Multidisciplinary Management of Pediatric Hepatoblastoma: A 20-Year Single-Center Experience

Abstract

Background: 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. Methods: 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). Results: 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). Conclusion: 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.

Development and validation of nomogram-based models for personalized survival assessment in pediatric hepatoblastoma patients

“…Recent advancements in therapy have resulted in notable enhancements in the prognosis of pediatric patients diagnosed with HB. Integrating surgical and chemotherapeutic interventions has yielded noteworthy 5-year overall survival (OS) and disease-free survival rates, attaining 88.9% and 80.8%, respectively ( 5 , 6 ). The achievement of total elimination of liver tumors is of utmost importance in ensuring the efficacy of the treatment ( 7 ).…”

The effect of donor graft type on survival after liver transplantation for hepatoblastoma in children

“…[6][7][8] Conversely, 2%-33% of children with HBL still require LT for complete resection. [9][10][11][12][13] A recent analysis of the National Cancer Database showed that 23% of children with HBL managed at Commission on Cancer US hospitals are not offered any type of surgical treatment. 14 Although total hepatectomy and LT is the only potentially curative option ensuring complete tumor extirpation in many children with unresectable HBL, LT was not considered a feasible treatment modality until the surge of publications showing favorable survival after LT between 1999 and 2002.…”

“…Data suggest that about 10%–30% of children with HBL that undergo non‐transplant liver resection will have disease recurrence 6–8 . Conversely, 2%–33% of children with HBL still require LT for complete resection 9–13 . A recent analysis of the National Cancer Database showed that 23% of children with HBL managed at Commission on Cancer US hospitals are not offered any type of surgical treatment 14 …”

Quality of Life Outcomes for Patients Who Underwent Conventional Resection and Liver Transplantation for Locally Advanced Hepatoblastoma

“…Hepatoblastoma (HB) is the most common primary liver malignancy in children [1][2][3][4], with increasing incidence in the United States [2,3,5]. It is most frequently diagnosed in children younger than 3 years of age [6]. The staging system for HB is based on pretreatment extent of disease or PRETEXT staging.…”