E2A-PBX1 exhibited a promising prognosis in pediatric acute lymphoblastic leukemia treated with the CCLG-ALL2008 protocol.
E2A-PBX1 exhibited a promising prognosis in pediatric acute lymphoblastic leukemia treated with the CCLG-ALL2008 protocol.
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DOI：
10.2147/ott.s115257
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发表时间：
2016
影响因子：
4
通讯作者：
Hu S
中科院分区：
文献类型：
--
作者：
Hu Y;He H;Lu J;Wang Y;Xiao P;Li J;Li J;Sun Y;Lv H;Fan J;Yao Y;Chai Y;Hu S
本研究的目的是观察采用CCLG-ALL2008方案治疗的e2a - pbx1阳性急性淋巴细胞白血病（ALL）患儿的预后。从2008年12月至2013年9月，349名中国儿童b细胞前ALL患者被纳入本研究。其中，20例E2A-PBX1表达患者和223例无E2A-PBX1基因表达患者被分为两组。分析比较两组患者的临床和生物学特征以及5年无事件生存期（EFS）、无复发生存期（RFS）和总生存期（OS）。349例患者中有20例（5.7%）检测到E2A-PBX1融合转录物。与基因阴性亚组相比，E2A-PBX1患者年龄更年轻，但在初诊时白细胞计数和性别分布没有显着差异。E2A-PBX1亚组存在较多的劣等核型（P=0.035）。采用CCLG-ALL2008治疗方案，E2A-PBX1患者在时间点1 （TP1， P=0.039）表现出较低的最小残留病（MRD）水平（<10−4），但没有较好的类固醇反应或组织学缓解。我们还观察到一个有希望的生存结果，5年EFS达到95.0%±4.9%，而基因阴性组为66.3%±3.9% （P=0.039）。然而，我们没有发现RFS （P=0.061）和OS （P=0.113）有显著差异。我们的数据提供了中国儿科患者的临床观察。e2a - pbx1阳性ALL患者受益于CCLG-ALL2008方案，这是一项基于风险的强化治疗试验，MRD水平较低，RFS持续时间较长，尽管他们在初级诊断时没有有利的特征。
The objective of this study was to observe the prognosis of pediatric patients with E2A-PBX1-positive acute lymphoblastic leukemia (ALL) from the treatment with the CCLG-ALL2008 protocol. Three hundred and forty-nine Chinese pediatric patients with pre-B-cell ALL were enrolled in this study from December 2008 to September 2013. Of these, 20 patients with E2A-PBX1 expression and 223 without the gene expression were stratified into two cohorts. Clinical and biological characteristics and 5-year event-free survival (EFS), relapse-free survival (RFS), and overall survival (OS) were analyzed and compared between these two groups. The E2A-PBX1 fusion transcript was detected in 20 of 349 (5.7%) patients. Compared with the gene-negative subgroup, patients with E2A-PBX1 were younger in age but did not show significant differences in white blood cell (WBC) count or gender distribution at primary diagnosis. Moreover, there were more inferior karyotypes detected in the E2A-PBX1 subgroup (P=0.035). With the CCLG-ALL2008 treatment protocol, patients with E2A-PBX1 showed a favorable treatment response with lower minimal residual disease (MRD) levels (<10−4) at time point 1 (TP1, P=0.039) but no superior steroid response or histological remission. We also observed a promising survival outcome, with a 5-year EFS reaching 95.0%±4.9% versus 66.3%±3.9% in the gene-negative group (P=0.039). However, we did not find significant differences in RFS (P=0.061) and OS (P=0.113). Our data provided clinical observation of Chinese pediatric patients. Patients with E2A-PBX1-positive ALL benefited well from the CCLG-ALL2008 protocol, a risk-based intensified treatment trial, with lower levels of MRD and longer RFS duration though they had no favorable characteristics at primary diagnosis.