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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 Relevant Treatment Approaches Therapy Name Approval Status Evidence Type Efficacy Evidence References
PDGFRA L221F gastrointestinal stromal tumor sensitive PDGFR Inhibitor (Pan) Imatinib Phase I Actionable In a Phase I retrospective study, treatment with Gleevec (imatinib) demonstrated a prolonged response in a patient with a gastrointestinal stromal tumor harboring a PDGFRA L221F mutation (PMID: 22718859). 22718859
PDGFRA exon18 gastrointestinal stromal tumor sensitive PDGFR-alpha Inhibitor 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 exon18 gastrointestinal stromal tumor sensitive PDGFR-alpha Inhibitor 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 D846V osteosarcoma predicted - sensitive Sorafenib Case Reports/Case Series Actionable In a clinical case study, a pediatric patient with metastatic osteosarcoma demonstrated a partial response to treatment with Nexavar (sorafenib) for 51 months prior to recurrence, and analysis of the pre-progression tumor demonstrated presence of PDGFRA D846V, which was not present in the post-progression sample, suggesting that the PDGFRA variant may have conferred sensitivity to Nexavar (sorafenib) (PMID: 30318721). 30318721
PDGFRA D846V gastrointestinal stromal tumor predicted - resistant PDGFR Inhibitor (Pan) Imatinib Case Reports/Case Series Actionable In a clinical study, none of 4 gastrointestinal stromal tumor patients harboring PDGFRA D842V (n=3) or PDGFRA D846V (n=1) mutations treated on a Phase III clinical trial demonstrated a response to treatment with Gleevec (imatinib), compared to 5 of 6 patients harboring PDGFRA sensitizing mutations (PMID: 16624552). 16624552
KIT A502_Y503dup PDGFRA H687Y gastrointestinal stromal tumor predicted - resistant PDGFR Inhibitor (Pan) Imatinib Case Reports/Case Series Actionable In a clinical study, PDGFRA H687Y was identified as a secondary mutation in a patient with gastrointestinal stromal tumor harboring primary KIT A502_Y503dup mutation, who developed resistance to Gleevec (imatinib mesylate) (PMID: 18488160). 18488160
PDGFRA positive lung non-small cell carcinoma no benefit MEDI-575 Preclinical - Cell line xenograft Actionable In a preclinical study, MEDI-575 treatment did not inhibit tumor growth in a cell line xenograft model of PDGFRA-positive non-small cell lung cancer (PMID: 23558446). 23558446
PDGFRA over exp malignant glioma sensitive MEDI-575 Preclinical - Cell line xenograft Actionable In a preclinical study, MEDI-575 treatment inhibited tumor growth in a cell line xenograft model of glioma overexpressing PDGFRA (PMID: 23558446). 23558446
PDGFRA S566_E571delinsR Advanced Solid Tumor sensitive PDGFR Inhibitor (Pan) Imatinib Preclinical - Cell culture Actionable In a preclinical study, Gleevec (imatinib) inhibited Pdgfra phosphorylation in transformed cells over expressing PDGFRA S566_E571delinsR in culture (PMID: 21224473). 21224473
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...
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 R748G myeloid leukemia sensitive PDGFR Inhibitor (Pan) Imatinib Preclinical - Cell line xenograft Actionable In a preclinical study, Gleevec (imatinib) inhibited growth of transformed mouse myeloid cells over expressing PDGFRA R748G in culture and prolonged survival in xenograft models (PMID: 21224473). 21224473
PDGFRA D842_I843delinsIM Advanced Solid Tumor resistant PDGFR Inhibitor (Pan) Imatinib Preclinical - Cell culture Actionable In a preclinical study, transformed cells over expressing PDGFRA D842_I843delinsIM were resistant to inhibition of Pdgfra phosphorylation by Gleevec (imatinib) in culture (PMID: 21224473). 21224473
PDGFRA Y288C Advanced Solid Tumor resistant PDGFR Inhibitor (Pan) Imatinib Preclinical - Cell culture Actionable In a preclinical study, transformed cells expressing PDGFRA Y288C demonstrated resistance to Gleevec (imatinib) in culture (PMID: 30389923). 30389923
PDGFRA Y288C Advanced Solid Tumor resistant PDGFR Inhibitor (Pan) Sunitinib Preclinical - Cell culture Actionable In a preclinical study, transformed cells expressing PDGFRA Y288C demonstrated resistance to Sutent (sunitinib) in culture (PMID: 30389923). 30389923
PDGFRA Y288C Advanced Solid Tumor sensitive GSK2126458 Preclinical - Cell culture Actionable In a preclinical study, transformed cells expressing PDGFRA Y288C demonstrated sensitivity to GSK2126458 (omipalisib) in culture, resulting in decreased cell proliferation (PMID: 30389923). 30389923
PDGFRA Y288C Advanced Solid Tumor sensitive Trametinib Preclinical - Cell culture Actionable In a preclinical study, Mekinist (trametinib) treatment resulted in decreased proliferation of transformed cells expressing PDGFRA Y288C in culture (PMID: 30389923). 30389923
PDGFRA Y288C Advanced Solid Tumor resistant PDGFR Inhibitor (Pan) Crenolanib Preclinical - Cell culture Actionable In a preclinical study, transformed cells expressing PDGFRA Y288C demonstrated resistance to Crenolanib in culture (PMID: 30389923). 30389923
PDGFRA Y288C Advanced Solid Tumor no benefit AZD9150 Preclinical - Cell culture Actionable In a preclinical study, ISIS-STAT3rx (AZD9150) did not decrease viability of transformed cells expressing PDGFRA Y288C in culture (PMID: 30389923). 30389923
PDGFRA Y288C Advanced Solid Tumor resistant PDGFR Inhibitor (Pan) Nilotinib Preclinical - Cell culture Actionable In a preclinical study, transformed cells expressing PDGFRA Y288C demonstrated resistance to Tasigna (nilotinib) in culture (PMID: 30389923). 30389923
PDGFRA D842Y Advanced Solid Tumor sensitive PDGFR Inhibitor (Pan) Crenolanib Preclinical Actionable In a preclinical study, Crenolanib inhibited Pdgfra phosphorylation and decreased proliferation in transformed human cells expressing PDGFRA D842Y in culture (PMID: 24132921). 24132921
PDGFRA D842Y Advanced Solid Tumor sensitive PDGFR Inhibitor (Pan) Imatinib Preclinical Actionable In a preclinical study, Gleevec (imatinib) inhibited Pdgfra phosphorylation and decreased proliferation in transformed human cells expressing PDGFRA D842Y in culture (PMID: 24132921). 24132921
PDGFRA N659S myeloid leukemia decreased response PDGFR Inhibitor (Pan) Imatinib Preclinical - Cell culture Actionable In a preclinical study, transformed mouse myeloid cells over expressing PDGFRA N659S demonstrated reduced sensitivity to Gleevec (imatinib) in culture (PMID: 21224473). 21224473
PDGFRA R841_D842delinsKI Advanced Solid Tumor resistant PDGFR Inhibitor (Pan) Imatinib Preclinical - Cell culture Actionable In a preclinical study, transformed cells over expressing PDGFRA R841_D842delinsKI were resistant to inhibition of Pdgfra phosphorylation by Gleevec (imatinib) in culture (PMID: 21224473). 21224473
PDGFRA H650Q leukemia sensitive PDGFR Inhibitor (Pan) Imatinib Preclinical Actionable In a preclinical study, mice injected with transformed cells harboring PDGFRA H650Q developed leukemia-like disease that is sensitive to Gleevec (imatinib) (PMID: 21224473). 21224473
PDGFRA G853D Advanced Solid Tumor sensitive PDGFR Inhibitor (Pan) Imatinib Preclinical Actionable In a preclinical study, Gleevec (imatinib) inhibited Pdgfra phosphorylation and decreased proliferation in transformed human cells expressing PDGFRA G853D in culture (PMID: 24132921). 24132921
PDGFRA G853D Advanced Solid Tumor sensitive PDGFR Inhibitor (Pan) Crenolanib Preclinical Actionable In a preclinical study, crenolanib inhibited Pdgfra phosphorylation and decreased proliferation in transformed human cells expressing PDGFRA G853D in culture (PMID: 24132921). 24132921
PDGFRA I843del Advanced Solid Tumor sensitive PDGFR Inhibitor (Pan) Imatinib Preclinical Actionable In a preclinical study, Gleevec (imatinib) decreased phosphorylation of Pdgfra in cell lines expressing PDGFRA I843del mutation in culture (PMID: 14645423). 14645423
PDGFRA H845_N848delinsP Advanced Solid Tumor sensitive PDGFR Inhibitor (Pan) Imatinib Preclinical - Cell culture Actionable In a preclinical study, Gleevec (imatinib) inhibited Pdgfra phosphorylation in transformed cells over expressing PDGFRA H845_N848delinsP in culture (PMID: 21224473). 21224473
PDGFRA H845_N848delinsP gastrointestinal stromal tumor predicted - sensitive PDGFR Inhibitor (Pan) Imatinib Case Reports/Case Series Actionable In a clinical case study, Gleevec (imatinib) treatment resulted in a complete metabolic response in all lesions in a patient with gastric gastrointestinal stromal tumor harboring PDGFRA H845_N848delinsP (PMID: 30778083). 30778083
PDGFRA P577S Advanced Solid Tumor sensitive PDGFR Inhibitor (Pan) Crenolanib Preclinical Actionable In a preclinical study, crenolanib inhibited Pdgfra phosphorylation and decreased proliferation in cells expressing PDGFRA P577S in culture (PMID: 24132921). 24132921
PDGFRA P577S Advanced Solid Tumor sensitive PDGFR Inhibitor (Pan) Imatinib Preclinical Actionable In a preclinical study, Gleevec (imatinib) inhibited Pdgfra phosphorylation and decreased proliferation in cells expressing PDGFRA P577S in culture (PMID: 24132921). 24132921
PDGFRA V658A Advanced Solid Tumor sensitive PDGFR Inhibitor (Pan) Crenolanib Preclinical Actionable In a preclinical study, crenolanib inhibited Pdgfra phosphorylation and decreased proliferation in cells expressing PDGFRA V658A in culture (PMID: 24132921). 24132921
PDGFRA V658A Advanced Solid Tumor sensitive PDGFR Inhibitor (Pan) Imatinib Preclinical Actionable In a preclinical study, crenolanib inhibited Pdgfra phosphorylation and decreased proliferation in transformed human cells expressing PDGFRA V658A in culture (PMID: 24132921). 24132921
PDGFRA mutant gastrointestinal stromal tumor sensitive PDGFR Inhibitor (Pan) 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 mutant gastrointestinal stromal tumor predicted - sensitive PDGFR-alpha Inhibitor 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 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 D842X gastrointestinal stromal tumor sensitive PDGFR-alpha Inhibitor Avapritinib Phase I Actionable In a Phase I (NAVIGATOR) trial, Ayvakit (avapritinib) treatment resulted in an objective response rate of 70% (26/37, 2 complete responses, 24 partial responses) and a disease control rate of 97% (36/37) in patients with gastrointestinal stromal tumor harboring PDGFRA D842 mutations (The CTOS 2018 Annual Meeting, Nov 14-17, Rome Italy, Paper 012 3027631; NCT02508532). detail...
PDGFRA D842X gastrointestinal stromal tumor sensitive PDGFR-alpha Inhibitor Avapritinib Phase I Actionable In a Phase I trial, Ayvakit (avapritinib) demonstrated preliminary antitumor activity in gastrointestinal stromal tumor patients harboring PDGFRA D842 mutations, with partial responses in 50% (6/12) and stable disease in 50% (6/12) of patients (EORTC-NCI-AACR 2016, Abs 6LBA). detail...
PDGFRA H845Y Advanced Solid Tumor sensitive PDGFR Inhibitor (Pan) Imatinib Preclinical Actionable In a preclinical study, Gleevec (imatinib) inhibited Pdgfra phosphorylation in cells expressing PDGFRA H845Y in culture (PMID: 24132921). 24132921
PDGFRA H845Y Advanced Solid Tumor sensitive PDGFR Inhibitor (Pan) Crenolanib Preclinical Actionable In a preclinical study, crenolanib inhibited Pdgfra phosphorylation in cells expressing PDGFRA H845Y in culture (PMID: 24132921). 24132921
PDGFRA V561D Advanced Solid Tumor sensitive PDGFR-alpha Inhibitor AZD3229 Preclinical - Cell culture Actionable In a preclinical study, transformed cells expressing PDGFRA V561D demonstrated sensitivity to AZD3229 in culture, resulting in reduced cell viability (PMID: 32350132). 32350132
PDGFRA V561D Advanced Solid Tumor sensitive PDGFR-alpha Inhibitor Ripretinib Preclinical - Cell culture Actionable In a preclinical study, transformed cells expressing PDGFRA V561D demonstrated sensitivity to Qinlock (ripretinib) in culture, resulting in reduced cell viability (PMID: 32350132). 32350132
PDGFRA V561D Advanced Solid Tumor sensitive PDGFR Inhibitor (Pan) Regorafenib Preclinical - Cell culture Actionable In a preclinical study, transformed cells expressing PDGFRA V561D demonstrated sensitivity to Stivarga (regorafenib) in culture, resulting in reduced cell viability (PMID: 32350132). 32350132
PDGFRA V561D Advanced Solid Tumor sensitive PDGFR Inhibitor (Pan) Crenolanib Preclinical - Cell culture Actionable In a preclinical study, transformed cells expressing PDGFRA V561D demonstrated sensitivity to Crenolanib in culture, resulting in decreased cell proliferation (PMID: 30389923). 30389923
PDGFRA V561D Advanced Solid Tumor sensitive PDGFR-alpha Inhibitor Avapritinib Preclinical - Cell culture Actionable In a preclinical study, transformed cells expressing PDGFRA V561D demonstrated sensitivity to Ayvakit (avapritinib) in culture, resulting in reduced cell viability (PMID: 32350132). 32350132
PDGFRA V561D Advanced Solid Tumor sensitive PDGFR-alpha Inhibitor Avapritinib Preclinical - Cell culture Actionable In a preclinical study, Ayvakit (avapritinib) inhibited Kit phosphorylation in transformed cells overexpressing PDGFRA V561D in culture (PMID: 29093181). 29093181
PDGFRA V561D Advanced Solid Tumor sensitive GSK2126458 Preclinical - Cell culture Actionable In a preclinical study, transformed cells expressing PDGFRA V561D demonstrated sensitivity to GSK2126458 (omipalisib) in culture, resulting in decreased cell proliferation (PMID: 30389923). 30389923
PDGFRA V561D Advanced Solid Tumor sensitive PDGFR Inhibitor (Pan) Imatinib Preclinical - Cell culture Actionable In a preclinical study, cells expressing PDGFRA V561D demonstrated sensitivity to Gleevec (imatinib) in cell culture (PMID: 15928335). 15928335
PDGFRA V561D Advanced Solid Tumor sensitive PDGFR Inhibitor (Pan) Imatinib Preclinical - Cell culture Actionable In a preclinical study, transformed cells expressing PDGFRA V561D demonstrated sensitivity to Gleevec (imatinib) in culture, resulting in decreased cell proliferation (PMID: 30389923). 30389923
PDGFRA V561D Advanced Solid Tumor sensitive PDGFR Inhibitor (Pan) Nilotinib Preclinical - Cell culture Actionable In a preclinical study, transformed cells expressing PDGFRA V561D demonstrated sensitivity to Tasigna (nilotinib) in culture, resulting in decreased cell proliferation (PMID: 30389923). 30389923
PDGFRA V561D Advanced Solid Tumor sensitive PDGFR Inhibitor (Pan) Sunitinib Preclinical - Cell culture Actionable In a preclinical study, transformed cells expressing PDGFRA V561D demonstrated sensitivity to Sutent (sunitinib) in culture, resulting in decreased cell proliferation (PMID: 30389923). 30389923
PDGFRA V561D PDGFRA D842V Advanced Solid Tumor resistant PDGFR Inhibitor (Pan) Regorafenib Preclinical - Cell culture Actionable In a preclinical study, transformed cells expressing PDGFRA V561D and PDGFRA D842V demonstrated resistance to cell growth inhibition by Stivarga (regorafenib) in culture (PMID: 32350132). 32350132
PDGFRA V561D PDGFRA D842V Advanced Solid Tumor resistant PDGFR Inhibitor (Pan) Imatinib Preclinical - Cell culture Actionable In a preclinical study, transformed cells expressing PDGFRA V561D and PDGFRA D842V demonstrated resistance to cell growth inhibition by Gleevec (imatinib) in culture (PMID: 32350132). 32350132
PDGFRA V561D PDGFRA D842V gastrointestinal stromal tumor resistant PDGFR Inhibitor (Pan) Imatinib Case Reports/Case Series Actionable In a clinical study, PDGFRA D842V was identified as a secondary mutation in a patient with gastrointestinal stromal tumor harboring a primary PDGFRA V561D mutation, who developed resistance to Gleevec (imatinib mesylate) (PMID: 16954519). 16954519
PDGFRA V561D PDGFRA D842V Advanced Solid Tumor resistant PDGFR Inhibitor (Pan) Sunitinib Preclinical - Cell culture Actionable In a preclinical study, PDGFRA D842V conferred resistance to inhibition of PDGFRA phosphorylation by Sutent (sunitinib) in cells expressing PDGFRA V561D in culture (PMID: 18955458). 18955458
PDGFRA V561D PDGFRA D842V Advanced Solid Tumor resistant PDGFR Inhibitor (Pan) Sunitinib Preclinical - Cell culture Actionable In a preclinical study, transformed cells expressing PDGFRA V561D and PDGFRA D842V demonstrated resistance to cell growth inhibition by Sutent (sunitinib) in culture (PMID: 32350132). 32350132
PDGFRA V561D PDGFRA D842V Advanced Solid Tumor sensitive PDGFR-alpha Inhibitor Avapritinib Preclinical - Cell culture Actionable In a preclinical study, transformed cells expressing PDGFRA V561D and PDGFRA D842V demonstrated sensitivity to Ayvakit (avapritinib) in culture, resulting in reduced cell viability (PMID: 32350132). 32350132
PDGFRA V561D PDGFRA D842V Advanced Solid Tumor sensitive PDGFR-alpha Inhibitor AZD3229 Preclinical - Cell culture Actionable In a preclinical study, transformed cells expressing PDGFRA V561D and PDGFRA D842V demonstrated sensitivity to AZD3229 in culture, resulting in reduced cell viability and inhibition of PDGFRA signaling (PMID: 32350132). 32350132
PDGFRA V561D PDGFRA D842V Advanced Solid Tumor resistant PDGFR-alpha Inhibitor Ripretinib Preclinical - Cell culture Actionable In a preclinical study, transformed cells expressing PDGFRA V561D and PDGFRA D842V demonstrated resistance to cell growth inhibition by Qinlock (ripretinib) in culture (PMID: 32350132). 32350132
PDGFRA V561_I562insER Advanced Solid Tumor sensitive PDGFR Inhibitor (Pan) Imatinib Preclinical - Cell culture Actionable In a preclinical study, Gleevec (imatinib) inhibited Pdgfra phosphorylation in transformed cells over expressing PDGFRA V561_I562insER in culture (PMID: 21224473). 21224473
PDGFRA D842V Advanced Solid Tumor resistant PDGFR Inhibitor (Pan) Regorafenib Preclinical - Cell culture Actionable In a preclinical study, transformed cells expressing PDGFRA D842V demonstrated reduced response to growth inhibition by Stivarga (regorafenib) compared to GIST cells harboring KIT exon 11 deletion in culture (PMID: 31085175). 31085175
PDGFRA D842V Advanced Solid Tumor resistant PDGFR Inhibitor (Pan) Regorafenib Preclinical - Cell culture Actionable In a preclinical study, transformed cells expressing PDGFRA D842V demonstrated resistance to cell growth inhibition by Stivarga (regorafenib) in culture (PMID: 32350132). 32350132
PDGFRA D842V Advanced Solid Tumor sensitive PDGFR Inhibitor (Pan) Crenolanib Preclinical - Cell culture Actionable In a preclinical study, transformed cells expressing PDGFRA D842V demonstrated sensitivity to Crenolanib in culture, resulting in decreased cell proliferation (PMID: 30389923). 30389923
PDGFRA D842V Advanced Solid Tumor sensitive PDGFR Inhibitor (Pan) Crenolanib Preclinical - Cell culture Actionable In a preclinical study, Crenolanib inhibited Pdgfra phosphorylation and proliferation of transformed cells expressing PDGFRA D842V in culture (PMID: 22745105). 22745105
PDGFRA D842V Advanced Solid Tumor resistant PDGFR Inhibitor (Pan) Imatinib Preclinical - Cell culture Actionable In a preclinical study, transformed cells expressing PDGFRA D842V demonstrated resistance to Gleevec (imatinib) in culture (PMID: 30389923). 30389923
PDGFRA D842V Advanced Solid Tumor resistant PDGFR Inhibitor (Pan) Imatinib Preclinical Actionable In a preclinical study, expression of PDGFRA D842V conferred resistance to Gleevec (imatinib) in cell culture (PMID: 15928335). 15928335
PDGFRA D842V Advanced Solid Tumor sensitive PDGFR-alpha Inhibitor Avapritinib Preclinical - Cell culture Actionable In a preclinical study, Ayvakit (avapritinib) inhibited Kit phosphorylation in transformed cells overexpressing PDGFRA D842V in culture (PMID: 29093181). 29093181
PDGFRA D842V Advanced Solid Tumor sensitive PDGFR-alpha Inhibitor Avapritinib Preclinical - Cell culture Actionable In a preclinical study, transformed cells expressing PDGFRA D842V demonstrated sensitivity to Ayvakit (avapritinib) in culture, resulting in reduced cell viability (PMID: 32350132). 32350132
PDGFRA D842V Advanced Solid Tumor resistant PDGFR Inhibitor (Pan) Sunitinib Preclinical - Cell culture Actionable In a preclinical study, cells expressing PDGFRA D842V demonstrated resistance to inhibition of PDGFRA phosphorylation by Sutent (sunitinib) in culture (PMID: 18955458). 18955458
PDGFRA D842V Advanced Solid Tumor resistant PDGFR Inhibitor (Pan) Sunitinib Preclinical - Cell culture Actionable In a preclinical study, transformed cells expressing PDGFRA D842V demonstrated resistance to Sutent (sunitinib) in culture (PMID: 30389923). 30389923
PDGFRA D842V Advanced Solid Tumor sensitive GSK2126458 Preclinical - Cell culture Actionable In a preclinical study, transformed cells expressing PDGFRA D842V demonstrated sensitivity to GSK2126458 (omipalisib) in culture, resulting in decreased cell proliferation (PMID: 30389923). 30389923
PDGFRA D842V gastrointestinal stromal tumor sensitive PDGFR Inhibitor (Pan) Dasatinib Guideline Actionable Sprycel (dasatinib) is included in the guidelines for patients with gastrointestinal stromal tumor (GIST) with PDGFRA D842V mutations (NCCN.org). detail...
PDGFRA D842V gastrointestinal stromal tumor sensitive PDGFR Inhibitor (Pan) Dasatinib Phase 0 Actionable In a clinical trial, Sprycel (dasatinib) treatment resulted in a partial response in 25% (12/48) of patients with Gleevec (imatinib)-resistant gastrointestinal stromal tumor, with decreased tumor size in 2 of those patients, 1 of which harbored PDGFRA D842V (PMID: 29710216). 29710216
PDGFRA D842V gastrointestinal stromal tumor sensitive PDGFR-alpha Inhibitor Avapritinib Case Reports/Case Series Actionable In a Phase I trial, Ayvakit (avapritinib) treatment resulted in partial response lasted for more than 15 cycles of treatment, and sustained reduction of plasma PDGFRA D842V in a patient with gastrointestinal stromal tumor harboring PDGFRA D842V, whose disease had progressed on previous Gleevec (imatinib mesylate), Sprycel (dasatinib), and Crenolanib treatment (PMID: 29093181; NCT02508532). 29093181
PDGFRA D842V gastrointestinal stromal tumor sensitive PDGFR-alpha Inhibitor 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 D842V gastrointestinal stromal tumor sensitive PDGFR-alpha Inhibitor Avapritinib FDA approved Actionable In a Phase I trial (NAVIGATOR) that supported FDA approval, Ayvakit (avapritinib) treatment in patients with a gastrointestinal stromal tumor harboring PDGFRA D842V resulted in an overall response in 88% (49/56), with 5 patients experiencing a complete response (9%) and 44 patients with a partial response (79%) (PMID: 32615108; NCT02508532). detail... 32615108
PDGFRA D842V Advanced Solid Tumor conflicting PDGFR-alpha Inhibitor Ripretinib Preclinical - Cell culture Actionable In a preclinical study, transformed cells expressing PDGFRA D842V demonstrated resistance to cell growth inhibition by Qinlock (ripretinib) in culture (PMID: 32350132). 32350132
PDGFRA D842V Advanced Solid Tumor conflicting PDGFR-alpha Inhibitor Ripretinib Preclinical - Cell culture Actionable In a preclinical study, Qinlock (ripretinib) inhibited proliferation of transformed cells expressing PDGFRA D842V in culture (PMID: 31085175). 31085175
PDGFRA D842V Advanced Solid Tumor sensitive PDGFR-alpha Inhibitor AZD3229 Preclinical - Cell culture Actionable In a preclinical study, transformed cells expressing PDGFRA D842V demonstrated sensitivity to AZD3229 in culture, resulting in reduced cell viability (PMID: 32350132). 32350132
PDGFRA D842V Advanced Solid Tumor resistant PDGFR Inhibitor (Pan) Nilotinib Preclinical - Cell culture Actionable In a preclinical study, transformed cells expressing PDGFRA D842V demonstrated resistance to Tasigna (nilotinib) in culture (PMID: 30389923). 30389923
PDGFRA D842V Advanced Solid Tumor resistant PDGFR Inhibitor (Pan) Nilotinib Preclinical - Cell culture Actionable In a preclinical study, transformed cells expressing PDGFRA D842V demonstrated resistance to growth inhibition by Tasigna (nilotinib) in culture (PMID: 18794084). 18794084
PDGFRA D842V gastrointestinal stromal tumor resistant PDGFR Inhibitor (Pan) Imatinib Guideline Actionable Gleevec (imatinib) is not indicated for use in gastrointestinal stromal tumor (GIST) patients with PDGFRA D842V mutations (NCCN.org). detail...
PDGFRA D842V gastrointestinal stromal tumor sensitive PDGFR Inhibitor (Pan) Crenolanib Phase Ib/II Actionable In a Phase I/II trial, treatment with Crenolanib (CP-868596) resulted in a clinical benefit rate of 31% (5/16) in gastronintestinal stromal tumor patients harboring PDGFRA D842V, with partial response in 2/16 and stable disease in 3/16 patients (J Clin Oncol 34, 2016 (suppl; abstr 11010)). detail...
PDGFRA D842V Advanced Solid Tumor sensitive Midostaurin Preclinical - Cell culture Actionable In a preclinical study, Rydapt (midostaurin) inhibited proliferation of transformed cells expressing PDGFRA D842V in culture (PMID: 31085175). 31085175
PDGFRA D842V gastrointestinal stromal tumor predicted - sensitive PDGFR Inhibitor (Pan) Regorafenib Clinical Study - Cohort Actionable In a clinical study, Stivarga (regorafenib) treatment resulted in partial response in 11% (2/18) of patients and stable disease in 39% (7/18) of gastrointestinal stromal tumor patients, including prolonged stable disease for 12 months in a patient harboring PDGFRA D842V (PMID: 25905001). 25905001
PDGFRA R841K Advanced Solid Tumor sensitive PDGFR Inhibitor (Pan) Crenolanib Preclinical Actionable In a preclinical study, crenolanib inhibited Pdgfra phosphorylation and decreased proliferation in cells expressing PDGFRA R841K in culture (PMID: 24132921). 24132921
PDGFRA R841K Advanced Solid Tumor sensitive PDGFR Inhibitor (Pan) Imatinib Preclinical Actionable In a preclinical study, Gleevec (imatinib) inhibited Pdgfra phosphorylation and decreased proliferation in cells expressing PDGFRA R841K in culture (PMID: 24132921). 24132921
PDGFRA amp pleomorphic liposarcoma sensitive PDGFR Inhibitor (Pan) Pazopanib Preclinical - Pdx Actionable In a preclinical study, Votrient (pazopanib) treatment induced necrosis and reduced tumor growth in an Adriamycin (doxorubicin)-resistant orthotopic patient-derived xenograft (PDX) model of pleomorphic liposarcoma harboring PDGFRA amplification (PMID: 30060824). 30060824
PDGFRA amp lung small cell carcinoma sensitive PDGFR-alpha Inhibitor Ponatinib Preclinical Actionable In a preclinical study, Iclusig (ponatinib) inhibited growth of lung squamous cell cancer cells with PDGFRA amplification in culture (PMID: 22328973). 22328973
PDGFRA amp lung squamous cell carcinoma sensitive PDGFR Inhibitor (Pan) Dasatinib Preclinical - Cell line xenograft Actionable In a preclinical study, Sprycel (dasatinib) inhibited growth of a lung squamous cell cancer (SCC) cell line with amplification of PDGFRA in culture, and inhibited tumor growth in PDGFRA-amplified lung SCC xenograft models (PMID: 22328973). 22328973
PDGFRA amp lung squamous cell carcinoma sensitive PDGFR Inhibitor (Pan) Imatinib Preclinical Actionable In a preclinical study, Gleevec (imatinib) inhibited growth of lung squamous cell cancer cells with PDGFRA amplification in culture (PMID: 22328973). 22328973
PDGFRA amp malignant peripheral nerve sheath tumor sensitive PDGFR Inhibitor (Pan) Imatinib Preclinical Actionable In a preclinical study, a malignant peripheral nerve sheath tumor cell line harboring a PDGFRA amplification demonstrated cell growth inhibition when treated with Gleevec (imatinib) (PMID: 16357008). 16357008
PDGFRA amp lung non-small cell carcinoma sensitive PDGFR Inhibitor (Pan) Sunitinib Preclinical Actionable In a preclinical study, Sutent (sunitinib) decreased cell proliferation and inhibited downstream signaling in a NSCLC cell line harboring a PDGFRA amplification (PMID: 19366796). 19366796
PDGFRA amp lung cancer sensitive PDGFR-alpha Inhibitor Ripretinib Preclinical - Cell culture Actionable In a preclinical study, Qinlock (ripretinib) inhibited proliferation of lung cancer cells harboring PDGFRA amplification in culture, and resulted in tumor regression in cell line xenograft models (PMID: 31085175). 31085175
KIT amp PDGFRA amp glioblastoma multiforme sensitive PDGFR-alpha Inhibitor Ripretinib Phase I Actionable In a Phase I trial, Qinlock (ripretinib) demonstrated preliminary safety and efficacy in patients with advanced solid tumors, including a partial response in a glioblastoma multiforme patient harboring KIT and PDGFRA amplification (EORTC-NCI-AACR 2016, Abs 7LBA). detail...
ROS1 fusion KDR amp KIT amp PDGFRA amp lung non-small cell carcinoma predicted - resistant Crizotinib Case Reports/Case Series Actionable In a clinical study, a non-small cell lung carcinoma patient harboring a ROS1 fusion treated with Xalkori (crizotinib) responded, but eventually progressed, and was subsequently found to harbor presumed resistance alterations, including amplification of KIT, KDR, and PDGFRA (PMID: 29636358). 29636358
MLH1 M524I PDGFRA W559_R560del gastrointestinal stromal tumor predicted - sensitive PDGFR Inhibitor (Pan) Imatinib Case Reports/Case Series Actionable In a clinical case study, Gleevec (imatinib) treatment resulted in disease-free survival for over 10 months in a patient with epithelioid variant of gastrointestinal stromal tumor harboring PDGFRA W559_R560del and MLH1 M524I (PMID: 31273885). 31273885
PDGFRA Y849S Advanced Solid Tumor sensitive PDGFR Inhibitor (Pan) Imatinib Preclinical - Cell culture Actionable In a preclinical study, Gleevec (imatinib) inhibited Pdgfra phosphorylation and cell proliferation in transformed cells over expressing PDGFRA Y849S in culture (PMID: 21224473). 21224473
PDGFRA Y849S myeloid leukemia sensitive PDGFR Inhibitor (Pan) Imatinib Preclinical - Cell culture Actionable In a preclinical study, Gleevec (imatinib) inhibited growth of transformed mouse myeloid cells over expressing PDGFRA Y849S in culture (PMID: 21224473). 21224473
PDGFRA N848K lung non-small cell carcinoma predicted - sensitive PDGFR Inhibitor (Pan) Imatinib Case Reports/Case Series Actionable In a clinical case study, a non-small cell lung cancer patient harboring PDGFRA N848K achieved a durable response to Gleevec (imatinib), lasting more than 23 months (PMID: 30268485). 30268485
PDGFRA N659K Advanced Solid Tumor sensitive PDGFR Inhibitor (Pan) Imatinib Preclinical Actionable In a preclinical study, Gleevec (imatinib) decreased Pdgfra phosphorylation in cells expressing PDGFRA N659K in culture (PMID: 22745105). 22745105
PDGFRA N659K Advanced Solid Tumor sensitive PDGFR Inhibitor (Pan) Crenolanib Preclinical Actionable In a preclinical study, crenolanib decreased Pdgfra phosphorylation in cells expressing PDGFRA N659K in culture (PMID: 22745105). 22745105
PDGFRA D846Y Advanced Solid Tumor sensitive PDGFR Inhibitor (Pan) Imatinib Preclinical - Cell culture Actionable In a preclinical study, Gleevec (imatinib) inhibited Pdgfra phosphorylation and cell proliferation in transformed cells over expressing PDGFRA D846Y in culture (PMID: 21224473). 21224473
Clinical Trial Phase Therapies Title Recruitment Status