Treatment-free remission in CML: Who, how, and why?

Hematology

Volumn 2017, Issue 1, 2017, Pages 102-109

  Mahon, Francois Xavier ^a  

^a UNIVERSITÉ DE BORDEAUX   (France)

Author keywords

[No Author keywords available]

Indexed keywords

BIOLOGICAL FACTOR; PROTEIN TYROSINE KINASE INHIBITOR;

ARTICLE; CHRONIC MYELOID LEUKEMIA; CLINICAL FEATURE; CLINICAL OUTCOME; DRUG WITHDRAWAL; ECONOMIC ASPECT; FOLLOW UP; HISTORY; HUMAN; NONHUMAN; OVERALL SURVIVAL; PERSONALIZED MEDICINE; QUALITY OF LIFE; REMISSION; WITHDRAWAL SYNDROME; CHRONIC LYMPHATIC LEUKEMIA; DISEASE FREE SURVIVAL; METABOLISM; MORTALITY; SURVIVAL RATE;

DISEASE-FREE SURVIVAL; HUMANS; LEUKEMIA, LYMPHOCYTIC, CHRONIC, B-CELL; QUALITY OF LIFE; REMISSION, SPONTANEOUS; SURVIVAL RATE;

EID: 85038416041     PISSN: 15204391     EISSN: 15204383     Source Type: Journal    
DOI: 10.1182/asheducation-2017.1.102     Document Type: Article

References (55)

1. O'Hare T, Zabriskie MS, Eiring AM, Deininger MW. Pushing the limits of targeted therapy in chronic myeloid leukaemia. Nat Rev Cancer. 2012; 12(8):513-526.
2. Melo JV, Barnes DJ. Chronic myeloid leukaemia as a model of disease evolution in human cancer. Nat Rev Cancer. 2007;7(6):441-453.
3. Rowley JD. Letter: A new consistent chromosomal abnormality in chronic myelogenous leukaemia identified by quinacrine fluorescence and Giemsa staining. Nature. 1973;243(5405):290-293.
4. Daley GQ, Van Etten RA, Baltimore D. Induction of chronic myelogenous leukemia in mice by the P210bcr/abl gene of the Philadelphia chromosome. Science. 1990;247(4944):824-830.
5. Roeder I, Horn M, Glauche I, Hochhaus A, Mueller MC, Loeffler M. Dynamic modeling of imatinib-treated chronic myeloid leukemia: Functional insights and clinical implications. Nat Med. 2006;12(10): 1181-1184.
6. Deininger MW, Goldman JM, Melo JV. The molecular biology of chronic myeloid leukemia. Blood. 2000;96(10):3343-3356.
7. Kesarwani M, Kincaid Z, Gomaa A, et al. Targeting c-FOS and DUSP1 abrogates intrinsic resistance to tyrosine-kinase inhibitor therapy in BCR-ABL-induced leukemia. Nat Med. 2017;23(4):472-482.
8. Graham SM, Jørgensen HG, Allan E, et al. Primitive, quiescent, Philadelphia-positive stem cells from patients with chronic myeloid leukemia are insensitive to STI571 in vitro. Blood. 2002;99(1):319-325.
9. Hughes TP, Kaeda J, Branford S, et al; International Randomised Study of Interferon versus STI571 (IRIS) Study Group. Frequency of major molecular responses to imatinib or interferon alfa plus cytarabine in newly diagnosed chronic myeloid leukemia. N Engl J Med. 2003;349(15): 1423-1432.
10. Druker BJ, Talpaz M, Resta DJ, et al. Efficacy and safety of a specific inhibitor of the BCR-ABL tyrosine kinase in chronic myeloid leukemia. N Engl J Med. 2001;344(14):1031-1037.
11. Björkholm M, Ohm L, Eloranta S, et al. Success story of targeted therapy in chronic myeloid leukemia: A population-based study of patients diagnosed in Sweden from 1973 to 2008. J Clin Oncol. 2011;29(18): 2514-2520.
12. Kantarjian H, O'Brien S, Jabbour E, et al. Improved survival in chronic myeloid leukemia since the introduction of imatinib therapy: A singleinstitution historical experience. Blood. 2012;119(9):1981-1987.
13. van Rhee F, Szydlo RM, Hermans J, et al. Long-term results after allogeneic bone marrow transplantation for chronic myelogenous leukemia in chronic phase: A report from the Chronic Leukemia Working Party of the European Group for Blood and Marrow Transplantation. Bone Marrow Transplant. 1997;20(7):553-560.
14. Kaeda J, O'Shea D, Szydlo RM, et al. Serial measurement of BCR-ABL transcripts in the peripheral blood after allogeneic stem cell transplantation for chronic myeloid leukemia: An attempt to define patients who may not require further therapy. Blood. 2006;107(10):4171-4176.
15. Radich JP, Gooley T, Bryant E, et al. The significance of bcr-abl molecular detection in chronic myeloid leukemia patients "late, " 18 months or more after transplantation. Blood. 2001;98(6):1701-1707.
16. Pavlu J, Szydlo RM, Goldman JM, Apperley JF. Three decades of transplantation for chronic myeloid leukemia: what have we learned?. Blood. 2011;117(3):755-763.
17. Mahon FX, Fabères C, Pueyo S, et al. Response at three months is a good predictive factor for newly diagnosed chronic myeloid leukemia patients treated by recombinant interferon-alpha. Blood. 1998;92(11): 4059-4065.
18. Mahon FX, Delbrel X, Cony-Makhoul P, et al. Follow-up of complete cytogenetic remission in patients with chronic myeloid leukemia after cessation of interferon alfa. J Clin Oncol. 2002;20(1):214-220.
19. Mahon FX, Fort MP, Etienne G, et al. Interferon alpha alone is able to cure chronic myeloid leukemia in a small subset of patients despite the persistence of leukemic cells: Experience of long follow up after treatment discontinuation. Blood. 2010;116. Abstract 2299.
20. Goldman J, Gordon M. Why do chronic myelogenous leukemia stem cells survive allogeneic stem cell transplantation or imatinib: Does it really matter?. Leuk Lymphoma. 2006;47(1):1-7.
21. Bower H, Björkholm M, Dickman PW, Höglund M, Lambert PC, Andersson TM. Life expectancy of patients with chronic myeloid leukemia approaches the life expectancy of the general population. J Clin Oncol. 2016;34(24):2851-2857.
22. Experts in Chronic Myeloid Leukemia. The price of drugs for chronic myeloid leukemia (CML) is a reflection of the unsustainable prices of cancer drugs: From the perspective of a large group of CML experts. Blood. 2013;121(22):4439-4442.
23. Mahon FX. Is going for cure in chronic myeloid leukemia possible and justifiable?. Hematology Am Soc Hematol Educ Program. 2012;2012: 122-128.
24. Efficace F, Baccarani M, Breccia M, et al; GIMEMA. Health-related quality of life in chronic myeloid leukemia patients receiving long-term therapy with imatinib compared with the general population. Blood. 2011;118(17):4554-4560.
25. Efficace F, Baccarani M, Breccia M, et al. Chronic fatigue is the most important factor limiting health-related quality of life of chronic myeloid leukemia patients treated with imatinib. Leukemia. 2013; 27(7):1511-1519.
26. Rousselot P, Huguet F, Réa D, et al. Imatinib mesylate discontinuation in patients with chronic myelogenous leukemia in complete molecular remission for more than 2 years. Blood. 2007;109(1):58-60.
27. Mahon FX, Réa D, Guilhot J, et al; Intergroupe Français des Leucémies Myéloïdes Chroniques. 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(11):1029-1035.
28. Etienne G, Guilhot J, Réa D, et al. Long-term follow-up of the French Stop Imatinib (STIM1) study in patients with chronic myeloid leukemia. J Clin Oncol. 2017;35(3):298-305.
29. Ross DM, Branford S, Seymour JF, et al. Safety and efficacy of imatinib cessation for CML patients with stable undetectable minimal residual disease: Results from the TWISTER study. Blood. 2013;122(4):515-522.
30. Rousselot P, Charbonnier A, Cony-Makhoul P, et al. Loss of major molecular response as a trigger for restarting tyrosine kinase inhibitor therapy in patients with chronic-phase chronic myelogenous leukemia who have stopped imatinib after durable undetectable disease. J Clin Oncol. 2014;32(5):424-430.
31. Lee SE, Choi SY, Bang JH, et al. Predictive factors for successful imatinib cessation in chronic myeloid leukemia patients treated with imatinib. Am J Hematol. 2013;88(6):449-454.
32. Campiotti L, Suter MB, Guasti L, et al. Imatinib discontinuation in chronic myeloid leukaemia patients with undetectable BCR-ABL transcript level: A systematic review and a meta-analysis. Eur J Cancer. 2017; 77:48-56.
33. Mahon FX, Richter J, Guilhot J, et al. Cessation of tyrosine kinase inhibitors treatment in chronic myeloid leukemia patients with deep molecular response: Results of the Euro-Ski Trial. Blood. 2016;128. Abstract 787.
34. Clark RE, Polydoros F, Apperley JF, et al. De-escalation of tyrosine kinase inhibitor dose in patients with chronic myeloid leukaemia with stable major molecular response (DESTINY): An interim analysis of a non-randomised, phase 2 trial. Lancet Haematol. 2017;4(7):e310-e316.
35. Réa D, Nicolini FE, Tulliez M, et al; France Intergroupe des Leucémies Myéloïdes Chroniques. Discontinuation of dasatinib or nilotinib in chronic myeloid leukemia: Interim analysis of the STOP 2G-TKI study. Blood. 2017;129(7):846-854.
36. Imagawa J, Tanaka H, Okada M, et al; DADI Trial Group. Discontinuation of dasatinib in patients with chronic myeloid leukaemia who have maintained deep molecular response for longer than 1 year (DADI trial): A multicentre phase 2 trial. Lancet Haematol. 2015;2(12):e528-e535.
37. Hochhaus A, Masszi T, Giles FJ, et al. Treatment-free remission following frontline nilotinib in patients with chronic myeloid leukemia in chronic phase: Results from the ENESTfreedom study. Leukemia. 2017; 31(7):1525-1531.
38. Hughes TP, Boquimpani C, Kim DW, et al. Treatment-free remission (TFR) in patients with chronic myeloid leukemia in chronic phase (CML-CP) treated with second-line nilotinib (NIL): First results from the ENESTop study. J Clin Oncol. 2016;34(suppl). Abstract 7054.
39. Cortes JE, Gambacorti-Passerini C, Deininger MW, et al. Bosutinib (BOS) versus imatinib (IM) for newly diagnosed chronic myeloid leukemia (CML): Initial results from the BFORE trial. J Clin Oncol. 2017; 35(suppl). Abstract 7002.
40. Richter J, Söderlund S, Lübking A, et al. Musculoskeletal pain in patients with chronic myeloid leukemia after discontinuation of imatinib: A tyrosine kinase inhibitor withdrawal syndrome?. J Clin Oncol. 2014;32(25): 2821-2823.
41. Berger MG, Perieira B, Oris C, et al. Osteoarticular pain after discontinuation of tyrosine kinase inhibitors: A French cohort. Blood. 2015;126: 137. Abstract 137.
42. Paramarta JE, Turina MC, Noordenbos T, et al. A proof-of-concept study with the tyrosine kinase inhibitor nilotinib in spondyloarthritis. J Transl Med. 2016;14(1):308.
43. Saußele S, Richter J, Hochhaus A, Mahon FX. The concept of treatmentfree remission in chronic myeloid leukemia. Leukemia. 2016;30(8): 1638-1647.
44. Legros L, Nicolini FE, Etienne G, et al. Second tyrosine kinase inhibitor discontinuation attempt in patients with chronic myeloid leukemia [published online ahead of print 25 July 2017]. Cancer. doi:10.1002/cncr.30885.
45. Hughes TP, Ross DM. Moving treatment-free remission into mainstream clinical practice in CML. Blood. 2016;128(1):17-23.
46. Mori S, Vagge E, le Coutre P, et al. Age and dPCR can predict relapse in CML patients who discontinued imatinib: The ISAV study. Am J Hematol. 2015;90(10):910-914.
47. Takahashi N, Kyo T, Maeda Y, et al. Discontinuation of imatinib in Japanese patients with chronic myeloid leukemia. Haematologica. 2012; 97(6):903-906.
48. Ilander M, Olsson-Strömberg U, Schlums H, et al. Increased proportion of mature NK cells is associated with successful imatinib discontinuation in chronic myeloid leukemia. Leukemia. 2017;31(5):1108-1116.
49. Rea D, Henry G, Khaznadar Z, et al. Natural killer-cell counts are associated with molecular relapse-free survival after imatinib discontinuation in chronic myeloid leukemia: The IMMUNOSTIM study. Haematologica. 2017;102(8):1368-1377.
50. Schütz C, Inselmann S, Sausslele S, et al. Expression of the CTLA-4 ligand CD86 on plasmacytoid dendritic cells (pDC) predicts risk of disease recurrence after treatment discontinuation in CML. Leukemia. 2017;31(4):829-836.
51. National Comprehensive Cancer Network. NCCN Clinical Practice Guidelines in Oncology: Chronic Myelogenous Leukemia. Version 2. http://www.jnccn.org/content/14/12/1505.long.
52. Biernaux C, Loos M, Sels A, Huez G, Stryckmans P. Detection of major bcr-abl gene expression at a very low level in blood cells of some healthy individuals. Blood. 1995;86(8):3118-3122.
53. Bose S, Deininger M, Gora-Tybor J, Goldman JM, Melo JV. The presence of typical and atypical BCR-ABL fusion genes in leukocytes of normal individuals: Biologic significance and implications for the assessment of minimal residual disease. Blood. 1998;92(9):3362-3367.
54. Deeks SG, Lewin SR, Ross AL, et al; International AIDS Society Towards a CureWorking Group. International AIDS Society global scientific strategy: Towards an HIV cure 2016. Nat Med. 2016;22(8):839-850.
55. Viel E, Arbion F, Barbe C, Bougnoux P. Prolonged complete response after treatment withdrawal in HER2-overexpressed, hormone receptornegative breast cancer with liver metastases: The prospect of disappearance of an incurable disease. BMC Cancer. 2014;14:690.