IDH2型
异柠檬酸脱氢酶
髓系白血病
癌症研究
髓样
医学
IDH1
白血病
疾病
突变
癌症
生物
肿瘤科
内科学
酶
基因
遗传学
生物化学
作者
M.F. Amaya,Daniel A. Pollyea
标识
DOI:10.1158/1078-0432.ccr-18-0536
摘要
Abstract Acute myeloid leukemia (AML) is an aggressive disease with a poor prognosis. A large percentage of patients succumb to this disease in spite of aggressive treatments with chemotherapy. Recent advances with mutational analysis led to the discovery of isocitrate dehydrogenase (IDH) mutations in AML. IDH2 is an enzyme that catalyzes the oxidative decarboxylation of isocitrate to α-ketoglutarate; its mutated version leads to the accumulation of the oncometabolite (R)-2 hydroxyglutarate, which disrupts several cell processes and leads to a blockage in differentiation. Targeting IDH2 is compelling, as it is an early and stable mutation in AML. Enasidenib, a specific small-molecule inhibitor of IDH2, recently gained FDA approval for the treatment of patients with relapsed/refractory IDH2-mutated AML. In this review, we will focus on the indications and efficacy of enasidenib in the treatment of patients with IDH2-mutated AML. Clin Cancer Res; 24(20); 4931–6. ©2018 AACR.
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