, a new study suggests.
The multicenter observational study showed that AHSCT was associated with a better overall annual relapse rate and significantly greater disability improvement versus fingolimod. Stem cell transplantation also offered a slight advantage compared with natalizumab but showed no benefit over ocrelizumab.
“Based on these results, we can conclude that in this observational study AHSCT is substantially superior to fingolimod and marginally superior to natalizumab in reducing the risk of posttreatment relapses,” study investigator Tomas Kalincik, MD, PhD, said at the annual meeting of the European Committee for Treatment and Research in Multiple Sclerosis (ECTRIMS).
Research gap
Prior studies have suggested that AHSCT is associated with better disability outcomes than other immunotherapies in patients with active secondary progressive MS. Findings from another study on long-term efficacy in relapse prevention are also promising.
“The information that we are still lacking is the head-to-head comparison of the effectiveness of AHSCT in relapsing-remitting disease to concrete, highly effective therapies, which is the aim of this current study,” said Dr. Kalincik, professor of neurology at the University of Melbourne and head of the Multiple Sclerosis and Neuroimmunology Service at the Royal Melbourne Hospital, Australia.
The study included 167 patients with relapsing-remitting MS from six centers in Ottawa, Uppsala, Sheffield, Bergen, Sydney, and Melbourne, combined with 1,675 patients from MSBase. Patients were included if they were treated with AHSCT or fingolimod (n = 769), natalizumab (n = 730) or ocrelizumab (n = 343).
Researchers used propensity matching to ensure the AHSCT and disease-modifying therapy (DMT) groups had similar relapse rates and disability scores at baseline. At 1 year, patients who underwent AHSCT were 74% more likely than were those on fingolimod to be relapse free (hazard ratio [HR], 0.26; P < .0001).
The AHSCT group had a lower overall annualized relapse rate (0.09 vs. 0.19, respectively; P < .0001) and a higher cumulative probability of remaining relapse free at 2 years (90% vs. 68%, respectively) and 5 years (85% vs. 54%, respectively). AHSCT was associated with significantly better confirmed disability improvement at 6 months compared with fingolimod (HR, 2.70; P < .0001).
The researchers found better outcomes with AHSCT compared with natalizumab, but the results weren’t as promising. The annual relapse rate with AHSCT was statistically significant (P = .03) but the clinical benefit was marginal, Dr. Kalincik said. However, the confirmed disability improvement was significantly better with AHSCT (HR, 2.68; P < .0001).
There was no significant difference in outcomes with AHSCT compared to ocrelizumab.
Adverse events were common with stem cell transplantation, a sticking point for at least one conference attendee who raised safety concerns. Among those treated with AHSCT, 23% had febrile neutropenia, 11% had serum sickness, and 36% experienced complications after discharge, mostly infection. There was one AHSCT treatment-related death.
“We’re showing that AHSCT is a highly potent therapy that definitely is competitive vis a vis the most potent standard conventional disease modifying therapies,” said Dr. Kalincik, who noted the high rate of adverse events. Still, treatment-related mortality from AHSCT has improved since the therapy was first used in MS patients, he added.
“I’m hoping the data provides some evidence to convince you that this treatment still has a place in our armamentarium,” Dr. Kalincik said.