Predicting the response of CML patients to tyrosine kinase inhibitor therapy

Current Hematologic Malignancy Reports

Volumn 6, Issue 2, 2011, Pages 88-95

  White, Deborah L ^a   Hughes, Timothy P ^a  

^a Haematology Department   (Australia)

Author keywords

Chronic myelogenous leukemia; Drug resistance; Front line therapy; Imatinib; Tyrosine kinase inhibitors

Indexed keywords

DASATINIB; IMATINIB; NILOTINIB; ORGANIC CATION TRANSPORTER 1;

ARTICLE; CHRONIC MYELOID LEUKEMIA; DRUG BLOOD LEVEL; DRUG EFFICACY; DRUG INTERACTION; DRUG WITHDRAWAL; HUMAN; MICROARRAY ANALYSIS; PHASE 3 CLINICAL TRIAL (TOPIC); PROTEIN POLYMORPHISM; SINGLE NUCLEOTIDE POLYMORPHISM;

FUSION PROTEINS, BCR-ABL; HUMANS; LEUKEMIA, MYELOID, CHRONIC-PHASE; ORGANIC CATION TRANSPORTER 1; P-GLYCOPROTEIN; PIPERAZINES; POLYMORPHISM, SINGLE NUCLEOTIDE; PROGNOSIS; PROTEIN KINASE INHIBITORS; PYRIMIDINES; THIAZOLES;

EID: 79957523077     PISSN: 15588211     EISSN: 1558822X     Source Type: Journal    
DOI: 10.1007/s11899-011-0087-9     Document Type: Article

References (50)

1. Deininger M, O'Brien SG, Guilhot F, et al. International Randomized Study of Interferon Vs STI571 (IRIS) 8-Year Follow up: Sustained Survival and Low Risk for Progression or Events in Patients with Newly Diagnosed Chronic Myeloid Leukemia in Chronic Phase (CML-CP) Treated with Imatinib. Blood (ASH Annual Meeting Abstracts). 2009; 114: Abstract 1126.
2. N Heisterkamp JR Stephenson J Groffen, et al. 1983 Localization of the c-ab1 oncogene adjacent to a translocation break point in chronic myelocytic leukaemia Nature 306 239 42 6316147 10.1038/306239a0 1:CAS:528: DyaL2cXhvVKrsQ%3D%3D (Pubitemid 14232351)
3. GQ Daley RA Van Etten D Baltimore 1990 Induction of chronic myelogenous leukemia in mice by the P210bcr/abl gene of the Philadelphia chromosome Science 247 824 30 2406902 10.1126/science.2406902 1:CAS:528:DyaK3cXhsFaqs7c%3D
4. E Buchdunger J Zimmermann H Mett, et al. 1996 Inhibition of the Abl protein-tyrosine kinase in vitro and in vivo by a 2-phenylaminopyrimidine derivative Cancer Res 56 100 4 8548747 1:CAS:528:DyaK28Xit1Chsg%3D%3D
5. M Baccarani G Saglio J Goldman, et al. 2006 Evolving concepts in the management of chronic myeloid leukemia: recommendations from an expert panel on behalf of the European LeukemiaNet Blood 108 1809 20 16709930 10.1182/blood-2006-02-005686 1:CAS:528:DC%2BD28Xpsl2isrc%3D (Pubitemid 44394987)
6. Hughes T, Hochhaus A, Saglio G, et al. ENESTnd Update: Continued Superiority of Nilotinib Versus Imatinib In Patients with Newly Diagnosed Chronic Myeloid Leukemia In Chronic Phase (CML-CP). Blood (ASH Annual Meeting Abstracts). 2010; 116: Abstract 207.
7. G Saglio DW Kim S Issaragrisil, et al. 2010 Nilotinib versus imatinib for newly diagnosed chronic myeloid leukemia N Engl J Med 362 2251 9 20525993 10.1056/NEJMoa0912614 1:CAS:528:DC%2BC3cXnvVaktbk%3D
8. H Kantarjian NP Shah A Hochhaus, et al. 2010 Dasatinib versus imatinib in newly diagnosed chronic-phase chronic myeloid leukemia N Engl J Med 362 2260 70 20525995 10.1056/NEJMoa1002315 1:CAS:528:DC%2BC3cXnvVaktbY%3D
9. DL White TP Hughes 2009 Predicting the response of CML patients to tyrosine kinase inhibitor therapy Curr Hematol Malig Rep 4 59 65 20425416 10.1007/s11899-009-0009-2
10. JE Sokal EB Cox M Baccarani, et al. 1984 Prognostic discrimination in "good-risk" chronic granulocytic leukemia Blood 63 789 99 6584184 1:STN:280:DyaL2c7lsFSgtQ%3D%3D (Pubitemid 14141296)
11. J Hasford M Pfirrmann R Hehlmann, et al. 1998 A new prognostic score for survival of patients with chronic myeloid leukemia treated with interferon alfa. Writing Committee for the Collaborative CML Prognostic Factors Project Group J Natl Cancer Inst 90 850 8 9625174 10.1093/jnci/90.11.850 1:STN:280: DyaK1c3os1ygug%3D%3D (Pubitemid 28280552)
12. Hasford J, Rosti G, Hoffman V, et al. Only a high relative risk (Sokal or Euro) predicts for response to imatinib: An European LeukemiaNet Eutos study. Presented at the 15th Congress of the European Hematology Association. Barcelona, Spain; June10-13, 2010.
13. M Baccarani J Cortes F Pane, et al. 2009 Chronic myeloid leukemia: an update of concepts and management recommendations of European LeukemiaNet J Clin Oncol 27 6041 51 19884523 10.1200/JCO.2009.25.0779 1:CAS:528: DC%2BC3cXhtVWisLY%3D
14. J Thomas L Wang RE Clark M Pirmohamed 2004 Active transport of imatinib into and out of cells: implications for drug resistance Blood 104 3739 45 15315971 10.1182/blood-2003-12-4276 1:CAS:528:DC%2BD2cXhtVGltb3M (Pubitemid 39564452)
15. L Crossman B Druker M Deininger 2005 hOCT 1 and resistance to imatinib Blood 106 1133 4 16033955 10.1182/blood-2005-02-0694 1:CAS:528: DC%2BD2MXntVSmsL4%3D (Pubitemid 41076465)
16. L Wang A Giannoudis S Lane P Williamson M Pirmohamed RE Clark 2008 Expression of the uptake drug transporter hOCT1 is an important clinical determinant of the response to imatinib in chronic myeloid leukemia Clin Pharmacol Ther 83 258 64 17568400 10.1038/sj.clpt.6100268 1:CAS:528: DC%2BD1cXitFKjtb4%3D
17. D Marin A Bazeos FX Mahon, et al. 2010 Adherence is the critical factor for achieving molecular responses in patients with chronic myeloid leukemia who achieve complete cytogenetic responses on imatinib J Clin Oncol 28 2381 8 20385986 10.1200/JCO.2009.26.3087 1:CAS:528:DC%2BC3cXnsFOntrg%3D This is the first description of a detailed analysis of adherence in imatinib-treated patients. It demonstrates that adherence is a significant problem in CP-CML patients, and that failure to adhere significantly affects response
18. TP Hughes S Branford DL White, et al. 2008 Impact of early dose intensity on cytogenetic and molecular responses in chronic- phase CML patients receiving 600 mg/day of imatinib as initial therapy Blood 112 3965 73 18768781 10.1182/blood-2008-06-161737 1:CAS:528:DC%2BD1cXhsVSmsrzK
19. DL White P Dang J Engler, et al. 2010 Functional activity of the OCT-1 protein is predictive of long-term outcome in patients with chronic-phase chronic myeloid leukemia treated with imatinib J Clin Oncol 28 2761 7 20421539 10.1200/JCO.2009.26.5819 1:CAS:528:DC%2BC3cXoslyktbc%3D This paper describes an assay that is performed at diagnosis in CP-CML and is predictive of long-term response and survival. To our knowledge, this is the first assay described to do this
20. WW Zhang JE Cortes H Yao, et al. 2009 Predictors of primary imatinib resistance in chronic myelogenous leukemia are distinct from those in secondary imatinib resistance J Clin Oncol 27 3642 9 19506164 10.1200/JCO.2008.19.4076 1:CAS:528:DC%2BD1MXhtVOnu7bM
21. R Kerb U Brinkmann N Chatskaia, et al. 2002 Identification of genetic variations of the human organic cation transporter hOCT1 and their functional consequences Pharmacogenetics 12 591 5 12439218 10.1097/00008571-200211000-00002 1:CAS:528:DC%2BD38XovVeitLg%3D (Pubitemid 36005667)
22. DH Kim L Sriharsha W Xu, et al. 2009 Clinical relevance of a pharmacogenetic approach using multiple candidate genes to predict response and resistance to imatinib therapy in chronic myeloid leukemia Clin Cancer Res 15 4750 8 19584153 10.1158/1078-0432.CCR-09-0145 1:CAS:528:DC%2BD1MXosV2ltbg%3D
23. A Bazeos D Marin AG Reid, et al. 2010 hOCT1 transcript levels and single nucleotide polymorphisms as predictive factors for response to imatinib in chronic myeloid leukemia Leukemia 24 1243 5 20445576 10.1038/leu.2010.86 1:STN:280:DC%2BC3cvotlOgtQ%3D%3D
24. DL White VA Saunders P Dang J Engler TP Hughes 2010 OCT-1 activity measurement provides a superior imatinib response predictor than screening for single-nucleotide polymorphisms of OCT-1 Leukemia 24 1962 5 20811406 10.1038/leu.2010.188 1:STN:280:DC%2BC3cblvVOqtg%3D%3D
25. DL White VA Saunders P Dang, et al. 2006 OCT-1-mediated influx is a key determinant of the intracellular uptake of imatinib but not nilotinib (AMN107): reduced OCT-1 activity is the cause of low in vitro sensitivity to imatinib Blood 108 697 704 16597591 10.1182/blood-2005-11-4687 1:CAS:528: DC%2BD28XntFehur0%3D (Pubitemid 44061372)
26. DL White VA Saunders P Dang, et al. 2007 Most CML patients who have a suboptimal response to imatinib have low OCT-1 activity: higher doses of imatinib may overcome the negative impact of low OCT-1 activity Blood 110 4064 72 17761829 10.1182/blood-2007-06-093617 1:CAS:528:DC%2BD2sXhtl2qsbzM (Pubitemid 350248463)
27. X Jiang Y Zhao C Smith, et al. 2007 Chronic myeloid leukemia stem cells possess multiple unique features of resistance to BCR-ABL targeted therapies Leukemia 21 926 35 17330101 1:CAS:528:DC%2BD2sXksFeltL4%3D (Pubitemid 46672068)
28. JR Engler A Frede VA Saunders AC Zannettino TP Hughes DL White 2010 Chronic myeloid leukemia CD34+ cells have reduced uptake of imatinib due to low OCT-1 activity Leukemia 24 765 70 20147974 10.1038/leu.2010.16 1:CAS:528:DC%2BC3cXks1Klsrw%3D
29. FX Mahon F Belloc V Lagarde, et al. 2003 MDR1 gene overexpression confers resistance to imatinib mesylate in leukemia cell line models Blood 101 2368 73 12609962 10.1182/blood.V101.6.2368 1:CAS:528:DC%2BD3sXit1egt78%3D (Pubitemid 36304670)
30. N Widmer S Colombo T Buclin LA Decosterd 2003 Functional consequence of MDR1 expression on imatinib intracellular concentrations Blood 102 1142 12869489 10.1182/blood-2003-03-0993 1:CAS:528:DC%2BD3sXlvFCmtb8%3D (Pubitemid 36917818)
31. DL Kroetz C Pauli-Magnus LM Hodges, et al. 2003 Sequence diversity and haplotype structure in the human ABCB1 (MDR1, multidrug resistance transporter) gene Pharmacogenetics 13 481 94 12893986 10.1097/00008571-200308000-00006 1:CAS:528:DC%2BD3sXlvV2jt7Y%3D (Pubitemid 37034590)
32. D Wang AD Johnson AC Papp DL Kroetz W Sadee 2005 Multidrug resistance polypeptide 1 (MDR1, ABCB1) variant 3435 C∈>∈T affects mRNA stability Pharmacogenet Genomics 15 693 704 16141795 10.1097/01.fpc.0000178311. 02878.83 1:CAS:528:DC%2BD2MXpslahtb4%3D (Pubitemid 41345321)
33. H Gurney M Wong RL Balleine, et al. 2007 Imatinib disposition and ABCB1 (MDR1, P-glycoprotein) genotype Clin Pharmacol Ther 82 33 40 17495881 10.1038/sj.clpt.6100201 1:CAS:528:DC%2BD2sXotVGju78%3D (Pubitemid 46944197)
34. S Dulucq S Bouchet B Turcq, et al. 2008 Multidrug resistance gene (MDR1) polymorphisms are associated with major molecular responses to standard-dose imatinib in chronic myeloid leukemia Blood 112 2024 7 18524988 10.1182/blood-2008-03-147744 1:CAS:528:DC%2BD1cXhtVyksr7M
35. Deenik W, van der Holt B, Janssen JJ, et al. Polymorphisms in the multidrug resistance gene MDR1 (ABCB1) predict for molecular resistance in patients with newly diagnosed chronic myeloid leukemia receiving high-dose imatinib. Blood. 116: 6144-6145; author reply 6145-6146.
36. S Dulucq C Preudhomme F Guilhot F-X Mahon 2010 Is there really a relationship between Multidrug Resistance Gene (MDR1) polymorphisms and major molecular response to imatinib in chronic myeloid leukemia? Blood 116 6145 6 10.1182/blood-2010-08-298794 1:CAS:528:DC%2BC3MXktFynsw%3D%3D
37. EJ Yeoh ME Ross SA Shurtleff, et al. 2002 Classification, subtype discovery, and prediction of outcome in pediatric acute lymphoblastic leukemia by gene expression profiling Cancer Cell 1 133 43 12086872 10.1016/S1535- 6108(02)00032-6 1:CAS:528:DC%2BD38XivVegsb0%3D (Pubitemid 41039160)
38. PJ Valk R Delwel B Lowenberg 2005 Gene expression profiling in acute myeloid leukemia Curr Opin Hematol 12 76 81 15604895 10.1097/01.moh.0000149610. 14438.9a 1:CAS:528:DC%2BD2MXotFCjsg%3D%3D (Pubitemid 40189242)
39. SK McWeeney LC Pemberton MM Loriaux, et al. 2009 A gene expression signature of CD34+ cells to predict major cytogenetic response in chronic-phase chronic myeloid leukemia patients treated with imatinib Blood 115 315 25 19837975 10.1182/blood-2009-03-210732 This is a landmark microarray analysis, which demonstrates that even within the CP-CML setting there is a wide variation in disease genotype, with those patients failing to respond to imatinib showing gene patterns analogous to advanced disease
40. CH Jamieson LE Ailles SJ Dylla, et al. 2004 Granulocyte-macrophage progenitors as candidate leukemic stem cells in blast-crisis CML N Engl J Med 351 657 67 15306667 10.1056/NEJMoa040258 1:CAS:528:DC%2BD2cXms1Oktbg%3D (Pubitemid 39063268)
41. C Zheng L Li M Haak, et al. 2006 Gene expression profiling of CD34+ cells identifies a molecular signature of chronic myeloid leukemia blast crisis Leukemia 20 1028 34 16617318 10.1038/sj.leu.2404227 1:CAS:528: DC%2BD28XkvVymu7o%3D (Pubitemid 43797293)
42. AS Yong RM Szydlo JM Goldman JF Apperley JV Melo 2006 Molecular profiling of CD34+ cells identifies low expression of CD7, along with high expression of proteinase 3 or elastase, as predictors of longer survival in patients with CML Blood 107 205 12 16144796 10.1182/blood-2005-05-2155 1:CAS:528: DC%2BD28XhsVGmsQ%3D%3D (Pubitemid 43053545)
43. JP Radich H Dai M Mao, et al. 2006 Gene expression changes associated with progression and response in chronic myeloid leukemia Proc Natl Acad Sci USA 103 2794 9 16477019 10.1073/pnas.0510423103 1:CAS:528:DC%2BD28XksF2rtL8%3D
44. VG Oehler KY Yeung YE Choi RE Bumgarner AE Raftery JP Radich 2009 The derivation of diagnostic markers of chronic myeloid leukemia progression from microarray data Blood 114 3292 8 19654405 10.1182/blood-2009-03-212969 1:CAS:528:DC%2BD1MXhtlShtbnE
45. S Picard K Titier G Etienne, et al. 2007 Trough imatinib plasma levels are associated with both cytogenetic and molecular responses to standard-dose imatinib in chronic myeloid leukemia Blood 109 3496 9 17192396 10.1182/blood-2006-07-036012 1:CAS:528:DC%2BD2sXksFWhsrY%3D (Pubitemid 46572540)
46. RA Larson BJ Druker F Guilhot, et al. 2008 Imatinib pharmacokinetics and its correlation with response and safety in chronic-phase chronic myeloid leukemia: a subanalysis of the IRIS study Blood 111 4022 8 18256322 10.1182/blood-2007-10-116475 1:CAS:528:DC%2BD1cXkvFeisrg%3D
47. DL Forrest S Trainor RR Brinkman, et al. 2009 Cytogenetic and molecular responses to standard-dose imatinib in chronic myeloid leukemia are correlated with Sokal risk scores and duration of therapy but not trough imatinib plasma levels Leuk Res 33 271 5 18762338 10.1016/j.leukres.2008.07.015 1:CAS:528:DC%2BD1cXhsVChtrvL
48. D Marin D Milojkovic E Olavarria, et al. 2008 European LeukemiaNet criteria for failure or suboptimal response reliably identify patients with CML in early chronic phase treated with imatinib whose eventual outcome is poor Blood 112 4437 44 18716134 10.1182/blood-2008-06-162388 1:CAS:528: DC%2BD1cXhsVCltbzF
49. T Hughes G Saglio S Branford, et al. 2009 Impact of baseline BCR-ABL mutations on response to nilotinib in patients with chronic myeloid leukemia in chronic phase J Clin Oncol 27 4204 10 19652056 10.1200/JCO.2009.21.8230 1:CAS:528:DC%2BD1MXhtF2jtLrF
50. Cortes J, Talpaz M, Bixby D, et al. A phase 1 trial of oral ponatinib (AP24534) in patients with refractory chronic myelogenous leukemia (CML) and other hematologic malignancies: emerging safety and clinical response findings. Blood (ASH Annual Meeting Abstracts). 2010;116:Abstract 210.