Carboplatin is effective therapy for young children with progressive opticpathway tumors: A Pediatric Oncology Group phase II study

The Pediatric Oncology Group conducted a phase II study to evaluate the act ivity 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 elig ible and had either neuro-imaging or symptomatic evidence of progressive OP Ts. Twenty-one of 50 had evidence of neurofibromatosis type I (NF-1), Thera py consisted of carboplatin 560 mg/m(2) 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 s table disease or better, and 34 completed the 18-month therapy. Our data ar e sufficient to conclude that the proportion of objective responses (comple te, partial, or minor response or stable disease) exceeded 30% (P < 0.00001 ), and the approximate 95% confidence interval estimate of the objective re sponse rate was 0.665 to 0.895. Twenty-one patients went off protocol becau se of progressive disease, Fifteen patients progressed during the 18-month therapy, and 6 patients progressed after completing therapy, Six children d ied with progressive disease. Major toxicities were neutropenia and thrombo cytopenia, and 3 children experienced allergic reactions. Carboplatin is ac tive and safe for the treatment of young children with progressive OPTs, Th e addition of other potentially active drugs may further increase the event -free survival for these children.