Autologous haematopoietic stem cell transplantation in patients with multiple sclerosis: Experience from South Africa

Abstract

Background. Multiple sclerosis (MS) is a chronic immune-mediated demyelinating disease that remains a major cause of neurological disability in young adults. While disease-modifying therapies (DMTs) can delay progression, many patients with highly active relapsing- remitting MS (RRMS) continue to experience breakthrough disease. Autologous haematopoietic stem cell transplantation (ASCT) offers durable immune reconstitution and remission but remains underutilised in upper-middle-income settings such as South Africa (SA). Objective. To evaluate the safety, feasibility and clinical outcomes of ASCT for MS in a real-world SA cohort. Methods. We conducted a retrospective record review of all MS patients who underwent ASCT between 1 January 2009 and 31 December 2024 at two JACIE-accredited tertiary centres (Wits Donald Gordon Medical Centre and Netcare Pretoria East Hospital). Data were extracted from institutional transplant records and analysed using descriptive statistics and Kaplan-Meier survival estimates. The primary outcome was the safety of ASCT for MS, and the secondary outcomes were the clinical outcomes of patients who underwent ASCT for MS. Results. Thirty-two patients were included (median age 42 years; 53% female; 94% RRMS). Median disease duration prior to ASCT was 7.4 years, and 97% had received prior DMTs. Conditioning included cyclophosphamide (97%), anti-thymocyte globulin (69%) and fludarabine (34%). The median neutrophil and platelet engraftment occurred at 10 and 14 days, respectively. Relapse-free survival was 100% at 1 year and 91% (95% confidence interval 68 - 98) at 5 years. Approximately 90% of evaluable patients demonstrated neurological improvement or stabilisation at 1 year. Treatment-related adverse events occurred in 63% of cases, and 95% of these side-effects were grade 3 or less: most commonly neutropenic fever (50%) and mucositis (16%). Only 5% of adverse reactions were grade 4, with no transplant-related mortality. Conclusion. ASCT is safe, feasible and highly effective in aggressive, treatment-refractory RRMS in SA. Its earlier use in selected patients may offer durable remission and substantial cost savings compared with long-term DMTs. The expansion of accredited national ASCT programmes and registry-based outcome monitoring is warranted.