Autologous Hematopoietic Stem Cell Transplantation for the Treatment of Multiple Sclerosis

Messages clés

• Autologous hematopoietic stem cell transplantation is an emerging health technology for treating multiple sclerosis, in particular for relapse-remitting forms of the disease. In 2019, transplant societies in the US and Europe both indicated the procedure may be provided as a standard of care for a subset of people with multiple sclerosis based on age and clinical classification criteria. The Canadian Multiple Sclerosis Working Group indicated in their 2020 treatment optimization recommendations that autologous hematopoietic stem cell transplantation may be considered for younger people (aged 18 to 31) who are early in their treatment course. The procedure is offered in 2 provinces, Alberta and Ontario, as an experimental treatment.
• Results from a phase III randomized controlled trial, a systematic review of single-arm trials, and retrospective analyses from European transplant registries provides evidence that the procedure may provide effective disease control for patients who show high disease activity despite receiving disease-modifying therapies. Evidence also shows that the safety profile of the procedure has been improving over the past 25 years.
• Although cost-effectiveness studies are not yet available, the one-time procedure may have important economic implications and may have the potential to provide cost-savings to health systems, as it may reduce the need for ongoing disease-modifying therapies that may be required for patients throughout their lives.
• At least 3 additional phase III randomized controlled trials are ongoing and aim to provide a stronger evidence base to inform optimal treatment regimens and appropriate eligibility criteria. As results from these studies develop, this horizon scan aims to provide health care stakeholders in Canada with an early overview of the technology and existing evidence, while highlighting considerations related to health equity, the need for multidisciplinary care, and transplant centre infrastructure that would be important if there is to be wider use across Canada should emerging evidence demonstrate value.