Stem cell transplantation not superior to natalizumab in progressive MS

Immune ablation with autologous haematopoietic stem cell transplantation (AHSCT) was not superior to natalizumab in reducing disability progression or allowing reduction of disability in patients with progressive MS. This was the main result from a non-randomised study comparing pairwise-censored groups.

What is known about the effectiveness of AHSCT in progressive MS mostly comes from results of studies that focused on relapsing-remitting MS. In fact, at ECTRIMS 2022, Prof. Tomas Kalincik (Royal Melbourne Hospital, Australia) presented results of a study comparing AHSCT with 3 high-efficacy DMTs in relapsing-remitting MS [1]. Prof. Kalincik also explored the effectiveness of AHSCT in progressive MS and compared it with a single, high-efficacy therapy, namely natalizumab [2]. This comparator was chosen for pragmatic reasons.

Patients with secondary or primary progressive MS (SPMS and PPMS) from 6 AHSCT MS centres around the world plus patients from the MSBase registry (msbase.org) could participate. They were included if they received AHSCT or started natalizumab during progressive MS. A total of 39 participants treated with AHSCT (37 with SPMS, 2 with PPMS) were matched with 139 natalizumab users. The pairwise-censored groups were compared on annualised relapse rates (ARR), freedom from relapses, and cumulative hazards of 6-months confirmed Expanded Disability Status Scale (EDSS) worsening and improvement. Prof. Kalincik stressed that, on average, participants had moderately advanced disease, with a mean EDSS of 5.7 and a mean 0.5–0.6 relapses in the preceding year. There was a follow-up of up to 6 years.

ARR while on treatment was low in both groups (both 0.08). “This is significant when we consider that these patients had a relatively high relapse rate prior to baseline,” Prof. Kalincik commented. Hazard ratio for relapses was 1.05 (see Figure), a result that was corroborated by the similar cumulative hazards of having a relapse at year 2 (AHSCT 20%, natalizumab 14%) and at year 5 (31% and 34%, respectively). Confirmed EDSS worsening was relatively prevalent in both groups (HR 1.49) and HR for improvement, which rarely occurred, was 1.49.

Figure: Relapse results for AHSCT and natalizumab [2]



In the AHSCT group, 3 patients (7.7%) had febrile neutropenia during mobilisation, 9 (23%) had serum sickness, 6 (15%) required ICU admission, and 36 (92%) experienced complications after discharge, including 21 infections. There were no treatment-related deaths.

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.
2. Kalincik T, et al. Effectiveness of autologous haematopoietic stem cell transplantation in comparison with natalizumab in progressive MS. Abstract O181, ECTRIMS 2022, 26–28 October, Amsterdam, the Netherlands.

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Posted on 20 December 202225 March 2024