IDH1-mutated relapsed or refractory AML: current challenges and future prospects.

ABSTRACT: The prognosis of patients with relapsed or refractory acute myeloid leukemia (R/R AML) is discouraging with salvage standard approaches. Mutations of isocitrate dehydrogenase 1 (IDH1 ^mut ), present in 7-14% of AML patients, have been discovered recently, opening the door to targeted agents aiming to improve the outcomes in this setting. Several oral selective IDH1 ^mut inhibitors are under investigation, ivosidenib being the first approved for R/R AML. We performed a systematic review to analyze the clinical outcomes and safety reported with IDH1 ^mut inhibitors and other agents in adult patients with IDH1 ^mut R/R AML. Ivosidenib in monotherapy achieved complete remission (CR) of 24%, overall response of 42%, and median

SUBMITTER: Megias-Vericat JE 

PROVIDER: S-EPMC6663038 | biostudies-literature | 2019

REPOSITORIES: biostudies-literature