Prognostic stratification for children with hepatoblastoma: The SIOPEL experience
Prognostic stratification for children with hepatoblastoma: The SIOPEL experience
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DOI：
10.1016/j.ejca.2011.12.011
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发表时间：
2012-07-01
影响因子：
8.4
通讯作者：
Perilongo, Giorgio
中科院分区：
文献类型：
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作者：
Maibach, Rudolf;Roebuck, Derek;Perilongo, Giorgio
目的：为了确定新诊断的肝母细胞瘤的长期结果相关的因素，并定义亚组的临床研究，定制治疗的个别patient.Patients和方法：在1995年和2006年之间的SIOPEL组进行了两项临床试验，建立风险适应性治疗肝母细胞瘤患者。将患者分为高风险（AFP < 100 ng/mL和/或PRETEXT IV和/或血管浸润和/或肝外腹腔内疾病（V+/P+/E+）和/或转移）和标准风险（所有其他）。进一步探讨了这些因素加上多灶性、PRETEXT III、AFP > 1，200，000 ng/mL、患者年龄、血小板计数和组织学的层次结构。结果指标为无事件生存率（EFS）。在541例患者中，EFS降低与AFP < 100 ng/ml显著相关（风险比[HR] 4.09，95%置信区间2.16-7.75），AFP >= 1.2 x 10（6）ng/mL（2.48，1.47-4.17），转移性疾病（3.02、2.05-4.44），前言四（2.15，1.19-3.87），多灶性（1.59，1.01-2.50），年龄>5岁（2.76，1.68-4.53）;与小细胞未分化（SCU）组织学交界（2.29，95%置信区间0.91-5.77）;但未使用PRETEXT III，年龄30-60个月，血小板计数或V+/P+/E+。通过使用显著性因素和SCU对人群进行分层，我们确定了三个不同的预后组：PRETEXT I/II/III，无其他因素，3年EFS为90%，PRETEXT IV和/或多灶性肿瘤和/或年龄> 5岁和/或AFP > 1.2 x 10（6），3年EFS为71%，SCU和/或AFP < 100 ng/mL和/或结论：对新诊断肝母细胞瘤的临床研究进行预后分层时，应考虑PRETEXT、转移性疾病、AFP、多灶性、年龄和SCU组织学等因素。(C)2011爱思唯尔有限公司保留所有权利。
Purpose: To identify factors relevant to long-term outcome in newly diagnosed hepatoblastoma, and define subgroups for clinical research on tailoring treatment to the individual patient.Patients and methods: Between 1995 and 2006 the SIOPEL group conducted two clinical trials which established risk-adapted therapy for hepatoblastoma patients. Patients were stratified into high-risk (AFP < 100 ng/mL and/or PRETEXT IV and/or vascular invasion and/or extra-hepatic intra-abdominal disease (V+/P+/E+) and/or metastases) and standard-risk (all others). The hierarchy of these factors plus multifocality, PRETEXT III, AFP > 1,200,000 ng/mL, patient age, platelet count and histology were further explored. The outcome measure was event-free survival (EFS).Results: In 541 patients, reduced EFS correlated significantly with AFP < 100 ng/ml (hazard ratio [HR] 4.09, 95% confidence interval 2.16-7.75), AFP >= 1.2 x 10(6) ng/mL (2.48, 1.47-4.17), metastatic disease (3.02, 2.05-4.44), PRETEXT IV (2.15, 1.19-3.87), multifocality (1.59, 1.01-2.50), age>5 years (2.76, 1.68-4.53); borderline with small cell undifferentiated (SCU) histology (2.29, 95% confidence interval 0.91-5.77); but not with PRETEXT III, age 30-60 months, platelet count or V+/P+/E+. By using the significant factors and SCU to stratify the population, we have identified three distinct prognostic groups: PRETEXT I/II/III, and no other factors, have 3 year EFS of 90%, PRETEXT IV and/or multifocal tumour and/or age > 5 years and/or AFP > 1.2 x 10(6) have 3 year EFS of 71% and SCU and/or AFP < 100 ng/mL and/or metastatic have a 3 year EFS of 49%.Conclusion: Prognostic stratification for clinical research on newly diagnosed hepatoblastoma should take into consideration PRETEXT, metastatic disease, AFP, multifocality, age and SCU histology. (C) 2011 Elsevier Ltd. All rights reserved.