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Daniel A Pollyea, MD, MS

Arellano ML et al. A phase II, multicenter, open-label study of obatoclax mesylate in patients with previously untreated myelodysplastic syndromes with anemia or thrombocytopenia. Clin Lymphoma Myeloma Leuk 2014;14(6):534-9. Abstract

Campos L et al. High expression of bcl-2 protein in acute myeloid leukemia cells is associated with poor response to chemotherapy. Blood 1993;81(11):3091-6. Abstract

Certo M et al. Mitochondria primed by death signals determine cellular addiction to antiapoptotic BCL-2 family members. Cancer Cell 2006;9(5):351-65. Abstract

Cleary ML et al. Clustering of extensive somatic mutations in the variable region of an immunoglobulin heavy chain gene from a human B cell lymphoma. Cell 1986;44(1):97-106. Abstract

Cortes JE et al. Randomized comparison of low dose cytarabine with or without glasdegib in patients with newly diagnosed acute myeloid leukemia or high-risk myelodysplastic syndrome. Leukemia 2019;33(2):379-89. Abstract

Dai H et al. BCL-2 family, mitochondrial apoptosis, and beyond. Cancer Transl Med 2016;2(1):7-20. Abstract

DiNardo CD et al. Venetoclax combined with decitabine or azacitidine in treatment-naive, elderly patients with acute myeloid leukemia. Blood 2019;133(1):7-17. Abstract

Jones CL et al. Inhibition of amino acid metabolism selectively targets human leukemia stem cells. Cancer Cell 2018;34(5):724-40. Abstract

Karakas T et al. High expression of bcl-2 mRNA as a determinant of poor prognosis in acute myeloid leukemia. Ann Oncol 1998;9(2):159-65. Abstract

Lauria F et al. High bcl-2 expression in acute myeloid leukemia cells correlates with CD34 positivity and complete remission rate. Leukemia 1997;11(12):2075-8. Abstract

Marcucci G et al. High expression levels of the ETS-related gene, ERG, predict adverse outcome and improve molecular risk-based classification of cytogenetically normal acute myeloid leukemia: A Cancer and Leukemia Group B study. J Clin Oncol 2007;25(22):3337-43. Abstract

Marcucci G et al. Phase I study of oblimersen sodium, an antisense to Bcl-2, in untreated older patients with acute myeloid leukemia: Pharmacokinetics, pharmacodynamics, and clinical activity. J Clin Oncol 2005;23(15):3404-11. Abstract

Marcucci G et al. Phase 1 and pharmacodynamic studies of 3139, a Bcl-2 antisense oligonucleotide, in combination with chemotherapy in refractory or relapsed acute leukemia. Blood 2003;101(2):425-32. Abstract

Moore J et al. A phase II study of Bcl-2 antisense (oblimersen sodium) combined with gemtuzumab ozogamicin in older patients with acute myeloid leukemia in first relapse. Leuk Res 2006;30(7):777-83. Abstract

Pollyea DA et al. Venetoclax in combination with hypomethylating agents induces rapid, deep, and durable responses in patients with AML ineligible for intensive therapy. Proc ASH 2018;Abstract 285. Abstract

Pollyea DA et al. Venetoclax with azacitidine disrupts energy metabolism and targets leukemia stem cells in patients with acute myeloid leukemia. Nat Med 2018;24(12):1859-66. Abstract

Schimmer AD et al. A multicenter phase I/II study of obatoclax mesylate administered as a 3- or 24-hour infusion in older patients with previously untreated acute myeloid leukemia. PLoS One 2014;9(10):e108694. Abstract

Schimmer AD et al. A phase I study of the pan bcl-2 family inhibitor obatoclax mesylate in patients with advanced hematologic malignancies. Clin Cancer Res 2008;14(24):8295-301. Abstract

Tam CS et al. Progress in BCL2 inhibition for patients with chronic lymphocytic leukemia. Semin Oncol 2016;43(2):274-9. Abstract

Tsujimoto Y et al. Involvement of the bcl-2 gene in human follicular lymphoma. Science 1985;228(4706):1440-3. Abstract

Wei AH et al. Venetoclax combined with low-dose cytarabine for previously untreated patients with acute myeloid leukemia: Results from a phase Ib/II study. J Clin Oncol 2019;37(15):1277-84. Abstract

Weitzman JB. Agonizing hedgehog. J Biol 2002;1(2):7. Abstract

Richard M Stone, MD

Boddu P et al. Co-occurrence of FLT3-TKD and NPM1 mutations defines a highly favorable prognostic AML group. Blood Adv 2017;1(19):1546-50. Abstract

Breitenbuecher F et al. Identification of a novel type of ITD mutations located in nonjuxtamembrane domains of the FLT3 tyrosine kinase receptor. Blood 2009;113(17):4074-7. Abstract

Cortes JE et al. Phase 2b study of 2 dosing regimens of quizartinib monotherapy in FLT3-ITD-mutated, relapsed or refractory AML. Blood 2018;132(6):598-607. Abstract

Cortes J et al. Quizartinib significantly prolongs overall survival in patients with FLT-internal tandem duplication-mutated (MUT) relapsed/refractory AML in the phase 3, randomized, controlled QUANTUM-R trial. EHA 2018;Abstract LB2600.

Cortes JE et al. Phase I study of quizartinib administered daily to patients with relapsed or refractory acute myeloid leukemia irrespective of FMS-like tyrosine kinase 3-internal tandem duplication status. J Clin Oncol 2013;31(29):3681-7. Abstract

Gale RE et al. The impact of FLT3 internal tandem duplication mutant level, number, size, and interaction with NPM1 mutations in a large cohort of young adult patients with acute myeloid leukemia. Blood 2008;111(5):2776-84. Abstract

Kottaridis PD et al. The presence of a FLT3 internal tandem duplication in patients with acute myeloid leukemia (AML) adds important prognostic information to cytogenetic risk group and response to the first cycle of chemotherapy: Analysis of 854 patients from the United Kingdom Medical Research Council AML 10 and 12 trials. Blood 2001;98(6):1752-9. Abstract

Nakao M et al. Internal tandem duplication of the flt3 gene found in acute myeloid leukemia. Leukemia 1996;10(12):1911-8. Abstract

Perl AE et al. Gilteritinib or chemotherapy for relapsed or refractory FLT3-mutated AML. N Engl J Med 2019;381(18):1728-40. Abstract

Schlenk RF et al. Differential impact of allelic ratio and insertion site in FLT3-ITD-positive AML with respect to allogeneic transplantation. Blood 2014;124(23):3441-9. Abstract

Slovak ML et al. A retrospective study of 69 patients with t(6;9)(p23;q34) AML emphasizes the need for a prospective, multicenter initiative for rare ‘poor prognosis’ myeloid malignancies. Leukemia 2006;20:1295-1297. Abstract

Staudt D et al. Targeting oncogenic signaling in mutant FLT3 acute myeloid leukemia: The path to least resistance. Int J Mol Sci 2018;19(10). Abstract

Stone RM et al. Midostaurin plus chemotherapy for acute myeloid leukemia with a FLT3 mutation. N Engl J Med 2017;377(5):454-64. Abstract

Thiede C et al. Analysis of FLT3-activating mutations in 979 patients with acute myelogenous leukemia: Association with FAB subtypes and identification of subgroups with poor prognosis. Blood 2002;99(12):4326-35. Abstract

Whitman SP et al. Absence of the wild-type allele predicts poor prognosis in adult de novo acute myeloid leukemia with normal cytogenetics and the internal tandem duplication of FLT3: A Cancer and Leukemia Group B study. Cancer Res 2001;61(19):7233-9. Abstract

Mark Levis, MD, PhD

Abbas S et al. Acquired mutations in the genes encoding IDH1 and IDH2 both are recurrent aberrations in acute myeloid leukemia: Prevalence and prognostic value. Blood 2010;116(12):2122-6. Abstract

Birendra KC, DiNardo CD. Evidence for clinical differentiation and differentiation syndrome in patients with acute myeloid leukemia and IDH1 mutations treated with the targeted mutant IDH1 inhibitor, AG-120. Clin Lymphoma Myeloma Leuk 2016;16(8):460-5. Abstract

Bledea R et al. Functional and topographic effects on DNA methylation in IDH1/2 mutant cancers. Sci Rep 2019;9(1):16830. Abstract

Chen J-Y et al. The oncometabolite R-2-hydroxyglutarate activates NF-κB-dependent tumor-promoting stromal niche for acute myeloid leukemia cells. Sci Rep 2016;6:32428. Abstract

Chou W-C et al. Distinct clinical and biologic characteristics in adult acute myeloid leukemia bearing the isocitrate dehydrogenase 1 mutation. Blood 2010;115(14):2749-54. Abstract

Daigle SR et al. High rate of IDH1 mutation clearance and measurable residual disease negativity in patients with IDH1-mutant newly diagnosed acute myeloid leukemia treated with ivosidenib (AG-120) and azacitidine. Proc ASH 2019;Abstract 2706.

Dang L et al. Cancer-associated IDH1 mutations produce 2-hydroxyglutarate. Nature 2009;462(7274):739-44. Abstract

DiNardo CD et al. Enasidenib plus azacitidine significantly improves complete remission and overall response compared with azacitidine alone in patients with newly diagnosed acute myeloid leukemia (AML) with isocitrate dehydrogenase 2 (IDH2) mutations: Interim phase II results from an ongoing, randomized study. Proc ASH 2019;Abstract 643.

DiNardo CD et al. Durable remissions with ivosidenib in IDH1-mutated relapsed or refractory AML. N Engl J Med 2018;378(25):2386-98. Abstract

Fathi AT 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;4(8):1106-10. Abstract

Fathi AT et al. Prospective serial evaluation of 2-hydroxyglutarate, during treatment of newly diagnosed acute myeloid leukemia, to assess disease activity and therapeutic response. Blood 2012;120(23):4649-52. Abstract

Harding JJ et al. Isoform switching as a mechanism of acquired resistance to mutant isocitrate dehydrogenase inhibition. Cancer Discov 2018;8(12):1540-7. Abstract

Intlekofer AM et al. Acquired resistance to IDH inhibition through trans or cis dimer-interface mutations. Nature 2018;559(7712):125-9. Abstract

Losman J-A et al. (R)-2-hydroxyglutarate is sufficient to promote leukemogenesis and its effects are reversible. Science 2013;339(6127):1621-5. Abstract

Ma R, Yun C-H. Crystal structures of pan-IDH inhibitor AG-881 in complex with mutant human IDH1 and IDH2. Biochem Biophys Res Commun 2018;503(4):2912-7. Abstract

Mardis ER et al. Recurring mutations found by sequencing an acute myeloid leukemia genome. N Engl J Med 2009;361(11):1058-66. Abstract

McMahon CM et al. Clonal selection with RAS pathway activation mediates secondary clinical resistance to selective FLT3 inhibition in acute myeloid leukemia. Cancer Discov 2019;9(8):1050-63. Abstract

Parsons DW et al. An integrated genomic analysis of human glioblastoma multiforme. Science 2008;321(5897):1807-12. Abstract

Paschka P et al. IDH1 and IDH2 mutations are frequent genetic alterations in acute myeloid leukemia and confer adverse prognosis in cytogenetically normal acute myeloid leukemia with NPM1 mutation without FLT3 internal tandem duplication. J Clin Oncol 2010;28(22):3636-43. Abstract

Schnittger S et al. IDH1 mutations are detected in 6.6% of 1414 AML patients and are associated with intermediate risk karyotype and unfavorable prognosis in adults younger than 60 years and unmutated NPM1 status. Blood 2010;116(25):5486-96. Abstract

Stein EM et al. Molecular remission and response patterns in patients with mutant-IDH2 acute myeloid leukemia treated with enasidenib. Blood 2019;133(7):676-87. Abstract

Stein EM et al. Ivosidenib or enasidenib combined with induction and consolidation chemotherapy in patients with newly diagnosed AML with an IDH1 or IDH2 mutation is safe, effective, and leads to MRD-negative complete remissions. Proc ASH 2018;Abstract 560.

Stein EM et al. Enasidenib in mutant IDH2 relapsed or refractory acute myeloid leukemia. Blood 2017;130(6):722-31. Abstract

Thol F et al. Prognostic impact of IDH2 mutations in cytogenetically normal acute myeloid leukemia. Blood 2010;116(4):614-6. Abstract

Xu Q et al. Correlation between isocitrate dehydrogenase gene aberrations and prognosis of patients with acute myeloid leukemia: A systematic review and meta-analysis. Clin Cancer Res 2017;23(15):4511-22. Abstract

Xu W et al. Oncometabolite 2-hydroxyglutarate is a competitive inhibitor of α-ketoglutarate-dependent dioxygenases. Cancer Cell 2011;19:17-30. Abstract

Yamaguchi S et al. IDH1 and IDH2 mutations confer an adverse effect in patients with acute myeloid leukemia lacking the NPM1 mutation. Eur J Haematol 2014;92(6):471-7. Abstract

Andrew H Wei, MBBS, PhD

Benitez L et al. Multi-center retrospective evaluation of high-dose cytarabine based induction versus CPX-351 induction in patients with secondary AML. Proc ASH 2019;Abstract 2639.

Borate U et al. Phase Ib study of the anti-TIM-3 antibody MBG453 in combination with decitabine in patients with high-risk myelodysplastic syndrome (MDS) and acute myeloid leukemia (AML). Proc ASH 2019;Abstract 570.

Brune M et al. Improved leukemia-free survival after postconsolidation immunotherapy with histamine dihydrochloride and interleukin-2 in acute myeloid leukemia: Results of a randomized phase 3 trial. Blood 2006;108:88-96. Abstract

Burnett AK. Identification of patients with acute myeloblastic leukemia who benefit from the addition of gemtuzumab ozogamicin: Results of the MRC AML15 trial. J Clin Oncol 2011;29(4):369-77. Abstract

Castaigne S et al. Effect of gemtuzumab ozogamicin on survival of adult patients with de-novo acute myeloid leukaemia (ALFA-0701): A randomised, open-label, phase 3 study. Lancet 2012;379(9825):1508-16. Abstract

Cluzeau T et al. APR-246 combined with azacitidine (AZA) in TP53 mutated myelodysplastic syndrome (MDS) and acute myeloid leukemia (AML). A phase 2 study by the Groupe Francophone Des Myélodysplasies (GFM). Proc ASH 2019;Abstract 677.

Daver NG et al. Azacitidine (AZA) with nivolumab (Nivo), and AZA with nivo + ipilimumab (Ipi) in relapsed/refractory acute myeloid leukemia: A non-randomized, prospective, phase 2 study. Proc ASH 2019;Abstract 830.

DiNardo CD et al. Venetoclax combined with decitabine or azacitidine in treatment-naive, elderly patients with acute myeloid leukemia. Blood 2019;133(1):7-17. Abstract

Hills RK et al. Addition of gemtuzumab ozogamicin to induction chemotherapy in adult patients with acute myeloid leukaemia: A meta-analysis of individual patient data from randomised controlled trials. Lancet Oncol 2014;15(9):986-96. Abstract

Huls G et al. Azacitidine maintenance after intensive chemotherapy improves DFS in older AML patients. Blood 2019;133(13):1457-64. Abstract

Lancet JE et al. CPX-351 (cytarabine and daunorubicin) liposome for injection versus conventional cytarabine plus daunorubicin in older patients with newly diagnosed secondary acute myeloid leukemia. J Clin Oncol 2018 36(26):2684-92. Abstract

Pigneux A et al. Addition of androgens improves survival in elderly patients with acute myeloid leukemia: A GOELAMS study. J Clin Oncol 2017;35(4):387-93. Abstract

Ramos Perez JM et al. Liposomal cytarabine and daunorubicin (CPX-351) in combination with gemtuzumab ozogamicin (GO) in relapsed/refractory (R/R) patients with acute myeloid leukemia (AML) and post-hypomethylating agent (Post-HMA) failure high-risk myelodysplastic syndrome (HR-MDS). Proc ASH 2019;Abstract 2642.

Sallman DA et al. Phase 2 results of APR-246 and azacitidine (AZA) in patients with TP53 mutant myelodysplastic syndromes (MDS) and oligoblastic acute myeloid leukemia (AML). Proc ASH 2019;Abstract 676.

Sallman DA et al. The first-in-class anti-CD47 antibody Hu5F9-G4 is active and well tolerated alone or with azacitidine in AML and MDS patients: Initial phase 1b results. ASCO 2019;Abstract 7009.

Sallman DA et al. The first-in-class anti-CD47 antibody magrolimab (5F9) in combination with azacitidine is effective in MDS and AML patients: Ongoing phase 1b results. Proc ASH 2019;Abstract 569.

Short NJ et al. Treatment with a 5-day versus a 10-day schedule of decitabine in older patients with newly diagnosed acute myeloid leukemia: A randomized phase 2 trial. Lancet Haematol 2019;6(1):e29-37. Abstract

Subklewe M et al. Preliminary results from a phase 1 first-in-human study of AMG 673, a novel half-life extended (HLE) anti-CD33/CD3 BiTE® (bispecific T-cell engager) in patients with relapsed/refractory (R/R) acute myeloid leukemia (AML). Proc ASH 2019;Abstract 833.

Uy GL et al. Flotetuzumab, an investigational CD123 x CD3 bispecific Dart® protein, in salvage therapy for primary refractory and early relapsed acute myeloid leukemia (AML) patients. Proc ASH 2019;Abstract 733.

Vey N et al. Randomized phase 2 trial of lirilumab (anti-KIR monoclonal antibody, mAb) as maintenance treatment in elderly patients (pts) with acute myeloid leukemia (AML): Results of the Effikir trial. Proc ASH 2017;130(1):889. Abstract

Wei AH et al. The QUAZAR AML-001 maintenance trial: Results of a phase III international, randomized, double-blind, placebo-controlled study of CC-486 (oral formulation of azacitidine) in patients with acute myeloid leukemia (AML) in first remission. Proc ASH 2019;Abstract LBA-3.

Wei AH et al. Venetoclax combined with low-dose cytarabine for previously untreated patients with acute myeloid leukemia: Results from a phase Ib/II study. J Clin Oncol 2019;37(15):1277-84. Abstract

Welch JS et al. TP53 and decitabine in acute myeloid leukemia and myelodysplastic syndromes. N Engl J Med 2016;375(21):2023-36. Abstract

Westervelt P et al. Phase 1 first-in-human trial of AMV564, a bivalent bispecific (2:2) CD33/CD3 T-cell engager, in patients with relapsed/refractory acute myeloid leukemia (AML). Proc ASH 2019;Abstract 834.

Zeidan AM et al. Efficacy and safety of azacitidine (AZA) in combination with the Anti-PD-L1 durvalumab (durva) for the front-line treatment of older patients (pts) with acute myeloid leukemia (AML) who are unfit for intensive chemotherapy (IC) and pts with higher-risk myelodysplastic syndromes (HR-MDS): Results from a large, international, randomized phase 2 study. Proc ASH 2019;Abstract 829.

Zeidner JF et al. Final clinical results of a phase II study of high dose cytarabine followed by pembrolizumab in relapsed/refractory AML. Proc ASH 2019;Abstract 831.