Evidence for Clinical Differentiation and Differentiation Syndrome in Patients With Acute Myeloid Leukemia and IDH1 Mutations Treated With the Targeted Mutant IDH1 Inhibitor, AG-120

We describe 3 patients with relapsed/refractory acute myeloid leukemia who developed clinically-apparent differentiation concurrent with clinical response during monotherapy with AG-120, a novel, oral inhibitor of mutant isocitrate dehydrogenase 1. Symptoms included marked leukocytosis and exuberant neutrophil recovery among other clinically-apparent constitutional manifestations. Awareness of the potential for differentiation syndrome with such inhibitors, and prompt identification and intervention, are essential to facilitate clinical resolution.
Source: Clinical Lymphoma, Myeloma and Leukemia - Category: Hematology Authors: Tags: Original Study Source Type: research