Department of Pediatrics, Seoul National University College of Medicine.
Seoul National University Cancer Research Institute, Seoul, Korea.
J Pediatr Hematol Oncol. 2024 Jul 1;46(5):e265-e271. doi: 10.1097/MPH.0000000000002888. Epub 2024 May 31.
The standard-risk hepatoblastoma has a good prognosis in children; however, refractory or relapsed (R/R) hepatoblastoma has a poor prognosis and high mortality rate. This study aimed to demonstrate the efficacy of high-dose chemotherapy and autologous hematopoietic stem cell transplantation (HSCT) rescue in pediatric patients with R/R hepatoblastoma.
We retrospectively analyzed 6 pediatric patients with R/R hepatoblastoma who underwent autologous HSCT. The MEC conditioning regimen was used for all patients, comprising melphalan 140 mg/m 2 /day intravenously (IV) on day 7 and 70 mg/m 2 on day 6, etoposide 200 mg/m 2 IV on days 5 to 8, and carboplatin 400 mg/m 2 IV on days 5 to 8. One patient received a TopoThioCarbo regimen, comprising topotecan 2 mg/m 2 /day IV on days 4 to 8, thiotepa 300 mg/m 2 /day IV on days 6 to 8, and carboplatin 500 mg/m 2 /day IV on days 3 to 5, as the conditioning regimen for the first transplantation. This was followed by salvage chemotherapy for relapse, and the second transplantation was performed using MEC as the conditioning regimen.
We report the retrospective results of 6 patients with a median age of 1.8 (range 0.4 to 10.2) years who had R/R hepatoblastoma and underwent autologous HSCT. The median follow-up period was 58 (range 28 to 113) months after diagnosis. The median stage at diagnosis was 2.0 (range 2 to 4). Two patients had lung metastases during diagnosis. The median initial alpha-fetoprotein level was 292,888 (range 28,831 to 2,406,942) ng/mL, and the median number of chemotherapy lines before autologous HSCT was 3.5 (range 2 to 7). The disease status before HSCT was complete remission (CR) for all patients. The engraftment rate was 100%. No treatment-related mortality was reported. The 3-year event-free survival and overall survival rates were 83.3% and 100%, respectively. One patient relapsed after the second HSCT and achieved CR after salvage chemotherapy.
This study suggests autologous HSCT as an effective treatment in pediatric patients with R/R hepatoblastoma. Nevertheless, future large-scale prospective studies are warranted.
标准风险肝母细胞瘤患儿预后良好；然而，难治性或复发性（R/R）肝母细胞瘤预后较差，死亡率较高。本研究旨在证明高剂量化疗和自体造血干细胞移植（HSCT）解救在儿科 R/R 肝母细胞瘤患者中的疗效。
我们回顾性分析了 6 例接受自体 HSCT 的 R/R 肝母细胞瘤患儿。所有患者均采用 MEC 预处理方案，包括第 7 天静脉注射（IV）140mg/m2美法仑，第 6 天 70mg/m2美法仑，第 5 至 8 天 IV 依托泊苷 200mg/m2，第 5 至 8 天 IV 卡铂 400mg/m2。1 例患者接受 TopoThioCarbo 方案，包括第 4 至 8 天 IV 拓扑替康 2mg/m2/天，第 6 至 8 天 IV 噻替派 300mg/m2/天，第 3 至 5 天 IV 卡铂 500mg/m2/天，作为第一次移植的预处理方案。继之以复发后的挽救性化疗，第二次移植采用 MEC 作为预处理方案。
我们报告了 6 例中位年龄为 1.8 岁（范围 0.4 至 10.2 岁）的 R/R 肝母细胞瘤患儿接受自体 HSCT 的回顾性结果。诊断后中位随访时间为 58 个月（范围 28 至 113 个月）。诊断时的中位分期为 2.0 （范围 2 至 4）。2 例患者在诊断时发生肺部转移。中位初始甲胎蛋白水平为 292888ng/mL（范围 28831 至 2406942ng/mL），自体 HSCT 前接受的化疗线数中位数为 3.5 （范围 2 至 7）。HSCT 前所有患者均处于完全缓解（CR）状态。植入率为 100%。无治疗相关死亡。3 年无事件生存率和总生存率分别为 83.3%和 100%。1 例患者在第二次 HSCT 后复发，经挽救性化疗后达到 CR。
本研究表明自体 HSCT 是儿科 R/R 肝母细胞瘤患者的一种有效治疗方法。然而，仍需要未来进行大规模的前瞻性研究。
