Evaluating primary intra-arterial chemotherapy versus intravenous plus intra-arterial chemotherapy for advanced intraocular retinoblastoma

Purpose Although intra-arterial chemotherapy (IAC) is commonly used for treating intraocular retinoblastoma, it is not a systemic therapy. We aimed to investigate whether the addition of intravenous chemotherapy (IVC) before IAC administration had any effects (whether beneficial or adverse) on patient outcomes. Methods This multicenter retrospective cohort study included 213 patients with advanced intraocular retinoblastoma who received IVC plus IAC ( n = 103) or IAC alone ( n = 110) between April 2009 and January 2017. Eyes were grouped according to the International Intraocular Retinoblastoma Classification. Kaplan–Meier and Cox regression analyses were performed to compare survival outcomes between the two groups. Moreover, details regarding enucleation were recorded. Results The 3-year ocular survival rates were 62% in the IVC plus IAC group and 68% in the IAC group (hazard ratio (HR) 0.88, 95% confidence interval (CI) 0.55–1.43, P = 0.61). Moreover, the corresponding 3-year overall survival rates were 97% and 93%, respectively (HR 1.56, 95% CI 0.41–5.90, P = 0.51), while the 3-year event-free survival rates were 76% and 72%, respectively (HR 0.96, 95% CI 0.56–1.65, P = 0.89). Conclusions Within a 3-year follow-up period, IVC plus IAC produced no additional benefit over primary IAC for treating advanced intraocular retinoblastoma in terms of local tumor control and extending survival. Longer follow-up periods are required to assess long-term efficacy.