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采用8格雷全身照射、氟达拉滨和环磷酰胺进行低毒性预处理对血液系统恶性肿瘤儿童、青少年和青年成人的长期疗效

Long-Term Outcomes of Reduced-Toxicity Conditioning Using 8-Gray Total Body Irradiation, Fludarabine, and Cyclophosphamide in Children, Adolescents, and Young Adults With Hematological Malignancies.

作者信息

Morokawa Hirokazu, Hirabayashi Koichi, Furui Yu, Okura Eri, Saito Shoji, Nakazawa Yozo

机构信息

Department of Pediatrics, Shinshu University School of Medicine, Matsumoto, Japan.

出版信息

Hematol Oncol. 2025 Jan;43(1):e70026. doi: 10.1002/hon.70026.

Abstract

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.

摘要

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。

https://cdn.ncbi.nlm.nih.gov/pmc/blobs/08ba/11646108/86ed72ae6455/HON-43-e70026-g001.jpg

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