Department of Pediatrics, Peking University People's Hospital, Peking University, Beijing, China.
Department of Hematology, Peking University People's Hospital, Peking University Institute of Hematology, Beijing Key Laboratory of Hematopoietic Stem Cell Transplantation, Peking University, Beijing, China.
Leuk Res. 2020 Apr;91:106333. doi: 10.1016/j.leukres.2020.106333. Epub 2020 Feb 20.
B-cell acute lymphoblastic leukemia (B-ALL) with MLL-rearrangements (MLL-r) is rare in pediatric patients (aged >1 year), and optimal treatment strategies remain unclear. This study aimed to retrospectively evaluate the clinical characteristics, outcomes, and effects of allogeneic hematopoietic stem cell transplantation (allo-HSCT) of 37 non-infant children with t(v;11q23)/MLL-r B-ALL. Their 4-year overall survival (OS), event-free survival (EFS), and cumulative incidence of relapse (CIR) were 69.8 %, 58.2 %, and 39.1 %, respectively, and differed significantly between patients receiving allo-HSCT (18/19 cases received haploidentical [haplo]-HSCT) at the first complete remission (HSCT at CR1, n = 19; 87.4 %, 89.5 % and 5.3 %) and those continuing consolidation therapy (Non-HSCT at CR1, n = 18; 52.2 %, 25.9 %, and 74.1 %, respectively), and the p values were 0.022, <0.001 and <0.001, respectively. Of the 13 patients experiencing relapse during consolidation chemotherapy, the five continuing with chemotherapy only died within 44 months, and the eight patients opting for allo-HSCT after CR2 had a 4-year OS of 57.1 %. Multivariate analysis revealed HSCT at CR1 as the only independent protective factor for OS, EFS, and CIR. The present results indicate that allo-HSCT (especially haplo-HSCT) at CR1 may decrease the relapse rate and improve the prognosis of non-infant children with t(v;11q23)/MLL-r B-ALL.
B 细胞急性淋巴细胞白血病（B-ALL）伴 MLL 重排（MLL-r）在儿童患者（>1 岁）中较为罕见，且其最佳治疗策略仍不明确。本研究旨在回顾性评估 37 例 t(v;11q23)/MLL-r B-ALL 非婴儿患儿的临床特征、结局及异基因造血干细胞移植（allo-HSCT）的效果。其 4 年总生存（OS）、无事件生存（EFS）和累积复发率（CIR）分别为 69.8%、58.2%和 39.1%，接受 allo-HSCT（19 例接受单倍体[haplo]-HSCT）的患者与继续巩固治疗的患者（18 例在 CR1 时未接受 HSCT，分别为 87.4%、89.5%和 5.3%）相比差异有统计学意义（n=19；分别为 87.4%、89.5%和 5.3%），p 值分别为 0.022、<0.001 和<0.001。在巩固化疗期间发生复发的 13 例患者中，仅继续接受化疗的 5 例患者在 44 个月内死亡，而在 CR2 后选择 allo-HSCT 的 8 例患者的 4 年 OS 为 57.1%。多变量分析显示，CR1 时行 HSCT 是 OS、EFS 和 CIR 的唯一独立保护因素。本研究结果表明，CR1 时行 allo-HSCT（尤其是 haplo-HSCT）可能降低复发率并改善 t(v;11q23)/MLL-r B-ALL 非婴儿患儿的预后。
