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|Molecular Profile||Indication/Tumor Type||Response Type||Therapy Name||Approval Status||Evidence Type||Efficacy Evidence||References|
|IDH2 R140Q||acute myeloid leukemia||sensitive||Enasidenib||FDA approved - On Companion Diagnostic||Actionable||In a Phase I/II trial that supported FDA approval, Idhifa (enasidenib) treatment resulted in an overall response rate of 40.3% (71/176) with a median response duration of 5.8 months, complete remission in 19.3% (34/176), and stable disease in 48.3% (85/176) of acute myeloid leukemia patients harboring IDH2 mutations (PMID: 28588020; NCT01915498) and IDH2 R140Q is on the companion diagnostic.||detail... detail... 28588020|
|IDH2 R140Q||acute myeloid leukemia||sensitive||Enasidenib||Preclinical - Pdx & cell culture||Actionable||In a preclinical study, Enasidenib (AG-221) treatment reduced 2-hydroxyglutarate (2HG) levels and induced differentiation of leukemic blasts from acute myeloid leukemia (AML) patients harboring IDH2 R140Q in culture, and decreased 2HG levels and blast percentage and improved survival relative to Cytosar-U (cytarabine) treatment in IDH2 R140Q-mutant primary AML xenograft models (PMID: 28193778).||28193778|
|PubMed Id||Reference Title||Details|
|(28193778)||AG-221, a First-in-Class Therapy Targeting Acute Myeloid Leukemia Harboring Oncogenic IDH2 Mutations.||Full reference...|
|(28588020)||Enasidenib in mutant IDH2 relapsed or refractory acute myeloid leukemia.||Full reference...|
|Idhifa (enasidenib) FDA Drug Label||Full reference...|