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Abstract Number: 1586/P848 Effectiveness of autologous haematopoietic stem cell transplant in comparison with anti-CD20 therapies in relapsing-remitting multiple sclerosis
Tomas Kalincik et al.
Introduction: Autologous hematopoietic stem cell transplant (AHSCT) is a potent therapy available to patients with suboptimal response to high-efficacy immunotherapy such as anti-CD20 antibodies. A sufficiently powered headto-head comparison of these two therapies is needed. Objectives/Aims: Objectives/Aims: This study emulated a trial of comparative effectiveness of AHSCT vs. anti-CD20 therapies. Methods: Methods: This cohort/registry study of comparative treatment effectiveness included data recorded at 7 specialist MS centres with AHSCT programs and international MSBase registry during 2002-2023 (registration nr. ACTRN12605000455662). The study included patients with relapsing-remitting MS, treated with AHSCT or an anti-CD20 monoclonal antibody (ocrelizumab or rituximab), with on-treatment follow-up including ≥2disability assessments. Patients were matched on a propensity score derived from their clinical and demographic characteristics. The matched groups were compared on annualised relapse rates (ARR), freedom from relapses and 6-month confirmed disability worsening and improvement, using a negative binomial model or Cox proportional hazards models, adjusted for paired structure of the matched dataset. Treatment safety was reported in the AHSCT group.
Results: The matching of 152 AHSCT-treated patients and 2572 anti-CD20-treated patients reduced the measured differences between the groups by 99.6%. The matched cohorts had highly active MS (79-82% of patients had experienced relapses in the prior 2 years), mean EDSS exceeding 3.5, and were followed-up for a mean of 4.03 (AHSCT) and 2.37 years (anti-CD20 therapy). 13 patients from the AHSCT group (8.6%)) were previously treated with an anti-CD20 therapy. Compared with anti-CD20 therapy, AHSCT was associated with a lower risk of relapses (mean ARR ± SD 0.04±0.25 vs. 0.09±0.32, respectively, p<0.0001; number needed to treat 20 patient-years; hazard ratio 0.43, 95% confidence interval [95%CI]=0.26-0.71), similar risk of EDSS worsening (hazard ratio 0.91, 95%CI=0.50-1.66) and higher probability of EDSS improvement (hazard ratio 1.91; 95%CI=1.24-2.96). AHSCT was associated with a risk of infections. No treatment-associated deaths were reported. Conclusion:
Conclusion: Disclosures: Among patients with active relapsing-remitting MS and moderate disability, AHSCT is superior to anti-CD20 therapies ocrelizumab and rituximab at suppressing relapses and enabling recovery of neurological function.
Source: multiple-sclerosis-research.org