BACKGROUND: The prognosis of children with metastatic stage 4 neuroblastoma (NB) has remained poor in the past decade.<br /><br />PATIENTS AND METHODS: Using microarray analyses of 342 primary tumors, we here developed and validated an easy to use gene expression-based risk score including 18 genes, which can robustly predict the outcome of stage 4 patients.<br /><br />RESULTS: This classifier was a significant predictor of overall survival in two independent validation cohorts [cohort 1 (n = 214): P = 6.3 × 10(-5); cohort 2 (n = 27): P = 3.1 × 10(-2)]. The prognostic value of the risk score was validated by multivariate analysis including the established markers age and MYCN status (P = 0.027). In the pooled validation cohorts (n = 241), integration of the risk score with the age and/or MYCN status identified subgroups with significantly differing overall survival (ranging from 35 to 100 %).<br /><br />CONCLUSION: Together, the 18-gene risk score classifier can identify patients with stage 4 NB with favorable outcome and may therefore improve risk assessment and treatment stratification of NB patients with disseminated disease.
Formicola, D., Petrosino, G., Lasorsa, V. A., Pignataro, P., Cimmino, F., Vetrella, S., … Capasso, M. (2016). An 18 gene expression-based score classifier predicts the clinical outcome in stage 4 neuroblastoma. Journal of Translational Medicine, 14(1). https://doi.org/10.1186/s12967-016-0896-7