Enasidenib in mutant IDH2 relapsed or refractory acute myeloid leukaemia
Enasidenib, a pill blocking the mutant IDH2 enzyme, produced responses in about four in ten patients with relapsed IDH2-mutated acute myeloid leukaemia by making the leukaemic cells mature, and became the first IDH-targeted drug approved.
Overview
Phase 1/2 study of 239 patients with IDH2-mutated advanced myeloid malignancies, including 176 with relapsed or refractory AML, treated with enasidenib, mostly at 100 mg daily.
Overall response rate was 40.3 percent with complete remission in 19.3 percent and a median overall survival of 9.3 months; responses came through differentiation of blasts rather than cytotoxicity, with differentiation syndrome in a minority.
- Overall response 40.3 percent; complete remission 19.3 percent.
- Median overall survival 9.3 months; 19.7 months in complete responders.
IDH2 mutations, present in about one in eight AML cases, became actionable. Enasidenib is an option at relapse; its role in newly diagnosed disease is less established than ivosidenib's.
- The later randomised IDHENTIFY trial did not show a survival benefit over conventional care.
- Indirect hyperbilirubinaemia and differentiation syndrome are characteristic toxicities.
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