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异柠檬酸脱氢酶 (IDH) 2是三羧酸循环中的重要酶, 催化异柠檬酸氧化脱羧成α-酮戊二酸 (α-KG), 突变后的IDH2能催化α-KG转化为R-2-羟基戊二酸 (R-2-HG), R-2-HG会阻止造血干细胞的分化, 引发白血病.enasidenib是一种选择性IDH2抑制剂, 通过减低R-2-HG水平, 诱导髓系祖细胞的正常分化和成熟, 从而治疗急性髓系白血病.2017年8月1日enasidenib被美国FDA批准用于治疗携带IDH2基因突变的成人复发或难治性急性髓系白血病.其常见的不良反应为胆红素升高、食欲减低、恶心、呕吐、腹泻和IDH抑制剂相关的分化综合征.“,”Isocitrate dehydrogenase-2 (IDH2) is a critical enzyme in the tricarboxylic acid cycle, and catalyzes isocitrate oxidation decarboxylation into α-ketoglutarate (α-KG).The mutated IDH2 catalyzes the conversion of α-KG to 2-hydroxyglutarate (R-2-HG) which can block hematopoietic stem cellular differentiation and induce leukemia.Enasidenib is a selective inhibitor of mutant-IDH2 and used to treat acute myeloid leukemia (AML) by reducing the level of R-2-HG and inducing cellular differentiation and maturation of myeloid progenitor.Enasidenib has been approved by the U.S.FDA on 1 August 2017 for the treatment of relapsed or refractory AML in adults with mutation of IDH2 gene.The common adverse reactions of enasidenib include elevated levels of bilirubin, decreased appetite, nausea, vomiting, diarrhea, and IDH-inhibitor-associated differentiation syndrome.