The impact of multiple low-level BCR-ABL1 mutations on response to ponatinib

Blood

Volumn 127, Issue 15, 2016, Pages 1870-1880

  Parker, Wendy T ^a,b,c   Yeung, David T O ^a,d,e   Yeoman, Alexandra L ^a   Altamura, Haley K ^a   Jamison, Bronte A ^a   Field, Chani R ^a   Hodgson, J Graeme ^f   Lustgarten, Stephanie ^f   Rivera, Victor M ^f   Hughes, Timothy P ^a,d,e   Branford, Susan ^a,c,d  

^a Centre for Cancer Biology and Department of Haematology   (Australia)

^b CENTRE FOR CANCER BIOLOGY   (Australia)

^c UNIVERSITY OF SOUTH AUSTRALIA   (Australia)

^d UNIVERSITY OF ADELAIDE   (Australia)

^e SOUTH AUSTRALIAN HEALTH AND MEDICAL RESEARCH INSTITUTE   (Australia)

^f ARIAD PHARMACEUTICALS INC   (United States)

Author keywords

[No Author keywords available]

Indexed keywords

DASATINIB; IMATINIB; NILOTINIB; PONATINIB; PROTEIN TYROSINE KINASE INHIBITOR; ANTINEOPLASTIC AGENT; BCR ABL PROTEIN; BCR-ABL1 FUSION PROTEIN, HUMAN; IMIDAZOLE DERIVATIVE; PROTEIN KINASE INHIBITOR; PYRIDAZINE DERIVATIVE;

ACUTE LYMPHOBLASTIC LEUKEMIA; ARTICLE; CHRONIC MYELOID LEUKEMIA; HUMAN; MAJOR CLINICAL STUDY; MASS SPECTROMETRY; MUTATION; OUTCOME ASSESSMENT; PRIORITY JOURNAL; TREATMENT RESPONSE; ADOLESCENT; ADULT; AGED; CLINICAL TRIAL; DNA MUTATIONAL ANALYSIS; DRUG RESISTANCE; GENETICS; LEUKEMIA, MYELOGENOUS, CHRONIC, BCR-ABL POSITIVE; MIDDLE AGED; MULTIVARIATE ANALYSIS; PHASE 2 CLINICAL TRIAL; PROGNOSIS; TREATMENT OUTCOME; VERY ELDERLY; YOUNG ADULT;

ADOLESCENT; ADULT; AGED; AGED, 80 AND OVER; ANTINEOPLASTIC AGENTS; DNA MUTATIONAL ANALYSIS; DRUG RESISTANCE, NEOPLASM; FUSION PROTEINS, BCR-ABL; HUMANS; IMIDAZOLES; LEUKEMIA, MYELOGENOUS, CHRONIC, BCR-ABL POSITIVE; MASS SPECTROMETRY; MIDDLE AGED; MULTIVARIATE ANALYSIS; MUTATION; PROGNOSIS; PROTEIN KINASE INHIBITORS; PYRIDAZINES; TREATMENT OUTCOME; YOUNG ADULT;

EID: 84959076841     PISSN: 00064971     EISSN: 15280020     Source Type: Journal    
DOI: 10.1182/blood-2015-09-666214     Document Type: Article

References (29)

1. Hughes T, Saglio G, Branford S, et al. Impact of baseline BCR-ABL mutations on response to nilotinib in patients with chronic myeloid leukemia in chronic phase. J Clin Oncol. 2009;27(25):4204-4210.
2. Müller MC, Cortes JE, Kim D-W, et al. Dasatinib treatment of chronic-phase chronic myeloid leukemia: analysis of responses according to preexisting BCR-ABL mutations. Blood. 2009;114(24):4944-4953.
3. Soverini S, Hochhaus A, Nicolini FE, et al. BCR-ABL kinase domain mutation analysis in chronic myeloid leukemia patients treated with tyrosine kinase inhibitors: recommendations from an expert panel on behalf of European LeukemiaNet. Blood. 2011;118(5):1208-1215.
4. Parker WT, Lawrence RM, Ho M, et al. Sensitive detection of BCR-ABL1 mutations in patients with chronic myeloid leukemia after imatinib resistance is predictive of outcome during subsequent therapy. J Clin Oncol. 2011;29(32):4250-4259.
5. Parker WT, Ho M, Scott HS, Hughes TP, Branford S. Poor response to second-line kinase inhibitors in chronic myeloid leukemia patients with multiple low-level mutations, irrespective of their resistance profile. Blood. 2012;119(10):2234-2238.
6. Branford S, Melo JV, Hughes TP. Selecting optimal second-line tyrosine kinase inhibitor therapy for chronic myeloid leukemia patients after imatinib failure: does the BCR-ABL mutation status really matter? Blood. 2009;114(27):5426-5435.
7. Apperley JF. Part I: mechanisms of resistance to imatinib in chronic myeloid leukaemia. Lancet Oncol. 2007;8(11):1018-1029.
8. Nicolini FE, Ibrahim AR, Soverini S, et al. The BCR-ABLT315I mutation compromises survival in chronic phase chronic myelogenous leukemia patients resistant to tyrosine kinase inhibitors, in a matched pair analysis. Haematologica. 2013;98(10):1510-1516.
9. Cortes JE, Kim DW, Pinilla-Ibarz J, et al; PACE Investigators. A phase 2 trial of ponatinib in Philadelphia chromosome-positive leukemias. N Engl J Med. 2013;369(19):1783-1796.
10. Gibbons DL, Pricl S, Posocco P, et al. Molecular dynamics reveal BCR-ABL1 polymutants as a unique mechanism of resistance to PAN-BCR-ABL1 kinase inhibitor therapy. Proc Natl Acad Sci USA. 2014;111(9):3550-3555.
11. Deininger MW, Shah NP, Cortes JE, et al. Impact of baseline (BL) mutations, including low-level and compound mutations, on ponatinib response and end of treatment (EOT) mutation analysis in patients (pts) with chronic phase chronic myeloid leukemia (CP-CML) [abstract]. Blood. 2013;122(21). Abstract 652.
12. Zabriskie MS, Eide CA, Tantravahi SK, et al. BCR-ABL1 compound mutations combining key kinase domain positions confer clinical resistance to ponatinib in Ph chromosome-positive leukemia. Cancer Cell. 2014;26(3):428-442.
13. O'Hare T, Shakespeare WC, Zhu X, et al. AP24534, a pan-BCR-ABL inhibitor for chronic myeloid leukemia, potently inhibits the T315I mutant and overcomes mutation-based resistance. Cancer Cell. 2009;16(5):401-412.
14. Cortes JE, Kantarjian H, Shah NP, et al. Ponatinib in refractory Philadelphia chromosome-positive leukemias. N Engl J Med. 2012;367(22):2075-2088.
15. Scrucca L, Santucci A, Aversa F. Regression modeling of competing risk using R: an in depth guide for clinicians. Bone Marrow Transplant. 2010;45(9):1388-1395.
16. O'Brien SG, Guilhot F, Larson RA, et al; IRIS Investigators. Imatinib compared with interferon and low-dose cytarabine for newly diagnosed chronic-phase chronic myeloid leukemia. N Engl J Med. 2003;348(11):994-1004.
17. Apperley JF, Cortes JE, Kim DW, et al. Dasatinib in the treatment of chronic myeloid leukemia in accelerated phase after imatinib failure: the START a trial. J Clin Oncol. 2009;27(21):3472-3479.
18. Talpaz M, Shah NP, Kantarjian H, et al. Dasatinib in imatinib-resistant Philadelphia chromosome-positive leukemias. N Engl J Med. 2006;354(24):2531-2541.
19. Guilhot J, Baccarani M, Clark RE, et al. Definitions, methodological and statistical issues for phase 3 clinical trials in chronic myeloid leukemia: a proposal by the European LeukemiaNet. Blood. 2012;119(25):5963-5971.
20. Branford S, Hughes T. Detection of BCR-ABL mutations and resistance to imatinib mesylate. Methods Mol Med. 2006;125:93-106.
21. Deininger M, Cortes J, Kim D, et al. Impact of baseline mutations on response to ponatinib and end of treatment mutation analysis in patients with chronic myeloid leukemia [abstract]. J Clin Oncol. 2013;31(suppl). Abstract 7001.
22. Deininger MW, Hodgson JG, Shah NP, et al. Compound mutations in BCR-ABL1 are not major drivers of primary or secondary resistance to ponatinib in CP-CML patients. Blood. 2016;127(6):703-712.
23. Parker WT, Yeoman AL, Jamison BA, et al. BCR-ABL1 kinase domain mutations may persist at very low levels for many years and lead to subsequent TKI resistance. Br J Cancer. 2013;109(6):1593-1598.
24. Mroz EA, Rocco JW. MATH, a novel measure of intratumor genetic heterogeneity, is high in poor-outcome classes of head and neck squamous cell carcinoma. Oral Oncol. 2013;49(3):211-215.
25. Park SY, Gönen M, Kim HJ, Michor F, Polyak K. Cellular and genetic diversity in the progression of in situ human breast carcinomas to an invasive phenotype. J Clin Invest. 2010;120(2):636-644.
26. Hanfstein B, Müller MC, Kreil S, et al. Dynamics of mutant BCR-ABL-positive clones after cessation of tyrosine kinase inhibitor therapy. Haematologica. 2011;96(3):360-366.
27. Gruber FXE, Lamark T, Anonli A, et al. Selecting and deselecting imatinib-resistant clones: observations made by longitudinal, quantitative monitoring of mutated BCR-ABL. Leukemia. 2005;19(12):2159-2165.
28. Mauro MJ, Cortes JE, Kim D-W, et al. Multivariate analyses of the clinical and molecular parameters associated with efficacy and safety in patients with chronic myeloid leukemia (CML) and Philadelphia chromosome-positive acute lymphoblastic leukemia (Ph+ ALL) treated with ponatinib in the PACE trial [abstract]. Blood. 2012;120(21). Abstract 3747.
29. Nicolini FE, Mauro MJ, Martinelli G, et al. Epidemiologic study on survival of chronic myeloid leukemia and Ph(+) acute lymphoblastic leukemia patients with BCR-ABL T315I mutation. Blood. 2009;114(26):5271-5278.