Pediatric retinoblastoma and intra-arterial chemotherapy: promise, results, and limits of globe preservation

Abstract

Introduction: Retinoblastoma is the most common malignant intraocular tumor of childhood. Timely diagnosis is critical for survival, ocular preservation, and the likelihood of maintaining functional vision. In this context, intra-arterial chemotherapy (IAC) has emerged as a globe-sparing approach for advanced intraocular disease in specialized centers. Objective: To critically evaluate the evidence on IAC in pediatric retinoblastoma, with emphasis on its clinical rationale, efficacy, safety, and interpretive limitations regarding survival, globe salvage, and functional vision. Methods: A narrative review of biomedical literature and consensus or practice documents was conducted. Sources included pediatric studies, institutional series, systematic reviews, meta-analyses, and editorially traceable guidelines. Results: IAC has demonstrated effectiveness in achieving anatomic globe salvage in advanced intraocular disease. However, most reports focus on ocular survival rather than consistent preservation of functional vision or definitive oncologic cure. Global and regional data further indicate that child survival and globe salvage are strongly influenced by national income level, stage at diagnosis, and continuity of treatment. Conclusions: IAC represents a key component of conservative management for advanced intraocular retinoblastoma within specialized programs. Interpretation of outcomes requires careful distinction between oncologic, anatomic, and functional endpoints, and caution against extrapolating institutional series to population-level results.