Immunosuppression and stem cell transplants achieve sustained MS remission
medwireNews: High-dose immunosuppressive therapy (HDIT) and autologous hematopoietic cell transplantation (HCT) can induce long-term remission for patients with relapsing–remitting multiple sclerosis (MS), show findings from the HALT-MS trial.
Five years after receiving HDIT/HCT, 69.2% of 24 individuals with relapsing–remitting MS survived without the return of disease activity – disability progression, relapse, or new lesions on magnetic resonance imaging – and the need for maintenance therapy.
Individual rates were 91.3% for progression-free survival, 86.9% for relapse-free survival, and 86.3% for overall survival.
And the researchers note in Neurology that participants who survived and completed the study had a median 0.5-point improvement in Expanded Disability Status Scale score.
“These outcomes are highly promising, as compared to non-HCT treatments, and consistent with other contemporary investigations of autologous HCT for similarly afflicted individuals,” say Richard Nash (Colorado Blood Cancer Institute, Denver, USA) and colleagues.
Given that the study participants had previously failed to respond to between two and four nontransplant MS medications, the researchers believe that “HDIT/HCT may represent a potential therapeutic option for patients with [relapsing–remitting] MS who fail conventional immunotherapy.”
There was also evidence of brain volume stabilization at 3 years that was maintained throughout the 5 years of the study, which the team explains is “consistent with attenuation of brain tissue loss following resolution of brain inflammation.”
Among the seven patients who did not achieve event-free survival, two had a more than 0.5-point increase in EDSS score, three had clinical relapses, and two developed new lesions, but even these individuals showed some improvement compared with before treatment.
The findings expand on the interim 3-year outcomes, published in JAMA Neurology in 2015, which reported an event-free survival rate of 78% at that time.
Adverse events were consistent with expectations for this type of treatment and included predominantly cytopenias and infections, with no significant later neurologic effects. There were 15 grade 3 events and no grade 4 events between year 3 and 5. One death from cardiorespiratory arrest occurred at 4.5 years after transplant in a patient whose disease progressed, but this, like the two previously recorded in the 3-year interim analysis, was not attributed to treatment.
“For patients failing first-line treatments, significantly more potent options are becoming available,” highlights the team. “We suggest that HDIT/HCT may be a reasonable consideration for such patients.”
By Lucy Piper
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