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Background Preoperative cisplatin alone may be as effective as cisplatin plus doxorubicin in standard-risk hepatoblastoma (a tumor involving three or fewer sectors of the liver that is associated with an alpha-fetoprotein level of >100 ng per milliliter).
Methods Children with standard-risk hepatoblastoma who were younger than 16 years of age were eligible for inclusion in the study. After they received one cycle of cisplatin (80 mg per square meter of body-surface area per 24 hours), we randomly assigned patients to receive cisplatin (every 14 days) or cisplatin plus doxorubicin administered in three preoperative cycles and two postoperative cycles. The primary outcome was the rate of complete resection, and the trial was powered to test the noninferiority of cisplatin alone (<10% difference in the rate of complete resection).
Results Between June 1998 and December 2006, 126 patients were randomly assigned to receive cisplatin and 129 were randomly assigned to receive cisplatin plus doxorubicin. The rate of complete resection was 95% in the cisplatin-alone group and 93% in the cisplatin–doxorubicin group in the intention-to-treat analysis (difference, 1.4%; 95% confidence interval [CI], –4.1 to 7.0); these rates were 99% and 95%, respectively, in the per-protocol analysis. Three-year event-free survival and overall survival were, respectively, 83% (95% CI, 77 to 90) and 95% (95% CI, 91 to 99) in the cisplatin group, and 85% (95% CI, 79 to 92) and 93% (95% CI, 88 to 98) in the cisplatin–doxorubicin group (median follow-up, 46 months). Acute grade 3 or 4 adverse events were more frequent with combination therapy (74.4% vs. 20.6%).
Conclusions As compared with cisplatin plus doxorubicin, cisplatin monotherapy achieved similar rates of complete resection and survival among children with standard-risk hepatoblastoma. Doxorubicin can be safely omitted from the treatment of standard-risk hepatoblastoma. (ClinicalTrials.gov number, NCT00003912
[ClinicalTrials.gov]
.)
Source Information
From the Division of Hematology–Oncology, Department of Pediatrics, University Hospital of Padua, Padua, Italy (G.P.); the International Breast Cancer Study Group Coordinating Center (R.M.), the Department of Surgical Pathology, Institute of Pathology (A.Z.), and the Department of Surgery, University Children's Hospital (J. Plaschkes) — all in Bern, Switzerland; the Children's Cancer and Leukaemia Group Data Centre, Leicester (E.S., M.C.), Great Ormond Street Hospital, London (P.B., D.R.), Birmingham Children's Hospital, Birmingham (B.M.), and the Royal Hospital for Sick Children, Edinburgh (J. Pritchard, G.M.) — all in the United Kingdom; the Department of Pediatrics, Institut Gustave Roussy, Villejuif, France (L.B., V.L.); the Department of Research, Hospital do Cancer, São Paulo (B.C.); the Department of Pediatric Oncology, Emma Children's Hospital, Academic Medical Center, Amsterdam (J.Z.), and Radboud University, Nijmegen Medical Center, Nijmegen (D.A.) — both in the Netherlands; the Department of Pediatrics, Ullevål University Hospital, Oslo (E.W.); Servicio de Hemato–Oncologia, Prof. Dr. Juan P. Garrahan Children's Hospital, Buenos Aires (M.S.); and the Department of Surgery and Urology for Children and Adolescents, Medical University of Gdansk, Gdansk, Poland (P.C.).
Dr. Jon Pritchard is deceased.
Address reprint requests to Dr. Perilongo at the Department of Pediatrics, University Hospital of Padua, Via Giustiniani 3, 35128 Padua, Italy, or at giorgio.perilongo{at}unipd.it.
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