Enasidenib in mutant IDH2 relapsed or refractory acute myeloid leukemia
Open accessAbstract
Key Points: Enasidenib, a selective inhibitor of mutant IDH2 enzymes, was safe and well tolerated in patients with IDH2-mutated myeloid malignancies. Enasidenib induced hematologic responses in patients with relapsed/refractory AML in this dose-escalation and expansion study.
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At least in AML, dependence on mutant IDH enzymes persists after the cancer becomes clinically apparent because mutant IDH inhibitors have therapeutic efficacy in humans.
“At least in AML, dependence on these mutant enzymes persists after the cancer becomes clinically apparent because mutant IDH inhibitors have therapeutic efficacy in humans (27).”
From Metabolic reprogramming and cancer progression (2020) · cites this paper as [27]
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- Authors
- Stein, E. M., DiNardo, C. D., Pollyea, D. A., Fathi, A. T., Roboz, G. J., Altman, J. K., Stone, R. M., DeAngelo, D. J., Levine, R. L., Flinn, I. W., Kantarjian, H. M., Collins, R., Patel, M. R., Frankel, A. E., Stein, A., Sekeres, M. A., Swords, R. T., Medeiros, B. C., Willekens, C., Vyas, P., Tosolini, A., Xu, Q., Knight, R. D., Yen, K. E., Agresta, S., de Botton, S., & Tallman, M. S.
- Affiliations
- Memorial Sloan Kettering Cancer Center (US)
- The University of Texas MD Anderson Cancer Center (US)
- University of Colorado Anschutz (US)
- Harvard University (US)
- NewYork–Presbyterian Hospital (US)
- Northwestern University (US)
- Dana-Farber Cancer Institute (US)
- Sarah Cannon (US)
- The University of Texas Southwestern Medical Center (US)
- Florida Cancer Specialists & Research Institute (US)
- City Of Hope National Medical Center (US)
- Cleveland Clinic (US)
- University of Miami (US)
- Stanford Medicine (US)
- Université Paris-Sud (FR)
- National Institute for Health and Care Research (GB)
- Celgene (United States) (US)
- Agios Pharmaceuticals (United States) (US)
- Département d’Hématologie et Département d’Innovation Thérapeutique, Gustave Roussy, Villejuif, France;
- Corresponding author
- Stein, Eytan M.
- DiNardo, Courtney D.
- Tallman, Martin S.
- ORCID
- Stein, Eytan M.
- DiNardo, Courtney D.
- Pollyea, Daniel A.
- Fathi, Amir T.
- Roboz, Gail J.
- Altman, Jessica K.
- Stone, Richard M.
- DeAngelo, Daniel J.
- Levine, Ross L.
- Flinn, Ian W.
- Kantarjian, Hagop M.
- Collins, Robert
- Patel, Manish R.
- Frankel, Arthur E.
- Stein, Anthony
- Sekeres, Mikkael A.
- Swords, Ronan T.
- Medeiros, Bruno C.
- Willekens, Christophe
- Vyas, Paresh
- Xu, Qiang
- Knight, Robert D.
- Yen, Katharine E.
- Tallman, Martin S.
- Venue
- Blood
- Publisher
- American Society of Hematology
- Published
- 2017-08-10
- Volume
- 130
- Issue
- 6
- Pages
- 722-731
- ISSN
- 0006-4971 (ISSN-L)
- 1528-0020
- PMC
- PMC5572791
- OpenAlex
- W2623089849
- Cited by
- 1,516
- References
- 35
- Citations by year
- 2026: 98 · 2025: 103 · 2024: 126 · 2023: 146 · 2022: 179 · 2021: 226 · 2020: 214 · 2019: 213 · 2018: 155 · 2017: 50 · 2016: 3
- FWCI
- 131.83
- Type
- journal article
- Language
- en
- Open access
- green
- www.ncbi.nlm.nih.gov
- Locations
- Blood (publishedVersion)
- Europe PMC (PubMed Central) (publishedVersion)
- PubMed Central (publishedVersion)
- PubMed (publishedVersion)
- Funders
- National Institutes of Health (KL2TR000461)
- Medical Research Council (MR/L008963/1)
- National Cancer Institute (P30CA016672)
- National Center for Advancing Translational Sciences
- Topics
- Health Sciences › Medicine › Hematology › Acute Myeloid Leukemia Research
- Life Sciences › Biochemistry, Genetics and Molecular Biology › Molecular Biology › Retinoids in leukemia and cellular processes
- Life Sciences › Biochemistry, Genetics and Molecular Biology › Molecular Biology › Protein Degradation and Inhibitors
- Keywords
- enasidenib, IDH2 mutation, relapsed/refractory acute myeloid leukemia, IDH inhibitors, differentiation syndrome, hyperbilirubinemia, myeloid differentiation, complete remission, overall survival, drug safety, dose escalation
- MeSH terms
- Adult, Aged, Aged, 80 and over, Aminopyridines, Antineoplastic Agents, Bone Marrow, Enzyme Inhibitors, Female, Humans, Isocitrate Dehydrogenase, Male, Middle Aged, Mutation, Neoplasm Recurrence, Local, Triazines, Leukemia, Myeloid, Acute, Maximum Tolerated Dose, Young Adult
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