Effect of intra-arterial chemotherapy drug regimens on globe salvage outcomes of retinoblastoma patients

Abstract

Aims To compare the effect of three-drug regimen (melphalan, topotecan and carboplatin) with two-drug regimen (melphalan and topotecan) on the globe salvage outcomes of retinoblastoma (Rb) with intra-arterial chemotherapy (IAC). Methods This study included 83 patients with 87 eyes received two-drug regimen IAC, and 95 patients with 97 eyes received three-drug regimen IAC. Propensity score matching was applied, and 84 matched (42:42) patients (eyes) were analysed. All Rb patients underwent an ophthalmologic examination before IAC using a wide-angle digital fundus camera, which was repeated monthly after the globe salvage management. Progression-free ocular survival (PFOS), relapse rate and local complications were analysed per affected eye. Analysis of overall survival (OS) and systemic complications was based on Rb individuals. Results Eyes treated with three-drug regimen IAC presented with higher overall PFOS than those treated with two-drug regimen IAC (p=0.026). Stratified analysis showed PFOS rate in three-drug group was higher than two-drug group among group D eyes (88.5% vs 60.6%, p=0.009). Consistently, among group E eyes, three-drug group correlated with a better PFOS, compared with two-drug group (42.9% vs 23.5%, p=0.002). There did not present with significant differences in OS (p=0.853), relapse rate (p=0.291), incidence of moderate-severe myelosuppression (grade 3-4, p=0.564) or ophthalmic artery obstruction (p=0.998) between these two groups. Conclusions Three-drug regimen was a superior IAC management compared with two-drug regiment, with improved progression-free ocular outcome for eyes of advanced retinoblastomas.