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

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.

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Publication Details

Journal
Bone Marrow Transplantation
Published
2025-09-29
DOI
https://doi.org/10.1038/s41409-025-02715-x
Primary Topic
Multiple Sclerosis Research Studies
Type
article
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article

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

Basil Sharrack, Paolo Antonio Muraro, Raffaella Greco, Alice Mariottini et al.
Bone Marrow Transplantation
Multiple Sclerosis Research Studies
article

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

Basil Sharrack, Paolo Antonio Muraro, Raffaella Greco, Alice Mariottini, Elisa Roldán, John A. Snowden, Sara Gil‐Perotín, Marek Śmiłowski, Chiara Nozzoli, Jaime Sanz, Tobias Alexander, Barbara Withers, M. Kazmi, Riccardo Saccardi, Kristina Carlson, Maria Pia Sormani, Grzegorz Helbig, Matilde Inglese, Jennifer Massey, Manuela Badoglio, Riccardo Varaldo, Joachim Burman, Marta Ponzano, Alessio Signori, Ian Gabriel
article en

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.

Bone Marrow TransplantationVol. 60(12)
Uppsala University (SE), Vita-Salute San Raffaele University (IT), Inserm (FR), Universitat de València (ES), Medical University of Silesia (PL), Guy's and St Thomas' NHS Foundation Trust (GB), Instituto de Salud Carlos III (ES), Hammersmith Hospital (GB), London Bridge Hospital (GB), Hospital Universitari i Politècnic La Fe (ES), Leibniz Association (DE), Sorbonne Université (FR), St Vincent's Hospital Sydney (AU), German Rheumatism Research Centre (DE), Sheffield Teaching Hospitals NHS Foundation Trust (GB), Hôpital Saint-Antoine (FR), Centro de Investigación Biomédica en Red (ES), Azienda Ospedaliero-Universitaria Careggi (IT), Ospedale Policlinico San Martino (IT), King's College Hospital (GB), Centro de Investigación Biomédica en Red de Cáncer (ES), Instituto de Investigación Sanitaria La Fe (ES), Imperial College London (GB), Charité - Universitätsmedizin Berlin (DE), University of Genoa (IT), University of Sheffield (GB)
Openalex Percentile: Top 62%
Multiple Sclerosis Research Studies
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