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Ref Type
Authors Courtney D. DiNardo, Stephane De Botton, Eytan M. Stein, Gail J. Roboz, Alice S. Mims, Daniel A. Pollyea, Ronan T. Swords, Jessica K. Altman, Robert H. Collins, Gabriel N. Mannis, Geoffrey L. Uy, Will Donnellan, Arnaud Pigneux, Amir T. Fathi, et al.
Title Ivosidenib (AG-120) in Mutant IDH1 AML and Advanced Hematologic Malignancies: Results of a Phase 1 Dose Escalation and Expansion Study
Abstract Text Blood 2017 130:725


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Molecular Profile Treatment Approach
Gene Name Source Synonyms Protein Domains Gene Description Gene Role
Therapy Name Drugs Efficacy Evidence Clinical Trials
Drug Name Trade Name Synonyms Drug Classes Drug Description
Gene Variant Impact Protein Effect Variant Description Associated with drug Resistance
Molecular Profile Indication/Tumor Type Response Type Therapy Name Approval Status Evidence Type Efficacy Evidence References
IDH1 R132C acute myeloid leukemia sensitive Ivosidenib FDA approved - On Companion Diagnostic Actionable In a Phase I trial that supported FDA approval, Tibsovo (ivosidenib) treatment resulted in complete remission (CR) in 21.6% (27/125), CR with partial hematological recovery (CRh) in 8.8% (11/125), and overall response (OR) in 41.6% (52/125) of patients with relapsed or refractory acute myeloid leukemia harboring a susceptible IDH1 mutation (R132C/G/H/L/S) as detected by an FDA-approved test (PMID: 29860938; NCT02074839). detail... detail... detail... 29860938