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Enasidenib in the treatment of relapsed/refractory acute myeloid leukemia: an evidence-based review of its place in therapy

Authors Galkin M, Jonas BA

Received 23 December 2018

Accepted for publication 31 March 2019

Published 26 April 2019 Volume 2019:14 Pages 3—17

DOI https://doi.org/10.2147/CE.S172912

Checked for plagiarism Yes

Review by Single-blind

Peer reviewers approved by Dr Amy Norman

Peer reviewer comments 3

Editor who approved publication: Professor Garry Walsh


Maria Galkin,1 Brian A Jonas2

1Department of Internal Medicine, University of California Davis School of Medicine, Sacramento, CA, USA; 2Department of Internal Medicine, Division of Hematology and Oncology, University of California Davis School of Medicine, Sacramento, CA, USA

Introduction: Acute myeloid leukemia (AML) remains a disease with high mortality, especially for older patients and those with relapsed/refractory (R/R) disease. With recent advances in molecular testing, targeting particular leukemogenic mutations such as those occurring in isocitrate dehydrogenase (IDH) became possible. Enasidenib is a new small-molecule inhibitor of mutant isocitrate dehydrogenase-2 (IDH2).
Aim: The objective of this article is to review the evidence for the use of enasidenib in R/R AML, as well as to outline future directions of enasidenib therapy.
Evidence Review: Enasidenib was approved in August 2017, after a successful Phase I/II trial showing an overall response rate (ORR) of 40.3% in R/R disease, with 19.3% of patients achieving complete remission (CR). Enrollees in the trial were mostly older adults. The most prominent toxicities were hyperbilirubinemia and IDH-differentiation syndrome (IDH-DS), though the drug was generally well tolerated and the maximum tolerated dose was not reached. A Phase III trial is currently ongoing.
Conclusion: Enasidenib provides a new therapeutic option for patients with R/R AML. Further studies are ongoing to ascertain its role in combination with other agents and newly diagnosed disease.

Keywords: AML, enasidenib, IDH-2 inhibitor, Idhifa

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