Intra-arterial chemotherapy for retinoblastoma: a structured narrative review

Retinoblastoma (Rb) is the most common primary intraocular malignancy in children. Intra-arterial chemotherapy (IAC), often termed ophthalmic artery chemosurgery, has emerged as an important globe-preserving treatment modality for advanced intraocular Rb. We focused on technical delivery, clinical indications, regimen selection, oncological safety, globe salvage, functional preservation and complications. Current evidence indicates that, in appropriately selected patients and experienced centers, IAC can improve local tumor control and globe salvage while reducing systemic toxicity when compared with intravenous chemotherapy. While melphalan remains the backbone chemotherapeutic agent, escalation to topotecan and/or carboplatin should be guided by tumor burden, vitreous or subretinal seeding, prior treatment response, evidence strength and expected functional potential. Functional preservation should be defined as more than anatomical globe retention; it should include age-appropriate visual acuity or fixation, electroretinography, macular and foveal integrity, avoidance of retinal or choroidal ischemia, amblyopia management, binocular visual development and quality-of-life outcomes. IAC remains associated with ocular and vascular risks, including retinal or choroidal ischemia, cataract formation and ophthalmic artery stenosis or occlusion. Future multicenter prospective studies should use standardized outcome measures, scenario-specific recommendations and molecular monitoring, including aqueous humor cell-free DNA analysis, to refine patient selection and support function-preserving care.