肝母细胞瘤的治疗结果:单一机构15年的经验- Sheikh Khalifa Medical City, Abu Dhabi, UAE

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摘要

背景:肝母细胞瘤是儿童最常见的肝脏恶性肿瘤。治疗方案各不相同。在我中心,我们采用了儿童肿瘤组(COG) AHEP0731方案,以及一些复发性实体瘤的实验方案。方法:我们旨在调查本中心目前肝母细胞瘤治疗方案的结果。在2008年1月至2023年6月期间纳入了15名患者。回顾性回顾了临床表现、诊断时血清α-胎蛋白(AFP)水平、组织学亚型、治疗和结局。结果:12例(80%)患者在诊断时有症状,腹部肿块是最常见的主诉。9例患者(60%)采用3期托普分期系统。上皮组织病理学亚型为优势亚型。13例患者术前化疗,手术切除;只有1例患者接受了手术切除后化疗。术前化疗为顺铂、氟尿嘧啶、长春新碱、阿霉素(C5VD) 2 ~ 4个周期,手术切除。其中4名患者接受了新辅助实验性化疗,使用了Pazopanib、Pembrolizumab和Sorafenib等药物。随访期间,6例患者死于疾病进展。中位生存时间为42个月(95%可信区间:18-42%)。5年总生存率为44.09%(95%可信区间:18-42%)。结论:手术加化疗是治疗肝母细胞瘤的有效途径。使用新的靶向治疗和复发性实体瘤方案可能延长对标准治疗无反应的患者的生命。需要进一步的研究来验证其在晚期肝母细胞瘤患者中的应用。
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Treatment Outcomes for Hepatoblastoma: 15-Years-Experience of a Single Institution – Sheikh Khalifa Medical City, Abu Dhabi, UAE
Background: Hepatoblastoma is the most common malignant liver tumor in children. Treatment protocols varies. In our center, we have adopted the Children’s Oncology Group (COG) AHEP0731 protocol, in addition to some experimental regimens for relapsed solid tumors. Methods: We aimed to investigate the outcome of the current hepatoblastoma treatment protocol at our center. 15 patients were included between January 2008 and June 2023. A retrospective review was carried to review the clinical presentation, serum α-fetoprotein (AFP) level at diagnosis, histological subtype, treatment, and outcomes. Results: 12 patients (80%) were symptomatic at time of diagnosis, with abdominal mass being the most common presenting complaint. Nine patients (60%) presented in stage 3 PRETEXT staging system. Epithelial histopathological subtype was predominant subtype. Thirteen patients have received preoperative chemotherapy, followed by surgical resection; only one patient underwent upfront surgical resection followed by chemotherapy. Preoperative chemotherapy consists of 2 to 4 cycles of Cisplatin, Fluorouracil, Vincristine and Doxorubicin (C5VD), followed by surgical resection. Four of them underwent neoadjuvent experimental chemotherapy utilizing agents such as Pazopanib, Pembrolizumab and Sorafenib. During followup, six patients died of progressive disease. The median survival time was 42 months (95% confidence interval: 18–42%). Five-year overall survival was 44.09% (95% confidence interval: 18–42%). Conclusions: The combination of surgery and chemotherapy for hepatoblastoma is an effective approach. Utilization of new-targeted therapies and relapsed solid tumors regimens may prolong life in patients who did not respond to standard therapy. Further studies are required to validate its usage on patients with advanced hepatoblastoma.
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