Real-world effectiveness of autologous haematopoietic stem cell transplantation for multiple sclerosis in the UK
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Le résumé fourni par la source
BACKGROUND: Autologous haematopoietic stem cell transplantation (AHSCT) is increasingly used as a one-off disease-modifying therapy for aggressive forms of multiple sclerosis (MS). We report real-world effectiveness of AHSCT for MS in the UK. METHODS: This retrospective open-label study included patients with (pw)MS treated with AHSCT between 2002 and 2023 in 14 UK centres. Outcomes included relapse-free survival (RFS), MRI activity-free survival (MFS), progression-free survival (PFS) and no evidence of disease activity (NEDA-3). We assessed 6-month confirmed Expanded Disability Status Scale (EDSS) score progression or improvement compared with pre-treatment. Treatment-related mortality (TRM) was defined as death from any cause within 100 days post-autologous graft reinfusion. RESULTS: 364 pwMS were included (median age 40 years; 58% female). Of these, 271 pwMS had adequate neurological follow-up data: 168 (62%) had relapsing-remitting MS (pwRRMS) and 103 (38%) had progressive MS (pwPMS). Median disease duration from symptom onset was 10 years (IQR 6-14), EDSS 6 (IQR 4.0-6.5) and follow-up from AHSCT 46 months. At 2 and 5 years from AHSCT, RFS was 94.6% and 88.6%; MFS 93.1% and 80.1%; PFS 83.5% and 62.4%; NEDA-3 72.3% and 46.2%. pwRRMS had significantly higher rates of PFS (p=0.007) and NEDA-3 (p=0.001) than pwPMS. RRMS was a predictor of EDSS improvement, whose prevalence was 24.2% at 2 years and 20.4% at 5 years. TRM was 1.4% (n=5/364). CONCLUSIONS: In this cohort with high EDSS at baseline and including pwPMS, AHSCT led to durable remission of inflammatory activity and stabilisation or improvement of neurological disability, particularly in pwRRMS.
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Le contrôle bibliographique ouvert
DOI retrouvé dans Crossref DOI retrouvé ; titre concordant.
- Titre Crossref
- Real-world effectiveness of autologous haematopoietic stem cell transplantation for multiple sclerosis in the UK
- Date Crossref
- 05/09/2025
- Éditeur
- BMJ
- Type
- journal-article
Ce recoupement confirme des métadonnées liées au DOI. Il ne confirme ni la méthode ni les conclusions de l’étude, et il ne compte pas comme une seconde source scientifique indépendante.
Où se fait cette recherche
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Imperial College Healthcare NHS Trust Neurology pays non établi dans la noticeÉtablissement de santé
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Imperial College London pays non établi dans la noticeUniversité ou école supérieure
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Guy's and St Thomas' NHS Foundation Trust pays non établi dans la noticeÉtablissement de santé
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King's College Hospital NHS Foundation Trust pays non établi dans la noticeÉtablissement de santé
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Sheffield Teaching Hospitals NHS Foundation Trust Department of Haematology pays non établi dans la noticeOrganisme public
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NIHR Sheffield Biomedical Research Centre pays non établi dans la noticeStructure de recherche
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University of Sheffield Institute for Translational Neuroscience pays non établi dans la noticeUniversité ou école supérieure
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University of Florence Department of Neurosciences pays non établi dans la noticeUniversité ou école supérieure
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Queen Mary University of London pays non établi dans la noticeUniversité ou école supérieure
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National Institute for Health and Care Research pays non établi dans la noticeOrganisme public
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National Hospital for Neurology and Neurosurgery pays non établi dans la noticeÉtablissement de santé
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UCL Queen Square Institute of Neurology pays non établi dans la noticeUniversité ou école supérieure
Neurology — Imperial College Healthcare NHS Trust, Imperial College London et Guy's and St Thomas' NHS Foundation Trust, avec 9 autres affiliations.
Une affiliation ne permet pas de déduire la nationalité d’un auteur.