Carboplatin is effective therapy for young children with progressive optic pathway tumors: a Pediatric Oncology Group phase II study.


Journal Article

The Pediatric Oncology Group conducted a phase II study to evaluate the activity of carboplatin in children 5 years or younger with progressive optic pathway tumors (OPTs). Of the 51 patients accrued to this study, 1 was not eligible because the child was older than 6 years. Fifty patients were eligible and had either neuro-imaging or symptomatic evidence of progressive OPTs. Twenty-one of 50 had evidence of neurofibromatosis type I (NF-1). Therapy consisted of carboplatin 560 mg/m2 at 4-week intervals. Patients with stable disease or better after two courses were continued on therapy for 18 months or until progressive disease. Of the 50 eligible children, 39 had stable disease or better, and 34 completed the 18-month therapy. Our data are sufficient to conclude that the proportion of objective responses (complete, partial, or minor response or stable disease) exceeded 30% (P < 0.00001), and the approximate 95% confidence interval estimate of the objective response rate was 0.665 to 0.895. Twenty-one patients went off protocol because of progressive disease. Fifteen patients progressed during the 18-month therapy, and 6 patients progressed after completing therapy. Six children died with progressive disease. Major toxicities were neutropenia and thrombocytopenia, and 3 children experienced allergic reactions. Carboplatin is active and safe for the treatment of young children with progressive OPTs. The addition of other potentially active drugs may further increase the event-free survival for these children.

Full Text

Duke Authors

Cited Authors

  • Mahoney, DH; Cohen, ME; Friedman, HS; Kepner, JL; Gemer, L; Langston, JW; James, HE; Duffner, PK; Kun, LE

Published Date

  • October 2000

Published In

Volume / Issue

  • 2 / 4

Start / End Page

  • 213 - 220

PubMed ID

  • 11265230

Pubmed Central ID

  • 11265230

International Standard Serial Number (ISSN)

  • 1522-8517

Digital Object Identifier (DOI)

  • 10.1093/neuonc/2.4.213


  • eng

Conference Location

  • England