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Allogeneic stem cell transplantation for patients with acute myeloid leukemia (AML) in second complete remission (CR2) transplanted from unrelated donors with post-transplant cyclophosphamide (PTCy). A study on behalf of the Acute Leukemia Working Party of the European Society for Blood and Marrow Transplantation

Abstract

Post-transplant cyclophosphamide (PTCy) is being increasingly used as graft-versus-host disease (GVHD) prophylaxis post allogeneic hematopoietic stem cell transplantation (allo-HSCT) in patients with acute myeloid leukemia (AML) transplanted in first complete remission (CR1). However, results may differ in patients transplanted in CR2. We retrospectively evaluated transplant outcomes of adult AML patients transplanted between 2010–2019 from 9–10/10 human leukocyte antigen (HLA)-matched unrelated donor (UD) in CR2. In total, 127 patients were included (median age 45.5 years, 54% male). Median follow-up was 19.2 months. Conditioning was myeloablative (MAC) in 50.4% and the graft source was peripheral blood in 93.7% of the transplants. Incidence of acute (a)GVHD II-IV and III-IV was 26.2% and 9.2%. Two-year total and extensive chronic (c)GVHD were 34.3% and 13.8 %, respectively. Two-year non-relapse mortality (NRM), relapse incidence (RI), leukemia-free survival (LFS), overall survival (OS), and GVHD-free, relapse-free survival (GRFS) were 17.2%, 21.1%, 61.7, %, 65.2%, and 49.3%, respectively. Time from diagnosis to transplant (>18 months) was a favorable prognostic factor for RI, LFS, OS, and GRFS while favorable risk cytogenetics was a positive prognostic factor for OS. The patient’s age was a poor prognostic factor for NRM and cGVHD. Finally, the female-to-male combination and reduced intensity conditioning (RIC) were poor and favorable prognostic factors for cGVHD, respectively. We conclude that PTCy is an effective method for GVHD prophylaxis in AML patients undergoing allo-HCT in CR2 from UD.

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Fig. 1: Transplantation outcomes of allogeneic stem cell transplantation from an unrelated donor in patients with acute myeloid leukemia transplanted in second complete remission with post-transplant cyclophosphamide as anti-graft versus host disease prophylaxis.

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Professor Mohamad Mohty, and Dr Myriam Labopin from the ALWP will provide the data upon request.

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Acknowledgements

We thank all the EBMT centers and national registries for contributing patients to this study (see Appendix S1). We also thank the data managers for their excellent work.

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AN wrote the manuscript, designed the study, and interpreted the data. ML designed the study, performed the statistical analyses, interpreted the data, and edited the manuscript. MM designed the study, interpreted the data, and edited the manuscript. RS helped in writing the manuscript. AK, HLW, MR, DB, JV, IYA, GC, PR, YK, JS and FC reviewed the manuscript and provided clinical data. All authors approved the final version of the manuscript.

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Correspondence to Arnon Nagler.

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The authors declare no competing interests.

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The scientific boards of the ALWP of the EBMT approved this study.

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Nagler, A., Labopin, M., Swoboda, R. et al. Allogeneic stem cell transplantation for patients with acute myeloid leukemia (AML) in second complete remission (CR2) transplanted from unrelated donors with post-transplant cyclophosphamide (PTCy). A study on behalf of the Acute Leukemia Working Party of the European Society for Blood and Marrow Transplantation. Bone Marrow Transplant 58, 552–557 (2023). https://doi.org/10.1038/s41409-023-01940-6

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