HEPATOBLASTOMA TREATMENT

J Clin Oncol. 2010 Apr 20. [Epub ahead of print]
Successful Treatment of Childhood High-Risk Hepatoblastoma With Dose-Intensive Multiagent Chemotherapy and Surgery: Final Results of the SIOPEL-3HR Study.
Zsíros J, Maibach R, Shafford E, Brugieres L, Brock P, Czauderna P, Roebuck D, Childs M, Zimmermann A, Laithier V, Otte JB, de Camargo B, Mackinlay G, Scopinaro M, Aronson D, Plaschkes J, Perilongo G.

Departments of Pediatric Oncology and of Pediatric Surgery, Emma Children's Hospital/Academic Medical Centre, Amsterdam, the Netherlands; International Breast Cancer Study Group, Coordinating Center; and Department of Surgical Pathology, Institute of Pathology; and Department of Surgery, University Children's Hospital, Bern, Switzerland; Children's Cancer and Leukaemia Group (CCLG), Data Centre, Leicester; and Departments of Haematology and Oncology and of Radiology, Great Ormond St Hospital for Children, London; and Department of Paediatric Surgery, Royal Hospital for Sick Children, Edinburgh, United Kingdom; Department of Pediatrics, Institut Gustave Roussy, Villejuif Cédex; and Department of Pediatric Hemato-Oncology, Centre Hospitalier Universitaire, Besançon, France; Department of Surgery and Urology for Children and Adolescents, Medical University of Gdansk, Gdansk, Poland, Department of Pediatric Surgery, Université Catholique de Louvain, Saint-Luc University Clinics, Brussels, Belgium; Department of Research, Hospital do Cancer, Sao Paolo, Brazil; Department of Hemato-Oncology, Hospital Prof Dr J.P. Garrahan, Buenos Aires, Argentina; and Division of Hematology-Oncology, Department of Pediatrics, University Hospital of Padua, Padua, Italy.
Abstract

PURPOSE: The primary objective was to determine the efficacy of a newly designed preoperative chemotherapy regimen in an attempt to improve the cure rate of children with high-risk hepatoblastoma. PATIENTS AND METHODS: High risk was defined as follows: tumor in all liver sections (ie, Pretreatment Extension IV [PRETEXT-IV]), or vascular invasion (portal vein [P+], three hepatic veins [V+]), or intra-abdominal extrahepatic extension (E+), or metastatic disease, or alpha-fetoprotein less than 100 ng/mL at diagnosis. Patients were treated with alternating cycles of cisplatin and carboplatin plus doxorubicin (preoperatively, n = 7; postoperatively, n = 3) and delayed tumor resection. RESULTS: Of the 151 patients (150 evaluable for response) 118 (78.7%) achieved a partial response to chemotherapy. Complete resection of the liver tumor could be achieved in 115 patients (76.2%) either by partial hepatectomy (55.6%) or by liver transplantation (20.6%). In 106 children (70.2%), complete resection of all tumor lesions (including metastases) was achieved. Among the patients with initial lung metastases, 52.2% achieved complete remission of the lung lesions with chemotherapy alone. In half of the patients with initial PRETEXT-IV tumor as the only high-risk feature, the tumor could be completely resected with partial hepatectomy. Event-free (EFS) and overall survival (OS) estimates at 3 years were 65% (95% CI, 57% to 73%) and 69% (95% CI, 62% to 77%) for the whole group. EFS and OS for all patients with PRETEXT-IV tumor were 68% and 69%, respectively, and they were 56% and 62%, respectively, for patients with metastasis. CONCLUSION: The applied treatment rendered a great proportion of tumors resectable, and, in comparison with previously published results, led to an improved survival in patients with high-risk hepatoblastoma.