Objective: To avoid enucleation cannulating repeatedly the ophthalmic artery of young children with advanced retinoblastoma and giving melphalan that is tumoricidal for retinoblastoma when given intraarterially.

Materials: 8 children with advanced retinoblastoma (Reese-Ellsworth stage V) eyes who were indicated for enucleation were entered in phase two of one center open study-approved protocol of ophthalmic artery infusion of melphalan to avoid enucleation.

Methods: cannulation of the ophthalmic artery was performed by a femoral artery approach using microcatheters while the children were under anesthesia and anticoagulated. Chemotherapy (melphalan) was infused into the artery over a 30-minute period.

Main outcome measures: ophthalmic examinations, retinal photography, and electroretinograms were used to document local toxicity, whereas physical examinations and complete blood counts were used to measure systemic toxicity.

Results: the ophthalmic arteries were successfully cannulated in 6 cases (total, 11 times). Dramatic regression of relapses was obtained into 4 cases, and of vitreous and subretinal seeds was observed in two cases. No severe systemic side effects (sepsis, anemia, neutropenia, fever, or death) occurred. No transfusions were required (red cells or platelets). Three patients developed lid rush that resolved without treatment in two days. There was no toxicity to the cornea, anterior segment, pupil, or motility.

Conclusions: we are experimenting a technique of direct ophthalmic artery infusion of melphalan for children with retinoblastoma. The technique has minimal systemic side effects and local toxicity. Therapeutic results in these 6 cases treated with this technique are reported and discussed.