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Abstract Details

Real-world Effectiveness and Predictors of Response to Autologous Haematopoietic Stem Cell Transplantation in MS: The UK Experience in 271 Patients Treated During 2000-2023
Multiple Sclerosis
S3 - Multiple Sclerosis: Clinical Trials (2:36 PM-2:48 PM)
009

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

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).

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.

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).

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.

Authors/Disclosures
Gavin Brittain, MBBS
PRESENTER
Dr. Brittain has nothing to disclose.
Eleonora De Matteis, MD Dr. De Matteis has nothing to disclose.
Richard S. Nicholas, FRCP (Imperial College Healthcare Trust) Dr. Nicholas has nothing to disclose.
Eli Silber, MD (King's Neuroscience Centre) Dr. Silber has nothing to disclose.
Varun Mehra (King's College Hospital NHS Foundation Trust) No disclosure on file
Ian Gabriel, MBBS, PhD Dr. Gabriel has nothing to disclose.
Julia Lee Ms. Lee has nothing to disclose.
Rachel M. Pearce Ms. Pearce has nothing to disclose.
Maria Pia Sormani (University of Genoa) Maria Pia Sormani has received personal compensation in the range of $500-$4,999 for serving as a Consultant for Biogen. Maria Pia Sormani has received personal compensation in the range of $500-$4,999 for serving as a Consultant for Roche. Maria Pia Sormani has received personal compensation in the range of $500-$4,999 for serving as a Consultant for Sanofi. Maria Pia Sormani has received personal compensation in the range of $500-$4,999 for serving as a Consultant for Merck. Maria Pia Sormani has received personal compensation in the range of $500-$4,999 for serving as a Consultant for Celgene. Maria Pia Sormani has received personal compensation in the range of $500-$4,999 for serving as a Consultant for Novartis. Maria Pia Sormani has received personal compensation in the range of $500-$4,999 for serving as a Consultant for Geneuro. Maria Pia Sormani has received personal compensation in the range of $500-$4,999 for serving as a Consultant for Immunic. Maria Pia Sormani has received personal compensation in the range of $5,000-$9,999 for serving on a Scientific Advisory or Data Safety Monitoring board for Medday. Maria Pia Sormani has received personal compensation in the range of $500-$4,999 for serving on a Scientific Advisory or Data Safety Monitoring board for Sanofi. Maria Pia Sormani has received personal compensation in the range of $500-$4,999 for serving on a Scientific Advisory or Data Safety Monitoring board for Roche.
Alessio Signori, PhD Prof. Signori has nothing to disclose.
ruth Paul, SDM Mrs. Paul has nothing to disclose.
Ram Malladi (Cambridge University Hospitals NHS Foundation Trust) No disclosure on file
Victoria Potter (Kings College London) No disclosure on file
John Snowden John Snowden has received personal compensation in the range of $500-$4,999 for serving on a Scientific Advisory or Data Safety Monitoring board for Vertex. John Snowden has received personal compensation in the range of $500-$4,999 for serving on a Scientific Advisory or Data Safety Monitoring board for Jazz. John Snowden has received personal compensation in the range of $500-$4,999 for serving on a Scientific Advisory or Data Safety Monitoring board for BMS.
Majid A. Kazmi, MBBS Dr. Kazmi has received personal compensation in the range of $500-$4,999 for serving on a Speakers Bureau for Sanofi.
Basil Sharrack, MD, PhD, FAAN (Department of Neuroscience) Dr. Sharrack has nothing to disclose.
Paolo Muraro Paolo Muraro has received personal compensation in the range of $500-$4,999 for serving on a Scientific Advisory or Data Safety Monitoring board for Cellerys. Paolo Muraro has received personal compensation in the range of $50,000-$99,999 for serving as an Expert Witness for Pinsent Masons LLP. Paolo Muraro has received personal compensation in the range of $50,000-$99,999 for serving as an Expert Witness for Taylor Wessing LLP. Paolo Muraro has received personal compensation in the range of $10,000-$49,999 for serving as an Expert Witness for Bugge Valentin. Paolo Muraro has received personal compensation in the range of $10,000-$49,999 for serving as an Expert Witness for Lambert Hornby LLP. The institution of Paolo Muraro has received research support from NIHR-EME. The institution of Paolo Muraro has received research support from Benaroya Research Institute.