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Lower incidence of acute graft-versus-host disease with post-transplantation cyclophosphamide compared to anti-thymocyte globulin in higher-risk myelodysplastic syndrome

Bone Marrow Transplantation volume 59, pages 1329–1331 (2024)Cite this article

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The increasing use of allogeneic hematopoietic cell transplantation (allo-HCT) for myelodysplastic syndrome (MDS) has led to increased graft-versus-host disease (GVHD) incidences, especially with more elderly patients receiving alternative donor transplantation with reduced-intensity conditioning and peripheral blood stem cell grafts. Post-transplantation cyclophosphamide (PTCy) has become a widely used GVHD prophylaxis method, initially for human leukocyte antigen (HLA)-haploidentical transplantation, which has been extended to HLA-matched settings with varied conditioning regimens [1]. In a bone marrow transplantation mouse model, regulatory T cells were found to play a crucial in controlling GVHD, which is mediated by PTCy promoting the differentiation of myeloid-derived suppressor cells [2]. In recent studies comparing PTCy with ATG, allo-HCT using PTCy was associated with lower incidences of severe acute GVHD, chronic GVHD, and non-relapse mortality (NRM), but may be accompanied by a higher risk of relapse and delayed engraftment compared to ATG [3,4,5,6]. In this study, we aimed to evaluate the efficacy and safety of PTCy in allo-HCT for patients with higher-risk MDS prospectively and compare outcomes with a historical cohort using ATG.

Patients with higher-risk MDS were enrolled in two prospective trials, and their post-transplantation outcomes were compared to those of a historical control receiving allo-HCT with ATG. The primary endpoint was cumulative incidence of acute and chronic GVHD. Details of patient population, transplantation procedures, GVHD prophylaxis, endpoints, and analysis methods are described in the Supplementary material. Survival analyses were censored at 3 years due to significantly longer follow-up of survivors in the ATG group (157.7 vs. 41.6 months).

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Fig. 1: Cumulative incidences of acute graft-versus-host disease (GVHD) based on GVHD prophylaxis.

Data availability

The datasets generated during and/or analyzed during the current study are available from the corresponding author upon reasonable request.

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Acknowledgements

This work was supported by a National Research Foundation of Korea (NRF) grant funded by the Korean government (MSIT) (NRF-2021R1C1C1007973) and a grant (2022IF0002-1) from the Asan Institute for Life Sciences, Asan Medical Center, Seoul, Korea.

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Authors and Affiliations

  1. Department of Hematology, Asan Medical Center, University of Ulsan College of Medicine, Seoul, Republic of Korea

    Eun-Ji Choi, Jin-Hee Han, Hyunkyung Park, Han-Seung Park, Yunsuk Choi, Jung-Hee Lee, Young-Shin Lee, Young-Ah Kang, Mijin Jeon, Hyeran Kang, Ji Min Woo & Je-Hwan Lee

  2. Department of Hematology-Oncology, Ewha Womans University Medical Center, Mokdong Hospital, Seoul, Republic of Korea

    Kyoo-Hyung Lee

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Contributions

E-JC and J-HL designed the study and wrote the manuscript. E-JC, J-HH, and HP extracted and analyzed data. H-SP, YC, Jung-HL, and K-HL reviewed the protocol and provided feedback on the report. All authors reviewed and approved the final manuscript.

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Correspondence to Je-Hwan Lee.

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Choi, EJ., Han, JH., Park, H. et al. Lower incidence of acute graft-versus-host disease with post-transplantation cyclophosphamide compared to anti-thymocyte globulin in higher-risk myelodysplastic syndrome. Bone Marrow Transplant 59, 1329–1331 (2024). https://doi.org/10.1038/s41409-024-02364-6

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