[Efficacy and safety of ivosidenib-based therapy in 16 patients with IDH1-mutated acute myeloid leukemia].

Abstract

To evaluate the real-world application of ivosidenib (IVO) in patients with IDH1-mutated acute myeloid leukemia (AML), as well as its efficacy and safety. We retrospectively analyzed clinical data from 16 patients with IDH1-mutated AML who received IVO-based therapy at Nanfang Hospital between September 2021 and May 2025. The median age was 57 years (range, 25 - 80 years), with 7 males and 9 females. Six patients (37.5%) had an Eastern Cooperative Oncology Group performance status of 3 - 4, and 14 patients (87.5%) were classified as intermediate- or high-risk according to the European LeukemiaNet criteria. Among 5 patients (31.2%) receiving venetoclax (VEN) plus azacitidine (AZA) combined with IVO for induction, 3 achieved complete remission (CR), including 2 with measurable residual disease (MRD) -negative and IDH1 dPCR-negative status. Two patients (12.5%) received VA plus IVO for consolidation; both maintained CR, with 1 achieving MRD-negative and IDH1 dPCR-negative status. Four relapsed or refractory patients (25.0%) received VEN plus hypomethylating agents (HMA) combined with IVO (n=3) or IVO alone (n=1) for salvage therapy. All patients achieved CR, of whom 3 obtained MRD-negative results, and 1 was IDH1 dPCR-negative. Five patients (31.2%) received IVO as maintenance therapy, with a duration of response exceeding 12 months. The most common grade ≥3 adverse events were neutropenia (56.3%), anemia (43.8%), and thrombocytopenia (37.5%). IVO-related adverse events included differentiation syndrome (12.5%) and QT interval prolongation (12.5%), and no treatment-related deaths were observed. IVO-based regimens demonstrated favorable efficacy and a manageable safety profile across all treatment phases in patients with IDH1-mutated AML, supporting their broader clinical application.