Molecular monitoring 101: Helping your patients with chronic myeloid leukemia to understand the meaning of molecular response

Leukemia and Lymphoma

Volumn 53, Issue 8, 2012, Pages 1452-1460

  Jabbour, Elias J ^a   Quintás Cardama, Alfonso ^a  

^a UNIVERSITY OF TEXAS MD ANDERSON CANCER CENTER   (United States)

Author keywords

BCR ABL1; Chronic myeloid leukemia; tyrosine kinase inhibitor therapy

Indexed keywords

BCR ABL PROTEIN; DASATINIB; IMATINIB; NILOTINIB; PROTEIN TYROSINE KINASE INHIBITOR;

CANCER PROGNOSIS; CANCER SURVIVAL; CHROMOSOME ANALYSIS; CHRONIC MYELOID LEUKEMIA; CLINICAL DECISION MAKING; DRUG EFFICACY; EVENT FREE SURVIVAL; EVIDENCE BASED MEDICINE; FLUORESCENCE IN SITU HYBRIDIZATION; GENE MUTATION; HUMAN; OVERALL SURVIVAL; PATIENT MONITORING; POLYMERASE CHAIN REACTION; PRACTICE GUIDELINE; PRIORITY JOURNAL; PROGRESSION FREE SURVIVAL; REVERSE TRANSCRIPTION POLYMERASE CHAIN REACTION; REVIEW; STANDARDIZATION; TREATMENT OUTCOME; TREATMENT RESPONSE;

CYTOGENETICS; DNA MUTATIONAL ANALYSIS; FEMALE; FUSION PROTEINS, BCR-ABL; HUMANS; LEUKEMIA, MYELOGENOUS, CHRONIC, BCR-ABL POSITIVE; MEDICAL ONCOLOGY; MIDDLE AGED; PHYSICIAN-PATIENT RELATIONS; REAL-TIME POLYMERASE CHAIN REACTION; REPRODUCIBILITY OF RESULTS; RNA, MESSENGER;

EID: 84864253100     PISSN: 10428194     EISSN: 10292403     Source Type: Journal    
DOI: 10.3109/10428194.2012.659734     Document Type: Review

References (55)

1. Nowell PC, Hungerford DA. Chromosome studies on normal and leukemic human leukocytes. J Natl Cancer Inst 1960;25:85-109.
2. Rowley JD. Letter: A new consistent chromosomal abnormality in chronic myelogenous leukaemia identifi ed by quinacrine fl uorescence and Giemsa staining. Nature 1973;243:290-293.
3. Heisterkamp N, Stam K, Groff en J, et al. Structural organization of the bcr gene and its role in the Ph'translocation. Nature 1985;315: 758-761.
4. Q uintas-Cardama A, Cortes J. Molecular biology of bcr-abl1-positive chronic myeloid leukemia. Blood 2009;113:1619-1630.
5. H azlehurst LA, Bewry NN, Nair RR, et al. Signaling networks associated with BCR-ABL-dependent transformation. Cancer Control 2009;16:100-107.
6. Howlader N, Noone AM, Krapcho M, et al., editors. SEER Cancer Statistics Review, 1975 - 2008, based on November 2010 SEER data submission, posted to the SEER web site, 2011. Bethesda, MD: National Cancer Institute. Available from: http://seer.cancer.gov/ csr/1975-2008/
7. B accarani M, Dreyling M. Chronic myeloid leukaemia: ESMO Clinical Practice Guidelines for diagnosis, treatment and follow-up. Ann Oncol 2010;21(Suppl. 5):v165-v167.
8. N CCN. Clinical Practice Guidelines in Oncology (NCCN Guidelines ® ). Chronic myelogenous leukemia (version 2.2012). © 2011 National Comprehensive Cancer Network, Inc. Available from: www. NCCN.org
9. Cortes JE, Talpaz M, O'Brien S, et al. Staging of chronic myeloid leukemia in the imatinib era: an evaluation of the World Health Organization proposal. Cancer 2006;106:1306-1315.
10. B accarani M, Saglio G, Goldman J, et al. Evolving concepts in the management of chronic myeloid leukemia: recommendations from an expert panel on behalf of the European LeukemiaNet. Blood 2006;108:1809-1820.
11. H ughes TP, Hochhaus A, Branford S, et al. Long-term prognostic signifi cance of early molecular response to imatinib in newly diagnosed chronic myeloid leukemia: an analysis from the International Randomized Study of Interferon and STI571 (IRIS). Blood 2010;116:3758-3765.
12. B accarani M, Castagnetti F, Gugliotta G, et al. Response defi nitions and European LeukemiaNet management recommendations. Best Pract Res Clin Haematol 2009;22:331-341.
13. Wei G, Rafi yath S, Liu D. First-line treatment for chronic myeloid leukemia: dasatinib, nilotinib, or imatinib. J Hematol Oncol 2010;3:47.
14. P uttini M, Coluccia AM, Boschelli F, et al. In vitro and in vivo activity of SKI-606, a novel Src-Abl inhibitor, against imatinib-resistant Bcr-Abl+ neoplastic cells. Cancer Res 2006;66:11314-11322.
15. W eisberg E, Manley P, Mestan J, et al. AMN107 (nilotinib): a novel and selective inhibitor of BCR-ABL. Br J Cancer 2006;94:1765-1769.
16. K antarjian H, Shah NP, Hochhaus A, et al. Dasatinib versus imatinib in newly diagnosed chronic-phase chronic myeloid leukemia. N Engl J Med 2010;362:2260-2270.
17. K antarjian HM, Hochhaus A, Saglio G, et al. Nilotinib versus imatinib for the treatment of patients with newly diagnosed chronic phase, Philadelphia chromosome-positive, chronic myeloid leukaemia: 24-month minimum follow-up of the phase 3 randomised ENESTnd trial. Lancet Oncol 2011;12:841-851.
18. S aglio G, Kim DW, Issaragrisil S, et al. Nilotinib versus imatinib for newly diagnosed chronic myeloid leukemia. N Engl J Med 2010;362:2251-2259.
19. M ahon FX, Rea D, Guilhot J, et al. Discontinuation of imatinib in patients with chronic myeloid leukaemia who have maintained complete molecular remission for at least 2 years: the prospective, multicentre Stop Imatinib (STIM) trial. Lancet Oncol 2010;11:1029-1035.
20. R ohrbacher M, Hasford J. Epidemiology of chronic myeloid leukaemia (CML). Best Pract Res Clin Haematol 2009;22:295-302.
21. V igil CE, Griffi ths EA, Wang ES, et al. Interpretation of cytogenetic and molecular results in patients treated for CML. Blood Rev 2010;25:139-146.
22. P elz AF, Kroning H, Franke A, et al. High reliability and sensitivity of the BCR/ABL1 D-FISH test for the detection of BCR/ABL rearrangements. Ann Hematol 2002;81:147-153.
23. S choch C, Schnittger S, Bursch S, et al. Comparison of chromosome banding analysis, interphase- and hypermetaphase-FISH, qualitative and quantitative PCR for diagnosis and for follow-up in chronic myeloid leukemia: a study on 350 cases. Leukemia 2002;16:53-59.
24. H ochhaus A, Lin F, Reiter A, et al. Quantifi cation of residual disease in chronic myelogenous leukemia patients on interferon-alpha therapy by competitive polymerase chain reaction. Blood 1996;87:1549-1555.
25. C ross NC, Feng L, Chase A, et al. Competitive polymerase chain reaction to estimate the number of BCR-ABL transcripts in chronic myeloid leukemia patients after bone marrow transplantation. Blood 1993;82:1929-1936.
26. H unt MR. Real time PCR tutorial. In: Hunt RC, editor. Microbiology and immunoloy on-line. Columbia: University of South Carolina School of Medicine; 2010. Available from: http://pathmicro.med.sc.edu/pcr/ realtime-home.htm
27. G harabaghi F, Tellier R, Cheung R, et al. Comparison of a commercial qualitative real-time RT-PCR kit with direct immunofl uorescence assay (DFA) and cell culture for detection of infl uenza A and B in children. J Clin Virol 2008;42:190-193.
28. L ima L, Bernal-Mizrachi L, Saxe D, et al. Peripheral blood monitoring of chronic myeloid leukemia during treatment with imatinib, second-line agents, and beyond. Cancer 2011;117:1245-1252.
29. G oh HG, Lin M, Fukushima T, et al. Sensitive quantitation of minimal residual disease in chronic myeloid leukemia using nanofl uidic digital polymerase chain reaction assay. Leuk Lymphoma 2011;52:896-904.
30. B ranford S, Seymour JF, Grigg A, et al. BCR-ABL messenger RNA levels continue to decline in patients with chronic phase chronic myeloid leukemia treated with imatinib for more than 5 years and approximately half of all fi rst-line treated patients have stable undetectable BCR - ABL using strict sensitivity criteria. Clin Cancer Res 2007;13:7080-7085.
31. S hah N, Kantarjian H, Hochhaus A, et al. Dasatinib versus imatinib in patients with newly diagnosed chronic myeloid leukemia in chronic phase (CML-CP) in the DASISION trial: 18-month followup. Blood 2010;116(Suppl. 1): Abstract 206.
32. S obrinho-Simoes M, Wilczek V, Score J, et al. In search of the original leukemic clone in chronic myeloid leukemia patients in complete molecular remission after stem cell transplantation or imatinib. Blood 2010;116:1329-1335.
33. Hehlmann R, Lauseker M, Jung-Munkwitz S, et al. Tolerabilityadapted imatinib 800 mg/d versus 400 mg/d versus 400 mg/d plus interferon-{alpha} in newly diagnosed chronic myeloid leukemia. J Clin Oncol 2011;29:1634-1642.
34. B ranford S, Fletcher L, Cross NC, et al. Desirable performance characteristics for BCR-ABL measurement on an international reporting scale to allow consistent interpretation of individual patient response and comparison of response rates between clinical trials. Blood 2008;112:3330-3338.
35. Hughes TP, Kaeda J, Branford S, et al. Frequency of major molecular responses to imatinib or interferon alfa plus cytarabine in newly diagnosed chronic myeloid leukemia. N Engl J Med 2003;349:1423-1432.
36. Hughes T, Deininger M, Hochhaus A, et al. Monitoring CML patients responding to treatment with tyrosine kinase inhibitors: review and recommendations for harmonizing current methodology for detecting BCR-ABL transcripts and kinase domain mutations and for expressing results. Blood 2006;108:28-37.
37. Hughes T. ABL kinase inhibitor therapy for CML: baseline assessments and response monitoring. Hematology Am Soc Hematol Educ Program 2006:211-218.
38. W hite HE, Matejtschuk P, Rigsby P, et al. Establishment of the fi rst World Health Organization International Genetic Reference Panel for quantitation of BCR-ABL mRNA. Blood 2010;116:e111-e117.
39. Druker BJ, Guilhot F, O'Brien SG, et al. Five-year follow-up of patients receiving imatinib for chronic myeloid leukemia. N Engl J Med 2006;355:2408-2417.
40. Kantarjian HM, Shah NP, Cortes JE, et al. Dasatinib or imatinib in newly diagnosed chronic phase chronic myeloid leukemia: 2-year follow-up from a randomized phase 3 trial (DASISION). Blood 2011 Dec 9. [Epub ahead of print]
41. S korski T. BCR/ABL, DNA damage and DNA repair: implications for new treatment concepts. Leuk Lymphoma 2008;49:610-614.
42. Perrotti D, Jamieson C, Goldman J, et al. Chronic myeloid leukemia: mechanisms of blastic transformation. J Clin Invest 2010; 120:2254-2264.
43. Q uintas-Cardama A, Kantarjian H, Jones D, et al. Delayed achievement of cytogenetic and molecular response is associated with increased risk of progression among patients with chronic myeloid leukemia in early chronic phase receiving high-dose or standard-dose imatinib therapy. Blood 2009;113:6315-6321.
44. M arin D, Ibrahim AR, Lucas C, et al. Assessment of BCR-ABL1 transcript levels at 3 months is the only requirement for predicting outcome for patients with chronic myeloid leukemia treated with tyrosine kinase inhibitors. J Clin Oncol 2012;30:232-238.
45. H anfstein B, Muller MC, Erben P, et al. Molecular and cytogenetic response after 3 months of imatinib treatment is predictive for the risk of disease progression and death in newly diagnosed chronic myeloid leukemia patients - a follow-up analysis of the German CML Study IV. Blood 2011;118(Suppl. 1): Abstract 783.
46. Kantarjian HM, Giles FJ, Bhalla KN, et al. Nilotinib is eff ective in patients with chronic myeloid leukemia in chronic phase after imatinib resistance or intolerance: 24-month follow-up results. Blood 2011;117:1141-1145.
47. Rea D, Rousselot P, Nicolini FE, et al. Discontinuation of dasatinib or nilotinib in chronic myeloid leukemia (CML) patients (pts) withstable undetectable Bcr-Abl transcripts: results from the French CMLGroup (FILMC). Blood 2011;118(Suppl. 1): Abstract 604.
48. M arin D, Bazeos A, Mahon FX, et al. 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 2010;28:2381-2388.
49. Hughes TP, Branford S. Monitoring disease response to tyrosine kinase inhibitor therapy in CML. Hematology Am Soc Hematol Educ Program 2009:477-487.
50. C ross NC. Standardisation of molecular monitoring for chronic myeloid leukaemia. Best Pract Res Clin Haematol 2009;22: 355-365.
51. K antarjian HM, Shan J, Jones D, et al. Signifi cance of increasing levels of minimal residual disease in patients with Philadelphia chromosome-positive chronic myelogenous leukemia in complete cytogenetic response. J Clin Oncol 2009;27:3659-3663.
52. P ress RD, Willis SG, Laudadio J, et al. Determining the rise in BCR-ABL RNA that optimally predicts a kinase domain mutation in patients with chronic myeloid leukemia on imatinib. Blood 2009;114: 2598-2605.
53. E liasson L, Cliff ord S, Barber N, et al. Exploring chronic myeloid leukemia patients'reasons for not adhering to the oral anticancer drug imatinib as prescribed. Leuk Res 2011;35:626-630.
54. S overini 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;118:1208-1215.
55. 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 fi ndings. Blood 2010;116(Suppl. 1): Abstract 210.