Autologous haematopoietic stem cell transplantation versus DMTs

Results of a new study comparing autologous haematopoietic stem cell transplantation (AHSCT) to various highly efficacious disease-modifying treatments (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. From observational, single-arm cohorts, autologous haematopoietic stem cell transplantation (AHSCT) is known to be very effective in MS patients with highly active disease [1]. In the absence of direct, randomised, comparative trials, the presented study compared the effectiveness of AHSCT with fingolimod, ocrelizumab, and natalizumab therapy by emulating a series of pairwise trials [2]. Patient data was collected in 6 centres specialised in AHSCT from 5 different countries, and was paired with patients from the MSBase registry (msbase.org) 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 annualised relapse rate (ARR), freedom from relapse, and Expanded Disability Status Scale (EDSS) change. Prof. Tomas Kalincik (University of Melbourne, Australia) presented the results.

Matched participants had a mean of >0.9 relapses in the prior year and a mean EDSS of 3–4. Compared with 612 fingolimod users, 120 matched AHSCT participants experienced less relapses (mean ARR 0.20 vs 0.11), relapse risk (HR 0.55; 95% CI 0.37–0.91). The risk of EDSS worsening was not significantly different (HR 0.49; 95% CI 0.16–1.54) and AHSCT patients 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 participants: ARR 0.10 versus 0.08, relapse risk (HR 0.85; 95% CI 0.46–1.56), EDSS worsening (HR 0.41; 95% CI 0.09–1.90), and EDSS improvement (HR 2.31; 95% CI 0.63–8.48). Compared with 606 natalizumab users, 116 AHSCT participants had similar results in ARR (0.12 vs 0.09), relapse risk (HR 0.78; 95% CI 0.40–1.52), and EDSS worsening (HR 0.50; 95% CI 0.09–2.61). AHSCT was however associated with a higher rate of EDSS improvement (HR 1.82; 95% CI 1.19–2.78), despite the fact that natalizumab is known for the reduction of disability in clinical trials.

1. Willison AG, et al. J Neurol. 2022;269(7): 3937–3958.
2. 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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Posted on 20 December 202215 February 2024