From observational, single-arm cohorts, autologous hematopoietic stem cell transplantation (AHSCT) is known to be highly efficacious in patients with highly active MS. Results of a new study comparing AHSCT to various highly efficacious, disease-modifying therapies (DMTs) reveal it to be superior to fingolimod and comparable with ocrelizumab and natalizumab therapy in the prevention of relapses.

In AHSCT, patients’ stem cells are harvested and reinfused after high-dose chemotherapy, in an attempt to reset the immune system. In the absence of direct, randomized, comparative trials, this study compared the effectiveness of AHSCT with fingolimod, ocrelizumab, and natalizumab therapy by emulating a series of pairwise trials. Patient data were collected in 6 centers specialized in AHSCT from five countries, which were paired with patients from the MSBase registry using fingolimod, ocrelizumab, or natalizumab. The AHSCT and pharmaceutical groups were matched using a propensity score that was based on demographic factors, previous relapses, time since diagnosis, and most effective prior therapy. The main endpoints were annualized relapse rates (ARR), freedom from relapse, and Expanded Disability Status Scale (EDSS) change. Professor Tomas Kalincik (University of Melbourne, Australia) presented the results.

Matched patients had a mean of more than 0.9 relapses in the prior year and a mean EDSS of 3–4. Compared with 612 fingolimod users, 120 matched patients with AHSCT experienced less relapses (mean ARR 0.20 vs 0.11; HR, 0.55; 95% CI, 0.37–0.91). The risk for EDSS worsening was not significantly different (HR 0.49; 95% CI 0.16–1.54), and patients who underwent AHSCT were more likely to experience disability improvement (HR, 2.62; 95% CI, 1.46-4.72).

No significant differences were detected between 91 AHSCT- and 303 ocrelizumab-treated patients: ARR 0.10 versus 0.08, relapse risk 0.85 (0.46–1.56), EDSS worsening 0.41 (0.09–1.90), and EDSS improvement 2.31 (0.63–8.48). Compared with 606 natalizumab users, 116 AHSCT patients had similar results in ARR (0.12 vs 0.09), relapse risk (0.78; 0.40–1.52), and EDSS worsening (0.50; 0.09–2.61). AHSCT was, however, associated with a higher rate of EDSS improvement (1.82; 1.19–2.78), despite natalizumab being known for the reduction of disability in clinical trials.

  1. Kalincik T, et al. Comparative effectiveness of autologous haematopoietic stem cell transplantation vs. fingolimod, ocrelizumab and natalizumab in relapsing-remitting MS. Abstract O019, ECTRIMS 2022, 26–28 October, Amsterdam, the Netherlands.

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