单倍体相合造血干细胞移植可能改善首次完全缓解后高危 T 细胞急性淋巴细胞白血病儿童的长期生存。
Haploidentical hematopoietic stem cell transplantation may improve long-term survival for children with high-risk T-cell acute lymphoblastic leukemia in first complete remission.
机构信息
Department of Pediatrics, Peking University People's Hospital, Peking University, Beijing 100044, China.
Department of Hematology, Peking University People's Hospital, Peking University Institute of Hematology, National Clinical Research Center for Hematologic Disease, Beijing Key Laboratory of Hematopoietic Stem Cell Transplantation, Beijing 100044, China.
出版信息
Chin Med J (Engl). 2022 Apr 20;135(8):940-949. doi: 10.1097/CM9.0000000000001999.
BACKGROUND
The role of allogeneic hematopoietic stem cell transplantation (allo-HSCT) in children with high-risk (HR) T-cell acute lymphoblastic leukemia (T-ALL) in first complete remission (CR1) is still under evaluation. Moreover, relapse is the main factor affecting survival. This study aimed to explore the effect of allo-HSCT (especially haploidentical HSCT [haplo-HSCT]) on improving survival and reducing relapse for HR childhood T-ALL in CR1 and the prognostic factors of childhood T-ALL in order to identify who could benefit from HSCT.
METHODS
A total of 74 newly diagnosed pediatric T-ALL patients between January 1, 2012 and June 30, 2018 were enrolled in this retrospective study. Patients were stratified into the low-risk chemotherapy cohort (n = 16), HR chemotherapy cohort (n = 31), and HR transplant cohort (n = 27). Characteristics, survival outcomes, and prognostic factors of all patients were then analyzed.
RESULTS
Patient prognosis in the HR chemotherapy cohort was significantly worse than that in the low-risk chemotherapy cohort (5year overall survival [OS]: 58.5% vs. 100%, P = 0.003; 5-year event-free survival [EFS]: 54.1% vs. 83.4%, P = 0.010; 5-year cumulative incidence of relapse [CIR]: 45.2% vs. 6.3%, P = 0.011). In HR patients, allo-HSCT improved the 5-year EFS and CIR compared to that of chemotherapy (5-year EFS: 80.1% vs. 54.1%, P = 0.041; 5-year CIR: 11.6% vs. 45.2%, P = 0.006). The 5-year OS was higher in the HR transplant cohort than that in the HR chemotherapy cohort (81.0% vs. 58.5%, P = 0.084). Minimal residual disease re-emergence was an independent risk factor for 5-year OS, EFS, and CIR; age ≥10 years was an independent risk factor for OS and EFS; and high white blood cell count was an independent risk factor for EFS and CIR.
CONCLUSION
Allo-HSCT, especially haplo-HSCT, could effectively reduce relapse of children with HR T-ALL in CR1.
背景
异体造血干细胞移植（allo-HSCT）在初诊缓解（CR1）期高危（HR）T 细胞急性淋巴细胞白血病（T-ALL）儿童中的作用仍在评估中。此外，复发是影响生存的主要因素。本研究旨在探讨 allo-HSCT（尤其是单倍体 HSCT[haplo-HSCT]）对改善 HR 儿童 T-ALL 在 CR1 期的生存和降低复发率的作用，以及识别哪些患者可能受益于 HSCT，以确定儿童 T-ALL 的预后因素。
方法
本回顾性研究共纳入 2012 年 1 月 1 日至 2018 年 6 月 30 日期间诊断的 74 例新确诊的儿科 T-ALL 患者。患者分为低危化疗组（n=16）、高危化疗组（n=31）和高危移植组（n=27）。分析所有患者的特征、生存结果和预后因素。
结果
高危化疗组患者的预后明显差于低危化疗组（5 年总生存[OS]：58.5% vs. 100%，P=0.003；5 年无事件生存[EFS]：54.1% vs. 83.4%，P=0.010；5 年累积复发率[CIR]：45.2% vs. 6.3%，P=0.011）。在 HR 患者中，allo-HSCT 与化疗相比，可提高 5 年 EFS 和 CIR（5 年 EFS：80.1% vs. 54.1%，P=0.041；5 年 CIR：11.6% vs. 45.2%，P=0.006）。高危移植组的 5 年 OS 高于高危化疗组（81.0% vs. 58.5%，P=0.084）。微小残留病复发是 5 年 OS、EFS 和 CIR 的独立危险因素；年龄≥10 岁是 OS 和 EFS 的独立危险因素；白细胞计数高是 EFS 和 CIR 的独立危险因素。
结论
allo-HSCT，特别是 haplo-HSCT，可有效降低 HR T-ALL 患者 CR1 期的复发率。
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