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

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.

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Item Type:	Article
Authors/Creators:	Greco, R. https://orcid.org/0000-0002-6117-5328 Saccardi, R. Ponzano, M. Badoglio, M. https://orcid.org/0000-0003-3226-2024 Helbig, G. Smilowski, M. Mariottini, A. https://orcid.org/0000-0002-1869-7067 Burman, J. https://orcid.org/0000-0002-7045-1806 Carlson, K. Kazmi, M. https://orcid.org/0000-0002-6863-6297 Muraro, P.A. https://orcid.org/0000-0002-3822-1218 Gabriel, I. Withers, B. Massey, J. Varaldo, R. Inglese, M. Sanz, J. https://orcid.org/0000-0001-6934-4619 Gil-Perotin, S. Sharrack, B. Roldan, E. https://orcid.org/0000-0001-7242-170X Nozzoli, C. Signori, A. Sormani, M.P. Alexander, T. https://orcid.org/0000-0003-1193-0097 Snowden, J.A. https://orcid.org/0000-0001-6819-3476
Copyright, Publisher and Additional Information:	© The Author(s) 2025. This article is licensed under a Creative Commons Attribution-NonCommercial-NoDerivatives 4.0 International License, which permits any non-commercial use, sharing, distribution and reproduction in any medium or format, as long as you give appropriate credit to the original author(s) and thesource,providealinktotheCreativeCommonslicence,andindicateifyoumodified the licensed material. You do not have permission under this licence to share adapted materialderivedfromthisarticleorpartsofit.Theimagesorotherthirdpartymaterialin this article are included in the article’s Creative Commons licence, unless indicated otherwise in a credit line to the material. If material is not included in the article’s Creative Commons licence and your intended use is not permitted by statutory regulation or exceeds the permitted use, you will need to obtain permission directly from the copyright holder. To view a copy of this licence, visit http:// creativecommons.org/licenses/by-nc-nd/4.0/.
Keywords:	Humans; Cyclophosphamide; Multiple Sclerosis; Hematopoietic Stem Cell Transplantation; Transplantation Conditioning; Retrospective Studies; Male; Female; Adult; Middle Aged; Antilymphocyte Serum; Transplantation, Autologous; Melphalan; Etoposide; Adolescent; Thiotepa; Young Adult; Antineoplastic Combined Chemotherapy Protocols; Carmustine; Cytarabine
Dates:	Submitted: 19 May 2025 Accepted: 5 September 2025 Published (online): 29 September 2025 Published: December 2025
Institution:	The University of Sheffield
Academic Units:	The University of Sheffield > Faculty of Medicine, Dentistry and Health (Sheffield) > School of Medicine and Population Health
Date Deposited:	06 Feb 2026 14:33
Last Modified:	06 Feb 2026 14:33
Status:	Published
Publisher:	Springer Science and Business Media LLC
Refereed:	Yes
Identification Number:	10.1038/s41409-025-02715-x
Related URLs:	PubMed URL
Open Archives Initiative ID (OAI ID):	oai:eprints.whiterose.ac.uk:236933

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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

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