Successful Treatment of Childhood High-Risk Hepatoblastoma With Dose-Intensive Multiagent Chemotherapy and Surgery: Final Results of the SIOPEL-3HR Study
Successful Treatment of Childhood High-Risk Hepatoblastoma With Dose-Intensive Multiagent Chemotherapy and Surgery: Final Results of the SIOPEL-3HR Study
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DOI：
10.1200/jco.2009.22.4857
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发表时间：
2010-05-20
影响因子：
45.3
通讯作者：
Perilongo, Giorgio
中科院分区：
文献类型：
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作者：
Zsiros, Jozsef;Maibach, Rudolf;Perilongo, Giorgio
PurposeThe主要目的是确定一种新设计的术前化疗方案的疗效，以提高高危肝母细胞瘤患儿的治愈率。所有肝脏切片均存在肿瘤（即，治疗前扩展IV [PRETEXT-IV]）或血管浸润（门静脉[P+]，三条肝静脉[V+]），或腹腔内肝外延伸（E+），或转移性疾病，或诊断时甲胎蛋白低于100 ng/mL。患者进行了治疗，交替周期的顺铂和卡铂加阿霉素（术前，n = 7;术后，n = 3）和延迟肿瘤切除术。ResultsOf 151例（150评价反应）118（78.7%）实现了部分反应化疗。115例（76.2%）患者通过肝部分切除（55.6%）或肝移植（20.6%）实现了肝脏肿瘤的完全切除。在106名儿童（70.2%）中，实现了所有肿瘤病变（包括转移瘤）的完全切除。在初始肺转移的患者中，52.2%的患者在单独化疗的情况下获得了肺部病灶的完全缓解。在初始PRETEXT-IV肿瘤作为唯一高风险特征的患者中，有一半的肿瘤可以通过部分肝切除术完全切除。整个组3年时的无事件（EFS）和总生存率（OS）估计值分别为65%（95% CI，57%至73%）和69%（95% CI，62%至77%）。EFS和OS的所有患者与PRETEXT-IV肿瘤分别为68%和69%，分别为56%和62%，分别为patients.ConclusionThe应用的治疗提供了很大比例的肿瘤切除，并与以前发表的结果相比，导致提高生存率的高危肝母细胞瘤患者。
PurposeThe primary objective was to determine the efficacy of a newly designed preoperative chemotherapy regimen in an attempt to improve the cure rate of children with high-risk hepatoblastoma.Patients and MethodsHigh risk was defined as follows: tumor in all liver sections (ie, Pretreatment Extension IV [PRETEXT-IV]), or vascular invasion (portal vein [P+], three hepatic veins [V+]), or intra-abdominal extrahepatic extension (E+), or metastatic disease, or alpha-fetoprotein less than 100 ng/mL at diagnosis. Patients were treated with alternating cycles of cisplatin and carboplatin plus doxorubicin (preoperatively, n = 7; postoperatively, n = 3) and delayed tumor resection.ResultsOf the 151 patients (150 evaluable for response) 118 (78.7%) achieved a partial response to chemotherapy. Complete resection of the liver tumor could be achieved in 115 patients (76.2%) either by partial hepatectomy (55.6%) or by liver transplantation (20.6%). In 106 children (70.2%), complete resection of all tumor lesions (including metastases) was achieved. Among the patients with initial lung metastases, 52.2% achieved complete remission of the lung lesions with chemotherapy alone. In half of the patients with initial PRETEXT-IV tumor as the only high-risk feature, the tumor could be completely resected with partial hepatectomy. Event-free (EFS) and overall survival (OS) estimates at 3 years were 65% (95% CI, 57% to 73%) and 69% (95% CI, 62% to 77%) for the whole group. EFS and OS for all patients with PRETEXT-IV tumor were 68% and 69%, respectively, and they were 56% and 62%, respectively, for patients with metastasis.ConclusionThe applied treatment rendered a great proportion of tumors resectable, and, in comparison with previously published results, led to an improved survival in patients with high-risk hepatoblastoma.