Incidence and prognostic role of the ocular manifestations of neuroblastoma in children

Abstract

Purpose To describe the ocular manifestations of neuroblastoma in a large cohort of children. Design Retrospective cohort study. Methods The medical records of patients diagnosed with neuroblastoma between 1989 and 2017 at a tertiary care pediatric hospital were analyzed. The main outcome measurements were the incidence and prognostic role of ocular findings. Results There were 523 patients with neuroblastoma in the study cohort. Median age at diagnosis was 1.9 years, median follow-up was 4.0 years, and 57.2% were male. At last follow-up, 55.3% were in disease remission, 5.0% had stable disease, 28.1% were deceased, and 11.7% were on active or palliative treatment. A total of 86 patients (16.4%) had ocular manifestations of neuroblastoma, 58 at presentation and 29 during the disease course. The most common findings were orbital involvement in 37 (43.0%), opsoclonus in 20 (23.3%), and Horner syndrome in 24 (27.9%). In 16 patients (3.1%), there were only ocular findings at presentation, including 8 (1.5%) with orbital involvement, 7 (1.3%) with Horner syndrome, and 1 (0.2%) with orbital involvement and a cranial nerve palsy. On survival analysis, a favorable prognosis was associated with opsoclonus, female sex, and diagnosis before 12 months of age, whereas a worse prognosis was associated with orbital involvement. Conclusions In this cohort, approximately 1 in 6 patients with neuroblastoma had ocular manifestations, but only 3% presented with only ocular findings. Orbital involvement was common and associated with a poor prognosis, whereas opsoclonus, female sex, and younger age at diagnosis were associated with a favorable prognosis.