The effect of topotecan in advanced intraocular retinoblastoma with manageable toxicity.

Abstract

9540 Background: There is a need to identify new effective chemotherapeutic agents for the treatment of intraocular retinoblastoma (RB). Methods: An institutional protocol (RET5) for the study and treatment of patients with intraocular RB stratified patients into three groups according to laterality and stage of disease. The primary objective of RET5 was to estimate the response of window therapy for patients with bilateral RB in whom at least one eye was Reese-Ellsworth IV or V. Window therapy consisted of 2 courses of vincristine and topotecan (VT with G-CSF support). Patients with ≥ partial response received 3 more courses of VT alternating with 6 courses of vincristine and carboplatin. The topotecan dose started at 3 mg/m2/day, and was adjusted to attain a target systemic exposure of 140 ± 20 ng/ml*hr. Carboplatin was administered to achieve an area under the curve of 6.5 mg/ml/min. The dose of vincristine dose was 0.05 mg/kg if age at diagnosis 12 months...