Comparative analysis of five-year clinical outcomes of autologous hematopoietic stem cell transplantation and alemtuzumab in multiple sclerosis patients.

Background: Alemtuzumab (ALZ) and autologous hematopoietic stem cell transplantation (aHSCT) are potent immune reconstitution therapies for patients with highly active multiple sclerosis (MS). This study aims to compare the five-year efficacy of aHSCT and ALZ in MS patients with high disease activity.

Methods: This multicenter observational study evaluated MS patients treated with aHSCT and ALZ. Annual relapse rates (ARR), Expanded Disability Status Scale (EDSS) scores, and annual cumulative disease activity (NEDA-3) were compared between pre-treatment levels and outcomes at the five-year follow-up.

Results: The study included 39 patients treated with ALZ and 18 who underwent aHSCT. The median ages at treatment initiation were 25 years (IQR: 15) for the ALZ group and 25 years (IQR: 14) for the aHSCT group, with median baseline EDSS scores of 6.5 (IQR: 1.5) and 6.8 (IQR: 1.0), respectively. The median follow-up durations were 1 year (IQR: 3) for ALZ-treated patients and 5 years (IQR: 6) for aHSCT-treated patients. Comparative analysis showed no significant differences between the groups in time to first relapse, EDSS worsening, or time to NEDA-3 loss over the five-year follow-up period.

Conclusions: This study is the first to compare the effectiveness of aHSCT and ALZ treatments in MS patients in Türkiye. The findings reveal that both treatments demonstrate comparable efficacy, particularly in the short-term follow-up period.