Acute myeloid leukemia

New Drug: Enasidenib for IDH2 Mutant AML

International, multicenter, randomized, open-label, phase 3 trial

Age ≥60 years, IDH2 gene mutated, de novo or secondary AML

Previously 2 or 3 prior AML- directed therapies

Enasidenib 100 mg/day orally (N:158) vs CCR (azacitidine N:69; LDAC N:37; IDAC N:33; or BSC:N:22)

mOS: 6.5 m vs 6.2 m (HR:0.86; 95% CI, 0.67-1.10; P = .23)

1 year OS: 38% vs 26% (∆11%; 95% CI, 1%-22%)

mEFS:4.9 m vs 2.6 m (HR, 0.68; 95% CI, 0.52-0.91; P = .008)

All grades AE: 77% vs 61%, nausea: 22.3% vs 15.6%, blood bilirubin increased:19.7% vs 0.7%, thrombocytopenia:15.3% vs 10.6%

Grade ≥3 AEs 47% vs 35%

Serious AEs 22% vs 21%; febrile neutropenia, 2% vs 8%

Reviewed by Hasan Cagri Yildirim, MD on Sep 20, 2022

enasidenib