Morokawa Hirokazu, Hirabayashi Koichi, Furui Yu, Okura Eri, Saito Shoji, Nakazawa Yozo
Department of Pediatrics, Shinshu University School of Medicine, Matsumoto, Japan.
Hematol Oncol. 2025 Jan;43(1):e70026. doi: 10.1002/hon.70026.
Recent studies have indicated that total body irradiation (TBI)-based reduced-toxicity conditioning (RTC) may be a potential treatment modality, especially in adults with leukemia. However, its efficacy and safety in children with hematological malignancies remain unclear. To investigate the long-term outcomes and safety of allogeneic hematopoietic stem cell transplantation (allo-HSCT) using an 8-Gray (Gy) TBI/fludarabine (FLU)/cyclophosphamide (CY) RTC in children with hematological malignancies. We included 66 consecutive patients with leukemia, lymphoma, or myelodysplastic syndrome in this retrospective cohort study. Participants were < 25 years old and received an 8-Gy TBI/FLU/CY RTC regimen followed by the first allo-HSCT at Shinshu University Hospital between March 2004 and March 2021. The 5-year overall and relapse-free survival probabilities were 88.2% and 76.5%, respectively, in the lymphoid malignancy group. The myeloid malignancy group had probabilities of 72.4% and 58.6%, respectively. The 5-year cumulative incidences of relapse and non-relapse mortality were 20.6% and 2.9%, respectively, in the lymphoid malignancy group. These incidences were 37.9% and 3.4%, respectively, in the myeloid malignancy group. All patients had engraftment without early relapse and none developed grade 5 regimen-related toxicity within 28 days after allo-HSCT. Nonetheless, two patients had congenital abnormalities caused by chromosomal aberrations and died without relapse. 8-Gy TBI/FLU/CY RTC was safe in children with hematological malignancies, regardless of the donor source. However, safety concerns were noted in cases of chromosomal aberration-induced congenital abnormalities. Additionally, patients in the lymphoid and myeloid malignancy groups had favorable prognoses.
近期研究表明，基于全身照射（TBI）的低毒性预处理（RTC）可能是一种潜在的治疗方式，尤其是对于成年白血病患者。然而，其在儿童血液系统恶性肿瘤中的疗效和安全性仍不明确。为了研究采用8格雷（Gy）TBI/氟达拉滨（FLU）/环磷酰胺（CY）进行低毒性预处理的异基因造血干细胞移植（allo-HSCT）在儿童血液系统恶性肿瘤中的长期疗效和安全性。在这项回顾性队列研究中，我们纳入了66例连续的白血病、淋巴瘤或骨髓增生异常综合征患者。参与者年龄小于25岁，于2004年3月至2021年3月在信州大学医院接受了8-Gy TBI/FLU/CY低毒性预处理方案，随后进行了首次allo-HSCT。在淋巴系统恶性肿瘤组中，5年总生存率和无复发生存率分别为88.2%和76.5%。骨髓系统恶性肿瘤组的这两个概率分别为72.4%和58.6%。在淋巴系统恶性肿瘤组中，5年复发累积发生率和非复发死亡率分别为20.6%和2.9%。在骨髓系统恶性肿瘤组中，这些发生率分别为37.9%和3.4%。所有患者均实现植入且无早期复发，在allo-HSCT后28天内均未发生5级方案相关毒性。尽管如此，两名患者因染色体畸变导致先天性异常，未复发而死亡。8-Gy TBI/FLU/CY低毒性预处理对儿童血液系统恶性肿瘤患者是安全的，无论供体来源如何。然而，在染色体畸变引起先天性异常的病例中存在安全问题。此外，淋巴系统和骨髓系统恶性肿瘤组的患者预后良好。
