Ivosidenib in relapsed or refractory acute myeloid leukemia: a profile of its use in the USA
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Ivosidenib (Tibsovo®), a first-in-class, small molecule inhibitor of mutant isocitrate dehydrogenase 1 (IDH1), is an important new therapeutic option in the USA for treating adults with relapsed or refractory acute myeloid leukemia who have a susceptible IDH1 gene mutation. In such patients, oral ivosidenib is effective and generally well tolerated, producing encouraging rates and durations of complete remission (CR), CR or CR with partial hematologic recovery (CR/CRh), overall response, and overall survival. CR/CRh rates are consistent across most baseline characteristics. Ivosidenib also provides additional clinical benefits, including transfusion independence, improvements in hematologic variables, and deep molecular responses as measured by IDH1 mutation clearance in patients who achieve CR/CRh.
The manuscript was reviewed by: G. N. Mannis, Hematology/Blood and Marrow Transplant, Department of Medicine, University of California, San Francisco, CA, USA; A. S. Mims, Division of Hematology, Department of Medicine, Ohio State University, Columbus, OH, USA. During the peer review process, Agios Inc., the marketing-authorization holder of ivosidenib (Tibsovo®), was also offered an opportunity to provide a scientific accuracy review of their data. Changes resulting from comments received were made on the basis of scientific and editorial merit.
Compliance with ethical standards
The preparation of this review was not supported by any external funding.
Conflicts of interest
E.H. McCafferty and K.A. Lyseng-Williamson are employees of Adis/Springer, are responsible for the article content and declare no conflicts of interest.
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