Avapritinib in the treatment of PDGFRA exon 18 mutated gastrointestinal stromal tumors.
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Gastrointestinal stromal tumors (GIST) can be molecularly classified based on different subtypes including mutations in <i>KIT</i> and <i>PDGFRA</i>. Patients with <i>PDGFRA</i> mutations are an important subgroup that commonly arise in the stomach and are associated with a more indolent disease course. Importantly, the most common <i>PDGFRA</i> molecular subtype, the D842V mutation in exon 18 of the gene which alters the activation loop, is imatinib insensitive in <i>in vitro</i> studies. Poor responses to imatinib have been seen clinically compared with <i>PDGFRA</i> exon 18 non-D842V-mutated GIST. Avapritinib (BLU-285) is a potent <i>KIT and PDGFRA-</i>specific tyrosine kinase inhibitor which has shown >90% response rates in patients with <i>PDGFRA</i> exon 18 D842V-mutated GIST. Results from the Phase I trial of avapritinib have indicated that this drug should be the standard of care for patients with <i>PDGFRA</i> exon 18 D842V-mutated GIST.
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Molecular and Systems Oncology
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Future oncology (London, England), 2020, 16 (22), pp. 1639 - 1646