The following is a summary of “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,” published in the January 2025 issue of Hematology by Cortes et al.
Olutasidenib is a selective, oral, small molecule inhibitor of mutant IDH1 (mIDH1), inducing durable remissions in relapsed/refractory (R/R) mIDH1 acute myeloid leukemia (AML).
Researchers conducted a retrospective study on patients with R/R AML who received combination olutasidenib and azacitidine therapy.
They received 150 mg olutasidenib twice daily plus standard-of-care azacitidine (OLU + AZA). Response and safety were evaluated in adult patients with mIDH1R132 AML.
The results showed that 67 patients with R/R mIDH1R132 AML received OLU + AZA. Median age was 66 years, and 54% were male. CR/CRh was achieved in 21/67 (31%; 95% CI 21–44) patients, with a median duration of 14.7 months. CR was achieved in 18/67 (27%; 95% CI 17–39) patients, with median duration of 20.3 months. Overall response was achieved in 34/67 (51%; 95% CI 38–63) patients. Median overall survival (OS) was 12.9 months. In the subset analysis (N=51), CR/CRh was achieved in 19/51 (37%; 95% CI 24–52) patients, CR in 16/51 (31%; 95% CI 19–46), and overall response in 30/51 (59%; 95% CI 44–72). Transfusion independence was achieved in 64% (7/11) and 57% (4/7) of transfusion-dependent patients. The most common grade 3 or 4 adverse events (AEs) were decreased platelet count (37%), red blood cell count (25%), and neutrophil count (24%). About 6 patients (9%) experienced differentiation syndrome, and 4 (6%) discontinued treatment due to AEs.
Investigators found that OLU + AZA induced high response rates and durable remissions in patients with R/R mIDH1 AML. The combination provided a tolerable side effect profile and offered a promising therapeutic option.
Source: jhoonline.biomedcentral.com/articles/10.1186/s13045-024-01657-z
