title: Hepatocellular carcinoma in children and adolescents: results from the Pediatric Oncology Group and the Children's Cancer Group intergroup study.
abstract: OBJECTIVE
  To determine surgical resectability, event-free 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).
  
  
  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.
  
  
  CONCLUSIONS
  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.
authors.affiliation: Department of Pediatrics and Surgery, Northwestern University and Children's Memorial Hospital, Chicago, IL, USA. hmk476@northwestern.edu
authors.forename: Howard M
authors.initials: HM
authors.lastname: Katzenstein
authors.forename: Mark D
authors.initials: MD
authors.lastname: Krailo
authors.forename: Marcio H
authors.initials: MH
authors.lastname: Malogolowkin
authors.forename: Jorge A
authors.initials: JA
authors.lastname: Ortega
authors.forename: Wen
authors.initials: W
authors.lastname: Liu-Mares
authors.forename: Edwin C
authors.initials: EC
authors.lastname: Douglass
authors.forename: James H
authors.initials: JH
authors.lastname: Feusner
authors.forename: Marleta
authors.initials: M
authors.lastname: Reynolds
authors.forename: John J
authors.initials: JJ
authors.lastname: Quinn
authors.forename: Kurt
authors.initials: K
authors.lastname: Newman
authors.forename: Milton J
authors.initials: MJ
authors.lastname: Finegold
authors.forename: Joel E
authors.initials: JE
authors.lastname: Haas
authors.forename: Martha G
authors.initials: MG
authors.lastname: Sensel
authors.forename: Robert P
authors.initials: RP
authors.lastname: Castleberry
authors.forename: Laura C
authors.initials: LC
authors.lastname: Bowman
mesh_terms: D000293:Adolescent; D000971:Antineoplastic Combined Chemotherapy Protocols / Q000627:therapeutic use*; D006528:Carcinoma, Hepatocellular / Q000188:drug therapy* / Q000473:pathology / Q000601:surgery*; D017024:Chemotherapy, Adjuvant; D002648:Child; D002675:Child, Preschool; D002945:Cisplatin / Q000008:administration & dosage; D018572:Disease-Free Survival; D004317:Doxorubicin / Q000008:administration & dosage; D005260:Female; D005472:Fluorouracil / Q000008:administration & dosage; D006801:Humans; D007223:Infant; D007231:Infant, Newborn; D007262:Infusions, Intravenous; D008113:Liver Neoplasms / Q000188:drug therapy* / Q000473:pathology / Q000601:surgery*; D008297:Male; D009367:Neoplasm Staging; D011379:Prognosis; D016896:Treatment Outcome; D014750:Vincristine / Q000008:administration & dosage
chemical_list: D014750:Vincristine; D004317:Doxorubicin; D002945:Cisplatin; D005472:Fluorouracil