Khaderi Saira, Guiteau Jacfranz, Cotton Ronald T, O'Mahony Christine, Rana Abbas, Goss John A
Saira Khaderi, Jacfranz Guiteau, Ronald T Cotton, Christine O'Mahony, Abbas Rana, John A Goss, Division of Abdominal Transplantation, Michael E. DeBakey Department of Surgery, Baylor College of Medicine, Houston, TX 77030, United States.
World J Transplant. 2014 Dec 24;4(4):294-8. doi: 10.5500/wjt.v4.i4.294.
Hepatoblastoma (HB) is the most common primary liver tumor in children and accounts for two-thirds of all malignant liver neoplasms in the pediatric population. For patients with advanced HB (unresectable or unresponsive to chemotherapy), combined treatment with chemotherapy and liver transplantation is an excellent option. The etiology of HB is mostly obscure because of its extreme rarity although some inherited syndromes and very low birth weight have been associated with it. The prognosis for children with HB has significantly improved in the past three decades thanks to advancements in chemotherapy, surgical resection and postoperative care. In 2002 a surgical staging system called pretreatment extent of disease (PRETEXT) was designed to allow a universal, multidisciplinary approach to patients with HB. Between one-third to two-thirds of patients initially present with unresectable tumors or distant metastases, but up to 85% of these tumors become operable after neoadjuvant chemotherapy. Patients with PRETEXT categories 1, 2, and some 3 are referred for neoadjuvant chemotherapy followed by surgical resection with the goal of complete tumor removal. Classic treatments regimens include a combination of cisplatin, fluorouracil, and vincristine or cisplatin and doxorubicin. Liver transplantation is the only treatment option for unresectable HB. In 2010 the pediatric end-stage liver disease, a pediatric-specific scoring system that determines a patient's ranking on the liver transplant list, began to award additional "exception" points for patients with HB. We analyzed the Standard Transplant Analysis and Research dataset to assess the impact of changes in exception point criteria for HB on outcomes after liver transplantation at Texas Children's Hospital in Houston, Texas. We found that patients who were listed for transplantation with current HB exception criteria experienced a shorter waitlist time but survival was similar between the two eras.
肝母细胞瘤（HB）是儿童最常见的原发性肝脏肿瘤，占儿科人群所有恶性肝脏肿瘤的三分之二。对于晚期HB患者（不可切除或对化疗无反应），化疗与肝移植联合治疗是一个很好的选择。HB的病因大多不明，因为其极为罕见，尽管一些遗传综合征和极低出生体重与之有关。由于化疗、手术切除和术后护理的进展，过去三十年中HB患儿的预后有了显著改善。2002年，一种名为疾病术前范围（PRETEXT）的手术分期系统被设计出来，以便对HB患者采用通用的多学科方法。三分之一至三分之二的患者最初表现为不可切除的肿瘤或远处转移，但这些肿瘤中高达85%在新辅助化疗后变得可切除。PRETEXT分类为1、2和部分3的患者接受新辅助化疗，然后进行手术切除，目标是完全切除肿瘤。经典治疗方案包括顺铂、氟尿嘧啶和长春新碱或顺铂和阿霉素联合使用。肝移植是不可切除HB的唯一治疗选择。2010年，儿科终末期肝病（一种确定患者在肝移植名单上排名的儿科特定评分系统）开始为HB患者授予额外的“例外”分数。我们分析了标准移植分析和研究数据集，以评估德克萨斯州休斯顿市德克萨斯儿童医院HB例外点标准的变化对肝移植后结局的影响。我们发现，按照当前HB例外标准列入移植名单的患者等待名单时间较短，但两个时代的生存率相似。
