Institute of Organ Transplantation, Tongji Hospital, Tongji Medical College, Huazhong University of Science and Technology, Wuhan, China.
Department of Surgery, University of Heidelberg, Heidelberg, Germany.
JAMA Netw Open. 2019 Oct 2;2(10):e1912676. doi: 10.1001/jamanetworkopen.2019.12676.
The incidence of hepatoblastoma is increasing, and liver transplant (LT) provides a potential cure for pediatric patients with unresectable hepatoblastoma; however, the use of LT for hepatoblastoma has not been examined in a modern cohort. Moreover, data are lacking on the association between the type of surgical management received and overall risk of death among pediatric patients with hepatoblastoma.
To examine the receipt of LT among pediatric patients with hepatoblastoma and to assess overall survival of pediatric patients with hepatoblastoma who were treated with chemotherapy after LT or liver resection (LR) using data from a national cancer registry.
DESIGN, SETTING, AND PARTICIPANTS: This cohort study used data for 443 pediatric patients with histologically confirmed hepatoblastoma who received chemotherapy and surgical therapies, as documented in the Surveillance, Epidemiology, and End Results database of the National Cancer Institute, from 2004 to 2016, with follow-up through December 31, 2018. Multivariable logistic regression was used to determine factors associated with the use of LT. Cox proportional hazards models were used to assess factors associated with overall survival. Data analysis was performed from April 18, 2019, to July 25, 2019.
Overall survival.
Among 443 patients receiving chemotherapy (mean [SD] age, 1.8 [2.6] years; 167 [37.7%] female), 350 (79%) underwent LR and 93 (21%) underwent LT. Multivariable analysis showed that patients with multiple lesions were more likely to undergo LT than LR (31% vs 13%; P < .001) and that patients with higher stage tumors were more likely to undergo LT than LR (local disease, 20% vs 58%; regional disease, 58% vs 24%; distant disease, 22% vs 18%; P < .001). There was a statistically significant 19% increase in the receipt of LT from 8% in 1998 to 27% 2016 (trend test, P = .02). Overall survival at 10 years was not significantly different for the 2 surgical management strategies (87.2% [95% CI, 78.3%-97.1%] for patients undergoing LT vs 87.8% [95% CI, 83.5%-92.4%] for those undergoing LR; P = .92). The overall risk of death was not significantly different for LT compared with LR (hazard ratio, 0.716; 95% CI, 0.309-1.657; P = .44).
The use of LT for the management of hepatoblastoma has increased significantly over time. Among pediatric patients with hepatoblastoma receiving chemotherapy, LT was not associated with improved overall survival compared with LR. There was no significant different between treatments with regard to the outcome variable, but this finding cannot be interpreted as indicating equivalence or lack of superiority.
肝细胞瘤的发病率正在增加，肝移植（LT）为无法切除的肝细胞瘤患儿提供了潜在的治愈方法；然而，现代队列研究并未检查 LT 在肝细胞瘤患儿中的应用。此外，缺乏关于接受不同手术治疗类型与接受 LT 或肝切除（LR）后接受化疗的肝细胞瘤患儿总体死亡风险之间关系的数据。
检查肝细胞瘤患儿接受 LT 的情况，并使用国家癌症登记处的数据评估接受 LT 或 LR 后接受化疗的肝细胞瘤患儿的总体生存率。
设计、地点和参与者：这项队列研究使用了国家癌症研究所的监测、流行病学和最终结果数据库中记录的 443 名接受化疗和手术治疗的组织学证实的肝细胞瘤患儿的数据，这些患儿在 2004 年至 2016 年接受了治疗，随访至 2018 年 12 月 31 日。多变量逻辑回归用于确定与 LT 使用相关的因素。使用 Cox 比例风险模型评估与总体生存率相关的因素。数据分析于 2019 年 4 月 18 日至 2019 年 7 月 25 日进行。
总体生存率。
在接受化疗的 443 名患儿中（平均[标准差]年龄，1.8[2.6]岁；167[37.7%]为女性），350 名（79%）接受了 LR，93 名（21%）接受了 LT。多变量分析显示，多发病灶患儿更可能接受 LT 而不是 LR（31%比 13%；P < .001），且肿瘤分期较高的患儿更可能接受 LT 而不是 LR（局部疾病，20%比 58%；区域疾病，58%比 24%；远处疾病，22%比 18%；P < .001）。从 1998 年的 8%到 2016 年的 27%，LT 的接受率呈统计学显著增加（趋势检验，P = .02）。两种手术管理策略的 10 年总生存率无显著差异（接受 LT 的患者为 87.2%[95%CI，78.3%-97.1%]，接受 LR 的患者为 87.8%[95%CI，83.5%-92.4%]；P = .92）。与 LR 相比，LT 治疗的总体死亡风险无显著差异（风险比，0.716；95%CI，0.309-1.657；P = .44）。
LT 用于肝细胞瘤治疗的应用随着时间的推移显著增加。在接受化疗的肝细胞瘤患儿中，与 LR 相比，LT 并不能提高总体生存率。两种治疗方法在结局变量方面没有显著差异，但这一发现不能解释为表明等效或缺乏优势。
