Page 37 - 2019_08-Haematologica-web

Promising targets for MRD therapies in AML
13. Papaemmanuil E, Gerstung M, Bullinger L, et al. Genomic classification and prognosis in acute myeloid leukemia. N Engl J Med. 2016;374(23):2209-2221.
14. Frohling S, Schlenk RF, Breitruck J, et al. Prognostic significance of activating FLT3 mutations in younger adults (16 to 60 years) with acute myeloid leukemia and normal cytogenetics: a study of the AML Study Group Ulm. Blood. 2002;100(13):4372-4380.
15. Nagel G, Weber D, Fromm E, et al. Epidemiological, genetic, and clinical charac- terization by age of newly diagnosed acute myeloid leukemia based on an academic population-based registry study (AMLSG BiO). Ann Hematol. 2017;96(12):1993-2003.
16. Thiede C, Steudel C, Mohr B, et al. Analysis of FLT3-activating mutations in 979 patients with acute myelogenous leukemia: associa- tion with FAB subtypes and identification of subgroups with poor prognosis. Blood. 2002;99(12):4326-4335.
17. Kottaridis PD, Gale RE, Frew ME, et al. The presence of a FLT3 internal tandem duplica- tion 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- 1759.
18. Sengsayadeth SM, Jagasia M, Engelhardt BG, et al. Allo-SCT for high-risk AML-CR1 in the molecular era: impact of FLT3/ITD outweighs the conventional markers. Bone Marrow Transplant. 2012;47(12):1535-1537.
19. Perl A, Martinelli, G, Cortes, JE, et al. Gilteritinib significantly prolongs overall sur- vival in patients with FLT3-mutated (FLT3mut+) relapsed/refractory (R/R) acute myeloid leukemia (AML): results from the phase III ADMIRAL trial Abstract #CCT184. Presented at the American Association for Cancer Research Annual Meeting 2019.
20. Levis M, Perl, AE, Altman, JK, et al. Impact of minimal residual disease and achieve- ment of complete remission/complete remission with partial hematologic recovery (CR/CRh) on overall survival following treatment with gilteritinib in patients with relapsed/refractory (R/R) acute myeloid leukemia (AML) with FLT3 mutations. Blood. 2018;132.
21. Cortes J, Perl, AE, Dohner, H, et al. Quizartinib, an FLT3 inhibitor, as monother- apy in patients with relapsed or refractory acute myeloid leukaemia: an open-label, multicentre, single-arm, phase 2 trial. Lancet Oncol. 2018;19(7):889-903.
22. Propper DJ, McDonald AC, Man A, et al. Phase I and pharmacokinetic study of PKC412, an inhibitor of protein kinase C. J Clin Oncol. 2001;19(5):1485-1492.
23. Stone RM, Mandrekar SJ, Sanford BL, et al. Midostaurin plus chemotherapy for acute myeloid leukemia with a FLT3 mutation. N Engl J Med. 2017;377(5):454-464.
24. Stone RM, Mandrekar SJ, Sanford BL, et al. The addition of midostaurin to standard chemotherapy decreases cumulative inci- dence of relapse (CIR) in the international prospective randomized, placebo-con- trolled, double-blind trial (CALGB 10603 / RATIFY [Alliance]) for newly diagnosed acute myeloid leukemia (AML) patients with FLT3 mutations. Blood. 2017;130 (Suppl 1):2580.
25. Smith CC, Lin K, Stecula A, Sali A, Shah NP. FLT3 D835 mutations confer differential
resistance to type II FLT3 inhibitors.
Leukemia. 2015;29(12):2390-2392.
26. Wang E, Tallman M, Stone R, et al. Low relapse rate in younger patients ≤ 60 years old with newly diagnosed FLT3-mutated acute myeloid leukemia (AML) treated with crenolanib and cytarabine/anthracycline chemotherapy. ASH Annual Meeting 2017.
Blood. 2017;130(Suppl 1):566-566.
27. Stone R, Collins, R, Tallman, MS, et al. . Effect of cytarabine/anthracycline/ crenolanib induction on minimal residual disease (MRD) in newly diagnosed FLT3 mutant AML. ASCO Annual Meeting 2017.
J Clin Oncol. 2017;35(15_suppl): 7016.
28. Pratz KW, Cherry M, Altman JK, et al. updated results from a phase 1 study of gilteritinib in combination with induction and consolidation chemotherapy in subjects with newly diagnosed acute myeloid leukemia (AML). ASH Annual Meeting
2018. 2018;132(Suppl 1):564.
29. Altman JK, Foran JM, Pratz KW, Trone D,
Cortes JE, Tallman MS. Phase 1 study of quizartinib in combination with induction and consolidation chemotherapy in patients with newly diagnosed acute myeloid leukemia. Am J Hematol. 2018;93(2):213- 221.
30. Medeiros BC, Fathi AT, DiNardo CD, Pollyea DA, Chan SM, Swords R. Isocitrate dehydrogenase mutations in myeloid malig- nancies. Leukemia. 2017;31(2):272-281.
31. Marcucci G, Maharry K, Wu YZ, et al. IDH1 and IDH2 gene mutations identify novel molecular subsets within de novo cytoge- netically normal acute myeloid leukemia: a Cancer and Leukemia Group B study. J Clin Oncol. 2010;28(14):2348-2355.
32. Paschka P, Schlenk RF, Gaidzik VI, 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):3636-3643.
33. Ward PS, Patel J, Wise DR, et al. The com- mon feature of leukemia-associated IDH1 and IDH2 mutations is a neomorphic enzyme activity converting alpha-ketoglu- tarate to 2-hydroxyglutarate. Cancer Cell. 2010;17(3):225-234.
34. Mardis ER, Ding L, Dooling DJ, et al. Recurring mutations found by sequencing an acute myeloid leukemia genome. N Engl J Med. 2009;361(11):1058-1066.
35. Patel KP, Ravandi F, Ma D, et al. Acute myeloid leukemia with IDH1 or IDH2 mutation: frequency and clinicopathologic features. Am J Clin Pathol. 2011;135(1):35- 45.
36. Losman JA, Looper RE, Koivunen P, et al. (R)-2-hydroxyglutarate is sufficient to pro- mote leukemogenesis and its effects are reversible. Science. 2013;339(6127):1621- 1625.
37. Kats LM, Reschke M, Taulli R, et al. Proto- oncogenic role of mutant IDH2 in leukemia initiation and maintenance. Cell Stem Cell. 2014;14(3):329-341.
38. DiNardo CD, Ravandi F, Agresta S, et al. Characteristics, clinical outcome, and prog- nostic significance of IDH mutations in AML. Am J Hematol. 2015;90(8):732-736.
39. DiNardo CD, Stein EM, de Botton S, et al. Durable remissions with ivosidenib in IDH1-mutated relapsed or refractory AML. N Engl J Med. 2018;378(25):2386-2398.
40. Stein EM, DiNardo CD, Pollyea DA, et al. Enasidenib in mutant IDH2 relapsed or
refractory acute myeloid leukemia. Blood.
2017;130(6):722-731.
41. Amatangelo MD, Quek L, Shih A, et al.
Enasidenib induces acute myeloid leukemia cell differentiation to promote clinical response. Blood. 2017;130(6):732-741.
42. Stein E, DiNardo, CD, Fathi, AT, 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 remis- sions. ASH Annual Meeting. Blood. 2018:132(Suppl 1).
43. Schlenk RF, Weber D, Fiedler W, et al. Midostaurin added to chemotherapy and continued single-agent maintenance therapy in acute myeloid leukemia with FLT3-ITD. Blood. 2019;133(8):840.
44. Mehta SV, Shukla SN, Vora HH. Overexpression of Bcl2 protein predicts chemoresistance in acute myeloid leukemia: its correlation with FLT3. Neoplasma. 2013;60(6):666-675.
45. Luedtke DA, Niu X, Pan Y, et al. Inhibition of Mcl-1 enhances cell death induced by the Bcl-2-selective inhibitor ABT-199 in acute myeloid leukemia cells. Signal Transduct Target Ther. 2017;2:17012.
46. Lagadinou ED, Sach A, Callahan K, et al. BCL-2 inhibition targets oxidative phospho- rylation and selectively eradicates quiescent human leukemia stem cells. Cell Stem Cell. 2013;12(3):329-341.
47. Pollyea DA, Stevens BM, Jones CL, et al. Venetoclax with azacitidine disrupts energy metabolism and targets leukemia stem cells in patients with acute myeloid leukemia. Nat Med. 2018;24(12):1859-1866.
48. Souers AJ, Leverson JD, Boghaert ER, et al. ABT-199, a potent and selective BCL-2 inhibitor, achieves antitumor activity while sparing platelets. Nat Med. 2013;19(2):202- 208.
49. Konopleva M, Pollyea DA, Potluri J, et al. Efficacy and biological correlates of response in a phase II study of venetoclax monother- apy in patients with acute myelogenous leukemia. Cancer Discov. 2016;6(10):1106- 1117.
50. Wei A, Strickland, SA, Roboz, GJ, et al. Phase 1/2 study of venetoclax with low- dose cytarabine in treatment-naive, elderly patients with acute myeloid leukemia unfit for intensive chemotherapy: 1-year out- comes. ASH Annual Meeting 2017. Blood. 2017;130(Suppl 1):890.
51. DiNardo CD, Pratz KW, Letai A, et al. Safety and preliminary efficacy of venetoclax with decitabine or azacitidine in elderly patients with previously untreated acute myeloid leukaemia: a non-randomised, open-label, phase 1b study. Lancet Oncol. 2018;19(2):216-228.
52. Pollyea D, Pratz, KW, Jonas, BA, et al. Venetoclax in combination with hypomethylating agents induces rapid, deep, and durable responses in patients with AML ineligible for intensive therapy ASH Annual Meeting 2018.
53. Wei A, Strickland, SA, Hou, J, et al. Venetoclax with low-dose cytarabine induces rapid, deep, and durable responses in previously untreated older adults with AML ineligible for intensive chemotherapy ASH Annual Meeting 2018.
54. DiNardo C, Albitar, M, Kadia, TM, et al. Venetoclax in combination with FLAG-IDA chemotherapy (FLAG-V-I) for fit, relapsed/refractory AML patients: interim results of a phase 1b/2 dose escalation and
haematologica | 2019; 104(8)
1529