Hong Kyung Taek, Choi Jung Yoon, Kim Hyery, Im Ho Joon, Hahn Seung Min, Lyu Chuhl Joo, Ju Hee Young, Yoo Keon Hee, Yang Eu Jeen, Yoon Sung-Soo, Park Hyeon Jin, Choi Hyoung Soo, Chueh Hee Won, Yang Deok-Hwan, Moon Joon Ho, Lee Jae Min, Lee Jung-Hee, Kim Jeong-A, Won Jong-Ho, Kang Hyoung Jin
Department of Pediatrics, Seoul National University Children's Hospital, Seoul National University College of Medicine Seoul National University Cancer Research Institute Seoul Republic of Korea.
Department of Pediatrics University of Ulsan College of Medicine, Asan Medical Center Seoul Republic of Korea.
Hemasphere. 2025 Jun 17;9(6):e70158. doi: 10.1002/hem3.70158. eCollection 2025 Jun.
Acute lymphoblastic leukemia (ALL) is the predominant malignancy in pediatric patients, and allogeneic hematopoietic stem cell transplantation (HSCT) plays a critical role in high-risk cases. However, real-world nationwide data comparing the outcomes of conditioning regimens are limited. This nationwide registry-based study analyzed data from 270 Korean pediatric patients with high-risk or relapsed ALL who underwent their first allogeneic HSCT with myeloablative conditioning. Among all analyzed patients, 118 received total body irradiation-based conditioning (MAC-TBI) and 152 received chemotherapy-based conditioning (MAC-Chemotherapy), of whom 96.6% underwent busulfan-based regimens. MAC-TBI recipients were older at diagnosis and at HSCT. No significant differences were observed between groups in neutrophil or platelet engraftment times, or infused CD34+ cell doses. Acute graft-versus-host disease (GVHD) incidences (grades II-IV and III-IV) were comparable, although chronic GVHD incidence tended to be lower in the MAC-Chemotherapy group (21.0% vs. 31.1%, P = 0.072). Additionally, the 5-year event-free survival (EFS) rates for MAC-TBI versus MAC-Chemotherapy were 73.7% and 69.8% (P = 0.827), respectively; the 5-year overall survival (OS) rates were 76.3% and 80.2% (P = 0.941), respectively, indicating that conditioning regimen did not significantly impact survival. Pediatric disease risk index, recent HSCT era, haploidentical donor type, and pre-transplant disease status independently influenced EFS and OS, whereas anti-thymocyte globulin administration significantly improved moderate-to-severe chronic GVHD, leukemia-free survival. This nationwide real-world analysis demonstrated comparable outcomes between myeloablative TBI-based and chemotherapy-based conditioning regimens in pediatric patients with ALL. These findings may inform the development of improved treatment strategies for this patient population.
急性淋巴细胞白血病（ALL）是儿科患者中最主要的恶性肿瘤，异基因造血干细胞移植（HSCT）在高危病例中起着关键作用。然而，比较预处理方案疗效的全国性真实世界数据有限。这项基于全国登记处的研究分析了270例接受首次异基因HSCT清髓预处理的韩国高危或复发ALL儿科患者的数据。在所有分析的患者中，118例接受了基于全身照射的预处理（MAC-TBI），152例接受了基于化疗的预处理（MAC-化疗），其中96.6%接受了基于白消安的方案。MAC-TBI受者诊断时和HSCT时年龄更大。两组在中性粒细胞或血小板植入时间或输注的CD34+细胞剂量方面未观察到显著差异。急性移植物抗宿主病（GVHD）发生率（II-IV级和III-IV级）相当，尽管MAC-化疗组慢性GVHD发生率倾向于较低（21.0%对31.1%，P = 0.072）。此外，MAC-TBI与MAC-化疗的5年无事件生存率（EFS）分别为73.7%和69.8%（P = 0.827）；5年总生存率（OS）分别为76.3%和80.2%（P = 0.941），表明预处理方案对生存没有显著影响。儿科疾病风险指数、最近的HSCT时代、单倍体相合供体类型和移植前疾病状态独立影响EFS和OS，而抗胸腺细胞球蛋白的使用显著改善了中重度慢性GVHD、无白血病生存率。这项全国性真实世界分析表明，在ALL儿科患者中，基于清髓性TBI和基于化疗的预处理方案疗效相当。这些发现可能为该患者群体改进治疗策略的制定提供参考。
