This paper looks at the outcome of HSCT in the United Kingdom and I guess one question is what DMT can compare? NEDA is no relapse no progression and no MRI so how does the good news on no relapse, no progression and no MRI equate to about 55% having disease?
Muraro PA, Kazmi M, De Matteis E, Brittain G, Mariottini A, Nicholas R, Silber E, Mehra V, Gabriel I, Ciccarelli O, Lee J, Pearce R, Sormani MP, Signori A, Paul R, Malladi R, Potter V, Snowden J, Sharrack B.J Neurol Neurosurg Psychiatry. 2025 Sep 5:jnnp-2025-336755. doi: 10.1136/jnnp-2025-336755.
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
Source: multiple-sclerosis-research.org