Clinical outcomes of allogeneic hematopoietic stem cell transplant with different stem cell sources in infant acute myeloid leukemia

Abstract Acute Myeloid Leukemia (AML) in infants constitutes a rare and biologically distinct subgroup. Hematopoietic stem cell transplantation (HSCT) plays a pivotal role in the treatment of infant AML. However, the source of stem cells remains insufficiently explored. This study aimed to evaluate...

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Main Authors: Yuyang Wang, Yibing Guo, Xiuxin Ling, Junhui Li, Tao Hu, Zhaoxia Zhang, Mengze Hu, Zeliang Song, Mei Yue, Ruihong Tang, Dixiao Zhong, Rong Liu
Format: Article
Language:English
Published: Nature Portfolio 2025-07-01
Series:Scientific Reports
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Online Access:https://doi.org/10.1038/s41598-025-05602-z
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author Yuyang Wang
Yibing Guo
Xiuxin Ling
Junhui Li
Tao Hu
Zhaoxia Zhang
Mengze Hu
Zeliang Song
Mei Yue
Ruihong Tang
Dixiao Zhong
Rong Liu
author_facet Yuyang Wang
Yibing Guo
Xiuxin Ling
Junhui Li
Tao Hu
Zhaoxia Zhang
Mengze Hu
Zeliang Song
Mei Yue
Ruihong Tang
Dixiao Zhong
Rong Liu
author_sort Yuyang Wang
collection DOAJ
description Abstract Acute Myeloid Leukemia (AML) in infants constitutes a rare and biologically distinct subgroup. Hematopoietic stem cell transplantation (HSCT) plays a pivotal role in the treatment of infant AML. However, the source of stem cells remains insufficiently explored. This study aimed to evaluate the clinical outcomes of allogeneic HSCT from different sources. We conducted a single-center retrospective analysis of 27 infants with AML who underwent HSCT. We compared peripheral blood (PB) with/without bone marrow (BM) (Group 1, n = 16) versus umbilical cord blood (UCB) (Group 2, n = 12). There was no significant difference in 3-year overall survival (OS), disease-free survival (DFS), 3-year recurrence rates, or one-year cumulative graft-versus-host disease (GVHD) incidence between two groups (3-year OS: 80.36% for group 1 vs. 91.67% for group 2, p = 0.5474; DFS: 73.66% vs. 69.84%, p = 0.8232; GVHD: 56.25% for group 1 vs. 50.0% for group 2, p = 0.824). The group with pre-transplant minimal residual disease (MRD) status had a lower recurrence rate. This study emphasizes the efficacy of HSCT in the treatment of infant AML, with higher OS rates compared to childhood AML. It also supports UCB as a viable stem cell source. Achieving MRD-negative status before transplantation is crucial for improving post-transplant outcomes.
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spelling doaj-art-c0a6ce9fb58042b68c797c3f8568f4de2025-08-20T03:05:18ZengNature PortfolioScientific Reports2045-23222025-07-011511910.1038/s41598-025-05602-zClinical outcomes of allogeneic hematopoietic stem cell transplant with different stem cell sources in infant acute myeloid leukemiaYuyang Wang0Yibing Guo1Xiuxin Ling2Junhui Li3Tao Hu4Zhaoxia Zhang5Mengze Hu6Zeliang Song7Mei Yue8Ruihong Tang9Dixiao Zhong10Rong Liu11Department of Hematology, Capital Center for Children’s Health, Capital Medical UniversityBioengineeringBioengineeringDepartment of Hematology, Capital Center for Children’s Health, Capital Medical UniversityDepartment of Hematology, Capital Center for Children’s Health, Capital Medical UniversityDepartment of Hematology, Capital Center for Children’s Health, Capital Medical UniversityDepartment of Hematology, Capital Center for Children’s Health, Capital Medical UniversityDepartment of Hematology, Capital Center for Children’s Health, Capital Medical UniversityDepartment of Hematology, Capital Center for Children’s Health, Capital Medical UniversityDepartment of Hematology, Capital Center for Children’s Health, Capital Medical UniversityDepartment of Hematology, Capital Center for Children’s Health, Capital Medical UniversityDepartment of Hematology, Capital Center for Children’s Health, Capital Medical UniversityAbstract Acute Myeloid Leukemia (AML) in infants constitutes a rare and biologically distinct subgroup. Hematopoietic stem cell transplantation (HSCT) plays a pivotal role in the treatment of infant AML. However, the source of stem cells remains insufficiently explored. This study aimed to evaluate the clinical outcomes of allogeneic HSCT from different sources. We conducted a single-center retrospective analysis of 27 infants with AML who underwent HSCT. We compared peripheral blood (PB) with/without bone marrow (BM) (Group 1, n = 16) versus umbilical cord blood (UCB) (Group 2, n = 12). There was no significant difference in 3-year overall survival (OS), disease-free survival (DFS), 3-year recurrence rates, or one-year cumulative graft-versus-host disease (GVHD) incidence between two groups (3-year OS: 80.36% for group 1 vs. 91.67% for group 2, p = 0.5474; DFS: 73.66% vs. 69.84%, p = 0.8232; GVHD: 56.25% for group 1 vs. 50.0% for group 2, p = 0.824). The group with pre-transplant minimal residual disease (MRD) status had a lower recurrence rate. This study emphasizes the efficacy of HSCT in the treatment of infant AML, with higher OS rates compared to childhood AML. It also supports UCB as a viable stem cell source. Achieving MRD-negative status before transplantation is crucial for improving post-transplant outcomes.https://doi.org/10.1038/s41598-025-05602-zHematopoietic stem cell transplantationAcute myeloid leukemiaSurvivalRecurrence rates
spellingShingle Yuyang Wang
Yibing Guo
Xiuxin Ling
Junhui Li
Tao Hu
Zhaoxia Zhang
Mengze Hu
Zeliang Song
Mei Yue
Ruihong Tang
Dixiao Zhong
Rong Liu
Clinical outcomes of allogeneic hematopoietic stem cell transplant with different stem cell sources in infant acute myeloid leukemia
Scientific Reports
Hematopoietic stem cell transplantation
Acute myeloid leukemia
Survival
Recurrence rates
title Clinical outcomes of allogeneic hematopoietic stem cell transplant with different stem cell sources in infant acute myeloid leukemia
title_full Clinical outcomes of allogeneic hematopoietic stem cell transplant with different stem cell sources in infant acute myeloid leukemia
title_fullStr Clinical outcomes of allogeneic hematopoietic stem cell transplant with different stem cell sources in infant acute myeloid leukemia
title_full_unstemmed Clinical outcomes of allogeneic hematopoietic stem cell transplant with different stem cell sources in infant acute myeloid leukemia
title_short Clinical outcomes of allogeneic hematopoietic stem cell transplant with different stem cell sources in infant acute myeloid leukemia
title_sort clinical outcomes of allogeneic hematopoietic stem cell transplant with different stem cell sources in infant acute myeloid leukemia
topic Hematopoietic stem cell transplantation
Acute myeloid leukemia
Survival
Recurrence rates
url https://doi.org/10.1038/s41598-025-05602-z
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