2025 American Academy of Neurology Abstract Website

Gavin Brittain¹, Eleonora de Matteis², Richard Nicholas², Eli Silber³, Varun Mehra⁴, Ian Gabriel⁵, Julia Lee⁶, Rachel Pearce⁶, Maria Pia Sormani⁷, Alessio Signori⁷, Ruth Paul⁶, Ram Malladi⁸, Victoria Potter⁶, John Snowden¹, Majid Kazmi⁶, Basil Sharrack¹, Paolo Muraro²
¹Sheffield Teaching Hospitals NHS Foundation Trust and The University of Sheffield, ²Imperial College London, ³King's Neuroscience Centre, ⁴King's College Hospital NHS Foundation Trust, ⁵Chelsea And Westminster Hospital NHS Foundation Trust, ⁶Kings College London, ⁷University of Genoa, ⁸Cambridge University Hospitals NHS Foundation Trust

Objective:

To report on the effectiveness and predictors of response to autologous haematopoietic stem cell transplantation (AHSCT) for the treatment of multiple sclerosis (MS) in the United Kingdom (UK).

Background:

AHSCT is an increasingly used treatment for aggressive forms of MS, the most common indication for any autoimmune disease.

Design/Methods:

Retrospective analysis of a cohort of patients with (pw)MS treated with AHSCT between 2002 and 2023 at 14 UK centres, excluding subjects in active trials. Effectiveness outcomes included relapse free survival (RFS), progression independent of relapse activity (PIRA), relapse associated worsening (RAW) and no evidence of disease progression (NEDA). A multivariate analysis was used to examine factors associated with PIRA and NEDA.

Results:

271 patients, median age of 40 years (range 19-66), of whom 153 (57%) were female, with a disease duration of 10 (range 6-14 years) and median EDSS of 6 (4-6.5) were included with a median follow up of 46 months (range 25-65). At baseline, 168 (62%) patients had relapsing-remitting MS (RRMS), 64 (24%) had secondary progressive MS and 39 (14%) had primary progressive MS.

RFS was 94% and 89% at 2 and 4 years after AHSCT. At the same timepoints, PIRA free survival was 91.4% and 68.7%, freedom from RAW was 99.2% and 99.2%, and NEDA was 72.0% and 54.7%, respectively. Patients with progressive MS had lower PIRA free survival (57.3%) than relapsing remitting MS (69.6%). Progressive MS was the only factor associated with PIRA (HR 1.68, 95% CI 1.05-2.69; p=0.032) and failure of NEDA (HR 1.62, 95% CI 1.13-2.33, p=0.009).

Conclusions:

In this real-world cohort, AHSCT led to near-complete suppression of relapses and durable suppression of clinical progression. Ongoing randomised controlled trials are comparing the efficacy of AHSCT with high efficacy disease modifying therapy in RRMS, including STAR-MS in the UK.