Pritchard J, Brown J, Shafford E, Perilongo G, Brock P, Dicks-Mireaux C, Keeling J, Phillips A, Vos A, Plaschkes J
Institute of Child Health, St Bartholomew's Hospital, London, UK.
J Clin Oncol. 2000 Nov 15;18(22):3819-28. doi: 10.1200/JCO.2000.18.22.3819.
Hepatoblastoma (HB) is a rare malignant liver tumor which occurs almost exclusively in childhood. In the 1970s, survival was approximately 20% to 30%. Since the introduction of cisplatin (PLA) and doxorubicin (DO) into the chemotherapy regimens used to treat these patients, the survival rate has improved dramatically. In most recent studies, primary surgery preceded chemotherapy. In this study by the liver group of the International Society of Pediatric Oncology the aim was to improve survival and reduce operative morbidity and mortality by using preoperative chemotherapy.
After biopsy and assessment of pretreatment extent of disease all patients were treated with continuous 24-hour intravenous infusion of PLA 80 mg/m(2) followed by DO 60 mg/m(2) over 48 hours (PLADO). After four courses of this chemotherapy, patients were reassessed. Where possible, the primary tumor was resected and treatment completed with two more courses of chemotherapy.
One hundred fifty-four patients were registered in the study, and 138 received preoperative chemotherapy. One hundred thirteen (82%) showed a partial response with tumor shrinkage and serial decrease of serum alpha-fetoprotein levels. One hundred fifteen patients had delayed surgery, and 106 (including six with liver transplants) had complete resection of primary tumor. Five-year event-free survival was 66%, and overall survival was 75%.
This study demonstrates that international collaboration on a large scale is feasible. The toxicity of chemotherapy and morbidity of surgery were acceptable and the overall survival gratifyingly high. We now regard PLADO chemotherapy and delayed surgery to be the best available treatment for children with HB. Other treatment programs should be measured against this standard.
肝母细胞瘤（HB）是一种罕见的恶性肝脏肿瘤，几乎仅发生于儿童期。在20世纪70年代，生存率约为20%至30%。自从顺铂（PLA）和阿霉素（DO）被引入用于治疗这些患者的化疗方案后，生存率有了显著提高。在最近的大多数研究中，主要手术在化疗之前进行。在国际小儿肿瘤学会肝脏组的这项研究中，目的是通过使用术前化疗来提高生存率并降低手术发病率和死亡率。
在活检并评估疾病的预处理范围后，所有患者接受持续24小时静脉输注80mg/m²的PLA，随后在48小时内输注60mg/m² 的DO（PLADO）。经过四个疗程的这种化疗后，对患者进行重新评估。在可能的情况下，切除原发肿瘤并再进行两个疗程的化疗以完成治疗。
154例患者登记参加该研究，138例接受了术前化疗。113例（82%）显示部分缓解，肿瘤缩小且血清甲胎蛋白水平持续下降。115例患者手术延迟，106例（包括6例接受肝移植者）原发肿瘤得以完全切除。五年无事件生存率为66%，总生存率为75%。
本研究表明大规模的国际合作是可行的。化疗毒性和手术发病率是可接受的，且总生存率高得令人满意。我们现在认为PLADO化疗和延迟手术是治疗HB患儿的最佳可用疗法。其他治疗方案应以该标准进行衡量。
