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.