@article{Muraro:2026:10.1136/jnnp-2025-336755,
author = {Muraro, P and Kazmi, M and De, Matteis E and Brittain, G and Mariottini, A and Nicholas, R and Silber, E and Mehra, V and Gabriel, I and Ciccarelli, O and Lee, J and Pearce, R and Pia, Sormani M and Signori, A and Paul, R and Malladi, R and Potter, V and Snowden, JA and Sharrack, B},
doi = {10.1136/jnnp-2025-336755},
journal = {Journal of Neurology, Neurosurgery and Psychiatry},
pages = {146--155},
title = {Real-world effectiveness of autologous haematopoietic stem cell transplantation for MS in the UK},
url = {http://dx.doi.org/10.1136/jnnp-2025-336755},
volume = {97},
year = {2026}
}

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TY  - JOUR
AB  - 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.
AU  - Muraro,P
AU  - Kazmi,M
AU  - De,Matteis E
AU  - Brittain,G
AU  - Mariottini,A
AU  - Nicholas,R
AU  - Silber,E
AU  - Mehra,V
AU  - Gabriel,I
AU  - Ciccarelli,O
AU  - Lee,J
AU  - Pearce,R
AU  - Pia,Sormani M
AU  - Signori,A
AU  - Paul,R
AU  - Malladi,R
AU  - Potter,V
AU  - Snowden,JA
AU  - Sharrack,B
DO  - 10.1136/jnnp-2025-336755
EP  - 155
PY  - 2026///
SN  - 0022-3050
SP  - 146
TI  - Real-world effectiveness of autologous haematopoietic stem cell transplantation for MS in the UK
T2  - Journal of Neurology, Neurosurgery and Psychiatry
UR  - http://dx.doi.org/10.1136/jnnp-2025-336755
VL  - 97
ER  - 

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