Secondary Ophthalmic Artery Chemotherapy for Bilateral Retinoblastoma After Incomplete Response to Intravenous Chemotherapy: Initial Prospective Experience from a Resource-Limited Setting.

In 30 seconds

This prospective single-centre study assessed short-term outcomes of secondary ophthalmic artery chemotherapy (OAC) in 20 children with bilateral retinoblastoma whose eyes had responded incompletely to intravenous chemotherapy, with or without focal therapy. Among 21 treated eyes, overall globe salvage after a median follow-up of 19 months was 76.2% (16/21). Exploratory analysis found higher observed salvage with bridging than rescue OAC (100% vs. 37.5%), but the groups differed in baseline characteristics and treatment indication, limiting interpretation. The study supports feasibility in a resource-limited setting, while larger studies are needed.

Key findings

• Overall globe salvage was 76.2% (16/21) after a median follow-up of 19 months.
• Kaplan-Meier analysis estimated eye preservation at >80% at 12 months and ~70% at 24 months.
• Observed globe salvage was 100% with bridging OAC versus 37.5% with rescue OAC; subgroup differences may confound this comparison.
• Lack of favorable response after the first OAC cycle was associated with subsequent enucleation (RR = 6.00, 95% CI 1.03-35.91, p=0.001).

Why it matters

The findings provide prospective evidence that secondary OAC can be delivered as a globe-preserving approach after incomplete response to intravenous chemotherapy in a resource-limited setting. The observed outcomes and early-response association may help inform assessment of treatment response, but the small cohort and non-comparable subgroups mean they should not be treated as definitive estimates of benefit or timing effects.

What to keep in mind

This was a small, single-centre study with short-term follow-up. The bridging and rescue groups differed in baseline disease characteristics and treatment indication, limiting causal interpretation of their outcome comparison.