Ioannis A Ziogas1, Daniel J Benedetti2, W Kelly Wu3, Lea K Matsuoka4, Manhal Izzy5, Muhammad A Rauf4, Anita K Pai6, Christina E Bailey7, Sophoclis P Alexopoulos8. 1. Department of Surgery, Division of Hepatobiliary Surgery and Liver Transplantation, Vanderbilt University Medical Center, Nashville, TN. Electronic address: https://twitter.com/IA_Ziogas. 2. Department of Pediatrics, Division of Hematology-Oncology, Vanderbilt University Medical Center, Nashville, TN. 3. Department of Surgery, Division of Hepatobiliary Surgery and Liver Transplantation, Vanderbilt University Medical Center, Nashville, TN. Electronic address: https://twitter.com/WKellyWuMD. 4. Department of Surgery, Division of Hepatobiliary Surgery and Liver Transplantation, Vanderbilt University Medical Center, Nashville, TN. 5. Department of Medicine, Division of Gastroenterology, Hepatology and Nutrition, Vanderbilt University Medical Center, Nashville, TN. Electronic address: https://twitter.com/manhalizzy. 6. Department of Pediatrics, D. Brent Polk Division of Gastroenterology, Hepatology, and Nutrition, Vanderbilt University Medical Center, Nashville, TN. 7. Department of Surgery, Division of Surgical Oncology, Vanderbilt University Medical Center, Nashville, TN. Electronic address: https://twitter.com/TheRealDrBailey. 8. Department of Surgery, Division of Hepatobiliary Surgery and Liver Transplantation, Vanderbilt University Medical Center, Nashville, TN. Electronic address: sopho.alexopoulos@vumc.org.
Abstract
BACKGROUND: Hepatoblastoma is the most common type of liver cancer in children. Refined therapeutic approaches combining risk-adapted chemotherapy along with complete tumor resection has led to improved survival. We aimed to evaluate the current state of management and outcomes for hepatoblastoma in the United States. METHODS: We retrospectively reviewed 794 children (<18 years) with hepatoblastoma from the National Cancer Database (2004-2015). We assessed overall survival by means of Kaplan-Meier method, log-rank tests, and multivariable Cox regression. RESULTS: Median age was 1 year (interquartile range: 0-2) and 170 (21.4%) presented with metastatic disease. Surgical resection was included in the treatment of 614 (77.3%) children (resection in 66.8% and liver transplantation in 10.6%). In the entire cohort, 95.1% of children received chemotherapy. In the surgical cohort, 575 (93.6%) received chemotherapy (34.5% neoadjuvant, 28.7% adjuvant, 30.5% both neoadjuvant and adjuvant). The 5-year overall survival was 76.6% for the entire cohort (no-surgery group: 55.3% vs surgery group: 82.8%). In multivariable analysis for all children, age ≥8 years (P = .009), metastasis (P < .001), surgery only (P = .009), and chemotherapy only (P < .001) were risk factors for mortality. In multivariable analysis for the surgical cohort, metastasis (P = .001), multifocality (P = .02), no chemotherapy (P = .03), and margin-positive resection (P = .02) were risk factors for mortality. CONCLUSION: Excellent long-term overall survival is achievable with a combination of chemotherapy and surgical resection when a negative resection margin is achieved. However, nearly a quarter of children never received surgical treatment, representing a potential opportunity for improvement in care.
BACKGROUND:Hepatoblastoma is the most common type of liver cancer in children. Refined therapeutic approaches combining risk-adapted chemotherapy along with complete tumor resection has led to improved survival. We aimed to evaluate the current state of management and outcomes for hepatoblastoma in the United States. METHODS: We retrospectively reviewed 794 children (<18 years) with hepatoblastoma from the National Cancer Database (2004-2015). We assessed overall survival by means of Kaplan-Meier method, log-rank tests, and multivariable Cox regression. RESULTS: Median age was 1 year (interquartile range: 0-2) and 170 (21.4%) presented with metastatic disease. Surgical resection was included in the treatment of 614 (77.3%) children (resection in 66.8% and liver transplantation in 10.6%). In the entire cohort, 95.1% of children received chemotherapy. In the surgical cohort, 575 (93.6%) received chemotherapy (34.5% neoadjuvant, 28.7% adjuvant, 30.5% both neoadjuvant and adjuvant). The 5-year overall survival was 76.6% for the entire cohort (no-surgery group: 55.3% vs surgery group: 82.8%). In multivariable analysis for all children, age ≥8 years (P = .009), metastasis (P < .001), surgery only (P = .009), and chemotherapy only (P < .001) were risk factors for mortality. In multivariable analysis for the surgical cohort, metastasis (P = .001), multifocality (P = .02), no chemotherapy (P = .03), and margin-positive resection (P = .02) were risk factors for mortality. CONCLUSION: Excellent long-term overall survival is achievable with a combination of chemotherapy and surgical resection when a negative resection margin is achieved. However, nearly a quarter of children never received surgical treatment, representing a potential opportunity for improvement in care.