Summary

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• Enasidenib (Idhifa) is indicated for the treatment of adult patients with relapsed or refractory acute myeloid leukemia (AML) with an isocitrate dehydrogenase-2 (IDH2) mutation as detected by an FDA-approved test.
• Two studies focusing on evaluating the safety and efficacy of IDH inhibitors, including Enasidenib in treating AML with IDH mutations, were reviewed.
• For newly diagnosed patients, the efficacy of IDH inhibitors appears lower. However, combining these inhibitors with azacitidine showed a higher objective response in 63-74% of medically unfit patients.
• In contrast to newly diagnosed cases, relapsed or refractory AML patients show promising results when treated with Enasidenib - complete response rate at 21%, overall response rate at 40%, and median overall survival at approximately eight months.
• The most common adverse events associated with this drug are gastrointestinal, while hematologic adverse events tend to be more severe. Particular attention needs to be given to differentiation syndrome, which is a known side effect of IDH inhibitors like enasidenib.
• Both studies highlight that further high-quality randomized controlled trials are needed for a better understanding and validation regarding the effectiveness and safety profile across different patient subgroups, such as those who are newly diagnosed versus those having relapsed/refractory AML.