ABSTRACT
Introduction
Management of acute myeloid leukemia (AML) remains challenging, especially for relapsed or refractory (R/R) AML patients who display poor prognosis with conventional therapies. This underlined the need for new treatments in this population.
Areas covered
This review will focus on ivosidenib, an oral inhibitor of mutated isocitrate dehydrogenase 1 (IDH1) enzyme. Mechanisms of IDH1 mutations and their inhibition by ivosidenib will be cover, as well as clinical efficacy and safety of ivosidenib in R/R AML. Ivosidenib has been approved by the FDA for R/R AML patients with IDH1 mutation in July 2018 and for unfit AML patients with IDH1 mutation as first line treatment in May 2019.
Expert commentary
Ivosidenib induces impressive response rates in R/R AML, a population of bad prognosis. Nevertheless, primary and acquired resistances to ivosidenib have been recently described, underlining importance of the ongoing clinical trials with ivosidenib in combination with standard chemotherapy, hypomethylating agents or other targeted therapies.
Information resources
Resources are PubMed, Google scholar, ClinicalTrials.gov
Declaration of interest
S. de Botton has received research support from Agios and has served on the advisory boards of Agios and Forma therapeutics. Florence Pasquier, Claude Chahine and Christophe Marzac have no relevant affiliations or financial involvement with any organization or entity with a financial interest in or financial conflict with the subject matter or materials discussed in the manuscript. This includes employment, consultancies, honoraria, stock ownership or options, expert testimony, grants or patents received or pending, or royalties.
Reviewers disclosure
Peer reviewers on this manuscript have no relevant financial relationships or otherwise to disclose.