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Olutasidenib + azacitidine in patients with IDH1-mutated R/R AML: Pooled analysis from a phase I/II trial
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Olutasidenib, a mutant IDH1 inhibitor, is approved by the U.S. FDA for the treatment of patients with IDH1mut R/R AML. A multicenter, open-label, phase I/II trial (NCT02719574) evaluated the efficacy and safety of olutasidenib combined with azacitidine in patients with IDH1mut R/R AML. The AML Hub previously reported on the 5-year follow-up data and the impact of long-term data on treatment strategies. A pooled analysis from multiple cohorts of this trial in patients who received olutasidenib + azacitidine was published by Cortes, et al.1 in the Journal of Hematology & Oncology, reporting the efficacy and tolerability of the combination in patients with IDH1mut R/R AML (N = 67). The primary efficacy endpoint was CR/CRh rate; secondary endpoints included ORR, 56-day transfusion independence rate, time to response, DoR, and OS.1 |
| Key learnings |
| The ORR was 51% and median time to first OR was 1.95 months. The CR/CRh rate was 31%, CR rate was 27%, and median CR duration was 20.3 months. The median OS was 12.9 months, with a median OS of 30.6 months among patients achieving CR/CRh. |
| In patients without prior olutasidenib exposure (N = 51), CR/CRh rate was 37%, CR rate was 31%, and ORR was 59%. In patients who achieved CR/CRh and were transfusion-dependent at baseline, transfusion independence rates were 64% (RBCs) and 57% (platelets). |
| Grade 3/4 AEs reported in ≥5% of patients were decreased platelet count (37%), RBC count (25%), neutrophil count (24%), and febrile neutropenia (19%). SEAEs occurred in 70% of patients and treatment discontinuation due to TEAE was reported in 6%. |
| Overall, promising efficacy and safety results suggest that olutasidenib plus azacitidine could be a viable therapeutic option for patients with IDH1mut R/R AML, which is a population with limited treatment options and a poor prognosis. |
Abbreviations: AE, adverse event; AML, acute myeloid leukemia; CR, complete remission; CRh, CR with partial hematologic recovery; DoR, duration of response; OR, overall response; ORR, overall response rate; OS, overall survival; R/R, relapsed/refractory; RBC, red blood cell; SAE, serious adverse event, TEAE, treatment-emergent adverse event.
- Cortes JE, Roboz GJ, Baer MR, et al. Olutasidenib in combination with azacitidine induces durable complete remissions in patients with relapsed or refractory mIDH1 acute myeloid leukemia: a multicohort open‑label phase 1/2 trial. J Hematol Oncol. 2025;18(1):7. DOI: 10.1186/s13045-024-01657-z
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