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 - Report - MDSpire
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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
Comparison of BEAM/ATG and Cyclophosphamide/ATG Conditioning in MS HSCT
Overview
This retrospective EBMT study compared the safety and efficacy of BEAM/ATG versus cyclophosphamide/ATG conditioning regimens in autologous HSCT for multiple sclerosis. Both regimens showed comparable effectiveness in maintaining no evidence of disease activity (NEDA), with differences noted in toxicity profiles.
Background
Multiple sclerosis (MS) is the most common autoimmune disease treated with autologous hematopoietic stem cell transplantation (HSCT). Over 2000 MS patients have undergone HSCT reported to the EBMT registry since 1995, with increasing use in highly active relapsing-remitting MS refractory to high-efficacy disease-modifying therapies. Conditioning regimens of intermediate intensity, primarily BEAM/ATG and cyclophosphamide/ATG, are widely used, but comparative data on their safety and efficacy remain limited. This study aimed to retrospectively analyze EBMT registry data to compare these two regimens in MS patients undergoing autologous HSCT.
Data Highlights
Parameter
BEAM/ATG
Cyclophosphamide/ATG
Number of patients
Not specified
Not specified
NEDA at 5 years (Swedish study)
81% (CI 68–96%)
71% (CI 63–80%)
Transplant-related mortality (100-day)
1.1%
Not specified
Non-relapse mortality (2015-2020)
1% (95% CI 0.5–1.9)
1% (95% CI 0.5–1.9)
Relapse incidence (3 years)
34.4%
Not specified
Progression-free survival (3 years)
64%
Not specified
Overall survival (3 years)
95.5%
Not specified
Severe adverse events and hospitalization
More common
Less common
Key Findings
No statistically significant difference in NEDA rates at 5 years between BEAM/ATG (81%) and cyclophosphamide/ATG (71%) was observed in prior studies.
BEAM/ATG regimen is associated with higher rates of severe adverse events and prolonged hospitalization compared to cyclophosphamide/ATG.
Transplant-related mortality remains low (~1%) with both regimens, consistent with recent EBMT data.
Patient selection and timing of HSCT in relapsing-remitting MS are critical to optimize outcomes and minimize risks.
Clinical Implications
Clinicians can consider both BEAM/ATG and cyclophosphamide/ATG conditioning regimens as effective options for autologous HSCT in treatment-resistant relapsing-remitting MS. The choice of regimen may be influenced by toxicity profiles, with cyclophosphamide/ATG potentially offering better tolerability. Careful patient selection and monitoring remain essential to maximize benefits and minimize complications.
Conclusion
This EBMT retrospective analysis supports comparable efficacy of BEAM/ATG and cyclophosphamide/ATG conditioning regimens in autologous HSCT for MS, with differences in safety profiles guiding regimen selection. Further prospective studies are warranted to confirm these findings.
References
EBMT Autoimmune Diseases Working Party -- Comparison of BEAM/ATG and cyclophosphamide/ATG Conditioning Regimens in Autologous HSCT for MS
by Raffaella Greco, Riccardo Saccardi, Marta Ponzano, Manuela Badoglio, Grzegorz Helbig, Marek Smilowski, Alice Mariottini, Joachim Burman, Kristina Carlson, Majid Kazmi, Paolo A. Muraro, Ian Gabriel, Barbara Withers, Jennifer Massey, Riccardo Varaldo, Matilde Inglese, Jaime Sanz, Sara Gil-Perotin, Basil Sharrack, Elisa Roldan, Chiara Nozzoli, Alessio Signori, Maria Pia Sormani, Tobias Alexander, John A. Snowden
