Work

BEAM/ATG or cyclophosphamide/ATG as conditioning regimen in autologous haemopoietic stem cell transplantation for multiple sclerosis: a retrospective analysis of the EBMT autoimmune diseases working party

Year: 2025

Type: article

Abstract: Multiple Sclerosis (MS) is the most frequent autoimmune disease (AD) treated with autologous hematopoietic stem cell transplantation (HSCT). There are no prospective trials comparing the most used conditioning regimens, BEAM/Anti-thymocyte globulin (ATG) and cyclophosphamide (CYC)/ATG. Herein, we report a retrospective analysis of the EBMT database aimed to assess their risk/benefit ratio. We included 1114 MS patients who were conditioned with either BEAM/ATG or CYC/ATG. Neutrophil engraftment in BEAM/ATG- and CYC/ATG-treated patients occurred at a median time of 11 and 10 days after HSCT (p = 0.079). Overall, 2.0% of patients conditioned with BEAM/ATG and 1.0% with CYC/ATG died within 100 days from HSCT (p = 0.19). Overall, the incidence of NEDA (No-Evidence of Disease Activity) failure in BEAM/ATG and CYC/ATG at 5 years was 42.3% (95% CI: 36.3-48.8%) and 44.1% (95% CI: 30.8-60.1%), respectively (p = 0.081). No statistically significant differences between the two treatments [HR = 0.90 (95% CI = 0.61; 1.34), p = 0.60] were confirmed, when adjusting for disease type, disability at baseline and year of transplant. A clear difference between the regimens is lacking, both in terms of toxicity and efficacy, in this large population. Type of disease (relapsing/remitting vs progressive) is still the major determinant of neurological outcome.

Source: Bone Marrow Transplantation

DOI: 10.1038/s41409-025-02715-x