Stem Cell Transplant Superior to Meds for Secondary Progressive MS

Kelli Whitlock Burton

December 29, 2022

Autologous hematopoietic stem cell transplantation (AHSCT) is linked to less disability and lower relapse rates in secondary progressive multiple sclerosis (SPMS) vs other disease-modifying therapies (DMTs), new research suggests.

Results from a retrospective study show that more than 60% of patients with SPMS who received AHSCT were free from disability progression at 5 years. Also for these patients, improvement was more likely to be maintained for years after treatment.

Investigators note that patients with secondary progressive disease often show little benefit from other DMTs, so interest in other treatments is high. While AHSCT is known to offer good results for patients with relapsing remitting MS, studies of its efficacy for SPMS have yielded conflicting results.

The new findings suggest it may be time to take another look at this therapy for patients with active, more severe disease, the researchers write.

"AHSCT may become a treatment option in secondary progressive MS patients with inflammatory activity who have failed available treatments," co-investigator Matilde Inglese, MD, PhD, professor of neurology at the University of Genoa, Italy, told Medscape Medical News.

"Patients selection is very important to ensure the best treatment response and minimize safety issues, including transplant-related mortality," Inglese added.

The findings were published online December 21 in Neurology.

Class III Evidence

In the retrospective, propensity-matching study, researchers used two Italian registries to identify 79 patients who were treated off label with AHSCT and 1975 patients who received another therapy.

Other DMTs included in the control-group analysis were beta-interferons, azathioprine, glatiramer-acetate, mitoxantrone, fingolimod, natalizumab, methotrexate, teriflunomide, cyclophosphamide, dimethyl fumarate, or alemtuzumab.

Results showed that time to first disability progression was significantly longer for patients who had received transplants (hazard ratio [HR], 0.5; P = .005); 61.7% of the AHSCT group were free of disability progression at 5 years, vs 46.3% of the control group.

Among patients who received AHSCT, relapse rates were lower in comparison with those who received other DMTs (P < .001), and disability scores were lower over 10 years (P < .001).

The transplant group was also significantly more likely than the other-DMTs group to achieve sustained improvement in disability 3 years after treatment (34.7% vs 4.6%; P < .001).

"This study provides Class III evidence that autologous hematopoietic stem cell transplants prolonged the time to confirmed disability progression compared to other disease modifying therapies," the investigators write.

Extends the Treatment Population

Commenting for Medscape Medical News, Jeff Cohen, MD, director of experimental therapeutics at the Mellen Center for Multiple Sclerosis Treatment and Research at Cleveland Clinic, Ohio, said the research "extends the population for which hematopoietic stem cell transplant should be considered."

Although previous studies did not show a benefit for patients with severe progressive MS, participants in the current study had secondary progressive MS and superimposed relapse activity, said Cohen, who was not involved with the research.

"We think that indicates a greater likelihood of benefit" from AHSCT, he noted. "The fact that someone has overt progression or somewhat more severe disability doesn't preclude the use of stem cell transplant."

Cohen pointed out, however, that the study is not without limitations. The exclusion of patients taking B-cell therapies from the SPMS control group raises the question of whether similar results would come from a comparison to AHSCT, he said.

In addition, Cohen noted there are safety concerns about the therapy, which has yielded higher transplant-related mortality among patients with SPMS ― although only one patient in the current study died following the transplant.

Still, the findings are promising, Cohen added.

"I think as more data accumulate that supports its benefit and reasonable safety in a variety of populations, we'll see it used more," he said.

The study was funded by the Italian Multiple Sclerosis Foundation. Inglese has received fees for consultation from Roche, Genzyme, Merck, Biogen, and Novartis. Cohen reported no relevant financial relationships.

Neurology. Published online December 21, 2022. Abstract

Kelli Whitlock Burton is a reporter for Medscape Medical News covering neurology and psychiatry.

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