Current and emerging therapy for improving outcomes in patients with intraocular retinoblastoma

Introduction: Survival for patients with intraocular retinoblastoma (RB) is excellent. Over the past two decades, the focus of therapy has shifted to emphasize ocular salvage and vision preservation. The combination of chemoreduction and aggressive focal therapies has formed the new standard approach to most patients with advanced intraocular RB. However, seeding of the vitreous cavity continues to be the primary reason for treatment failure in these patients.Areas covered: Local delivery methods, including periocular injections, intra-ophthalmic artery infusions, and intravitreal injections, have been developed in the hope of controlling disease in advanced cases. This review highlights the current approach to therapy for patients and discusses these new local delivery techniques and the discovery of novel therapeutics that may help to further improve ocular outcomes for patients with intraocular RB.Expert opinion: Due to the rarity of this childhood disease, clinical efforts to improve ocular salvage and vision preservation must occur in parallel with translational research efforts that identify novel strategies for therapy of RB. A dedicated RB team provides guidance for a coordinated approach to address the key therapeutic, biologic, and long-term challenges in our patients.