Complete cytogenetic response and major molecular response as surrogate outcomes for overall survival in first-line treatment of chronic myelogenous leukemia: A case study for technology appraisal on the basis of surrogate outcomes evidence

Value in Health

Volumn 16, Issue 6, 2013, Pages 1081-1090

  Oriana, Ciani ^a   Martin, Hoyle ^a   Toby, Pavey ^b   Chris, Cooper ^a   Ruth, Garside ^a   Claudius, Rudin ^c   Rod, Taylor ^a  

^a UNIVERSITY OF EXETER MEDICAL SCHOOL   (United Kingdom)

^b UNIVERSITY OF QUEENSLAND   (Australia)

^c ROYAL DEVON AND EXETER NHS FOUNDATION TRUST   (United Kingdom)

Author keywords

chronic myeloid leukemia complete cytogenetic response dasatinib health technology assessment HTA imatinib intermediate outcomes major molecular response nilotinib surrogate end points systematic review technology appraisal

Indexed keywords

ALPHA INTERFERON; CYTARABINE; DASATINIB; IMATINIB; NILOTINIB;

ARTICLE; CANCER CHEMOTHERAPY; CANCER PATIENT; CANCER SURVIVAL; CASE STUDY; CHRONIC MYELOID LEUKEMIA; CYTOGENETICS; FOLLOW UP; FUNDING; HUMAN; INTERNAL VALIDITY; LICENSING; LIFE EXPECTANCY; LONG TERM SURVIVAL; META ANALYSIS; OVERALL SURVIVAL; PRIORITY JOURNAL; RANDOMIZED CONTROLLED TRIAL (TOPIC); SYSTEMATIC REVIEW; TREATMENT OUTCOME; TREATMENT RESPONSE;

CHRONIC MYELOID LEUKEMIA; COMPLETE CYTOGENETIC RESPONSE; DASATINIB; HEALTH TECHNOLOGY ASSESSMENT; HTA; IMATINIB; INTERMEDIATE OUTCOMES; MAJOR MOLECULAR RESPONSE; NILOTINIB; SURROGATE END POINTS; SYSTEMATIC REVIEW; TECHNOLOGY APPRAISAL;

ADOLESCENT; ADULT; AGED; AGED, 80 AND OVER; BIOLOGICAL MARKERS; CYTOGENETIC ANALYSIS; FEMALE; HUMANS; LEUKEMIA, MYELOGENOUS, CHRONIC, BCR-ABL POSITIVE; MALE; MIDDLE AGED; ORGANIZATIONAL CASE STUDIES; OUTCOME ASSESSMENT (HEALTH CARE); SURVIVAL ANALYSIS; TECHNOLOGY ASSESSMENT, BIOMEDICAL; YOUNG ADULT;

EID: 84884223108     PISSN: 10983015     EISSN: 15244733     Source Type: Journal    
DOI: 10.1016/j.jval.2013.07.004     Document Type: Article

References (71)

1. C.L. Sawyers Chronic myeloid leukemia N Engl J Med 340 1999 1330 1340
2. M. Baccarani, and M. Dreyling Chronic myeloid leukaemia: ESMO Clinical Practice Guidelines for diagnosis, treatment and follow-up Ann Oncol 21 Suppl. 5 2010 v165 v167
3. B. Calabretta, and D. Perrotti The biology of CML blast crisis Blood 103 2004 4010 4022
4. J.M. Goldman Chronic myeloid leukemia: reversing the chronic phase J Clin Oncol 28 2010 363 365
5. P.P. Piccaluga, S. Paolini, and C. Bertuzzi First-line treatment of chronic myeloid leukemia with nilotinib: critical evaluation J Blood Med 3 2012 151 156
6. X. Huang, J. Cortes, and H. Kantarjian Estimations of the increasing prevalence and plateau prevalence of chronic myeloid leukemia in the era of tyrosine kinase inhibitor therapy Cancer 118 2012 3123 3127
7. Haematological Malignancy Research Network. Chronic myelogenous leukaemia. Available from: http://www.hmrn.org/Statistics/Cancer-Info.aspx?id=1. [Accessed July 23, 2012].
8. B. Rachet, C. Maringe, and U. Nur Population-based cancer survival trends in England and Wales up to 2007: an assessment of the NHS cancer plan for England Lancet Oncol 10 2009 351 369
9. Cancer, Research, UK. Leukaemia incidence statistics. 2011. Available from: http://www.cancerresearchuk.org/cancer-info/cancerstats/types/leukaemia/ incidence/uk-leukaemia-incidence-statistics#Prevalence. [Accessed February 26, 2013].
10. P. Nowell A minute chromosome in human chronic granulocytic leukemia Science 132 1960 1497
11. R. Hehlmann, A. Hochhaus, and M. Baccarani Chronic myeloid leukaemia Lancet 370 2007 342 350
12. R. Ren Mechanisms of BCR-ABL in the pathogenesis of chronic myelogenous leukaemia Nat Rev Cancer 5 2005 172 183
13. B.J. Druker Translation of the Philadelphia chromosome into therapy for CML Blood 112 2008 4808 4817
14. S.G. O'Brien, F. Guilhot, and R.A. Larson Imatinib compared with interferon and low-dose cytarabine for newly diagnosed chronic-phase chronic myeloid leukemia N Engl J Med 348 2003 994 1004
15. M. Baccarani, J. Cortes, and F. Pane Chronic myeloid leukemia: an update of concepts and management recommendations of European LeukemiaNet J Clin Oncol 27 2009 6041 6051
16. A. Hochhaus, H.M. Kantarjian, and M. Baccarani Dasatinib induces notable hematologic and cytogenetic responses in chronic-phase chronic myeloid leukemia after failure of imatinib therapy Blood 109 2007 2303 2309
17. J. Cortes, P. Rousselot, and D.W. Kim Dasatinib induces complete hematologic and cytogenetic responses in patients with imatinib-resistant or -intolerant chronic myeloid leukemia in blast crisis Blood 109 2007 3207 3213
18. T.P. Hughes, J. Kaeda, and S. Branford Frequency of major molecular responses to imatinib or interferon alfa plus cytarabine in newly diagnosed chronic myeloid leukemia N Engl J Med 349 2003 1423 1432
19. R. Capdeville, E. Buchdunger, and J. Zimmermann Glivec (STI571, imatinib), a rationally developed, targeted anticancer drug Nat Rev Drug Discov 1 2002 493 502
20. National Institute for Health and Care Excellence. Leukaemia (Chronic Myeloid) - Imatinib (TA70). 2003. Available from: http://www.nice.org.uk/ta70. [Accessed July 23, 2012].
21. H. Kantarjian, N.P. Shah, and A. Hochhaus Dasatinib versus imatinib in newly diagnosed chronic-phase chronic myeloid leukemia N Engl J Med 362 2010 2260 2270
22. J.E. Cortes, D. Jones, and S. O'Brien Nilotinib as front-line treatment for patients with chronic myeloid leukemia in early chronic phase J Clin Oncol 28 2010 392 397
23. G. Rosti, F. Palandri, and F. Castagnetti Nilotinib for the frontline treatment of Ph(+) chronic myeloid leukemia Blood 114 2009 4933 4938
24. G. Saglio, D.W. Kim, and S. Issaragrisil Nilotinib versus imatinib for newly diagnosed chronic myeloid leukemia N Engl J Med 362 2010 2251 2259
25. J. Cortes, and H. Kantarjian How I treat newly diagnosed chronic phase CML Blood 120 7 2012 1390 1397
26. National Institute for Health and Care Excellence. NICE recommends nilotinib and standard dose imatinib for first line chronic myeloid leukaemia. 2012. Available from: http://www.nice.org.uk/newsroom/pressreleases/ NilotinibAndImatinibForCMLDraftGuidance.jsp. [Accessed July 23, 2012].
27. Biomarkers Definition Working Group Biomarkers and surrogate endpoints: preferred definitions and conceptual framework Clin Pharmacol Ther 69 2001 7
28. National Institute for Health and Care Excellence. Leukaemia (chronic myeloid, first line) - dasatinib, nilotinib and standard-dose imatinib: final appraisal determination. 2012. Available from: http://guidance.nice.org.uk/TA/ Wave24/15/FAD. [Accessed July 23, 2012].
29. D. Moher, A. Liberati, and J. Tetzlaff Preferred reporting items for systematic reviews and meta-analyses: the PRISMA statement BMJ 339 2009 b2535
30. K. Dalziel, A. Round, and K. Stein Effectiveness and cost-effectiveness of imatinib for first-line treatment of chronic myeloid leukaemia in chronic phase: a systematic review and economic analysis Health Technol Assess (Winchester, England) 8 iii 2004 1 120
31. Centre for Reviews and Dissemination. Undertaking Systematic Reviews of Research on Effectiveness: CRD's Guidance for Carrying Out or Commissioning Reviews (2nd ed.). CRD Report 4. York: NHS Centre for Reviews and Dissemination, 2001.
32. J. Elston, and R.S. Taylor Use of surrogate outcomes in cost-effectiveness models: a review of United Kingdom health technology assessment reports Int J Technol Assess Health Care 25 2009 6 13
33. J.R. Anderson, K.C. Cain, and R.D. Gelber Analysis of survival by tumor response and other comparisons of time-to-event by outcome variables J Clin Oncol 26 2008 3913 3915
34. E. Wilson Probable inference, the law of succession, and statistical inference J Am Stat Assoc 22 1927 4
35. G. Lu, and A.E. Ades Assessing evidence inconsistency in mixed treatment comparisons J Am Stat Assoc 101 2006 22
36. D.T.A. Spiegelhalter, N. Best, and D. Lunn WinBUGS User Manual: Version 1.4 2003 MRC Biostatistics Unit Cambridge
37. A. Sutton, K. Abrams, and D. Jones Methods for Meta-Analysis in Medical Research 2000 Wiley Chichester
38. T. Pavey, M. Hoyle, and O. Ciani Dasatinib, nilotinib and standard-dose imatinib for the first-line treatment of chronic myeloid leukaemia: systematic reviews and economic analyses Health Technol Assess (Winchester, England) 16 iii-iv 2012 1 277
39. GAD. Life tables. Available from: http://www.gad.gov.uk/ Demography%20Data/Life%20Tables/. [Accessed March 13, 2011].
40. H.M. Kantarjian, A. Hochhaus, and G. Saglio 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 12 2011 841 851
41. R.A. Larson, A. Hochhaus, and T.P. Hughes Nilotinib vs imatinib in patients with newly diagnosed Philadelphia chromosome-positive chronic myeloid leukemia in chronic phase: ENESTnd 3-year follow-up Leukemia 26 2012 2197 2203
42. H.M. Kantarjian, N.P. Shah, and J.E. Cortes Dasatinib or imatinib in newly diagnosed chronic-phase chronic myeloid leukemia: 2-year follow-up from a randomized phase 3 trial (DASISION) Blood 119 2012 1123 1129
43. T.P. Hughes, A. Hochhaus, and S. Branford Long-term prognostic significance 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 116 2010 3758 3765
44. L. Roy, J. Guilhot, and T. Krahnke Survival advantage from imatinib compared with the combination interferon-alpha plus cytarabine in chronic-phase chronic myelogenous leukemia: historical comparison between two phase 3 trials Blood 108 2006 1478 1484
45. E. Jabbour, H. Kantarjian, and S. O'Brien The achievement of an early complete cytogenetic response is a major determinant for outcome in patients with early chronic phase chronic myeloid leukemia treated with tyrosine kinase inhibitors Blood 118 2011 4541 4546 (quiz 759)
46. H. de Lavallade, J.F. Apperley, and J.S. Khorashad Imatinib for newly diagnosed patients with chronic myeloid leukemia: incidence of sustained responses in an intention-to-treat analysis J Clin Oncol 26 2008 3358 3363
47. H. Kantarjian, S. O'Brien, and J. Shan Cytogenetic and molecular responses and outcome in chronic myelogenous leukemia: need for new response definitions? Cancer 112 2008 837 845
48. R. Hehlmann, M. Lauseker, and S. Jung-Munkwitz Tolerability-adapted imatinib 800 mg/d versus 400 mg/d versus 400 mg/d plus interferon-alpha in newly diagnosed chronic myeloid leukemia J Clin Oncol 29 12 2011 1634 1642
49. S. Rajappa, L. Varadpande, and T. Paul Imatinib mesylate in early chronic phase chronic myeloid leukemia: experience from a developing country Leukemia Lymphoma 49 2008 554 558
50. A. Hochhaus, S.G. O'Brien, and F. Guilhot Six-year follow-up of patients receiving imatinib for the first-line treatment of chronic myeloid leukemia Leukemia 23 2009 1054 1061
51. M.W. Deininger, J.M. Goldman, and J.V. Melo The molecular biology of chronic myeloid leukemia Blood 96 2000 3343 3356
52. S. Chakraborty, J.M. Stark, and C.L. Sun Chronic myelogenous leukemia stem and progenitor cells demonstrate chromosomal instability related to repeated breakage-fusion-bridge cycles mediated by increased nonhomologous end joining Blood 119 2012 6187 6197
53. M. Carroll BCR/ABL and chromosomal instability: debate resolved Blood 119 2012 6180 6181
54. K. Futosi, T. Nemeth, and R. Pick Dasatinib inhibits proinflammatory functions of mature human neutrophils Blood 119 2012 4981 4991
55. A. Zarbock The shady side of dasatinib Blood 119 2012 4817 4818
56. H.C. Bucher, G.H. Guyatt, and D.J. Cook Users' guides to the medical literature, XIX: applying clinical trial results. A. How to use an article measuring the effect of an intervention on surrogate end points. Evidence-Based Medicine Working Group JAMA 282 1999 771 778
57. M.N. Lassere The Biomarker-Surrogacy Evaluation Schema: a review of the biomarker-surrogate literature and a proposal for a criterion-based, quantitative, multidimensional hierarchical levels of evidence schema for evaluating the status of biomarkers as surrogate endpoints Stat Methods Med Res 17 2008 303 340
58. T. Burzykowski, G. Molenberghs, and M. Buyse The Evaluation of Surrogate Endpoints 2005 Springer Science + Business Media, Inc. US
59. K.J. Anstrom, S.D. Reed, and A.S. Allen Long-term survival estimates for imatinib versus interferon-alpha plus low-dose cytarabine for patients with newly diagnosed chronic-phase chronic myeloid leukemia Cancer 101 2004 2584 2592
60. M. Baccarani, G. Rosti, and A. de Vivo A randomized study of interferon-alpha versus interferon-alpha and low-dose arabinosyl cytosine in chronic myeloid leukemia Blood 99 2002 1527 1535
61. F. Bonifazi, A. de Vivo, and G. Rosti Chronic myeloid leukemia and interferon-alpha: a study of complete cytogenetic responders Blood 98 2001 3074 3081
62. F. Guilhot, C. Chastang, and M. Michallet Interferon alfa-2b combined with cytarabine versus interferon alone in chronic myelogenous leukemia. French Chronic Myeloid Leukemia Study Group N Engl J Med 337 1997 223 229
63. H.M. Kantarjian, S. O'Brien, and J.E. Cortes Complete cytogenetic and molecular responses to interferon-alpha-based therapy for chronic myelogenous leukemia are associated with excellent long-term prognosis Cancer 97 2003 1033 1041
64. R.J. Schrover, M.A. Adena, and R. De Abreu Lourenco Development of a predictive population survival model according to the cytogenetic response rate for patients with chronic myeloid leukemia in the chronic phase Leukemia Lymphoma 47 2006 1069 1081
65. R. Gurion, A. Gafter-Gvili, and L. Vidal Has the time for first-line treatment with second generation tyrosinekinase inhibitors in patients with chronic myelogenous leukemia already come? Systematic review and meta-analysis Haematologica 98 1 2013 95 102
66. European Medicines Agency. Glivec: EPAR - scientific discussion. 2005. Available from: http://www.ema.europa.eu/ema/index.jsp?curl=pages/medicines/ human/medicines/000406/human-med-000808.jsp&mid=WC0b01ac058001d124. [Accessed July 23, 2012].
67. R.D. Riley, P.C. Lambert, and G. Abo-Zaid Meta-analysis of individual participant data: rationale, conduct, and reporting BMJ 340 2010 c221
68. J.E. Cortes Not only response but early response to tyrosine kinase inhibitors in chronic myeloid leukemia J Clin Oncol 30 2012 223 224
69. F.M. Clement, A. Harris, and J.J. Li Using effectiveness and cost-effectiveness to make drug coverage decisions: a comparison of Britain, Australia, and Canada JAMA 302 2009 1437 1443
70. Institute for Quality and Efficiency in Healthcare. Validity of surrogate endpoints in oncology. 2011. Available from: https://www.iqwig.de/a10-05- validity-of-surrogate-endpoints-in.986.en.html?tid=1325&random=f6799f. [Accessed July 23, 2012].
71. National Institute for Health and Care Excellence. Review of the Guide to the Methods of Technology Appraisal. 2012. Available from: http://www.nice.org. uk/aboutnice/howwework/devnicetech/TAMethodsGuideReview.jsp?domedia=1&mid= CB13DD0D-19B9-E0B5-D4AB0011AEE2B0E7. [Accessed July 23, 2012].