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

92 Citations (Scopus)

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

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Hepatocellular Carcinoma
Pediatrics
Neoplasms
Survival
Vincristine
Adjuvant Chemotherapy
Fluorouracil
Doxorubicin
Cisplatin
Therapeutics
Confidence Intervals
Biopsy
Drug Therapy
Regimen B

ASJC Scopus subject areas

  • Oncology
  • Cancer Research

Cite this

Hepatocellular carcinoma in children and adolescents : Results from the Pediatric Oncology Group and the Children's Cancer Group intergroup study. / Katzenstein, Howard M.; Krailo, Mark D.; Malogolowkin, Marcio; Ortega, Jorge A.; Liu-Mares, Wen; Douglass, Edwin C.; Feusner, James H.; Reynolds, Marleta; Quinn, John J.; Newman, Kurt; Finegold, Milton J.; Haas, Joel E.; Sensel, Martha G.; Castleberry, Robert P.; Bowman, Laura C.

In: Journal of Clinical Oncology, Vol. 20, No. 12, 15.06.2002, p. 2789-2797.

Research output: Contribution to journalArticle

Katzenstein, HM, Krailo, MD, Malogolowkin, M, Ortega, JA, Liu-Mares, W, Douglass, EC, Feusner, JH, Reynolds, M, Quinn, JJ, Newman, K, Finegold, MJ, Haas, JE, Sensel, MG, Castleberry, RP & Bowman, LC 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, vol. 20, no. 12, pp. 2789-2797. https://doi.org/10.1200/JCO.2002.06.155
Katzenstein, Howard M. ; Krailo, Mark D. ; Malogolowkin, Marcio ; Ortega, Jorge A. ; Liu-Mares, Wen ; Douglass, Edwin C. ; Feusner, James H. ; Reynolds, Marleta ; Quinn, John J. ; Newman, Kurt ; Finegold, Milton J. ; Haas, Joel E. ; Sensel, Martha G. ; Castleberry, Robert P. ; Bowman, Laura C. / Hepatocellular carcinoma in children and adolescents : Results from the Pediatric Oncology Group and the Children's Cancer Group intergroup study. In: Journal of Clinical Oncology. 2002 ; Vol. 20, No. 12. pp. 2789-2797.
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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.",
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T1 - Hepatocellular carcinoma in children and adolescents

T2 - Results from the Pediatric Oncology Group and the Children's Cancer Group intergroup study

AU - Katzenstein, Howard M.

AU - Krailo, Mark D.

AU - Malogolowkin, Marcio

AU - Ortega, Jorge A.

AU - Liu-Mares, Wen

AU - Douglass, Edwin C.

AU - Feusner, James H.

AU - Reynolds, Marleta

AU - Quinn, John J.

AU - Newman, Kurt

AU - Finegold, Milton J.

AU - Haas, Joel E.

AU - Sensel, Martha G.

AU - Castleberry, Robert P.

AU - Bowman, Laura C.

PY - 2002/6/15

Y1 - 2002/6/15

N2 - 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.

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