A phase I/II study of subconjunctival carboplatin for intraocular retinoblastoma

Purpose: To evaluate the efficacy and toxicity of subconjunctival carboplatin for intraocular retinoblastoma. Design: A phase I/II clinical trial (noncomparative case series). Participants: Eleven children with bilateral retinoblastoma who had 13 eyes containing active tumor. Methods: Subconjunctival carboplatin (1.4-2.0 ml of a 10-mg/ml solution) was administered. Ophthalmologic examinations with the patient under anesthesia were performed before each injection, and response and toxicity were assessed. Main Outcome Measures: Tumor response and toxicities. Results: A median of three injections per eye was administered (range, 1-7 injections per eye) at a median interval of 21 days between injections (range, 14-35 days). The median dose was 20 mg/injection (range, 14-20 mg). Toxicities included transient periorbital edema in four eyes and optic atrophy in one eye that also received laser photocoagulation and cryotherapy. No nonocular toxicities were noted. Three of five eyes with vitreous disease that could be evaluated had major responses. Two of five eyes with retinal tumors had a response, but neither eye with subretinal disease responded. Conclusions: Subconjunctival carboplatin as used in this protocol is effective for intraocular retinoblastoma. It appears to be safe, but additional study and longer follow-up are required, particularly to determine the risk of optic atrophy.