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Post-transplant cyclophosphamide in one-antigen mismatched unrelated donor transplantation versus haploidentical transplantation in acute myeloid leukemia: a study from the Acute Leukemia Working Party of the EBMT


Whether to choose Haploidentical (Haplo) or one-antigen mismatched unrelated donor (1Ag-MMUD) hematopoietic cell transplantation (HCT) with post-transplant cyclophosphamide (PTCy) remains an unanswered question. We compared PTCy- Haplo-HCT to PTCy-1Ag-MMUD-HCT for acute myeloid leukemia (AML) in complete remission (three groups: 1Ag-MMUD using peripheral blood (1Ag-MMUD-PB; n = 155); Haplo using bone marrow (Haplo-BM; n = 647) or peripheral blood (Haplo-PB; n = 949)). Haplo-BM and Haplo-PB had a higher non-relapse mortality (NRM) compared to 1Ag-MMUD-PB (HR 2.28, 95% CI 1.23–4.24, p < 0.01; HR 2.65, 95% CI 1.46–4.81, p < 0.01, respectively). Haplo groups experienced a lower leukemia-free survival (LFS) compared to 1Ag-MMUD-PB (Haplo-BM: HR 1.51, 95% CI 1.06–2.14, p = 0.02; Haplo-PB: 1.47, 95% CI 1.05–2.05, p = 0.02); overall survival (OS) was also lower in Haplo-HCT (Haplo-BM: HR 1.50, 95% CI 1.02–2.21, p = 0.04; Haplo-PB: HR 1.51, 95% CI 1.05–2.19, p = 0.03). No differences were observed for graft-versus-host/relapse-free survival (GRFS) and relapse incidence (RI). Haplo-BM was associated with a lower risk of grade III-IV acute graft-versus-host disease (GVHD) (HR 0.44, 95% CI 0.24–0.81; p < 0.01), while no statistical differences were observed between groups for grade II-IV aGVHD and for cGVHD. Use of PTCy in 1Ag-MMUD-HCT is a valid alternative to consider when using alternative donors. Larger analysis of 1Ag-MMUD versus Haplo-HCT are warranted.

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Fig. 1: Non-relapse mortality and relapse incidence according to donor type and stem cell source.
Fig. 2: Overall survival, leukemia-free survival and graft-versus-host disease/relapse-free survival according to donor type and stem cell source.


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The authors thank Emmanuelle Polge from the office of the ALWP of the EBMT, and the clinical staff and investigators involved in this research. They especially thank the patients who took part.

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GB designed the study and wrote the manuscript, JEG performed the statistical analysis. ML, FC, AN AR, and MMo revised the manuscript and all the authors reviewed and accepted its final version. AMR, DB, AR, YK, ZG, AV, SS, JLDM, LC, BB, MR, IM, MM, GG, MCA, CEB, SN, GS, MA, JP, JT, HM, LLC, GC, EF, AMD, FP, and AB were the principal investigators at the centers recruiting the highest number of patients for the study.

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Correspondence to Giorgia Battipaglia.

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Battipaglia, G., Galimard, JE., Labopin, M. et al. Post-transplant cyclophosphamide in one-antigen mismatched unrelated donor transplantation versus haploidentical transplantation in acute myeloid leukemia: a study from the Acute Leukemia Working Party of the EBMT. Bone Marrow Transplant 57, 562–571 (2022).

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