The addition of topotecan to melphalan in the treatment of retinoblastoma with intra-arterial chemotherapy

Purpose: To evaluate the effect of adding topotecan to melphalan for the treatment of retinoblastoma using intra-arterial chemotherapy (IAC). Design: Single-center, consecutive case series. Participants: All eyes treated with IAC at the University of Siena, Siena, Italy, from 2008 to 2019. Methods: Eyes were treated via IAC with either melphalan monotherapy or melphalan plus topotecan. The characteristics and outcomes of these 2 groups were compared. Main Outcome Measures: The main outcome measure was globe salvage rate. Additionally, a complete summary of all adverse events for all eyes was compared between groups and included local, regional, and systemic events causing both transient and permanent effects. Results: A total of 193 patients and 208 eyes were treated with IAC between April 2008 and October 2019. Melphalan alone (MA) was used to treat 44 patients and 50 eyes for a total of 191 procedures. The combination of melphalan plus topotecan (MPT) was used to treat 149 patients and 158 eyes for a total of 780 procedures. Groups were similar in terms of age at presentation. The MPT group included more advanced eyes (P < 0.001) and had shorter follow-up time (mean 47 vs. 120 months in the MA group, P < 0.001). The MPT group required less laser and cryotherapy after treatment (32% of eyes vs. 50% of eyes in the MA group, P < 0.001); there was no other difference in the number of adjuvant treatments required between groups. There was no difference in the number of acute adverse events, both systemic and local, between groups. There was no difference in the number of transient or permanent intraocular side effects between groups. Kaplan–Meier survival analysis estimated a better globe salvage rate in the MPT group (66%) compared with the MA group (58%, P = 0.05). Conclusions: In this case series, the addition of topotecan to melphalan did not alter the IAC side effect profile and may contribute to improved globe salvage. © 2020 American Academy of Ophthalmology