Primary Enucleation for Intraocular Unilateral Retinoblastoma Can Save Life in Lower-Income Settings

To investigate the outcomes of primary enucleation and adjuvant systemic chemotherapy, when deemed appropriate, on a cohort of children with unilateral intraocular Rb (AJCC 8th edition, cT2 and cT3) from diverse economic groupings.

A prospective analysis including treatment-naïve Rb patients were presented to 11 centers from 10 countries from January 1 to December 31, 2019, and were followed-up thereafter. Only children with unilateral intraocular Rb that underwent primary enucleation were included in the present analysis. Systemic metastasis and survival were investigated.

Of the 692 children with Rb, 191 (27.6%) were included in the study cohort. Among them, 24 (12.6%) were from low-income countries (LICs), 89 (46.6%) from lower-middle-income countries (LMICs), 59 (30.9%) from upper-middle-income countries (UMICs), and 19 (9.9%) from high-income countries (HICs). High-risk histopathological features were observed in 110 eyes (57.6%) following enucleation, and 102 of these children (92.7%) received adjuvant intravenous chemotherapy. The three-year survival rate for the entire cohort was 95.0%. Stratified by economic grouping, survival rates were 87.5% (LIC), 96.6% (LMIC), 93.2% (UMIC), and 100% (HIC). Children from LICs demonstrated a higher prevalence of HRHF compared to HICs. Residing in a lower-income country was associated with a higher risk of systemic metastasis and poorer outcomes.

In the present multinational cohort of children with unilateral intraocular Rb who underwent upfront enucleation coupled with adjuvant chemotherapy as needed, overall survival was favorable, especially for children from low-income countries. Prompting early diagnosis, while the tumor remains intraocular can be life-saving, particularly in low-resource settings where primary enucleation and adjuvant chemotherapy can cure unilateral Rb.