Successful treatment of childhood high-risk hepatoblastoma with dose-intensive multiagent chemotherapy and surgery: final results of the SIOPEL-3HR study.

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.

• Authors: József Zsíros, Rudolf Maibach, Elizabeth Shafford, Laurence Brugieres, Penelope Brock, Piotr Czauderna, Derek Roebuck, Margaret Childs, Arthur Zimmermann, Veronique Laithier, Jean-Bernard Otte, Beatriz de Camargo, Gordon MacKinlay, Marcelo Scopinaro, Daniel Aronson, Jack Plaschkes, Giorgio Perilongo
• Journal: Journal of clinical oncology : official journal of the American Society of Clinical Oncology (J Clin Oncol) Vol. 28 Issue 15 Pg. 2584-90 (May 20 2010) ISSN: 1527-7755 [Electronic] United States
• PMID: 20406943 (Publication Type: Clinical Trial, Journal Article, Multicenter Study)

Chemical References

• Doxorubicin
• Carboplatin
• Cisplatin

Topics

• Adolescent
• Antineoplastic Combined Chemotherapy Protocols (adverse effects, therapeutic use)
• Carboplatin (administration & dosage)
• Chemotherapy, Adjuvant
• Child
• Child, Preschool
• Cisplatin (administration & dosage)
• Doxorubicin (administration & dosage)
• Female
• Hepatoblastoma (drug therapy, surgery)
• Humans
• Infant
• Liver Neoplasms (drug therapy, surgery)
• Male
• Preoperative Care
• Prospective Studies
• Risk Factors
• Survival Analysis
• Treatment Outcome