Objective
Assess the utility of second-course ophthalmic artery chemosurgery (OAC) for patients with intra-ocular retinoblastoma that recurred following prior ophthalmic artery chemosurgery. This study evaluates the efficacy and toxicity of second-course OAC.
Design
Single-arm, retrospective study of 29 eyes in 30 patients treated with second-course OAC at Memorial Sloan-Kettering Cancer Center between May 2006 and July 2013, with a median 25.9 months follow-up.
Participants
Retinoblastoma patients who underwent a course of OAC, with a minimum of 2 months of progression-free follow-up at monthly examinations, but who subsequently received additional OAC for recurrent tumor.
Methods
Efficacy- Kaplan-Meier survival estimates were generated and the Mantel-Cox test was used to compare curves. Toxicity- Electroretinogram (ERG) amplitudes were measured in response to 30-Hz photopic flicker stimulation before and after OAC treatment; systemic adverse events were graded according to CTCAE 4.0.
Main Outcome Measure
Efficacy- Ocular progression free survival, ocular event (enucleation, external beam radiation or intravitreal melphalan) free survival and ocular survival. Toxicity- Peak-to-peak comparisons between ERG studies before and after OAC treatment; CTCAE 4.0 graded systemic adverse events.
Results
50% of all recurrences were within 4.4 months and 90% were within 16 months of completion of first course OAC. The 2-year Kaplan-Meier ocular survival, event free survival and progression free survival estimates following second-course OAC were 82.8% (95% confidence interval [CI], 60.1–93.2%), 57.3% (95%CI, 36.1–73.7%) and 26.5% (95% confidence interval [CI], 11.0–45.0%), respectively. All eyes without vitreous seeding are progression free, while eyes with vitreous seeding were significantly associated with worse ocular survival following second- course OAC (p=0.03). Following second-course OAC, 90% of eyes had stable or improved electroretinogram responses. Of all evaluable cases, there was no increased risk of systemic toxicity during second course compared to initial course OAC.
Conclusions
Retinoblastoma eyes requiring second-course OAC following initial OAC treatment have good salvage rates and the treatment has an acceptable ocular and systemic toxicity profile. However, these eyes often require additional (third or fourth-course) OAC or other treatment modalities due to progression of disease after second-line OAC, particularly if vitreous seeds are present at the time of initial OAC failure.