the use of AHSCT to control disability in progressive MS with advanced disability and low relapse activity.
CORe Department of Medicine University of Melbourne Parkville Victoria Australia
Department of Haematology 1st Faculty of Medicine Charles University Prague Czech Republic
Department of Haematology 3rd Faculty of Medicine Charles University Prague Czech Republic
Department of Haematology Haukeland University Hospital Bergen Norway
Department of Haematology Sheffield Teaching Hospitals NHS Foundation Trust Sheffield UK
Department of Haematology St Vincent's Hospital Sydney Darlinghurst New South Wales Australia
Department of Medical Sciences Neurology Uppsala University Uppsala Sweden
Department of Medicine Ottawa Hospital Research Institute Ottawa Ontario Canada
Department of Neurology Austin Health Melbourne Victoria Australia
Department of Neurology Haukeland University Hospital Bergen Norway
Department of Neurology St Vincent's Hospital Sydney Darlinghurst New South Wales Australia
Department of Oncology and Metabolism Medical School The University of Sheffield Sheffield UK
General University Hospital Prague Prague Czech Republic
Neuroimmunology Centre Department of Neurology Royal Melbourne Hospital Parkville Victoria Australia
St Vincent's Clinical School University of New South Wales Sydney New South Wales Australia
University Hospital Kralovske Vinohrady Prague Czech Republic
University of Melbourne Melbourne Victoria Australia
University of New South Wales Sydney New South Wales Australia
References provided by Crossref.org
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