Prognostic implications of CEBPA mutations in pediatric acute myeloid leukemia: a report from the Japanese Pediatric Leukemia/Lymphoma Study Group.
Prognostic implications of CEBPA mutations in pediatric acute myeloid leukemia: a report from the Japanese Pediatric Leukemia/Lymphoma Study Group.
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DOI：
10.1038/bcj.2014.47
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发表时间：
2014-07-11
影响因子：
12.8
通讯作者：
Adachi, S.
中科院分区：
文献类型：
--
作者：
Matsuo, H.;Kajihara, M.;Tomizawa, D.;Watanabe, T.;Saito, A. M.;Fujimoto, J.;Horibe, K.;Kodama, K.;Tokumasu, M.;Itoh, H.;Nakayama, H.;Kinoshita, A.;Taga, T.;Tawa, A.;Taki, T.;Tanaka, S.;Adachi, S.
CCAAT/增强子结合蛋白α（CEBPA）突变是成人急性髓系白血病（AML）患者的有利预后因素；然而，很少有研究探讨它们对儿童 AML 患者的重要性。在这里，我们检查了 AML-05 研究中治疗的儿科 AML 患者的 CEBPA 突变状态和临床结果。我们发现 315 名可评估患者中有 47 名 (14.9%) 携带 CEBPA 突变； 26 例 (8.3%) 存在单突变 (CEBPA-single)，21 例 (6.7%) 存在双或三突变 (CEBPA-double)。排除核心结合因子-AML病例后，与野生型患者相比，携带CEBPA突变的患者表现出更好的总生存期（OS；P=0.048），但无事件生存期（EFS；P=0.051）。多变量分析确定CEBPA-单项和CEBPA-双项是整个队列中EFS的独立有利预后因素（风险比（HR）：0.47和0.33；P分别为0.02和0.01）。 CEBPA-double也是OS的独立有利预后因素（HR：0.30；P=0.04）。在正常核型队列中，CEBPA-double 仍然是 EFS 的独立有利因素（HR：0.28；P=0.04）。这些结果表明CEBPA突变，特别是CEBPA双突变，是儿科AML患者的一个独立的有利预后因素，这将对风险分层治疗产生重要影响。
CCAAT/enhancer-binding protein alpha (CEBPA) mutations are a favorable prognostic factor in adult acute myeloid leukemia (AML) patients; however, few studies have examined their significance in pediatric AML patients. Here we examined the CEBPA mutation status and clinical outcomes of pediatric AML patients treated in the AML-05 study. We found that 47 (14.9%) of the 315 evaluable patients harbored mutations in CEBPA; 26 cases (8.3%) harbored a single mutation (CEBPA-single) and 21 (6.7%) harbored double or triple mutations (CEBPA-double). After excluding core-binding factor-AML cases, patients harboring CEBPA mutations showed better overall survival (OS; P=0.048), but not event-free survival (EFS; P=0.051), than wild-type patients. Multivariate analysis identified CEBPA-single and CEBPA-double as independent favorable prognostic factors for EFS in the total cohort (hazard ratio (HR): 0.47 and 0.33; P=0.02 and 0.01, respectively). CEBPA-double was also an independent favorable prognostic factor for OS (HR: 0.30; P=0.04). CEBPA-double remained an independent favorable factor for EFS (HR: 0.28; P=0.04) in the normal karyotype cohort. These results suggest that CEBPA mutations, particularly CEBPA-double, are an independent favorable prognostic factor in pediatric AML patients, which will have important implications for risk-stratified therapy.
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影响因子：
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作者：
Staffas, Anna;Kanduri, Meena;Ehrencrona, Hans
通讯作者：
Ehrencrona, Hans
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