Predictors of underlying neuroblastoma and neurodevelopmental outcomes in children with opsoclonus-myoclonus syndrome: a retrospective cohort study in China

Aim To explore whether distinct clinical features can serve as predictive indicators for the underlying NB and examine the neurodevelopmental outcomes in children diagnosed with opsoclonus-myoclonus syndrome (OMS). Methods We retrospectively enrolled children with a definitive diagnosis of OMS at Peking University First Hospital between January 2010 to January 2025. Demographic data, clinical characteristics were collected to compare based on with or without tumor. Neurodevelopmental outcomes were classified based on adaptive behavior assessments. Results Seventy-three children were totally included, including 30 with NB and 43 without NB. 52 children (71%) had neurological signs onset concentrated between 1 and 2 years of age. There was significant difference in the composition ratio of initial symptoms between the two groups of OMS patients. The proportion of opsoclonus as the initial symptom in the OMS with NB group (20%) was higher than that in the OMS without NB group (2%). 48 patients with neurological remission underwent assessment of adaptive behavior. By the last follow-up, 28 (58%) patients had good adaptive behavior, and 20 (42%) patients had poor adaptive behavior. The main variables influencing the neurodevelopmental prognosis in OMS patients, ranked from highest to lowest importance, are as follows: chemotherapy, rituximab, myoclonus at onset, immunotherapy initiation, tumor, age at onset, gender, ataxia at onset, OMS severity at onset, opsoclonus at onset, and ataxia at onset. The multivariate logistic regression model further confirmed that myoclonus at onset (OR = 14.33, 95% CI: 1.77–116.4, p = 0.01) was an independent risk factor for poor outcomes, whereas neuroblastoma (OR = 0.11, 95% CI: 0.02–0.68, p = 0.02) and chemotherapy (OR = 0.07, 95% CI: 0.01–0.58, p = 0.02) were independent protective factors. The ROC curve yielded an AUC of 0.856, indicating good discriminative ability of the model. Interpretation Opsoclonus as the initial symptom may be a predictive indicator for the underlying NB. With NB and chemotherapy are the key factors for the favorable neurodevelopmental prognosis of OMS patients.