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Cancer Research and Treatment > Accepted Articles
doi: https://doi.org/10.4143/crt.2024.155    [Accepted]
The Effect of Hematopoietic Stem Cell Transplantation on Treatment Outcome in Children with Acute Lymphoblastic Leukemia
Hee Young Ju1 , Na Hee Lee2 , Eun Sang Yi3, Young Bae Choi4, So Jin Kim1, Ju Kyung Hyun1, Hee Won Cho1, Jae Kyung Lee1, Ji Won Lee1, Ki Woong Sung1, Hong Hoe Koo5, Keon Hee Yoo1,6,7
1Department of Pediatrics, Samsung Medical Center, Sungkyunkwan University School of Medicine, Seoul, Korea
2Department of Pediatrics, CHA Bundang Medical Center, CHA University, Seongnam, Korea
3Department of Pediatrics, Korea University Guro Hospital, Korea University College of Medicine, Seoul, Korea
4Department of Pediatrics, Ajou University Hospital, Suwon, Korea
5Korea Hemophilia Foundation, Seoul, Korea
6Cell & Gene Therapy Institute, Samsung Medical Center, Seoul, Korea
7Department of Health Sciences and Technology, SAIHST, Sungkyunkwan University, Seoul, Korea
Correspondence  Keon Hee Yoo ,Tel: 82-2-3410-3532, Fax: 82-2-3410-0043, Email: hema2170@skku.edu
Received: February 14, 2024;  Accepted: July 4, 2024.  Published online: July 5, 2024.
*Hee Young Ju and Na Hee Lee contributed equally to this work.
ABSTRACT
Purpose
Hematopoietic stem cell transplantation (HSCT) has been an important method of treatment in the advance of pediatric acute lymphoblastic leukemia (ALL). The indications for HSCT are evolving and require updated establishment. In this study, we aimed to investigate the efficacy of HSCT on the treatment outcome of pediatric ALL, considering the indications for HSCT and subgroups.
Materials and Methods
A retrospective analysis was conducted on ALL patients diagnosed and treated at a single center. Risk groups were categorized based on age at diagnosis, initial white blood cell count, disease lineage (B/T), and cytogenetic study results. Data on the patients’ disease status at HSCT and indications of HSCT were collected. Indications for HSCT were categorized as upfront HSCT at 1st complete remission, relapse, and refractory disease.
Results
Among the 549 screened patients, a total of 418 patients were included in the study; B-ALL (n=379) and T-ALL (n=39). HSCT was conducted on a total of 106 patients (25.4%), with a higher frequency as upfront HSCT in higher risk groups and specific cytogenetics. The overall survival (OS) was significantly better when done upfront than in relapsed or refractory state in T-ALL patients (p=0.0016). The KMT2A-rearranged ALL patients showed superior event-free survival (p=0.0023) and OS (p=0.0221) when HSCT was done as upfront treatment.
Conclusion
HSCT had a substantial positive effect in a specific subset of pediatric ALL. In particular, frontline HSCT for T-ALL and KMT2A-rearranged ALL offered a better prognosis than when HSCT was conducted in a relapsed or refractory setting.
Key words: Precursor cell lymphoblastic leukemia-lymphoma, Hematopoietic stem cell transplantation, Survival, KMT2A, Precursor T-cell lymphoblastic leukemia-lymphoma
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