Acute myeloid leukemia with IDH1 and IDH2 mutations: 2021 treatment algorithm

被引:119
作者
Issa, Ghayas C. [1 ]
DiNardo, Courtney D. [1 ]
机构
[1] Univ Texas MD Anderson Canc Ctr, Dept Leukemia, Houston, TX 77030 USA
关键词
MUTANT IDH2; PROGNOSTIC-SIGNIFICANCE; INHIBITOR IVOSIDENIB; AZACITIDINE; ENASIDENIB; DIFFERENTIATION; VENETOCLAX; DEPENDS; IMPACT; PLUS;
D O I
10.1038/s41408-021-00497-1
中图分类号
R73 [肿瘤学];
学科分类号
100214 ;
摘要
Acute myeloid leukemia is a genetically heterogeneous hematologic malignancy; approximately 20% of AML harbors a mutation in the isocitrate dehydrogenase (IDH) genes, IDH1 or IDH2. These recurrent mutations in key metabolic enzymes lead to the production of the oncometabolite 2-hydroxyglutarate, which promotes leukemogenesis through a block in normal myeloid differentiation. Since this discovery, selective oral inhibitors of mutant IDH1 and IDH2 have subsequently been developed and are now approved as single agent therapy, based on clinical efficacy observed within the original first-in-human trials. The investigation of IDH inhibitors in combination with standard therapies such as azacytidine, with intensive chemotherapy, and with other small molecule targeted therapies in rational combinations are currently under evaluation to further improve upon clinical efficacy.
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页数:7
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共 43 条
[1]   The role of enasidenib in the treatment of mutant IDH2 acute myeloid leukemia [J].
Abou Dalle, Iman ;
DiNardo, Courtney D. .
THERAPEUTIC ADVANCES IN HEMATOLOGY, 2018, 9 (07) :163-173
[2]   Enasidenib induces acute myeloid leukemia cell differentiation to promote clinical response [J].
Amatangelo, Michael D. ;
Quek, Lynn ;
Shih, Alan ;
Stein, Eytan M. ;
Roshal, Mikhail ;
David, Muriel D. ;
Marteyn, Benoit ;
Farnoud, Noushin Rahnamay ;
de Botton, Stephane ;
Bernard, Olivier A. ;
Wu, Bin ;
Yen, Katharine E. ;
Tallman, Martin S. ;
Papaemmanuil, Elli ;
Penard-Lacronique, Virginie ;
Thakurta, Anjan ;
Vyas, Paresh ;
Levine, Ross L. .
BLOOD, 2017, 130 (06) :732-741
[3]   Targeting IDH Mutations in AML: Wielding the Double-edged Sword of Differentiation [J].
Becker, Justin S. ;
Fathi, Amir T. .
CURRENT CANCER DRUG TARGETS, 2020, 20 (07) :490-500
[4]   Prognostic Impact of Isocitrate Dehydrogenase Enzyme Isoforms 1 and 2 Mutations in Acute Myeloid Leukemia: A Study by the Acute Leukemia French Association Group [J].
Boissel, Nicolas ;
Nibourel, Olivier ;
Renneville, Aline ;
Gardin, Claude ;
Reman, Oumedaly ;
Contentin, Nathalie ;
Bordessoule, Dominique ;
Pautas, Cecile ;
de Revel, Thierry ;
Quesnel, Bruno ;
Huchette, Pascal ;
Philippe, Nathalie ;
Geffroy, Sandrine ;
Terre, Christine ;
Thomas, Xavier ;
Castaigne, Sylvie ;
Dombret, Herve ;
Preudhomme, Claude .
JOURNAL OF CLINICAL ONCOLOGY, 2010, 28 (23) :3717-3723
[5]   Structure-Based Design and Identification of FT-2102 (Olutasidenib), a Potent Mutant-Selective IDH1 Inhibitor [J].
Caravella, Justin A. ;
Lin, Jian ;
Diebold, R. Bruce ;
Campbell, Ann-Marie ;
Ericsson, Anna ;
Gustafson, Gary ;
Wang, Zhongguo ;
Castro, Jennifer ;
Clarke, Andrea ;
Gotur, Deepali ;
Josephine, Helen R. ;
Katz, Marie ;
Kershaw, Mark ;
Yao, Lili ;
Toms, Angela, V ;
Barr, Kenneth J. ;
Dinsmore, Christopher J. ;
Walker, Duncan ;
Ashwell, Susan ;
Lu, Wei .
JOURNAL OF MEDICINAL CHEMISTRY, 2020, 63 (04) :1612-1623
[6]   Isocitrate dehydrogenase 1 and 2 mutations induce BCL-2 dependence in acute myeloid leukemia [J].
Chan, Steven M. ;
Thomas, Daniel ;
Corces-Zimmerman, M. Ryan ;
Xavy, Seethu ;
Rastogi, Suchita ;
Hong, Wan-Jen ;
Zhao, Feifei ;
Medeiros, Bruno C. ;
Tyvoll, David A. ;
Majeti, Ravindra .
NATURE MEDICINE, 2015, 21 (02) :178-184
[7]   Molecular mechanisms mediating relapse following ivosidenib monotherapy in IDH1-mutant relapsed or refractory AML [J].
Choe, Sung ;
Wang, Hongfang ;
DiNardo, Courtney D. ;
Stein, Eytan M. ;
de Botton, Stephane ;
Roboz, Gail J. ;
Altman, Jessica K. ;
Mims, Alice S. ;
Watts, Justin M. ;
Pollyea, Daniel A. ;
Fathi, Amir T. ;
Tallman, Martin S. ;
Kantarjian, Hagop M. ;
Stone, Richard M. ;
Quek, Lynn ;
Konteatis, Zenon ;
Dang, Lenny ;
Nicolay, Brandon ;
Nejad, Parham ;
Liu, Guowen ;
Zhang, Vickie ;
Liu, Hua ;
Goldwasser, Meredith ;
Liu, Wei ;
Marks, Kevin ;
Bowden, Chris ;
Biller, Scott A. ;
Attar, Eyal C. ;
Wu, Bin .
BLOOD ADVANCES, 2020, 4 (09) :1894-1905
[8]   The prognostic impact and stability of Isocitrate dehydrogenase 2 mutation in adult patients with acute myeloid leukemia [J].
Chou, W-C ;
Lei, W-C ;
Ko, B-S ;
Hou, H-A ;
Chen, C-Y ;
Tang, J-L ;
Yao, M. ;
Tsay, W. ;
Wu, S-J ;
Huang, S-Y ;
Hsu, S-C ;
Chen, Y-C ;
Chang, Y-C ;
Kuo, K-T ;
Lee, F-Y ;
Liu, M-C ;
Liu, C-W ;
Tseng, M-H ;
Huang, C-F ;
Tien, H-F .
LEUKEMIA, 2011, 25 (02) :246-253
[9]   Cancer-associated IDH1 mutations produce 2-hydroxyglutarate [J].
Dang, Lenny ;
White, David W. ;
Gross, Stefan ;
Bennett, Bryson D. ;
Bittinger, Mark A. ;
Driggers, Edward M. ;
Fantin, Valeria R. ;
Jang, Hyun Gyung ;
Jin, Shengfang ;
Keenan, Marie C. ;
Marks, Kevin M. ;
Prins, Robert M. ;
Ward, Patrick S. ;
Yen, Katharine E. ;
Liau, Linda M. ;
Rabinowitz, Joshua D. ;
Cantley, Lewis C. ;
Thompson, Craig B. ;
Heiden, Matthew G. Vander ;
Su, Shinsan M. .
NATURE, 2009, 462 (7274) :739-U52
[10]   Azacitidine and Venetoclax in Previously Untreated Acute Myeloid Leukemia [J].
DiNardo, C. D. ;
Jonas, B. A. ;
Pullarkat, V. ;
Thirman, M. J. ;
Garcia, J. S. ;
Wei, A. H. ;
Konopleva, M. ;
Doehner, H. ;
Letai, A. ;
Fenaux, P. ;
Koller, E. ;
Havelange, V. ;
Leber, B. ;
Esteve, J. ;
Wang, J. ;
Pejsa, V. ;
Hajek, R. ;
Porkka, K. ;
Illes, A. ;
Lavie, D. ;
Lemoli, R. M. ;
Yamamoto, K. ;
Yoon, S. -S. ;
Jang, J. -H. ;
Yeh, S. -P. ;
Turgut, M. ;
Hong, W. -J. ;
Zhou, Y. ;
Potluri, J. ;
Pratz, K. W. .
NEW ENGLAND JOURNAL OF MEDICINE, 2020, 383 (07) :617-629