ACTRIMS 2026 | HSCT in multiple sclerosis: current evidence and patient selection

Well, so there’s been interest in hematopoietic stem cell transplant for autoimmune diseases, including multiple sclerosis, for now 20 or 30 years. And there’s a rather sizable literature that indicates that it has very potent efficacy in multiple sclerosis and that that benefit is durable. It lasts for many years in most patients without the need for restarting disease therapies. However, that literature has some shortcomings. Most of the studies were uncontrolled case series or Phase II trials. And there only are two randomized trials supporting the use of hematopoietic stem cell transplant. Both of those studies showed superior efficacy of transplant, but the comparator group had some issues in both studies. In one study, the comparator group was mitoxantrone, novantrone, a disease-modifying therapy that we no longer use because of toxicity. And the other randomized trial, the MIST trial, only about half of the patients in the medication arm in that trial received what we would currently consider a high-efficacy therapy. So as a result, precisely where to place hematopoietic stem cell transplant in our overall treatment sequence remains somewhat uncertain. The accumulated evidence suggests that hematopoietic stem cell transplant is most effective for treating relapses and ongoing MRI activity, and that it’s less effective for slowing or preventing disability worsening. So as a result, the people that are most likely to benefit from transplant are those who are relatively young with relatively recent disease onset, active disease, by which I mean recent relapses or MRI changes, and who have had continued activity despite treatment with one or more medications, but who have only mild or moderate accumulated disability. Conversely, people who are less likely to benefit and are more likely to sustain harm are older people with more severe non-active progressive MS and who have other comorbidities that might increase the medical risk of the procedure.

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