The role of stem cell transplantation for chronic myelogenous leukemia in the 21st century

Blood

Volumn 125, Issue 21, 2015, Pages 3230-3235

  Barrett, A John ^a   Ito, Sawa ^a  

^a NATIONAL HEART LUNG AND BLOOD INSTITUTE   (United States)

Author keywords

[No Author keywords available]

Indexed keywords

BCR ABL PROTEIN; PROTEIN TYROSINE KINASE INHIBITOR;

ALLOGENEIC STEM CELL TRANSPLANTATION; ARTICLE; CANCER CHEMOTHERAPY; CANCER IMMUNOTHERAPY; CHILDHOOD CANCER; CHRONIC MYELOID LEUKEMIA; CORD BLOOD STEM CELL TRANSPLANTATION; COST EFFECTIVENESS ANALYSIS; DONOR; DRUG COST; GRAFT VERSUS HOST REACTION; HUMAN; LEUKEMIA RELAPSE; MONOZYGOTIC TWINS; PREGNANCY; PRIORITY JOURNAL; QUALITY ADJUSTED LIFE YEAR; REDUCED INTENSITY CONDITIONING; STEM CELL TRANSPLANTATION; TIME; TREATMENT INDICATION; ADULT; CHILD; HEMATOPOIETIC STEM CELL TRANSPLANTATION; LEUKEMIA, MYELOGENOUS, CHRONIC, BCR-ABL POSITIVE; PROCEDURES;

ADULT; CHILD; HEMATOPOIETIC STEM CELL TRANSPLANTATION; HUMANS; LEUKEMIA, MYELOGENOUS, CHRONIC, BCR-ABL POSITIVE;

EID: 84929999516     PISSN: 00064971     EISSN: 15280020     Source Type: Journal    
DOI: 10.1182/blood-2014-10-567784     Document Type: Article

References (93)

1. Buckner CD, Clift RA, Fefer A, Neiman PE, Storb R, Thomas ED. Treatment of blastic transformation of chronic granulocytic leukemia by high dose cyclophosphamide, total body irradiation and infusion of cryopreserved autologous marrow. Exp Hematol. 1974;2(3):138-146.
2. Buckner CD, Stewart P, Clift RA, et al. Treatment of blastic transformation of chronic granulocytic leukemia by chemotherapy, total body irradiation and infusion of cryopreserved autologous marrow. Exp Hematol. 1978;6(1):96-109.
3. Goldman JM, Catovsky D, Hows J, Spiers AS, Galton DA. Cryopreserved peripheral blood cells functioning as autografts in patients with chronic granulocytic leukaemia in transformation. BMJ. 1979;1(6174):1310-1313.
4. Fefer A, Cheever MA, Thomas ED, et al. Disappearance of Ph1-positive cells in four patients with chronic granulocytic leukemia after chemotherapy, irradiation and marrow transplantation from an identical twin. N Engl J Med. 1979;300(7):333-337.
5. Clift RA, Buckner CD, Thomas ED, et al. Treatment of chronic granulocytic leukaemia in chronic phase by allogeneic marrow transplantation. Lancet. 1982;320(8299):621-623.
6. Goldman JM, Baughan AS, McCarthy DM, et al. Marrow transplantation for patients in the chronic phase of chronic granulocytic leukaemia. Lancet. 1982;320(8299):623-625.
7. McGlave PB, Arthur DC, Kim TH, Ramsay NK, Hurd DD, Kersey J. Successful allogeneic bone-marrow transplantation for patients in the accelerated phase of chronic granulocytic leukaemia. Lancet. 1982;320(8299):625-627.
8. 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.
9. Horowitz MM, Gale RP, Sondel PM, et al. Graft-versus-leukemia reactions after bone marrow transplantation. Blood. 1990;75(3):555-562.
10. Kolb HJ, Mittermüller J, Clemm C, et al. Donor leukocyte transfusions for treatment of recurrent chronic myelogenous leukemia in marrow transplant patients. Blood. 1990;76(12):2462-2465.
11. van Rhee F, Lin F, Cullis JO, et al. Relapse of chronic myeloid leukemia after allogeneic bone marrow transplant: the case for giving donor leukocyte transfusions before the onset of hematologic relapse. Blood. 1994;83(11):3377-3383.
12. Mackinnon S, Papadopoulos EB, Carabasi MH, et al. Adoptive immunotherapy evaluating escalating doses of donor leukocytes for relapse of chronic myeloid leukemia after bone marrow transplantation: separation of graft-versus-leukemia responses from graft-versus-host disease. Blood. 1995;86(4):1261-1268.
13. Adekola K, Popat U, Ciurea SO. An update on allogeneic hematopoietic progenitor cell transplantation for myeloproliferative neoplasms in the era of tyrosine kinase inhibitors. Bone Marrow Transplant. 2014;49(11):1352-1359.
14. Gratwohl A. The EBMT risk score. Bone Marrow Transplant. 2012;47(6):749-756.
15. Druker BJ, Guilhot F, O'Brien SG, et al IRIS Investigators. Five-year follow-up of patients receiving imatinib for chronic myeloid leukemia. N Engl J Med. 2006;355(23):2408-2417.
16. 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.
17. Baccarani M, Deininger MW, Rosti G, et al. European LeukemiaNet recommendations for the management of chronic myeloid leukemia: 2013. Blood. 2013;122(6):872-884.
18. Jabbour E, Kantarjian H. Chronic myeloid leukemia: 2014 update on diagnosis, monitoring, and management. Am J Hematol. 2014;89(5):547-556.
19. O'Brien S, Radich JP, Abboud CN, et al; Ntational comprehensive cancer network. Chronic Myelogenous Leukemia, Version 1.2014. J Natl Compr Canc Netw. 2013;11(11):1327-1340.
20. Cortes JE, Kantarjian H, Shah NP, et al. Ponatinib in refractory Philadelphia chromosome-positive leukemias. N Engl J Med. 2012;367(22):2075-2088.
21. Cortes J, Lipton JH, Rea D, et al Omacetaxine 202 Study Group. Phase 2 study of subcutaneous omacetaxine mepesuccinate after TKI failure in patients with chronic-phase CML with T315I mutation. Blood. 2012;120(13):2573-2580.
22. Hamilton A, Helgason GV, Schemionek M, et al. Chronic myeloid leukemia stem cells are not dependent on Bcr-Abl kinase activity for their survival. Blood. 2012;119(6):1501-1510.
23. Copland M, Hamilton A, Elrick LJ, et al. Dasatinib (BMS-354825) targets an earlier progenitor population than imatinib in primary CML but does not eliminate the quiescent fraction. Blood. 2006;107(11):4532-4539.
24. Saussele S, Lauseker M, Gratwohl A, et al German CML Study Group. Allogeneic hematopoietic stem cell transplantation (allo SCT) for chronic myeloid leukemia in the imatinib era: evaluation of its impact within a subgroup of the randomized German CML Study IV. Blood. 2010;115(10):1880-1885.
25. Jiang Q, Xu LP, Liu DH, et al. Imatinib mesylate versus allogeneic hematopoietic stem cell transplantation for patients with chronic myelogenous leukemia in the accelerated phase. Blood. 2011;117(11):3032-3040.
26. Jabbour E, Cortes J, Santos FP, et al. Results of allogeneic hematopoietic stem cell transplantation for chronic myelogenous leukemia patients who failed tyrosine kinase inhibitors after developing BCR-ABL1 kinase domain mutations. Blood. 2011;117(13):3641-3647.
27. Khoury HJ, Kukreja M, Goldman JM, et al. Prognostic factors for outcomes in allogeneic transplantation for CML in the imatinib era: a CIBMTR analysis. Bone Marrow Transplant. 2012;47(6):810-816.
28. Lee SJ, Kukreja M, Wang T, et al. Impact of prior imatinib mesylate on the outcome of hematopoietic cell transplantation for chronic myeloid leukemia. Blood. 2008;112(8):3500-3507.
29. Oehler VG, Gooley T, Snyder DS, et al. The effects of imatinib mesylate treatment before allogeneic transplantation for chronic myeloid leukemia. Blood. 2007;109(4):1782-1789.
30. Jabbour E, Cortes J, Kantarjian H, et al. Novel tyrosine kinase inhibitor therapy before allogeneic stem cell transplantation in patients with chronic myeloid leukemia: no evidence for increased transplant-related toxicity. Cancer. 2007;110(2):340-344.
31. Sloand E, Childs RW, Solomon S, Greene A, Young NS, Barrett AJ. The graft-versus-leukemia effect of nonmyeloablative stem cell allografts may not be sufficient to cure chronic myelogenous leukemia. Bone Marrow Transplant. 2003;32(9):897-901.
32. Or R, Shapira MY, Resnick I, et al. Nonmyeloablative allogeneic stem cell transplantation for the treatment of chronic myeloid leukemia in first chronic phase. Blood. 2003;101(2):441-445.
33. Das M, Saikia TK, Advani SH, Parikh PM, Tawde S. Use of a reduced-intensity conditioning regimen for allogeneic transplantation in patients with chronic myeloid leukemia. Bone Marrow Transplant. 2003;32(2):125-129.
34. Kebriaei P, Detry MA, Giralt S, et al. Long-term follow-up of allogeneic hematopoietic stem-cell transplantation with reduced-intensity conditioning for patients with chronic myeloid leukemia. Blood. 2007;110(9):3456-3462.
35. Topcuoglu P, Arat M, Ozcan M, et al. Case-matched comparison with standard versus reduced intensity conditioning regimen in chronic myeloid leukemia patients. Ann Hematol. 2012;91(4):577-586.
36. Warlick E, Ahn KW, Pedersen TL, et al. Reduced intensity conditioning is superior to nonmyeloablative conditioning for older chronic myelogenous leukemia patients undergoing hematopoietic cell transplant during the tyrosine kinase inhibitor era. Blood. 2012;119(17):4083-4090.
37. Stern M, de Wreede LC, Brand R, et al. Sensitivity of hematological malignancies to graft-versus-host effects: an EBMT megafile analysis. Leukemia. 2014;28(11):2235-2240.
38. Zuckerman T, Katz T, Haddad N, et al. Allogeneic stem cell transplantation for patients with chronic myeloid leukemia: risk stratified approach with a long-term follow-up. Am J Hematol. 2012;87(9):875-879.
39. Poiré X, Artz A, Larson RA, et al. Allogeneic stem cell transplantation with alemtuzumab-based conditioning for patients with advanced chronic myelogenous leukemia. Leuk Lymphoma. 2009;50(1):85-91.
40. Arpinati M, Tolomelli G, Bochicchio MT, et al. Molecular monitoring of BCR-ABL transcripts after allogeneic stem cell transplantation for chronic myeloid leukemia. Biol Blood Marrow Transplant. 2013;19(5):735-740.
41. 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.
42. Heaney NB, Copland M, Stewart K, et al. Complete molecular responses are achieved after reduced intensity stem cell transplantation and donor lymphocyte infusion in chronic myeloid leukemia. Blood. 2008;111(10):5252-5255.
43. Carpenter PA, Snyder DS, Flowers ME, et al. Prophylactic administration of imatinib after hematopoietic cell transplantation for high-risk Philadelphia chromosome-positive leukemia. Blood. 2007;109(7):2791-2793.
44. Nakasone H, Kanda Y, Takasaki H, et al Kanto Study Group for Cell Therapy. Prophylactic impact of imatinib administration after allogeneic stem cell transplantation on the incidence and severity of chronic graft versus host disease in patients with Philadelphia chromosome-positive leukemia. Leukemia. 2010;24(6):1236-1239.
45. Luo Y, Lai XY, Tan YM, et al. Reduced-intensity allogeneic transplantation combined with imatinib mesylate for chronic myeloid leukemia in first chronic phase. Leukemia. 2009;23(6):1171-1174.
46. Goldman JM, Majhail NS, Klein JP, et al. Relapse and late mortality in 5-year survivors of myeloablative allogeneic hematopoietic cell transplantation for chronic myeloid leukemia in first chronic phase. J Clin Oncol. 2010;28(11):1888-1895.
47. Goldman JM, Melo JV. Chronic myeloid leukemia - advances in biology and new approaches to treatment. N Engl J Med. 2003;349(15):1451-1464.
48. Ullmann AJ, Hess G, Kolbe K, et al. Current results on the use of imatinib mesylate in patients with relapsed Philadelphia chromosome positive leukemia after allogeneic or syngeneic hematopoietic stem cell transplantation. Keio J Med. 2003;52(3):182-188.
49. Olavarria E, Ottmann OG, Deininger M, et al Chronic Leukaemia Working Party of the European Group of Bone and Marrow Transplantation (EBMT). Response to imatinib in patients who relapse after allogeneic stem cell transplantation for chronic myeloid leukemia. Leukemia. 2003;17(9):1707-1712.
50. Kim YJ, Kim DW, Lee S, et al. Cytogenetic clonal evolution alone in CML relapse post-transplantation does not adversely affect response to imatinib mesylate treatment. Bone Marrow Transplant. 2004;33(2):237-242.
51. DeAngelo DJ, Hochberg EP, Alyea EP, et al. Extended follow-up of patients treated with imatinib mesylate (gleevec) for chronic myelogenous leukemia relapse after allogeneic transplantation: durable cytogenetic remission and conversion to complete donor chimerism without graft-versus-host disease. Clin Cancer Res. 2004;10(15):5065-5071.
52. Kantarjian HM, O'Brien S, Cortes JE, et al. Imatinib mesylate therapy for relapse after allogeneic stem cell transplantation for chronic myelogenous leukemia. Blood. 2002;100(5):1590-1595.
53. Savani BN, Montero A, Kurlander R, Childs R, Hensel N, Barrett AJ. Imatinib synergizes with donor lymphocyte infusions to achieve rapid molecular remission of CML relapsing after allogeneic stem cell transplantation. Bone Marrow Transplant. 2005;36(11):1009-1015.
54. Bansal D, Shava U, Varma N, Trehan A, Marwaha RK. Imatinib has adverse effect on growth in children with chronic myeloid leukemia. Pediatr Blood Cancer. 2012;59(3):481-484.
55. Giona F, Mariani S, Gnessi L, et al. Bone metabolism, growth rate and pubertal development in children with chronic myeloid leukemia treated with imatinib during puberty. Haematologica. 2013;98(3):e25-e27.
56. Hobernicht SL, Schweiger B, Zeitler P, Wang M, Hunger SP. Acquired growth hormone deficiency in a girl with chronic myelogenous leukemia treated with tyrosine kinase inhibitor therapy. Pediatr Blood Cancer. 2011;56(4):671-673.
57. de la Fuente J, Baruchel A, Biondi A, et al International BFM Group (iBFM) Study Group Chronic Myeloid Leukaemia Committee. Managing children with chronic myeloid leukaemia (CML): recommendations for the management of CML in children and young people up to the age of 18 years. Br J Haematol. 2014;167(1):33-47.
58. Andolina JR, Neudorf SM, Corey SJ. How I treat childhood CML. Blood. 2012;119(8):1821-1830.
59. 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.
60. Bevans MF, Mitchell SA, Barrett JA, et al. Symptom distress predicts long-term health and well-being in allogeneic stem cell transplantation survivors. Biol Blood Marrow Transplant. 2014;20(3):387-395.
61. Bevans M. Health-related quality of life following allogeneic hematopoietic stem cell transplantation. Hematology Am Soc Hematol Educ Program. 2010;2010:248-254.
62. Millot F, Guilhot J, Baruchel A, et al. Impact of early molecular response in children with chronic myeloid leukemia treated in the French Glivec phase 4 study. Blood. 2014;124(15):2408-2410.
63. Apperley J. Issues of imatinib and pregnancy outcome. J Natl Compr Canc Netw. 2009;7(10): 1050-1058.
64. Pye SM, Cortes J, Ault P, et al. The effects of imatinib on pregnancy outcome. Blood. 2008;111(12):5505-5508.
65. Kuwabara A, Babb A, Ibrahim A, et al. Poor outcome after reintroduction of imatinib in patients with chronic myeloid leukemia who interrupt therapy on account of pregnancy without having achieved an optimal response. Blood. 2010;116(6):1014-1016.
66. Borgmann-Staudt A, Rendtorff R, Reinmuth S, et al. Fertility after allogeneic haematopoietic stem cell transplantation in childhood and adolescence. Bone Marrow Transplant. 2012;47(2):271-276.
67. Loren AW, Chow E, Jacobsohn DA, et al. Pregnancy after hematopoietic cell transplantation: a report from the late effects working committee of the Center for International Blood and Marrow Transplant Research (CIBMTR). Biol Blood Marrow Transplant. 2011;17(2):157-166.
68. Wu KN, Luo Y, Liu LZ, et al. Twin pregnancy and childbirth after reduced-intensity conditioning allogeneic haematopoietic stem cell transplantation combined with imatinib mesylate for chronic myeloid leukaemia: case report and literature review. J Int Med Res. 2012;40(6):2409-2415.
69. Kim MK, Lee DR, Han JE, et al. Live birth with vitrified-warmed oocytes of a chronic myeloid leukemia patient nine years after allogenic bone marrow transplantation. J Assist Reprod Genet. 2011;28(12):1167-1170.
70. Gale RP, Horowitz MM, Ash RC, et al. Identicaltwin bone marrow transplants for leukemia. Ann Intern Med. 1994;120(8):646-652.
71. Barrett AJ, Ringdén O, Zhang MJ, et al. Effect of nucleated marrow cell dose on relapse and survival in identical twin bone marrow transplants for leukemia. Blood. 2000;95(11):3323-3327.
72. Zheng C, Tang B, Yao W, et al. Comparison of unrelated cord blood transplantation and HLA-matched sibling hematopoietic stem cell transplantation for patients with chronic myeloid leukemia in advanced stage. Biol Blood Marrow Transplant. 2013;19(12):1708-1712.
73. Adekola K, di Stasi A, Ferro R, et al. Safety and efficacy of haploidentical stem cell transplantation for advanced chronic myeloid leukemia. Biol Blood Marrow Transplant. 2014;20(2):S213-S214.
74. 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.
75. Ruiz-Argüelles GJ, Tarin-Arzaga LC, Gonzalez-Carrillo ML, et al. Therapeutic choices in patients with Ph-positive CML living in Mexico in the tyrosine kinase inhibitor era: SCT or TKIs? Bone Marrow Transplant. 2008;42(1):23-28.
76. Ohm L, Lundqvist A, Dickman P, et al. Real-world cost-effectiveness in chronic myeloid leukemia: the price of success during four decades of development from non-targeted treatment to imatinib [published online ahead of print October 21, 2014]. Leuk Lymphoma.
77. Goldman JM. The significance of BCR-ABL transcripts after allogeneic stem cell transplantation for chronic myeloid leukemia. Biol Blood Marrow Transplant. 2013;19(5):679-680.
78. Cortes J, Goldman JM, Hughes T. Current issues in chronic myeloid leukemia: monitoring, resistance, and functional cure. J Natl Compr Canc Netw. 2012;10(suppl 3):S1-S13.
79. 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.
80. 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.
81. Rojas JM, Knight K, Wang L, Clark RE. Clinical evaluation of BCR-ABL peptide immunisation in chronic myeloid leukaemia: results of the EPIC study. Leukemia. 2007;21(11):2287-2295.
82. Rezvani K, Yong AS, Mielke S, et al. Leukemia-associated antigen-specific T-cell responses following combined PR1 and WT1 peptide vaccination in patients with myeloid malignancies. Blood. 2008;111(1):236-242.
83. Rezvani K. Posttransplantation vaccination: concepts today and on the horizon. Hematology Am Soc Hematol Educ Program. 2011;2011:299-304.
84. Pinilla-Ibarz J, Cathcart K, Korontsvit T, et al. Vaccination of patients with chronic myelogenous leukemia with bcr-abl oncogene breakpoint fusion peptides generates specific immune responses. Blood. 2000;95(5):1781-1787.
85. Bocchia M, Defina M, Aprile L, et al. Complete molecular response in CML after p210 BCR-ABL1-derived peptide vaccination. Nat Rev Clin Oncol. 2010;7(10):600-603.
86. Weber G, Gerdemann U, Caruana I, et al. Generation of multi-leukemia antigen-specific T cells to enhance the graft-versus-leukemia effect after allogeneic stem cell transplant. Leukemia. 2013;27(7):1538-1547.
87. Bornhäuser M, Thiede C, Platzbecker U, et al. Prophylactic transfer of BCR-ABL-, PR1-, and WT1-reactive donor T cells after T cell-depleted allogeneic hematopoietic cell transplantation in patients with chronic myeloid leukemia. Blood. 2011;117(26):7174-7184.
88. Gill S, Tasian SK, Ruella M, et al. Preclinical targeting of human acute myeloid leukemia and myeloablation using chimeric antigen receptor-modified T cells. Blood. 2014;123(15):2343-2354.
89. Ochi T, Fujiwara H, Suemori K, et al. Aurora-A kinase: a novel target of cellular immunotherapy for leukemia. Blood. 2009;113(1):66-74.
90. Yong AS, Stephens N, Weber G, et al. Improved outcome following allogeneic stem cell transplantation in chronic myeloid leukemia is associated with higher expression of BMI-1 and immune responses to BMI-1 protein. Leukemia. 2011;25(4):629-637.
91. Järås M, Johnels P, Hansen N, et al. Isolation and killing of candidate chronic myeloid leukemia stem cells by antibody targeting of IL-1 receptor accessory protein. Proc Natl Acad Sci USA. 2010;107(37):16280-16285.
92. Herrmann H, Sadovnik I, Cerny-Reiterer S, et al. Dipeptidylpeptidase IV (CD26) defines leukemic stem cells (LSC) in chronic myeloid leukemia. Blood. 2014;123(25):3951-3962.
93. Gooley TA, Chien JW, Pergam SA, et al. Reduced mortality after allogeneic hematopoietic-cell transplantation. N Engl J Med. 2010;363(22):2091-2101.