Browne Marybeth, Sher Dani, Grant David, Deluca Enza, Alonso Estella, Whitington Peter F, Superina Riccardo A
Department of Surgery, Children's Memorial Hospital, Feinberg School of Medicine, Northwestern University, Chicago, IL 60614, USA.
J Pediatr Surg. 2008 Nov;43(11):1973-81. doi: 10.1016/j.jpedsurg.2008.05.031.
Complete resection with adjuvant chemotherapy is the accepted treatment for hepatoblastoma. The aim of this study is to evaluate our results of liver transplantation (LT) for tumors still unresectable after adequate chemotherapy.
All patients transplanted for hepatoblastoma from 2 institutions between 1990 and 2004 were included. Variables reviewed to determine impact on survival included the following: previous tumor resection, metastatic disease at diagnosis, microscopic vascular invasion, alpha-fetoprotein (AFP) levels at diagnosis and at transplant, tumor histology, and administration of posttransplantation chemotherapy. Effectiveness of pretransplantation chemotherapy was defined as a drop of more than 99% in peak AFP levels.
Fourteen patients were transplanted: 9 boys and 5 girls (age range, 18 months-13 years; mean age, 57 +/- 48 months). Patients were transplanted a mean of 4 +/- 1 months after diagnosis. Overall survival was 71% (10/14) with a mean follow-up of 46 months. All deaths were secondary to recurrent tumor. Of 10 patients who underwent a primary LT, 9 survived compared to only 1 of 4 transplanted for unresectable tumor recurrence after primary resection (90% vs 25%; P = .02). Decline in peak AFP of more than 99% was also associated with better survival (100% vs 56%; P = .08). Similarly, patients who received posttransplantation chemotherapy had 100% survival compared with 56% without chemotherapy (P = .08). Other variables had little effect on survival.
Liver transplantation is a successful treatment option for children with unresectable hepatoblastoma with a 90% survival rate for primary transplantation. Rescue LT for recurrent hepatoblastoma after previous resection has a poor survival outcome and should be considered a relative contraindication. Posttransplantation chemotherapy improves survival. A prospective multicenter collaboration to validate these findings with a larger patient population is necessary. Until that time, patients who receive rescue transplants should receive posttransplantation chemotherapy.
根治性切除联合辅助化疗是肝母细胞瘤公认的治疗方法。本研究旨在评估我们对经充分化疗后仍无法切除的肿瘤进行肝移植（LT）的结果。
纳入1990年至2004年间在2家机构接受肝母细胞瘤移植的所有患者。为确定对生存的影响而回顾的变量包括以下内容：既往肿瘤切除情况、诊断时的转移性疾病、显微镜下血管侵犯、诊断时及移植时的甲胎蛋白（AFP）水平、肿瘤组织学以及移植后化疗的应用。移植前化疗的有效性定义为AFP峰值水平下降超过99%。
14例患者接受了移植：9例男孩和5例女孩（年龄范围18个月至13岁；平均年龄57±48个月）。患者在诊断后平均4±1个月接受移植。总体生存率为71%（10/14），平均随访46个月。所有死亡均继发于肿瘤复发。在10例接受初次肝移植的患者中，9例存活，而在4例因初次切除后不可切除的肿瘤复发而接受移植的患者中，仅1例存活（90%对25%；P = 0.02）。AFP峰值下降超过99%也与更好的生存率相关（100%对56%；P = 0.08）。同样，接受移植后化疗的患者生存率为100%，未接受化疗的患者为56%（P = 0.08）。其他变量对生存影响不大。
肝移植是无法切除的肝母细胞瘤患儿的一种成功治疗选择，初次移植的生存率为90%。既往切除后复发的肝母细胞瘤进行挽救性肝移植的生存结果较差，应被视为相对禁忌证。移植后化疗可提高生存率。有必要进行一项前瞻性多中心合作研究，以更大的患者群体验证这些发现。在此之前，接受挽救性移植的患者应接受移植后化疗。
