Intraarterial Chemotherapy for Retinoblastoma, Outcomes Analysis in 357 Eyes: Thirteen Years of Experience in a Referral Center in Brazil.

Abstract

Objective: To evaluate the outcomes of intraarterial chemotherapy (IAC) for the treatment of naive and nonnaive retinoblastoma eyes. Ocular survival rates, risk factors for enucleation, ocular complications, metastatic disease, and overall survival were analyzed.

Design: A retrospective, single-institution study.

Participants: A total of 300 patients treated with IAC between April 2010 and April 2023 were included.

Interventions: During IAC infusions, 1 to 3 drugs were used (melphalan, 3.0-7.5 mg; topotecan, 0.3-2.0 mg; and carboplatin, 20-50 mg). Adjuvant therapy was used as needed to consolidate treatment.

Main outcome measures: Ocular survival rates, ocular complications, and the risk factors for enucleation were measured.

Results: A total of 357 eyes were treated with 1536 IAC infusions, with a median of 4 cycles per eye, and followed for 60.69 months. The Kaplan-Meier estimates for the overall ocular survival were 90% at 1 year, 89% at 2 years, and 86% at 5 years. No difference in ocular survival was found between IAC indications (primary 88% vs. secondary 85% vs. bridge 89%; P = 0.52) and the use of tandem therapy (tandem 85% vs. no tandem 87%; P = 0.93). Intravitreal chemotherapy was used as adjuvant therapy in 31.37% and plaque therapy in 5% of the eyes. The group did not receive external beam radiation. Univariable and multivariable analyses showed that the presence of subretinal seeds was significantly associated with an increased risk of enucleation, and the use of ophthalmic artery (OA) ostium in >50% of infusions per eye was a protective factor to avoid enucleation. Retinal and choroidal vascular, ischemic, or atrophic effects were the most frequent complications found in 5.0% of the eyes. Metastatic disease was observed in 0.33% of the patients. The overall 5-year patient survival was 99.3%.

Conclusions: The use of IAC in different indications (primary, secondary, bridge, and tandem) to treat naive or recurrent-refractory retinoblastomas showed successful results. Most eyes were preserved. Subretinal seeds at presentation were associated with a high enucleation risk. The use of the OA ostium for drug delivery avoided enucleation.

Financial disclosure(s): Proprietary or commercial disclosure may be found in the Footnotes and Disclosures at the end of this article.