Therapeutic Options for Retinoblastoma

Pia R. Mendoza, MD; Hans E. Grossniklaus, MD


Cancer Control. 2016;23(2):99-109. 

In This Article


Management of retinoblastoma is individualized according to patient characteristics and preferences, with the main goals being to save the patient's life and to preserve useful vision, if possible. Therapeutic options for retinoblastoma have rapidly evolved in recent years, with a paradigm shift in standard treatment protocols toward the targeted delivery of chemotherapeutic agents. Increased use of intra-arterial and intravitreal types of chemotherapy with focal consolidation, along with the reduced use of systemic chemotherapy, enucleation, and external beam radiotherapy, has contributed to improved rates of survival and globe salvage while also minimizing unwanted treatment-related adverse events. However, these approaches are not without complications, and a need still exists to weigh the risks and benefits of treatment and to evaluate their long-term effects. New therapies being investigated include novel carriers and routes for local drug delivery as well as molecularly targeted therapies. Our understanding of tumor biology and how to develop safe and effective therapy continues to progress, thus helping to ensure that retinoblastoma will be a curable childhood cancer worldwide in the future.