Exosomal microRNAs from Longitudinal Liquid Biopsies for the Prediction of Response to Induction Chemotherapy in High-Risk Neuroblastoma Patients: A Proof of Concept SIOPEN Study

Martina Morini, Davide Cangelosi, Daniela Segalerba, Danilo Marimpietri, Federica Raggi, Aurora Castellano, Doriana Fruci, Jaime Font de Mora, Adela Cañete, Yania Yáñez, Virginie Viprey, Maria Valeria Corrias, Barbara Carlini, Annalisa Pezzolo, Gudrun Schleiermacher, Katia Mazzocco, Ruth Ladenstein, Angela Rita Sementa, Massimo Conte, Alberto GaraventaSusan Burchill, Roberto Luksch, Maria Carla Bosco, Alessandra Eva, Luigi Varesio

Research output: Contribution to journalArticle


Despite intensive treatment, 50% of children with high-risk neuroblastoma (HR-NB) succumb to their disease. Progression through current trials evaluating the efficacy of new treatments for children with HR disease usually depends on an inadequate response to induction chemotherapy, assessed using imaging modalities. In this study, we sought to identify circulating biomarkers that might be detected in a simple blood sample to predict patient response to induction chemotherapy. Since exosomes released by tumor cells can drive tumor growth and chemoresistance, we tested the hypothesis that exosomal microRNA (exo-miRNAs) in blood might predict response to induction chemotherapy. The exo-miRNAs expression profile in plasma samples collected from children treated in HR-NBL-1/SIOPEN before and after induction chemotherapy was compared to identify a three exo-miRs signature that could discriminate between poor and good responders. Exo-miRNAs expression also provided a chemoresistance index predicting the good or poor prognosis of HR-NB patients.

Original languageEnglish
Issue number10
Publication statusPublished - Sep 30 2019


Cite this