To compare the efficacy and safety of intra-arterial chemotherapy (IAC) and intravenous plus intra-arterial chemotherapy (IV-IAC) for patients with late-stage retinoblastoma.

A total of 80 patients diagnosed with retinoblastoma between September 2013 and February 2016 without any treatment history were included. Forty-two patients treated with primary intra-arterial chemotherapy were defined as IAC group. Three-eight patients treated with primary twice intravenous plus intra-arterial chemotherapy were defined as IV-IAC group. Eye salvage rate, enucleation-free survival and mid-to-long term complications were assessed.

(1) Clinical results: A total of 46 eyes in group IAC were included. During the medium follow-up period of 20.2 months, the overall eye salvage rate was 84.7%, 1 patient was dead due to metastasis (2.2%) , 6 eyes developed recurrence (13.0%) ; Totally 49 eyes in group IV-IAC were included. Over all eye salvage rate was 77.3% with a medium follow-up period of 21.4 months. One patient died due to metastasis (2.3%) , 7 eyes developed recurrence (15.9%) . (2) Complications: Complications in group IAC were enophthalmos (n=19) , cataract (n=4) , vitreous haemorrhage (n=1) , orbital edema (n=17) and ptosis (n=12) . Complications in group IV-IAC were enophthalmos (n=10) , orbital edema (n=15) and ptosis (n=12) . The difference of the overall eye salvage rate, recurrence rate, metastatic rate and death rate between group IAC and group IV-IAC were not statistically significant (P>0.05) , but the enophthalmos rate was higher in group IAC (P=0.006) .

The primary IV-IAC for retinoblastoma could not effectively lower the eye salvage rate, but it will reduce the incidence of enophthalmos and cataracts.