Abstract
Twenty-five consecutive children (median age 7.4 years) were enrolled according to the standard MRI inclusion criteria in a treatment strategy, initially begun on a compassionate basis, including standard dose radiotherapy, and re-irradiation at relapse with 19.8 Gy, and concomitant and adjuvant nimotuzumab and vinorelbine. One-year OS was 30 ± 10 %; median PFS and OS were 8.5 and 15 months, respectively. Serum specimens were collected at baseline, at each therapy administration and during follow-up. We used a high-throughput microRNA screening approach to assess miRNA profile in serum samples obtained from 24 of those patients with the main aim to identify novel non-invasive biomarkers able to improve the prediction of disease outcome and therapeutic sensitivity. Here we present the results of serum miRNA profiling at baseline. microRNA expression profiling was performed using Agilent platform and Human miRNA SureSelect 8x60K containing 2006 miRNAs annotated on miRBase19.0. Primary data analysis yielded a matrix containing 330 detectable miRNA. Association with PFS allowed us to disclose a signature of 10 miRNAs able to stratify high and low risk patients (HR=4.33, 95%CI 1.49–12.54; p=4.27E-05). Our signature was evaluated in longitudinal series and associated to clinical data in order to improve prediction of disease progression. At present, this is the first study that aims at investigating circulating miRNA levels in DIPG pediatric patients.