Autologous haematopoietic stem cell transplantation as a first-line disease-modifying therapy in patients with ‘aggressive’ multiple sclerosis

J. Das*, J. A. Snowden, J. Burman, M. S. Freedman, H. Atkins, M. Bowman, R. K. Burt, R. Saccardi, C. Innocenti, S. Mistry, P. J. Laud, H. Jessop, B. Sharrack

*Corresponding author for this work

Research output: Contribution to journalArticlepeer-review

22 Scopus citations

Abstract

Background: Autologous haematopoietic stem cell transplantation (AHSCT) is an effective treatment for patients with multiple sclerosis (MS) who have highly active disease, despite the use of standard disease-modifying therapies (DMTs). However, the optimal time for offering AHSCT to patients with ‘aggressive’ MS is yet to be established. Objectives: The objective was to explore the safety and efficacy of AHSCT as a first-line DMT in patients with ‘aggressive’ MS. Methods: All patients with ‘aggressive’ MS who received AHSCT as a first-line DMT in five European and North American centres were retrospectively evaluated. Results: Twenty patients were identified. The median interval between diagnosis and AHSCT was 5 (1–20) months. All had multiple poor prognostic markers with a median pre-transplant Expanded Disability Status Scale (EDSS) score of 5.0 (1.5–9.5). After a median follow-up of 30 (12–118) months, the median EDSS score improved to 2.0 (0–6.5, p < 0.0001). No patient had further relapses. Three had residual magnetic resonance imaging (MRI) disease activities in the first 6 months post-transplant, but no further new or enhancing lesions were observed in subsequent scans. Conclusion: AHSCT is safe and effective as a first-line DMT in inducing rapid and sustained remission in patients with ‘aggressive’ MS.

Original languageEnglish (US)
Pages (from-to)1198-1204
Number of pages7
JournalMultiple Sclerosis Journal
Volume27
Issue number8
DOIs
StatePublished - Jul 2021

Keywords

  • Autologous haematopoietic stem cell transplantation
  • aggressive multiple sclerosis
  • disease-modifying therapy
  • multiple sclerosis
  • no evidence of disease activity

ASJC Scopus subject areas

  • Clinical Neurology
  • Neurology

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