Systemic Chemotherapy

Adjuvant setting for patients with high-risk pathology. Different regimens have been used in the management of patients with high-risk pathology in the enucleated specimen. Most regimens include a three-drug combination of vincristine, etoposide, and carboplatin, either alone or alternating with cyclophosphamide and an anthracycline.[5,9-11]; [4][Level of evidence C1 and C2]

Treatment of patients with extraocular and metastatic disease. Patients with extraocular disease benefit from more intensive therapy. While a standard treatment has not been determined, responses to cisplatin-based regimens, with consolidation using high-dose chemotherapy and autologous hematopoietic stem cell rescue for patients with extraorbital disease, have been reported.[12-15]

Chemoreductive treatment in conjunction with aggressive local treatment for patients undergoing ocular salvage treatments. During the past two decades, the standard of care has been systemic chemotherapy to reduce tumor volume (chemoreduction) to facilitate the use of local treatments and to avoid the long-term effects of radiation therapy.[16] The success rate for eye salvage varies from center to center, but overall good ocular outcomes are consistently obtained for discrete tumors without vitreous seeding. In a large cohort analysis of 994 eyes in 554 patients who were treated with intravenous chemotherapy and had long-term outcome data, investigators found that tumor control was strongly dependent on the International Classification of Retinoblastoma group designation per eye. Frontline intravenous chemotherapy consisting of six cycles of vincristine, etoposide, and carboplatin plus additional intra-arterial chemotherapy and/or plaque radiation therapy led to tumor control for Groups A (96%), B (91%), C (91%), D (71%), and E (32%) by year 2. With the aforementioned treatment, enucleation or external-beam radiation therapy could be avoided, and the tumor-controlling effect lasted up to 20 years.[17][Level of evidence C1]Eye grouping, as defined by the International Classification of Retinoblastoma, is the best predictor of ocular salvage using this approach, with salvage rates ranging from 60% to 100%.[16] Prolonged chemotherapy instead of enucleation, in the context of persistent intraocular disease activity, should be used cautiously because this approach has been associated with an increased risk of metastatic disease.[18]; [19][Level of evidence C2]