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

96 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.",
author = "Katzenstein, {Howard M.} and Krailo, {Mark D.} and Marcio Malogolowkin and Ortega, {Jorge A.} and Wen Liu-Mares and Douglass, {Edwin C.} and Feusner, {James H.} and Marleta Reynolds and Quinn, {John J.} and Kurt Newman and Finegold, {Milton J.} and Haas, {Joel E.} and Sensel, {Martha G.} and Castleberry, {Robert P.} and Bowman, {Laura C.}",
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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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