Tian Yu, Chen Xinghai, Yu Fan, Feng Jiayi, Huang Guimin, Ren Xianghai, Hu Huimin, Zhang Weiling, Li Long
Department of Pediatric Surgery, Capital Institute of Pediatrics-Peking University Teaching Hospital, Beijing, China.
Research Unit of Minimally Invasive Pediatric Surgery on Diagnosis and Treatment, Chinese Academy of Medical Sciences 2021RU015, Beijing, China.
Pediatr Blood Cancer. 2023 Jun 7:e30470. doi: 10.1002/pbc.30470.
We retrospectively investigated the role of neoadjuvant chemotherapy in low-risk patients with hepatoblastoma (HB) who underwent curative resection between February 2009 and December 2017. We also verified the feasibility of the risk stratification system to select the optimal patients for upfront surgery.
We compared 5-year overall survival (OS) and event-free survival (EFS) between the upfront surgery (n = 26) and neoadjuvant chemotherapy (n = 104) groups at three oncology centers in Beijing, China. To reduce the effect of covariate imbalance, propensity score matching (PSM) was used. We explored whether preoperative chemotherapy affected surgical outcomes and identified the risk factors for events and death, including resection margin status, PRETreatment EXTent of disease stages, age, sex, pathology classification, and α-fetoprotein levels.
The median follow-up period was 64 (interquartile range 60-72) months. After PSM, 22 pairs of patients were identified, and the patient characteristics were similar for all variables included in PSM. In the upfront surgery group, the 5-year EFS and OS rates were 81.8% and 86.3%, respectively. In the neoadjuvant chemotherapy group, the 5-year EFS and OS rates were 81.8% and 90.9%, respectively. No significant differences in EFS or OS were observed between the groups. Pathological classification was the only risk factor for death, disease progression, tumor recurrence, other tumors found during HB diagnosis, and death from any cause (p = .007 and .032, respectively).
Upfront surgery achieved long-term disease control in low-risk patients with resectable HB, thus reduced the cumulative toxicity of platinum-based chemotherapy drugs.
我们回顾性研究了2009年2月至2017年12月期间接受根治性切除的低风险肝母细胞瘤（HB）患者中，新辅助化疗的作用。我们还验证了风险分层系统选择最佳患者进行直接手术的可行性。
我们比较了中国北京三个肿瘤中心直接手术组（n = 26）和新辅助化疗组（n = 104）的5年总生存率（OS）和无事件生存率（EFS）。为减少协变量不平衡的影响，采用了倾向评分匹配（PSM）。我们探讨了术前化疗是否影响手术结果，并确定了事件和死亡的风险因素，包括切缘状态、治疗前疾病范围分期、年龄、性别、病理分类和甲胎蛋白水平。
中位随访期为64（四分位间距60 - 72）个月。PSM后，确定了22对患者，PSM纳入的所有变量的患者特征相似。在直接手术组中，5年EFS率和OS率分别为81.8%和86.3%。在新辅助化疗组中，5年EFS率和OS率分别为81.8%和90.9%。两组之间在EFS或OS方面未观察到显著差异。病理分类是死亡、疾病进展、肿瘤复发、HB诊断期间发现的其他肿瘤以及任何原因导致死亡的唯一风险因素（分别为p = 0.007和0.032）。
直接手术在可切除的低风险HB患者中实现了长期疾病控制，从而降低了铂类化疗药物的累积毒性。
