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For Relapsing-Remitting MS, Autologous Hematopoietic Stem Cell Transplant May Be Superior Disease-Modifying Therapy
For the treatment of relapsing-remitting multiple sclerosis (MS), a new study has found the comparative clinical effectiveness of autologous hematopoietic stem cell transplant (AHSCT) “considerably superior” to fingolimod and “marginally superior” to natalizumab. Findings were published in JAMA Neurology.
“We show that over 5 years, AHSCT is associated with a lower risk of relapses and a higher chance of recovery from disability in highly active relapsing-remitting MS when compared with fingolimod and natalizumab,” wrote Tomas Kalincik, MD, PhD, professorial fellow, Neurological Outcomes, Royal Melbourne Hospital, and co-authors.
“Although AHSCT requires a complex treatment procedure, its one-off nature may offer practical advantages over the continuously administered therapies. AHSCT is associated with considerable risks, but the risk of treatment-associated mortality is low.”
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The observational comparative effectiveness study included 4915 individuals using a composite cohort from specialized MS centers and the MSBase international registry with relapsing-remitting MS, high frequency of relapse, and moderate disability. Authors compared the effectiveness of AHSCT with 1 medium-efficacy and 2 high-efficacy disease-modifying therapies (DMTs) (fingolimod, natalizumab, and ocrelizumab) in patients with 2 or more years of study follow-up, including 2 or more disability assessments. Matched patients were compared on annualized relapse rates (ARR), freedom from relapses, and 6-month confirmed Expanded Disability Status Scale (EDSS) score.
Compared with the fingolimod group (769 [30.0%]), AHSCT (144 [86.2%]) was associated with fewer relapses (ARR: mean [SD], 0.09 [0.30] vs 0.20 [0.44]), similar risk of disability worsening (hazard ratio [HR], 1.70; 95% CI, 0.91-3.17), and higher chance of disability improvement (HR, 2.70; 95% CI, 1.71-4.26) over 5 years. Compared with natalizumab (730 [49.0%]), AHSCT (146 [87.4%]) was associated with marginally lower ARR (mean [SD], 0.08 [0.31] vs 0.10 [0.34]), similar risk of disability worsening (HR, 1.06; 95% CI, 0.54-2.09), and a higher chance of disability improvement (HR, 2.68; 95% CI, 1.72-4.18) over 5 years. AHSCT (110 [65.9%]) and ocrelizumab (343 [49.0%]) were associated with similar ARR (mean [SD], 0.09 [0.34] vs 0.06 [0.32]), disability worsening (HR, 1.77; 95% CI, 0.61-5.08), and disability improvement (HR, 1.37; 95% CI, 0.66-2.82) over 3 years. AHSCT-related mortality occurred in 1 of 159 patients (0.6%).
“The observation that AHSCT showed superiority in clinical outcomes over fingolimod and, to a lesser extent, natalizumab, but not ocrelizumab, is intriguing,” the authors noted in the study discussion. “While this may be attributed to the shorter study follow-up available in the ocrelizumab cohort, it may also be due to the differences in the mechanisms of action among the therapies.”
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