Autologous hematopoietic stem cell transplantation (AHSCT) is associated with reduced disability and lower relapse rates in secondary progressive multiple sclerosis (SPMS) compared with other disease-modifying therapies (DMT). New research suggests.
Results from a retrospective study show that more than 60% of SPMS patients who underwent AHSCT were free of disability progression after 5 years. These patients were also more likely to maintain improvement for years after treatment.
Researchers note that patients with secondary progressive disease often show little benefit from other DMTs, so other treatments are of high interest. Although AHSCT is known to produce good results in patients with relapsing relapsing MS, studies of its efficacy for SPMS have yielded conflicting results.
The new findings suggest that it may be time to reconsider this treatment for patients with active, more serious disease, the researchers wrote.
“AHSCT may be a treatment option for patients with secondary progressive MS with inflammatory activity who have failed available therapies,” said co-investigator Matilde, professor of neurology at the University of Genoa, Italy. Inglese, M.D. said. Medscape Medical News.
“Patient selection is critical to ensuring the best treatment response and minimizing safety issues, including transplant-related mortality,” Inglese added.
The survey results were published online on December 21st. Neurology.
Class III evidence
In a retrospective propensity-matching study, researchers used two Italian registries to identify 79 patients who received off-label AHSCT treatment and 1975 patients who received another treatment.
Other DMTs included in the control group analysis were beta-interferon, azathioprine, glatiramer acetate, mitoxantrone, fingolimod, natalizumab, methotrexate, teriflunomide, cyclophosphamide, dimethyl fumarate, or alemtuzumab.
Results showed that time to first disability progression was significantly longer in transplanted patients (hazard ratio) [HR]0.5; P. = .005); 61.7% of the AHSCT group were free of disability progression at 5 years compared to 46.3% of the control group.
Patients who underwent AHSCT had a lower recurrence rate compared with those who underwent other DMTs (P. < .001) and disability scores were lower over the decade (P. < .001).
The transplant group was also significantly more likely than other DMT groups 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 transplantation extended the time to documented disease progression compared with other disease-modifying therapies,” the researchers wrote. increase.
Expand treatment population
Comments on Medscape Medical News, Jeff Cohen, M.D., director of the Experimental Therapies Division at the Mellen Multiple Sclerosis Treatment and Research Center at the Cleveland Clinic in Ohio, said the study “expands the population that should be considered for hematopoietic stem cell transplantation.”
Previous studies showed no benefit in patients with severe progressive multiple sclerosis, but participants in the current study had secondary progressive multiple sclerosis and had overlapping relapsing activity. , said Cohen, who was not involved in the study.
“I think it shows a high potential benefit” from AHSCT, he pointed out. It’s nothing.”
But Cohen pointed out that the research is not without limitations. His exclusion of patients receiving B-cell therapy from the SPMS control group raises the question of whether comparisons with AHSCT yield similar results, he said.
Additionally, Cohen noted safety concerns with this treatment, which has a high transplant-related mortality rate in patients with SPMS, although only one patient died after transplantation in the current study.
Still, the findings are encouraging, Cohen added.
“I think it will see more use as data accumulates supporting its benefits and reasonable safety in different populations,” he said.
This study was funded by the Italian Multiple Sclerosis Foundation. Inglese receives consultation fees from Roche, Genzyme, Merck, Biogen, and Novartis. Cohen has not reported any related financial relationships.
Neurology. Published online on December 21, 2022.
Kelli Whitlock Burton is a Medscape Medical News reporter covering neurology and psychiatry.
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