Avapritinib shrank tumours in almost nine in ten patients with PDGFRA D842V-mutant gastrointestinal stromal tumour, a subtype completely resistant to imatinib and every other kinase inhibitor, and became its first effective treatment.
Phase 1 dose-escalation and expansion study; this analysis covers 56 patients with PDGFRA D842V-mutant advanced GIST treated with avapritinib at 300 or 400 mg daily.
Objective response was 88 percent (9 percent complete), median duration of response not reached, and 12-month progression-free survival 81 percent; cognitive effects, periorbital oedema and anaemia were the characteristic toxicities, with rare intracranial bleeding.
Avapritinib 300 mg is the standard first-line treatment for advanced PDGFRA D842V GIST, and mutation testing before starting imatinib is essential to identify these patients.
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Shares PDGFRA D842V-mutant GIST, Avapritinib.
Shares PDGFRA D842V-mutant GIST, Avapritinib.
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