Transcriptome based individualized therapy of refractory pediatric sarcomas: feasibility, tolerability and efficacy.

ABSTRACT: Survival rates of pediatric sarcoma patients stagnated during the last two decades, especially in adolescents and young adults (AYAs). Targeted therapies offer new options in refractory cases. Gene expression profiling provides a robust method to characterize the transcriptome of each patient's tumor and guide the choice of therapy. Twenty patients with refractory pediatric sarcomas (age 8-35 years) were assessed with array profiling: ten had Ewing sarcoma, five osteosarcoma, and five soft tissue sarcoma. Overexpressed genes and deregulated pathways were identified as actionable targets and an individualized combination of targeted therapies was recommended. Disease status, survival, adverse events (AEs), and quality of life (QOL) were assessed in patients receiving targeted therapy (TT) a

SUBMITTER: Weidenbusch B 

PROVIDER: S-EPMC5945512 | biostudies-literature | 2018 Apr

REPOSITORIES: biostudies-literature