Differentiating the Differentiation Syndrome Associated with IDH Inhibitors in AML

Isocitrate dehydrogenase (IDH) inhibitors have clinical activity in acute myeloid leukemia, in part, by differentiating blasts to mature myeloid cells. In the largest systematic analysis to date, differentiation syndrome was seen in 19% of patients treated with IDH inhibitors. Early recognition with uniform diagnostic criteria, as utilized in acute promyelocytic leukemia, may reduce subsequent complications.