title: Phase II study of a protracted irinotecan schedule in children with refractory or recurrent soft tissue sarcoma.
abstract: BACKGROUND
  Irinotecan (CPT-11) is a novel antineoplastic agent that takes effect by inhibiting topoisomerase I. The Italian Soft Tissue Sarcoma (STS) Committee performed a multiinstitutional Phase II study to evaluate its effect on STS.
  
  
  METHODS
  Over a 2-year period between 2002 and 2004, 32 heavily pretreated patients were administered 60-minute infusions of irinotecan at 20 mg/m2/day, for 5 days a week, for 2 consecutive weeks. The courses were repeated every 4 weeks for at least 2 courses, unless there were signs of toxicity or disease progression. Thirty patients, 13 with peripheral primitive neuroectodermal tumor (PNET), 12 with rhabdomyosarcoma (RMS), 3 with desmoplastic small round cell tumor (DSRCT), and 2 with other STS were evaluable for response.
  
  
  RESULTS
  A total of 79 cycles were delivered. The main regimen-related toxicity was diarrhea, occurring in 58% of cycles with 9 episodes graded as 3 or 4. Grade 3-4 neutropenia was recorded in 10% of cycles. The overall response rate was 23% (2 complete remissions +5 partial remissions of 30 patients), 38% for PNET and 16% for RMS. In addition, 4 minor responses were noted.
  
  
  CONCLUSIONS
  As a single agent in the treatment of recurrent and refractory STS, irinotecan administered on a daily x5 x2 schedule revealed a noteworthy response rate in a population of heavily pretreated patients, especially in the subset of patients with PNET. Its hematologic toxicity profile warrants further investigation in association with other myelotoxic agents.
authors.affiliation: Division of Hematology/Oncology, Department of Pediatrics, University-Hospital of Padova, Italy. gianni.bisogno@unipd.it
authors.forename: Gianni
authors.initials: G
authors.lastname: Bisogno
authors.forename: Riccardo
authors.initials: R
authors.lastname: Riccardi
authors.forename: Antonio
authors.initials: A
authors.lastname: Ruggiero
authors.forename: Giampaolo
authors.initials: G
authors.lastname: Arcamone
authors.forename: Arcangelo
authors.initials: A
authors.lastname: Prete
authors.forename: Gianmarco
authors.initials: G
authors.lastname: Surico
authors.forename: Massimo
authors.initials: M
authors.lastname: Provenzi
authors.forename: Patrizia
authors.initials: P
authors.lastname: Bertolini
authors.forename: Paolo
authors.initials: P
authors.lastname: Paolucci
authors.forename: Modesto
authors.initials: M
authors.lastname: Carli
mesh_terms: D000293:Adolescent; D000972:Antineoplastic Agents, Phytogenic / Q000008:administration & dosage* / Q000009:adverse effects; D002166:Camptothecin / Q000008:administration & dosage / Q000009:adverse effects / Q000031:analogs & derivatives*; D002648:Child; D002675:Child, Preschool; D018450:Disease Progression; D004334:Drug Administration Schedule; D005260:Female; D006801:Humans; D007223:Infant; D007262:Infusions, Intravenous; D000077146:Irinotecan; D008297:Male; D009503:Neutropenia / Q000139:chemically induced; D012509:Sarcoma / Q000188:drug therapy* / Q000473:pathology; D012983:Soft Tissue Neoplasms / Q000188:drug therapy* / Q000473:pathology; D016896:Treatment Outcome
chemical_list: D000972:Antineoplastic Agents, Phytogenic; D000077146:Irinotecan; D002166:Camptothecin