Transcriptome based individualized therapy of refractory pediatric cancer in adolescents and young adults.

10060 Background: Progress in pediatric oncology had little impact on adolescents and young adults (AYAs). Survival rates stagnated and are even worse than in younger and older age groups. Tumor biology in refractory patients is highly heterogeneous inter- and even intra-individually .New approaches are therefore urgently needed. Gene expression analysis may aid in medical decision making in this setting. Methods: We enrolled patients aged 10-40 years for whom standard of care and current clinical trials provided no further treatment options in a one arm open label prospective study to assess survival. Tumor samples obtained for diagnosis underwent transcriptome analysis with Affymetrix arrays. We focused on genes with > 1.5 fold expression vs. normal tissue, identified as drivers by TARGETgene. Targets ranked between 1-100 were considered for therapy. Drug selection criteria were: delivery, no previous use in the patient, citations related to disease, citations related to other cancers, side effects, dru...