Carboplatin therapy for optic pathway tumors in children with neurofibromatosis type-1

Symptomatic optic pathway tumors (OPT) occur in 7% of children with neurofi bromatosis type-1 (NF-1). Although turner progression following diagnosis i s unusual in such children, specific therapy may be necessary for patients with either severe or progressive disease. We reviewed the records of 9 chi ldren (6 girls, 3 boys) with NF-1 associated OPT who were treated with the second generation platinum compound carboplatin. Carboplatin was given at a dose of 560 mg/mm(2) every 4 weeks for a mean of 15 cycles. The mean age a t presentation of the OPT was 3.4 years. Eight children had abnormal ophtha lmologic examinations at the time of diagnosis. Only 4 patients had documen ted evidence of progressive disease prior to the institution of therapy. No patient had evidence of progressive disease following therapy. Four patien ts had radiologic evidence of tumor shrinkage and 2 patients had definite i mprovement in vision. There was only minimal toxicity. In conclusion, carbo platin is a safe and effective treatment for OPT in children with NF-1. How ever, as disease stabilization of NF-1 associated OPT often occurs followin g clinical presentation. the clinician should document tumor progression or visual deterioration prior to the institution of therapy.