BEAM or cyclophosphamide in autologous haematopoietic stem cell transplantation for relapsing-remitting multiple sclerosis
Thomas Silfverberg, Christina Zjukovskaja, Yassine Noui, Kristina Carlson, AutoMS-Swe Investigators, Adjmal Nahimi, A. Erik, Cherif Honar, Dreimane Arta, Sigrun Einarsdottir, Fagius Jan, Hägglund Hans, Iacobaeus Ellen, Lange Niclas, Lenhoff Stig, Ljungman Per, Jan Lycke, Mellergård Johan, Piehl Fredrik, Anders Svenningsson, Tolf Andreas, Joachim Burman
Abstract
The most widely used conditioning regimens in autologous haematopoietic stem cell transplantation (ASCT) for multiple sclerosis (MS) are BEAM with anti-thymocyte globulin (ATG) and high-dose cyclophosphamide with ATG (Cy/ATG). In this retrospective study, we compare efficacy and safety of these regimens when used for relapsing-remitting MS. We assessed 231 patients treated in Sweden before January 1, 2020. The final cohort comprised 33 patients treated with BEAM/ATG and 141 with Cy/ATG. Prospectively collected data from the Swedish MS registry were used for efficacy, and electronic health records for procedure-related safety. The Kaplan-Meier estimate of 'no evidence of disease activity' (NEDA) at 5 years was 81% (CI 68-96%) with BEAM/ATG and 71% (CI 63-80%) with Cy/ATG, p = 0.29. Severe adverse events were more common with BEAM/ATG, mean 3.1 vs 1.4 per patient, p = <0.001. Febrile neutropaenia occurred in 88% of BEAM/ATG patients and 68% of Cy/ATG patients, p = 0.023. Average hospitalisation was 3.0 days longer in BEAM/ATG patients from day of stem-cell infusion, p < 0.001. While both regimens showed similar efficacy, BEAM/ATG was associated with more severe adverse events and prolonged hospitalisation. In the absence of randomised controlled trials, Cy/ATG may be preferable for ASCT in patients with relapsing-remitting MS due to its favourable safety profile.