Baylor College of Medicine, Texas Children's Hospital, 6701 Fannin, Houston, TX 77030, United States.
Primary Children's Hospital, University of Utah, 100N. Mario Capecchi Dr., Salt Lake City, UT 84113, United States.
J Pediatr Surg. 2022 Oct;57(10):251-256. doi: 10.1016/j.jpedsurg.2022.05.022. Epub 2022 Jun 9.
Hepatoblastoma (HB) requires surgical resection for cure, but only 20-30% of patients have resectable disease at diagnosis. Patients who undergo partial hepatectomy at diagnosis have historically received 4-6 cycles of adjuvant chemotherapy; however, those with 100% well-differentiated fetal histology (WDF) have been observed to have excellent outcomes when treated with surgery alone.
Patients on the Children's Oncology Group non randomized, multicenter phase III study, AHEP0731, were stratified based on Evan's stage, tumor histology, and serum alpha-fetoprotein level at diagnosis. Patients were eligible for the very low risk stratum of surgery and observation if they had a complete resection at diagnosis and rapid central histologic review demonstrated HB with 100% WDF histology.
A total of 8 eligible patients were enrolled on study between September 14, 2009 and May 28, 2014. Outcome current to 06/30/2020 was used in this analysis. The median age at enrollment was 22.5 months (range: 8-84 months) and the median AFP at enrollment was 714 ng/ml (range: 18-77,747 ng/mL). With a median follow-up of 6.6 years (range: 3.6-9.8 years), the 5-year event-free (EFS) and overall survival (OS) were both 100%.
This report supports that HB with 100% WDF histology completely resected at diagnosis is curable with surgery only. The development of evidence-based surgical guidelines utilizing criteria based on PRETEXT group, vascular involvement (annotation factors), tumor-specific histology and corresponding biology will be crucial for optimizing which patients are candidates for resection at diagnosis followed by observation.
Prognosis study, Level I evidence.
肝母细胞瘤（HB）需要手术切除才能治愈，但只有 20-30%的患者在诊断时具有可切除的疾病。在诊断时接受部分肝切除术的患者历史上接受了 4-6 个周期的辅助化疗；然而，当仅用手术治疗时，具有 100%良好分化的胎儿组织学（WDF）的患者观察到具有极好的结果。
在儿童肿瘤学组非随机、多中心 III 期研究 AHEP0731 中，根据 Evan 分期、肿瘤组织学和诊断时的血清甲胎蛋白（AFP）水平对患者进行分层。如果患者在诊断时进行了完全切除，并且快速中心组织学检查显示 HB 具有 100% WDF 组织学，则有资格在手术和观察中进入非常低风险分层。
共有 8 名符合条件的患者于 2009 年 9 月 14 日至 2014 年 5 月 28 日入组研究。本分析采用截至 2020 年 6 月 30 日的结果。入组时的中位年龄为 22.5 个月（范围：8-84 个月），入组时 AFP 的中位值为 714ng/ml（范围：18-77747ng/ml）。中位随访 6.6 年（范围：3.6-9.8 年），5 年无事件生存（EFS）和总生存（OS）均为 100%。
本报告支持诊断时完全切除的具有 100% WDF 组织学的 HB 仅通过手术即可治愈。利用 PRETEXT 组、血管受累（注释因素）、肿瘤特异性组织学和相应生物学的标准制定基于证据的手术指南对于优化哪些患者适合在诊断时进行切除，然后进行观察至关重要。
预后研究，I 级证据。
