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Ref Type Journal Article
PMID (40594997)
Authors Watts J, Nong T, Micin K, Soong D, Madarang E, Affer M, Mehra S, Lesmes JA, Chapman J, Zhou Y, Thomassen A, Bradley T, Totiger T, Swords R, Taylor J
Title Functional cure with single agent olutasidenib in relapsed IDH1/NPM1 co-mutated AML.
Journal Vol Issue Date Pages Journal Short Title
NPJ precision oncology 9 1 2025 Jul 01 211 NPJ Precis Oncol
URL
Abstract Text Olutasidenib is a potent, selective, oral, small-molecule inhibitor of mutant isocitrate dehydrogenase 1 (IDH1) that was recently approved by the US FDA for adult patients with relapsed or refractory acute myeloid leukemia (AML) harboring mutant IDH1. In the pivotal Phase II trial of olutasidenib, the median duration of complete response (CR) was 28.1 months. Here we report the first patient in the world to receive olutasidenib, for relapsed NPM1 and IDH1 co-mutated AML, who remains in continuous CR for over 7 years on olutasidenib monotherapy. We detail the clinical course as well as the pathologic and genomic evolution of the disease. Furthermore, using a novel single cell measurable residual disease assay and digital PCR and qPCR for the detection of IDH1 and NPM1 mutations, we found no evidence of residual detectable leukemia. To our knowledge, this is the first report of an AML patient functionally cured by IDH1 inhibitor monotherapy.

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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 Olutasidenib Case Reports/Case Series Actionable In a clinical case study, Rezlidhia (olutasidenib) treatment resulted in a complete response with treatment ongoing for at least 87 months in a patient with relapsed acute myeloid leukemia harboring IDH1 R132C, along with NPM1 W299Cfs*12 (PMID: 40594997). 40594997