Molecular Profile Detail

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Profile Name PDGFRA R841_D842delinsKI
Gene Variant Detail

PDGFRA R841_D842delinsKI (gain of function - predicted)

Relevant Treatment Approaches PDGFR Inhibitor (Pan) PDGFR-alpha Inhibitor

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Molecular Profile Indication/Tumor Type Response Type Therapy Name Approval Status Evidence Type Efficacy Evidence References
PDGFRA exon18 gastrointestinal stromal tumor sensitive Avapritinib FDA approved Actionable In a Phase I trial (NAVIGATOR) that supported FDA approval, Ayvakit (avapritinib) treatment resulted in an objective response in 86% (37/43, 3 complete response, 34 partial response) and stable disease in 12% (5/43) of patients with unresectable or metastatic gastrointestinal stromal tumor harboring a PDGFRA exon 18 mutation, including D842V, with median duration of response not reached (J Clin Oncol 37, no. 15_suppl (May 20, 2019) 11022-11022; NCT02508532). detail... detail...
PDGFRA exon18 gastrointestinal stromal tumor sensitive Avapritinib Guideline Actionable Ayvakit (avapritinib) is included in the guidelines for patients with unresectable/metastatic gastrointestinal stromal tumor (GIST) with PDGFRA exon 18 mutations, including PDGFRA D842V (NCCN.org). detail...
PDGFRA mutant gastrointestinal stromal tumor not applicable N/A Clinical Study Diagnostic PDGFRA mutations are used in the diagnosis of gastrointestinal stromal tumors (PMID: 26276366, PMID: 25729899). 25729899 26276366
PDGFRA mutant gastrointestinal stromal tumor predicted - sensitive Ripretinib Phase I Actionable In a Phase I trial, Qinlock (ripretinib) was well tolerated, resulted in an objective response in 11.3% (16/142, 16 partial responses) and stable disease in 61.3% (87/142) of patients with advanced gastrointestinal stromal tumor harboring KIT (n=135) or PDGFRA mutations (n=7), with a median progression-free survival of 5.6 months (PMID: 32804590; NCT02571036). 32804590
PDGFRA mutant gastrointestinal stromal tumor sensitive Imatinib Guideline Actionable Gleevec (imatinib) is included in the guidelines as first-line therapy for patients with gastrointestinal stromal tumor (GIST) with PDGFRA mutations (NCCN.org). detail...
PDGFRA act mut pancreatic cancer predicted - sensitive Vistusertib Phase I Actionable In a Phase I trial, Vistusertib (AZD2014) demonstrated safety and preliminary efficacy in patients with advanced solid tumors, including a patient with pancreatic cancer harboring a PDGFRA activating mutation (PMID: 25805799). 25805799
PDGFRA act mut gastrointestinal stromal tumor not applicable N/A Guideline Diagnostic PDGFRA activating mutations aid the diagnosis of gastrointestinal stromal tumor (NCCN.org). detail...
Clinical Trial Phase Therapies Title Recruitment Status