Hepatocellular carcinoma in children and adolescents: Results from the Pediatric Oncology Group and the Children's Cancer Group intergroup study

Howard M. Katzenstein, Mark D. Krailo, Marcio Malogolowkin, Jorge A. Ortega, Wen Liu-Mares, Edwin C. Douglass, James H. Feusner, Marleta Reynolds, John J. Quinn, Kurt Newman, Milton J. Finegold, Joel E. Haas, Martha G. Sensel, Robert P. Castleberry, Laura C. Bowman

Research output: Contribution to journalArticle

102 Scopus citations

Abstract

Purpose: To determine surgical resectability, eventfree survival (EFS), and toxicity in children with hepatocellular carcinoma (HCC) randomized to treatment with either cisplatin (CDDP), vincristine, and fluorouracil (regimen A) or CDDP and continuous-infusion doxorubicin (regimen B). Patients and Methods: Forty-six patients were enrolled onto Pediatric Intergroup Hepatoma Protocol INT-0098 (Pediatric Oncology Group (POG) 8945/Children's Cancer Group (CCG) 8881). After initial surgery or biopsy, children with stage I (n = 8), stage III (n = 25), and stage IV (n = 13) HCC were randomly assigned to receive regimen A (n = 20) or regimen B (n = 26). Results: For the entire cohort, the 5-year EFS estimate was 19% (SD = 6%). Patients with stage I, III, and IV had 5-year EFS estimates of 88% (SD = 12%), 8% (SD = 5%), and 0%, respectively. Five-year EFS estimates were 20% (SD = 9%) and 19% (SD = 8%) for patients on regimens A and B, respectively (P = .78), with a relative risk of 1.2 (95% confidence interval, 0.60 to 2.3) for regimen B when compared with regimen A. Outcome was similar for either regimen within disease stages. Events occurred before postinduction surgery I in 18 (47%) of 38 patients with stage III or IV disease, and tumor resection was possible in two (10%) of the remaining 20 children with advanced-stage disease after chemotherapy. Conclusion: Children with initially resectable HCC have a good prognosis and may benefit from the use of adjuvant chemotherapy. Outcome was uniformly poor for children with advanced-stage disease treated with either regimen. New therapeutic strategies are needed for the treatment of advanced-stage pediatric HCC.

Original languageEnglish (US)
Pages (from-to)2789-2797
Number of pages9
JournalJournal of Clinical Oncology
Volume20
Issue number12
DOIs
StatePublished - Jun 15 2002
Externally publishedYes

ASJC Scopus subject areas

  • Oncology
  • Cancer Research

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    Katzenstein, H. M., Krailo, M. D., Malogolowkin, M., Ortega, J. A., Liu-Mares, W., Douglass, E. C., Feusner, J. H., Reynolds, M., Quinn, J. J., Newman, K., Finegold, M. J., Haas, J. E., Sensel, M. G., Castleberry, R. P., & Bowman, L. C. (2002). Hepatocellular carcinoma in children and adolescents: Results from the Pediatric Oncology Group and the Children's Cancer Group intergroup study. Journal of Clinical Oncology, 20(12), 2789-2797. https://doi.org/10.1200/JCO.2002.06.155