Survival Outcomes and Complications Among Canadian Children With Retinoblastoma: A Population-Based Report From CYP-C

Retinoblastoma (RB) is the most common pediatric ocular cancer, yet population-based data on survival and risk factors remain limited. This study aimed to describe survival in a large national RB cohort and identify predictors of death and complications.

We performed a retrospective, population-based cohort study using the Cancer in Young People in Canada database of all children diagnosed with RB from 2001 to 2019. Primary outcomes were 5-year overall survival (OS) and grade 3-4 complications. Univariate and multivariable regression analyses were performed to assess associations between clinical characteristics and the outcomes. Kaplan-Meier survival analyses were conducted to estimate time-to-event outcomes.

Among the 484 patients, the median age at diagnosis was 1.2 years (interquartile range: 0.4-2.2). Eight (1.7%) patients had metastatic disease at diagnosis, and 273 (56.4%) underwent enucleation. Overall, 96.1% of patients in the cohort were alive at last follow-up. The cohort's 5-year OS, as estimated by the Kaplan-Meier method, was 94.2% (95% CI 90.8-96.3). Compared with localized disease, metastatic disease was associated with inferior 5-year OS (12.5% vs. 96.2%, p < 0.001). Non-White patients (vs. White patients) had inferior 5-year OS (88.8% vs. 96.3%, p = 0.003). The prevalence of grade 3 and 4 complications was 4.8% across the entire cohort (n = 31 events, n = 23 patients). Eight (1.7%) patients developed grade 3 or 4 hearing loss.

This large population-based study reported few complications in children with RB but highlighted key survival disparities among non-White patients and patients with metastatic disease.