Division of Pediatric Hematology, Oncology and BMT, Department of Pediatrics, Akdeniz University Faculty of Medicine, Antalya, Turkey.
Department of Hematology-Oncology, Clinic of Pediatrics, Konya City Hospital, Konya, Turkey.
Turk J Gastroenterol. 2022 Dec;33(12):1069-1078. doi: 10.5152/tjg.2022.21827.
Hepatoblastoma is rare cancer that responds well to risk-based chemotherapy, and surgical treatment is needed to achieve complete remission and satisfactory survival rates in hepatoblastoma patients. In this study, we evaluated the clinical features and treatment outcomes of pediatric hepatoblastoma patients treated in our clinic.
Eighteen patients with hepatoblastoma who were treated and followed up in our center between June 1999 and June 2020 were analyzed retrospectively. All patients were evaluated by a multidisciplinary team and managed using a risk-based protocol (SIOPEL-1 and SIOPEL-3).
The patients' mean age at diagnosis was 38.33 ± 52.34 months. Sixteen patients (89%) received neoadjuvant chemotherapy, and 2 patients (11%) who underwent complete mass excision at diagnosis received adjuvant chemotherapy. After neoadjuvant therapy, the tumor was completely resected in 8 patients (45%), while liver transplantation was performed in 6 patients (34%) because complete resection of the tumor was not possible. Two patients died before surgical treatment. One patient relapsed with lung metastasis after salvage chemotherapy. She is alive without disease at 64 months. The mean follow-up time was 59.3 ± 49.8 months; 5-year overall and disease-free survival rates were 88.9% and 80.8%, respectively. The 5-year overall survival rate was 100% for both liver transplant and resected patients, whereas 5-year disease-free survival was lower in transplant patients (75% vs 100%, P < .001).
Multidisciplinary follow-up is especially important for patients who may need liver transplantation. Some patients may benefit from new treatment options such as radiofrequency ablation and cyberknife treatment.
肝母细胞瘤是一种罕见的癌症，对基于风险的化疗反应良好，手术治疗是实现肝母细胞瘤患者完全缓解和满意生存率的必要手段。在本研究中，我们评估了在我们诊所接受治疗和随访的儿童肝母细胞瘤患者的临床特征和治疗结果。
回顾性分析了 1999 年 6 月至 2020 年 6 月期间在我们中心接受治疗和随访的 18 例肝母细胞瘤患者。所有患者均由多学科团队评估，并采用基于风险的方案（SIOPEL-1 和 SIOPEL-3）进行管理。
患者诊断时的平均年龄为 38.33 ± 52.34 个月。16 例患者（89%）接受了新辅助化疗，2 例（11%）诊断时完全切除肿块的患者接受了辅助化疗。新辅助治疗后，8 例患者（45%）肿瘤完全切除，6 例患者（34%）因无法完全切除肿瘤而行肝移植。2 例患者在手术治疗前死亡。1 例患者在挽救性化疗后复发伴肺转移，无病生存 64 个月。平均随访时间为 59.3 ± 49.8 个月；5 年总生存率和无病生存率分别为 88.9%和 80.8%。肝移植和切除患者的 5 年总生存率均为 100%，而移植患者的 5 年无病生存率较低（75%比 100%，P<0.001）。
对于可能需要肝移植的患者，多学科随访尤为重要。一些患者可能受益于新的治疗选择，如射频消融和 Cyberknife 治疗。
