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Original Article
Allogeneic Hematopoietic Stem Cell Transplantation in Pediatric and Young Adult Patients with Chronic Myeloid Leukemia in Tyrosine Kinase Inhibitor Era: A Study of the Korean Blood and Marrow Transplantation Registry
Hee Young Ju1orcid , Hyoung Soo Choi2orcid , Hyeon Jin Park3, Keon Hee Yoo1, Chuhl Joo Lyu4, Ho Joon Im5, Min Kyoung Kim6, Yeung-Chul Mun7, Joon Ho Moon8, Sung-Soo Yoon9, Eunyoung Lee10, Jae Hoon Lee11, Je-Hwan Lee12, So Young Chong13, June-Won Cheong14, Seunghyun Won15, on behalf of the Korean Society of Blood and Marrow Transplantation

DOI: https://doi.org/10.4143/crt.2024.1186 [Accepted]
Published online: May 7, 2025
1Department of Pediatrics, Samsung Medical Center, Sungkyunkwan University School of Medicine, Seoul, Korea
2Department of Pediatrics, Seoul National University Bundang Hospital, Seoul National University College of Medicine, Seongnam, Korea
3Center for Pediatric Cancer, Department of Pediatrics, National Cancer Center, Goyang, Korea
4Division of Pediatric Hematology and Oncology, Department of Pediatrics, Yonsei University College of Medicine, Seoul, Korea
5Division of Pediatric Hematology/Oncology, Department of Pediatrics, Asan Medical Center Children's Hospital, University of Ulsan College of Medicine, Seoul, Korea
6Department of Medicine, Yeungnam University College of Medicine, Daegu, Korea
7Department of Internal Medicine, Ewha Womans University School of Medicine, Seoul, Korea
8Department of Hematology/Oncology, Kyungpook National University Hospital, Kyungpook National University School of Medicine, Daegu, Korea
9Department of Internal Medicine, Seoul National University Hospital, Seoul National University College of Medicine, Seoul, Korea
10Department of Internal Medicine, Center for Hematologic Malignancy, National Cancer Center, Goyang, Korea
11Department of Internal Medicine, Gachon University Gil Hospital, Incheon, Korea
12Department of Hematology, Asan Medical Center, University of Ulsan College of Medicine, Seoul, Korea
13Department of Internal Medicine, CHA Bundang Medical Center, CHA University, Seongnam, Korea
14Department of Internal Medicine, Yonsei University College of Medicine, Seoul, Korea
15Medical Research Collaborating Center, Seoul National University Bundang Hospital, Seongnam, Korea
Corresponding author:  Hyoung Soo Choi
Tel: 82-31-787-7797 
Email: choihs1786@snubh.org
Received: 10 December 2024   • Accepted: 29 April 2025
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Purpose
Chronic myeloid leukemia (CML) in children, adolescents, and young adults is rare and differs from older adults. This study evaluated the outcomes of allogeneic hematopoietic stem cell transplantation (HSCT) in young Korean CML patients during the tyrosine kinase inhibitor (TKI) era.
Materials and Methods
A retrospective analysis of 35 CML patients aged <40 years who underwent allogeneic HSCT from 2009 to 2019 was conducted using Korean Blood and Marrow Transplantation Registry data. Patients were grouped by age <20 years at HSCT (Group 1, n=15) and 20-40 years at HSCT (Group 2, n=20). Survival outcomes including overall survival (OS), relapse-free survival (RFS), and event-free survival (EFS) were analyzed using the Kaplan–Meier method.
Results
The median time between diagnosis and HSCT was 8.9 months. All the patients achieved engraftment but platelet recovery was significantly slower in Group 1 (p=0.034). Acute and chronic graft-versus-host disease occurred in 54.3% and 34.3%, respectively. Five-year OS, RFS, and EFS rates of total patients were 66.8%, 50.8%, and 47.6%, with better OS was observed in Group 1 by multivariable analysis (p=0.048). Disease status at HSCT was a significant predictor of OS (p=0.028), RFS (p=0.003) and EFS (p=0.004). Disease progression occurred in 13 out of 35 patients (37.1%); treatment-related mortality accounted for 63.6% of deaths (7 out of 11).
Conclusion
When performed at a younger age, allogeneic HSCT result in superior outcome in CML. Achieving remission before HSCT is critical for improved outcomes, highlighting the importance of pretransplant remission via optimal TKI strategies and minimal residual disease monitoring.

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    Allogeneic Hematopoietic Stem Cell Transplantation in Pediatric and Young Adult Patients with Chronic Myeloid Leukemia in Tyrosine Kinase Inhibitor Era: A Study of the Korean Blood and Marrow Transplantation Registry
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