SOHO State of the Art Update and Next Questions: IDH Therapeutic Targeting in AML

Mutations in isocitrate dehydrogenase isoform (IDH) 1 and 2 occur in approximately 25% of patients with acute myeloid leukemia (AML). These mutations lead to a block in myeloid differentiation and ultimately, to the development of AML. Inhibitors of mutant IDH1 and 2 have recently been approved by the US Food and Drug Administration and their use has led to clinical responses with prolonged duration of response. IDH inhibitors in combination with standard-of-care therapy and other small molecular inhibitors are now being used.