Intra-arterial chemotherapy as second-line treatment for advanced retinoblastoma: A 2-year single-center study in China.

Purpose: To evaluate the effectiveness and complications of intra-arterial chemotherapy (IAC) for treating advanced refractory retinoblastoma (RB) in a large single-center cohort.

Patients and Methods: Eighty-four eyes of 62 consecutive patients with advanced refractory RB who received IAC were included in the study during January 2013 and April 2015. These patients failed to respond adequately to a standard systemic chemotherapy (i.e., carboplatin, vincristine, and etoposide) with or without local therapy. Clinical outcomes and complications of these patients were reviewed.

Results: All of these patients received IAC of melphalan combined with topotecan. The mean follow-up period was 14.2 months after final IAC (ranged from 3 to 28 months). The rate of eye preservation was 41.67% in Group D and 20.83% in Group E of this study. Short-term ocular adverse events included eyelid edema (n = 12, 14.29%), bulbar conjunctiva congestion (n = 25, 29.76%), and excessive tearing (n = 10, 11.90%). Long-term complications included vitreous hemorrhage (n = 7, 8%), subretinal hemorrhage (n = 9, 11%), retinal vasculopathy (n = 6, 7%), and ophthalmic artery spasm with reperfusion (n = 11, 13%). Fever was observed after IAC in 14 patients; transient vomiting was observed in 17 patients; there were 8 cases of transient myelosuppression.

Conclusion: IAC can be an optional treatment to save eyes of Group D RB that failed in systemic chemotherapy and were destined for enucleation. However, it should be cautioned for Group E. Both the ocular and systemic toxicities of IAC were within tolerance.