Ophthalmic Artery Chemosurgery for Retinoblastoma Babies Less than 3 Months Old or under 6-kg Weight.
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OBJECTIVE: To determine the feasibility, safety, and efficacy of ophthalmic artery chemosurgery (OAC) for very young children who have unilateral and bilateral retinoblastoma. DESIGN: Retrospective, consecutive review of all children with retinoblastoma (unilateral and bilateral) treated with OAC in the first 3 months of life or, if weighing <6 kg, treated at the Memorial Sloan Kettering Cancer Center. PARTICIPANTS: All children with retinoblastoma (unilateral or bilateral) treated with OAC (17 patients) in the first 3 months of life or weighing <6 kg. METHODS: Ophthalmic artery chemosurgery delivered by microcatheter inserted in the femoral artery and through the internal carotid artery. Combinations of melphalan, carboplatin, and topotecan were delivered in 60 sessions via the ophthalmic artery in 17 patients. MAIN OUTCOME MEASURES: Ocular salvage, serial electroretinograms, patient survival, second cancers, fever/neutropenia, transfusion of any blood product, groin bleeding or femoral artery occlusion, and anesthetic complications. RESULTS: Seventeen retinoblastoma patients under the age of 3 months or weighing <6 kg were successfully canulated and treated a total of 60 times (11 bilateral patients and 6 unilateral patients) with combinations of Melphalan, Carboplatin, and Topotecan. All patients are alive; the mean follow-up 4 years. No patient developed metastatic disease, second cancer, or trilateral retinoblastoma and all eyes were salvaged. There were no cases of chemotherapy induced fever/neutropenia or need for transfusion of any blood product. Electroretinogram was not impaired by treatment, significant improvement in 30Hz function was seen (P = 0.02; mean before 61 μV, after 71 μV, SEM: 5.25 vs. 5.82). CONCLUSIONS: Very young children (<3 months old and <6 kg weight) with unilateral or bilateral retinoblastoma can be safely and effectively treated with OAC (intra-arterial chemotherapy) without significant complications even though the majority had advanced intraocular disease (Reese-Ellsworth Vb or International Classification of Retinoblastoma "D" and "E"). This includes treating both eyes in the same session and as young as 5 weeks and weighing 3.6 kg. This eliminates the need for systemic chemotherapy, which is well known to be toxic to the youngest children. FINANCIAL DISCLOSURE(S): Proprietary or commercial disclosure may be found in the Footnotes and Disclosures at the end of this article.