Unrelated cord blood transplantation with myeloablative conditioning for pediatric acute lymphoblastic leukemia in remission: prognostic factors

Abstract

The number of individuals undergoing unrelated cord blood transplantation (UCBT) has increased in recent years; however, information on prognostic factors is limited. We retrospectively analyzed data from 475 children and adolescents receiving UCBT with myeloablative conditioning for acute lymphoblastic leukemia (ALL) in complete remission (CR), based on a nationwide registry. In the total patient cohort, 5-year leukemia-free survival (LFS) and overall survival (OS) rates after UCBT were 61.1% and 67.7%, respectively. UCBT at first CR and UCBT after 2007 were associated with good survival, while grade II–IV acute graft-versus-host disease (GVHD) was associated with low relapse rate but did not affect survival. Analysis according to human leukocyte antigen (HLA) disparity revealed that tacrolimus-based GVHD prophylaxis resulted in higher OS and lower relapse rate and nonrelapse mortality (NRM) than cyclosporine-based GVHD prophylaxis in patients transplanted with 6/6 and ≤4/6 HLA-matched umbilical cord blood. Furthermore, grade II–IV acute GVHD was associated with good LFS and low relapse rate, without high NRM, in patients receiving 5/6 HLA-matched UCBT. These data indicate that prognostic factors for ALL differ depending on HLA disparity in UCBT.

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Fig. 1: Outcomes of children and adolescents with ALL in complete remission after unrelated cord blood transplantation with myeloablative conditioning regimen.
Fig. 2: Outcomes of children and adolescents with ALL in complete remission after unrelated cord blood transplantation with myeloablative conditioning regimen.

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Acknowledgements

We thank all the staff at the hospitals and centers who contributed valuable data to the JSHCT registry. We also thank all the members of the data management committees of the JSHCT. This work was supported in part by the Practical Research Project for Allergic Diseases and Immunology (Research Technology of Medical Transplantation) from Japan Agency for Medical Research and Development, AMED, under Grant Number 19ek0510023h0002.

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YK is the principal investigator and takes principal responsibility for the paper. YK, AM, JI, KoKa, FK, and MK designed the study. KKo, MN, HG, NY, YC, TH, MH, KeKa, AO, YH, MI, KoKa, and YA recruited the patients and collected the data. YK analyzed the data. YK and AM wrote the manuscript. All authors discussed the results, commented on the manuscript, and gave their final approval of the manuscript.

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Correspondence to Yuta Kawahara.

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Kawahara, Y., Morimoto, A., Inagaki, J. et al. Unrelated cord blood transplantation with myeloablative conditioning for pediatric acute lymphoblastic leukemia in remission: prognostic factors. Bone Marrow Transplant (2020). https://doi.org/10.1038/s41409-020-01019-6

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