Encouraging real-world results of AHSCT to treat aggressive MS

In a large-scale real-world cohort, autologous haematopoietic stem cell transplantation (AHSCT) appeared to be effective and beneficial for patients with severe forms of MS. AHSCT led to overall durable remission of inflammatory activity and clinical stability, even though 38% were patients with progressive MS.

AHSCT is increasingly used to treat people with aggressive forms of MS. To establish its relative efficacy, randomised-controlled trials comparing AHSCT with high-efficacy treatments are necessary. Of such trials, 4 are ongoing. Currently, the main source of information on the efficacy of AHSCT comes from observational studies. Prof. Paolo Muraro (London Bridge Hospital, UK) presented a large retrospective cohort analysis including patients with MS who were treated with AHSCT between 2002 and 2023 at 14 UK centres [1].

The study cohort consisted of 363 patients with MS, 61.8% of whom had relapsing-remitting MS and 38.2% had progressive MS. Their median age was 40 years, median EDSS 6.0, and median disease duration 10 years. The results were focused on effectiveness. Outcomes used were relapse-free survival (RFS), MRI-activity-free survival (MFS), progression-free survival (PFS), no evidence of disease activity (NEDA), and confirmed improvement of the Expanded Disability Status Scale (EDSS).

Treatment-related mortality occurred in 4 patients (1.1%). At 2 and 4 years after AHSCT, RFS was 94.6% and 88.6%, respectively. MFS was 88.2% and 78.8%, PFS 83.5% and 62.4%, and NEDA 72.0% and 48.5%, respectively. Prof. Muraro considered the PFS results, which are probably the most important for patients, to be good, as well as the NEDA results. He considered the cumulative EDSS improvement an even greater success: 24.6% at 2 years and 26.8% at 5 years. The prevalence of this improvement was 24.2% and 20.4%, respectively.

Ongoing randomised-controlled trials will further compare the efficacy of AHSCT with high-efficacy disease-modifying therapy, including for patients with relapsing-remitting MS in the STAR-MS trial (ISRCTN88667898) in the UK [2].

1. Muraro P, et al. Real-world effectiveness and safety of autologous haematopoietic stem cell transplantation in MS: The UK experience in 363 patients treated during 2002-2023. Abstract O018, ECTRIMS 2024, 18–20 September, Copenhagen, Denmark.
2. Brittain G, et al. BMJ Open. 2024;14(2):e083582.

 

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Posted on 18 November 202418 November 2024