title: Efficacy of irinotecan single drug treatment in children with refractory or recurrent hepatoblastoma--a phase II trial of the childhood liver tumour strategy group (SIOPEL).
abstract: OBJECTIVE
  To assess the clinical activity of irinotecan as single drug in children with refractory or recurrent hepatoblastoma.
  
  
  METHODS
  Four cycles of irinotecan were administered (20mg/m(2)/day intravenous (i.v.) infusion on days 1-5 and 8-12, every 21days) unless tumour progression occurred or resectability was achieved earlier. Tumour response was assessed according to modified SIOPEL and Response Evaluation Criteria In Solid Tumours (RECIST) criteria. Main end-points were best overall response rate (RR), early progression rate (EPR) and progression free survival (PFS).
  
  
  RESULTS
  Twenty-four eligible patients (median age 58.0months; 19 boys) were enrolled in the study (11 relapses, 13 refractory diseases). Of the 23 evaluable patients six had an overall partial response, 11 stable disease and six progressive disease, of which four were early progression (RR: 26%, EPR: 17%). In eight patients the residual tumour could be completely resected; seven patients became tumour free. At last follow-up 12 patients were alive (six with no evidence of disease, six with disease). PFS at 1year was 24%. Patients with relapse had a higher RR than patients with refractory disease (46% versus 8%) and patients with isolated lung lesions showed a better response than patients with other tumour localisations (50% versus 13%). The main grade 3-4 toxicities, diarrhoea and neutropenia, occurred in half of the patients.
  
  
  CONCLUSIONS
  Irinotecan has a significant anti-tumour activity and acceptable toxicity in patients with relapsed hepatoblastoma and therefore should be considered for the treatment of these patients. Exploration of the role of irinotecan in the initial treatment of hepatoblastoma is warranted.
authors: Academic Medical Center, Amsterdam, The Netherlands. j.zsiros@amc.uva.nl
authors: József
authors: J
authors: Zsíros
authors: Laurence
authors: L
authors: Brugières
authors: Penelope
authors: P
authors: Brock
authors: Derek
authors: D
authors: Roebuck
authors: Rudolf
authors: R
authors: Maibach
authors: Margaret
authors: M
authors: Child
authors: Bruce
authors: B
authors: Morland
authors: Michela
authors: M
authors: Casanova
authors: Daniele
authors: D
authors: Pariente
authors: Claudia
authors: C
authors: Paris
authors: Beatriz
authors: B
authors: de Camargo
authors: Milind
authors: M
authors: Ronghe
authors: Arthur
authors: A
authors: Zimmermann
authors: Jack
authors: J
authors: Plaschkes
authors: Piotr
authors: P
authors: Czauderna
authors: Giorgio
authors: G
authors: Perilongo
mesh_terms: D000293:Adolescent; D018906:Antineoplastic Agents, Alkylating / Q000009:adverse effects / Q000627:therapeutic use*; D014408:Biomarkers, Tumor / Q000097:blood; D002166:Camptothecin / Q000009:adverse effects / Q000031:analogs & derivatives* / Q000627:therapeutic use; D002648:Child; D002675:Child, Preschool; D003131:Combined Modality Therapy; D003967:Diarrhea / Q000139:chemically induced; D005260:Female; D006498:Hepatectomy; D018197:Hepatoblastoma / Q000097:blood / Q000188:drug therapy* / Q000556:secondary / Q000601:surgery; D006801:Humans; D007223:Infant; D000077146:Irinotecan; D008113:Liver Neoplasms / Q000097:blood / Q000188:drug therapy* / Q000601:surgery; D016031:Liver Transplantation; D008175:Lung Neoplasms / Q000188:drug therapy / Q000556:secondary*; D008297:Male; D009364:Neoplasm Recurrence, Local / Q000188:drug therapy*; D009503:Neutropenia / Q000139:chemically induced; D011446:Prospective Studies; D016879:Salvage Therapy; D016896:Treatment Outcome; D000509:alpha-Fetoproteins / Q000032:analysis
chemical_list: D018906:Antineoplastic Agents, Alkylating; D014408:Biomarkers, Tumor; D000509:alpha-Fetoproteins; D000077146:Irinotecan; D002166:Camptothecin