Trametinib appears to be a suitable option for refractory pediatric low-grade glioma and warrants further investigations in case of progression.

No abstract

s vi185 NEURO-ONCOLOGY • NOVEMBER 20175-year EFS/OS rates of 62.8 ± 9.9% and 78.5 ± 8.3%, respectively, while patients with molecularly-classified HGG had an EFS/OS of 5.6 ± 3.8% and 12.0 ± 6.5%, respectively. Although the study was likely underpowered due to the early closure of CNS-PNET/PBL enrollment, there was no evidence that carboplatin or isotretinoin significantly altered outcomes for these patients. Survival for patients with HGG were similar to studies that do not use craniospinal irradiation or intensive chemotherapy. CONCLU-SIONS: This trial highlights the challenges of histology-based pathology for brain tumors in children and indicates that molecular profiling should become a standard component of initial diagnosis. For patients with CNS-PNETs/PBLs, prognosis is considerably better than previously assumed when molecularly-confirmed HGG were removed from the cohort. Identification of molecular HGG will spare affected children from unhelpful intensive treatment.

Aim: We previously conducted exome-wide association study in acute lymphoblastic leukemia patients and identified association of five SNPs with asparaginase-related thrombosis. Here we aimed to replicate these findings in an independent patient cohort and through analyses in vitro. Patients & methods: SNPs located in IL16, MYBBP1A, PKD2L1, RIN3 and MPEG1 genes were analyzed in patients receiving Dana-Farber Cancer Institute acute lymphoblastic leukemia treatment protocols 05-001 and 11-001. Thrombophilia-related variations were also analysed. Results: IL16 rs11556218 conferred higher risk of thrombosis and higher in vitro sensitivity to asparaginase. The association was modulated by the treatment protocol, risk group and immunophenotype. A crosstalk between factor V Leiden, non-O blood groups and higher risk of thrombosis was also seen. Conclusion: IL16 and factor V Leiden variations are implicated in asparaginase-related thrombosis.