Enasidenib for the treatment of acute myeloid leukemia

References

• Surveillance, epidemiology, and end results (SEER) program (www.seer.cancer.gov) research data (1975-2014). Leukemia – Acute myeloid leukemia (AML), National Cancer Institute, DCCPS, Surveillance Research Program, based on the 2007-2013 SEER 18 data.
   Google Scholar
• Rowe JM, Tallman MS. How I treat acute myeloid leukemia. Blood. 2010;116:3147–3156.
   PubMed Web of Science ®Google Scholar
• Walter RB, Estey EH. Management of older or unfit patients with acute myeloid leukemia. Leukemia. 2015;29:770–775.
   PubMed Web of Science ®Google Scholar
• Kantarjian H, O’brien S, Cortes J, et al. Results of intensive chemotherapy in 998 patients age 65 years or older with acute myeloid leukemia or high-risk myelodysplastic syndrome: predictive prognostic models for outcome. Cancer. 2006;106:1090–1098.
   PubMed Web of Science ®Google Scholar
• Martin MG, Abboud CN. Induction therapy for elderly patients with acute myeloid leukemia. Blood Rev. 2008;22:311–320.
   PubMed Web of Science ®Google Scholar
• Baraldi-Junkins CA, Beck AC, Rothstein G. Hematopoiesis and cytokines. Relevance to cancer and aging. Hematol Oncol Clin North Am. 2000;14: 45–61. viii.
   PubMed Web of Science ®Google Scholar
• Ossenkoppele G, Löwenberg B. How I treat the older patient with acute myeloid leukemia. Blood. 2015;125:767–774.
   PubMed Web of Science ®Google Scholar
• Clark O, Yen K, Mellinghoff IK. Molecular pathways: isocitrate dehydrogenase mutations in cancer. Clin Cancer Res. 2016;22:1837–1842.
   PubMed Web of Science ®Google Scholar
• McKenney AS, Levine RL. Isocitrate dehydrogenase mutations in leukemia. J Clin Invest. 2013;123:3672–3677.
   PubMed Web of Science ®Google Scholar
• DiNardo CD, Jabbour E, Ravandi F, et al. IDH1 and IDH2 mutations in myelodysplastic syndromes and role in disease progression. Leukemia. 2016;30:980–984.
   PubMed Web of Science ®Google Scholar
• Medeiros BC, Fathi AT, DiNardo CD, et al. Isocitrate dehydrogenase mutations in myeloid malignancies. Leukemia. 2017;31:272–281.
   PubMed Web of Science ®Google Scholar
• Papaemmanuil E, Gerstung M, Bullinger L, et al. Genomic classification and prognosis in acute myeloid leukemia. N Engl J Med. 2016;374:2209–2221.
   PubMed Web of Science ®Google Scholar
• Yan H, Parsons DW, Jin G, et al. IDH1 and IDH2 mutations in gliomas. N Engl J Med. 2009;360:765–773.
   PubMed Web of Science ®Google Scholar
• Ward PS, Patel J, Wise DR, et al. The common feature of leukemia-associated IDH1 and IDH2 mutations is a neomorphic enzyme activity converting alpha-ketoglutarate to 2-hydroxyglutarate. Cancer Cell. 2010;17:225–234.
   PubMed Web of Science ®Google Scholar
• Showalter MR, Hatakeyama J, Cajka T, et al. Replication Study: the common feature of leukemia-associated IDH1 and IDH2 mutations is a neomorphic enzyme activity converting alpha-ketoglutarate to 2-hydroxyglutarate. Elife. 2017Jun 27;6. pii: e26030. doi: 10.7554/eLife.26030. PMID: 28653623
   Google Scholar
• Ye D, Ma S, Xiong Y, et al. R-2-hydroxyglutarate as the key effector of IDH mutations promoting oncogenesis. Cancer Cell. 2013;23:274–276.
   PubMed Web of Science ®Google Scholar
• Figueroa ME, Abdel-Wahab O, Lu C, et al. Leukemic IDH1 and IDH2 mutations result in a hypermethylation phenotype, disrupt TET2 function, and impair hematopoietic differentiation. Cancer Cell. 2010;18:553–567.
   PubMed Web of Science ®Google Scholar
• Lu C, Ward PS, Kapoor GS, et al. IDH mutation impairs histone demethylation and results in a block to cell differentiation. Nature. 2012;483:474–478.
   PubMed Web of Science ®Google Scholar
• Losman J-A, Looper R, Koivunen P, et al. (R)-2-Hydroxyglutarate is sufficient to promote leukemogenesis and its effects are reversible. Science. 2013;339:1621–1625.
   PubMed Web of Science ®Google Scholar
• Wang F, Travins J, DeLaBarre B, et al. Targeted inhibition of mutant IDH2 in leukemia cells induces cellular differentiation. Science. 2013;340:622–626.
   PubMed Web of Science ®Google Scholar
• Kats LM, Reschke M, Taulli R, et al. Proto-oncogenic role of mutant IDH2 in leukemia initiation and maintenance. Cell Stem Cell. 2014;14:329–341.
   PubMed Web of Science ®Google Scholar
• Kickingereder P, Sahm F, Radbruch A, et al. IDH mutation status is associated with a distinct hypoxia/angiogenesis transcriptome signature which is non-invasively predictable with rCBV imaging in human glioma. Sci Rep. 2015;5:16238.
   PubMed Web of Science ®Google Scholar
• Cairns RA, Mak TW. Oncogenic isocitrate dehydrogenase mutations: mechanisms, models, and clinical opportunities. Cancer Discovery. 2013;3:730–741.
   PubMed Web of Science ®Google Scholar
• Celgene. IDHIFA (enasidenib) tablets: US prescribing information. https://wwwfdagov Accessed Nov 1 2017.
   Google Scholar
• Shih AH, Shank KR, Meydan C, et al. AG-221, a small molecule mutant idh2 inhibitor, remodels the epigenetic state of IDH2-mutant cells and induces alterations in self-renewal/differentiation in IDH2-mutant AML model in vivo. Blood. 2014;124:437.
   PubMed Web of Science ®Google Scholar
• Shih AH, Meydan C, Shank K, et al. Combination targeted therapy to disrupt aberrant oncogenic signaling and reverse epigenetic dysfunction in IDH2- and TET2-mutant acute myeloid leukemia. Cancer Discovery. 2017;7:494–505.
   PubMed Web of Science ®Google Scholar
• Quivoron C, David M, Straley K, et al. AG-221, an oral, selective, first-in-class, potent IDH2-R140Q mutant inhibitor, induces differentiation in a xenotransplant model. Blood. 2014;124:3735.
   Web of Science ®Google Scholar
• Amatangelo MD, Quek L, Shih A, et al. Enasidenib induces acute myeloid leukemia cell differentiation to promote clinical response. Blood. 2017;130:732–741.
   PubMed Web of Science ®Google Scholar
• Stein EM, DiNardo CD, Pollyea DA, et al. Enasidenib in mutant IDH2 relapsed or refractory acute myeloid leukemia. Blood. 2017;130:722–731.
   PubMed Web of Science ®Google Scholar
• Agios. Agios announces initiation of phase 1/2 frontline combination study of AG-221 or AG-120 with Vidaza (azacitadine for injection) in newly diagnosed acute myeloid leukemia (AML) patients not eligible for intensive chemotherapy [media release]. http://wwwagioscom Mar 30 2017. [cited 2018 Mar 23].
   Google Scholar
• Agios. Agios announces initiation of phase 1b frontline trial of AG-221 or AG-120 in combination with intensive chemotherapy in newly diagnosed acute myeloid leukemia (AML) patients [media release]. http://wwwagioscom Dec 18 2015. [cited 2018 Mar 23].
   Google Scholar
• Montesinos P, Bergua JM, Vellenga E, et al. Differentiation syndrome in patients with acute promyelocytic leukemia treated with all-trans retinoic acid and anthracycline chemotherapy: characteristics, outcome, and prognostic factors. Blood. 2009;113:775–783.
   PubMed Web of Science ®Google Scholar
• Fathi AT, DiNardo CD, Kline I, et al. Differentiation syndrome associated with enasidenib, a selective inhibitor of mutant isocitrate dehydrogenase 2: analysis of a phase 1/2 study. JAMA Oncol. 2018 Jan 18. doi: 10.1001/jamaoncol.2017.4695. [Epub ahead of print].
   PubMedGoogle Scholar
• Tallman MS, Andersen JW, Schiffer CA, et al. All-trans-retinoic acid in acute promyelocytic leukemia. N Engl J Med. 1997;337:1021–1028.
   PubMed Web of Science ®Google Scholar
• De Botton S, Dombret H, Sanz M, et al. Incidence, clinical features, and outcome of all trans-retinoic acid syndrome in 413 cases of newly diagnosed acute promyelocytic leukemia. The European APL Group. Blood. 1998;92:2712–2718.
   PubMed Web of Science ®Google Scholar
• Roboz GJ, Rosenblat T, Arellano M, et al. International randomized phase III study of elacytarabine versus investigator choice in patients with relapsed/refractory acute myeloid leukemia. J Clin Oncol. 2014;32:1919–1926.
   PubMed Web of Science ®Google Scholar
• Koivunen P, Lee S, Duncan CG, et al. Transformation by the (R)-enantiomer of 2-hydroxyglutarate linked to EGLN activation. Nature. 2012;483:484–488.
   PubMed Web of Science ®Google Scholar