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Ivosidenib
Source: Acute Myeloid Leukemia Treatment (PDQ®)–Health Professional Version, National Cancer Institute.
Source updated: March 14, 2025 · Captured 2026-09-09.
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Ivosidenib is an oral small molecule inhibitor with activity against the altered IDH1 enzyme.
Evidence (ivosidenib):
In a phase I/II study, 179 patients with relapsed or refractory AML were treated with ivosidenib.[19]
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In a phase I/II study, 179 patients with relapsed or refractory AML were treated with ivosidenib.[19]
The overall response rate was 39.1% (95% CI, 31.9%–46.7%) with a median duration response of 6.5 months (range, 4.6–9.3). This included 54 patients (30.2%) with a CR or a CRi.
In a phase I/II study, 179 patients with relapsed or refractory AML were treated with ivosidenib.[19]
Over one-third of patients who initially required transfusions became transfusion independent.
In a phase I/II study, 179 patients with relapsed or refractory AML were treated with ivosidenib.[19]
Grade 3 or higher prolongation of the QT interval was seen in 14 patients (7.8%), and grade 3 or higher differentiation was seen in 7 patients (3.9%). The FDA added a boxed warning to ivosidenib, which alerts prescribers that differentiation syndrome can occur.[19][Level of evidence C3]
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Preserved source evidence · Not medical advice