Avapritinib: preclinical studies of a promising novel therapeutic option for gastrointestinal stromal tumors

Milena Urbini


Gastrointestinal stromal tumors (GISTs) represent the most common mesenchymal tumor of the gastrointestinal tract (80%), with an annual incidence of 10–15 cases per million people (1). GISTs have become a paradigm for the use of molecular diagnostics and targeted therapy, since its molecular classification is able to predict treatment response. Most GISTs are driven by activating mutations in KIT (70–80% of cases) or PDGFRA (10%) receptor tyrosine kinases. This discovery represents the rationale of the efficacy of target therapies efficacy using small molecule tyrosine kinase inhibitors (TKIs) for this malignancy (1,2).