메뉴 건너뛰기




Volumn 65, Issue 9, 2012, Pages 940-945

Treatment effects on patient-important outcomes can be small, even with large effects on surrogate markers

Author keywords

Biostatistics; Clinical trials; Methodology; Patient important outcomes; Surrogate markers; Treatment effect

Indexed keywords

ALPHA INTERFERON; LAMIVUDINE;

EID: 84864286590     PISSN: 08954356     EISSN: 18785921     Source Type: Journal    
DOI: 10.1016/j.jclinepi.2012.02.012     Document Type: Article
Times cited : (16)

References (41)
  • 1
    • 0024520844 scopus 로고
    • Surrogate endpoints in clinical trials: Definition and operational criteria
    • R.L. Prentice Surrogate endpoints in clinical trials: definition and operational criteria Stat Med 8 1989 431 440
    • (1989) Stat Med , vol.8 , pp. 431-440
    • Prentice, R.L.1
  • 2
    • 0000232101 scopus 로고
    • On the exact variance of products
    • L.A. Goodman On the exact variance of products J Am Stat Assoc 55 1960 708 713
    • (1960) J Am Stat Assoc , vol.55 , pp. 708-713
    • Goodman, L.A.1
  • 3
    • 33751514752 scopus 로고    scopus 로고
    • Interferon therapy in HBeAG positive chronic hepatitis reduces progression to cirrhosis and hepatocellular carcinoma
    • S.M. Lin, M.L. Yu, C.M. Lee, R.N. Chien, I.S. Sheen, and C.M. Chu Interferon therapy in HBeAG positive chronic hepatitis reduces progression to cirrhosis and hepatocellular carcinoma J Hepatol 46 2007 45 52
    • (2007) J Hepatol , vol.46 , pp. 45-52
    • Lin, S.M.1    Yu, M.L.2    Lee, C.M.3    Chien, R.N.4    Sheen, I.S.5    Chu, C.M.6
  • 4
    • 0034962643 scopus 로고    scopus 로고
    • Long-term follow-up of interferon alfa treatment in Chinese patients with chronic hepatitis b infection: The effect on hepatitis b e antigen seroconversion and the development of cirrhosis-related complications
    • M.F. Yuen, C.K. Hui, C.C. Cheng, C.H. Wu, Y.P. Lai, and C.L. Lai Long-term follow-up of interferon alfa treatment in Chinese patients with chronic hepatitis b infection: the effect on hepatitis b e antigen seroconversion and the development of cirrhosis-related complications J Hepatol 34 2001 139 145
    • (2001) J Hepatol , vol.34 , pp. 139-145
    • Yuen, M.F.1    Hui, C.K.2    Cheng, C.C.3    Wu, C.H.4    Lai, Y.P.5    Lai, C.L.6
  • 5
    • 0034105307 scopus 로고    scopus 로고
    • Lamivudine and alpha interferon combination treatment of patients with chronic hepatitis B infection: A randomised trial
    • S.W. Schalm, J. Heathcote, J. Cianciara, G. Farrell, M. Sherman, and B. Willems Lamivudine and alpha interferon combination treatment of patients with chronic hepatitis B infection: a randomised trial Gut 46 2000 562 568
    • (2000) Gut , vol.46 , pp. 562-568
    • Schalm, S.W.1    Heathcote, J.2    Cianciara, J.3    Farrell, G.4    Sherman, M.5    Willems, B.6
  • 6
    • 21244447705 scopus 로고    scopus 로고
    • Peginterferon alfa-2a, lamivudine, and the combination for HBeAG-positive chronic hepatitis b
    • G.K. Lau, T. Piratvisuth, K.X. Luo, P. Marcellin, S. Thongsawat, and G. Cooksley Peginterferon alfa-2a, lamivudine, and the combination for HBeAG-positive chronic hepatitis b N Engl J Med 352 2005 2682 2695
    • (2005) N Engl J Med , vol.352 , pp. 2682-2695
    • Lau, G.K.1    Piratvisuth, T.2    Luo, K.X.3    Marcellin, P.4    Thongsawat, S.5    Cooksley, G.6
  • 7
    • 0030777744 scopus 로고    scopus 로고
    • Meta-analysis for the evaluation of potential surrogate markers
    • M.J. Daniels, and M.D. Hughes Meta-analysis for the evaluation of potential surrogate markers Stat Med 16 1996 1965 1982
    • (1996) Stat Med , vol.16 , pp. 1965-1982
    • Daniels, M.J.1    Hughes, M.D.2
  • 8
    • 0031708453 scopus 로고    scopus 로고
    • Criteria for the validation of surrogate endpoints in randomized experiments
    • M. Buyse, and G. Molenbergs Criteria for the validation of surrogate endpoints in randomized experiments Biometrics 54 1998 1014 1029
    • (1998) Biometrics , vol.54 , pp. 1014-1029
    • Buyse, M.1    Molenbergs, G.2
  • 10
    • 2942525565 scopus 로고    scopus 로고
    • A perspective on surrogate endpoints in controlled clinical trials
    • G. Molenberghs, T. Burzykowski, and A. Alonso A perspective on surrogate endpoints in controlled clinical trials Stat Methods Med Res 13 2004 177 206
    • (2004) Stat Methods Med Res , vol.13 , pp. 177-206
    • Molenberghs, G.1    Burzykowski, T.2    Alonso, A.3
  • 11
    • 34547120211 scopus 로고    scopus 로고
    • Quantitative science methods for biomarker validation in chemoprevention trials
    • L. Freedman Quantitative science methods for biomarker validation in chemoprevention trials Cancer Biomark 3 2007 135 140
    • (2007) Cancer Biomark , vol.3 , pp. 135-140
    • Freedman, L.1
  • 12
    • 84859003632 scopus 로고    scopus 로고
    • Surrogates under scrutiny: Fallible correlations, fatal consequences
    • R. Moynihan Surrogates under scrutiny: fallible correlations, fatal consequences BMJ 343 2011 d5160
    • (2011) BMJ , vol.343 , pp. 5160
    • Moynihan, R.1
  • 13
    • 0030268577 scopus 로고    scopus 로고
    • Surrogate end points in clinical trials: Are we being misled?
    • T.R. Fleming, and D.L. DeMets Surrogate end points in clinical trials: are we being misled? Ann Intern Med 125 1996 605 613
    • (1996) Ann Intern Med , vol.125 , pp. 605-613
    • Fleming, T.R.1    Demets, D.L.2
  • 14
    • 82955210363 scopus 로고    scopus 로고
    • The long march to surrogates of meaningful clinical outcomes in MS trials - Are we there yet?
    • J.S. Wolinsky, and C.A. Beck The long march to surrogates of meaningful clinical outcomes in MS trials - are we there yet? Neurology 77 2011 1658
    • (2011) Neurology , vol.77 , pp. 1658
    • Wolinsky, J.S.1    Beck, C.A.2
  • 16
    • 60749130280 scopus 로고    scopus 로고
    • Potential surrogate endpoints for prostate cancer survival: Analysis of a phase III randomized trial
    • M.E. Ray, K. Bae, M.H.A. Hussain, G.E. Hanks, W.U. Shipley, and H.M. Snadler Potential surrogate endpoints for prostate cancer survival: analysis of a phase III randomized trial J Natl Cancer Inst 101 2009 228 236
    • (2009) J Natl Cancer Inst , vol.101 , pp. 228-236
    • Ray, M.E.1    Bae, K.2    Hussain, M.H.A.3    Hanks, G.E.4    Shipley, W.U.5    Snadler, H.M.6
  • 17
    • 63149095503 scopus 로고    scopus 로고
    • Surrogate endpoints for overall survival in locally advanced head and neck cancer: Meta-analyses of individual patient data
    • S. Michiels, A. Le Maitre, M. Buyse, T. Burzykowski, E. Maillard, and J. Bogaerts Surrogate endpoints for overall survival in locally advanced head and neck cancer: meta-analyses of individual patient data Lancet Oncol 10 2009 341 350
    • (2009) Lancet Oncol , vol.10 , pp. 341-350
    • Michiels, S.1    Le Maitre, A.2    Buyse, M.3    Burzykowski, T.4    Maillard, E.5    Bogaerts, J.6
  • 18
    • 63149103474 scopus 로고    scopus 로고
    • Surrogate endpoints in head and neck cancer
    • L. Chen, and E.E.W. Cohen Surrogate endpoints in head and neck cancer Lancet Oncol 10 2009 309 310
    • (2009) Lancet Oncol , vol.10 , pp. 309-310
    • Chen, L.1    Cohen, E.E.W.2
  • 20
    • 70350156608 scopus 로고    scopus 로고
    • Use of meta-analysis for the validation of surrogate endpoints and biomarkers in cancer trials
    • M. Buyse Use of meta-analysis for the validation of surrogate endpoints and biomarkers in cancer trials Cancer J 15 2009 421 425
    • (2009) Cancer J , vol.15 , pp. 421-425
    • Buyse, M.1
  • 21
    • 77949321968 scopus 로고    scopus 로고
    • Progression-free survival as surrogate and as true end point: Insights from the breast and colorectal cancer literature
    • E.D. Saad, A. Katz, P.M. Hoff, and M. Buyse Progression-free survival as surrogate and as true end point: insights from the breast and colorectal cancer literature Ann Oncol 21 2010 7 12
    • (2010) Ann Oncol , vol.21 , pp. 7-12
    • Saad, E.D.1    Katz, A.2    Hoff, P.M.3    Buyse, M.4
  • 22
    • 79961097283 scopus 로고    scopus 로고
    • Leukemia-free survival as a surrogate end point for overall survival in the evaluation of maintenance therapy for patients with acute myeloid leukemia in complete remission
    • M. Buyse, S. Michiels, P. Squifflet, K.J. Lucchesi, K. Hellstrand, and M.L. Brune Leukemia-free survival as a surrogate end point for overall survival in the evaluation of maintenance therapy for patients with acute myeloid leukemia in complete remission Haematologica 96 2011 1106 1112
    • (2011) Haematologica , vol.96 , pp. 1106-1112
    • Buyse, M.1    Michiels, S.2    Squifflet, P.3    Lucchesi, K.J.4    Hellstrand, K.5    Brune, M.L.6
  • 23
    • 0041564148 scopus 로고    scopus 로고
    • Biochemical failure as a determinant of distant metastasis and death in prostate cancer treated with radiotherapy
    • A. Pollack, A.L. Hanlon, B. Movsas, G.E. Hanks, R. Uzzo, and E.M. Horwitz Biochemical failure as a determinant of distant metastasis and death in prostate cancer treated with radiotherapy Int J Radiat Oncol Biol Phys 57 2003 19 23
    • (2003) Int J Radiat Oncol Biol Phys , vol.57 , pp. 19-23
    • Pollack, A.1    Hanlon, A.L.2    Movsas, B.3    Hanks, G.E.4    Uzzo, R.5    Horwitz, E.M.6
  • 24
    • 33750348034 scopus 로고    scopus 로고
    • Posttreatment prostatic-specific antigen doubling time as a surrogate endpoint for prostate cancer-specific survival: An analysis of radiation therapy oncology group protocol 92-02
    • R.K. Valicenti, M. DeSilvio, G.E. Hanks, A. Porter, H. Brereton, and S.A. Rosenthal Posttreatment prostatic-specific antigen doubling time as a surrogate endpoint for prostate cancer-specific survival: an analysis of radiation therapy oncology group protocol 92-02 Int J Radiat Oncol Biol Phys 66 2006 1064 1071
    • (2006) Int J Radiat Oncol Biol Phys , vol.66 , pp. 1064-1071
    • Valicenti, R.K.1    Desilvio, M.2    Hanks, G.E.3    Porter, A.4    Brereton, H.5    Rosenthal, S.A.6
  • 26
    • 0024321898 scopus 로고
    • Preliminary Report: Effect of encainide and flecainide on mortality in a randomized trial of arrhythmia suppression after myocardial infarction
    • The Cardiac Arrhythmia Suppression Trial (CAST) Investigators
    • The Cardiac Arrhythmia Suppression Trial (CAST) Investigators Preliminary Report: effect of encainide and flecainide on mortality in a randomized trial of arrhythmia suppression after myocardial infarction N Engl J Med 321 1989 406 412
    • (1989) N Engl J Med , vol.321 , pp. 406-412
  • 27
    • 0026752875 scopus 로고
    • Effect of the antiarrhythmic agent moricizine on survival after myocardial infarction
    • The Cardiac Arrhythmia Suppression Trial II (CAST) Investigators
    • The Cardiac Arrhythmia Suppression Trial II (CAST) Investigators Effect of the antiarrhythmic agent moricizine on survival after myocardial infarction N Engl J Med 327 1992 227 233
    • (1992) N Engl J Med , vol.327 , pp. 227-233
  • 28
    • 0033603785 scopus 로고    scopus 로고
    • Surrogate end points, health outcomes, and the drug-approval process for the treatment of risk factors for cardiovascular disease
    • B.M. Psaty, N.S. Weiss, C.D. Furberg, T.D. Koepsell, D.S. Siscovick, and F.R. Rosendaal Surrogate end points, health outcomes, and the drug-approval process for the treatment of risk factors for cardiovascular disease J Am Med Assoc 282 2007 786 795
    • (2007) J Am Med Assoc , vol.282 , pp. 786-795
    • Psaty, B.M.1    Weiss, N.S.2    Furberg, C.D.3    Koepsell, T.D.4    Siscovick, D.S.5    Rosendaal, F.R.6
  • 29
    • 0033603814 scopus 로고    scopus 로고
    • Are surrogate markers adequate to assess cardiovascular disease drugs?
    • R. Temple Are surrogate markers adequate to assess cardiovascular disease drugs? J Am Med Assoc 282 1999 790 795
    • (1999) J Am Med Assoc , vol.282 , pp. 790-795
    • Temple, R.1
  • 31
    • 79957864517 scopus 로고    scopus 로고
    • Strengths and weaknesses of renal markers as risk factors and surrogate markers
    • K.R. Johnson Strengths and weaknesses of renal markers as risk factors and surrogate markers Kidney Int 79 2011 1272 1274
    • (2011) Kidney Int , vol.79 , pp. 1272-1274
    • Johnson, K.R.1
  • 32
    • 42949145995 scopus 로고    scopus 로고
    • Evaluation of tumor response, disease control, progression-free survival, and time to progression as potential surrogate end points in metastatic breast cancer
    • T. Burzykowski, M. Buyse, M.J. Piccart-Gebhard, G. Sledge, J. Carmichael, and H.-J. Lück Evaluation of tumor response, disease control, progression-free survival, and time to progression as potential surrogate end points in metastatic breast cancer J Clin Oncol 26 2008 987 992
    • (2008) J Clin Oncol , vol.26 , pp. 987-992
    • Burzykowski, T.1    Buyse, M.2    Piccart-Gebhard, M.J.3    Sledge, G.4    Carmichael, J.5    Lück, H.-J.6
  • 34
    • 66949158002 scopus 로고    scopus 로고
    • Related causal frameworks for surrogate outcomes
    • M.M. Joffee, and T. Greene Related causal frameworks for surrogate outcomes Biometrics 65 2008 530 538
    • (2008) Biometrics , vol.65 , pp. 530-538
    • Joffee, M.M.1    Greene, T.2
  • 35
    • 84864286778 scopus 로고    scopus 로고
    • The promise and peril of surrogate endpoints in cancer research
    • T. Burzykowski, G. Molenberghs, M. Buyse, Springer New York, NY
    • A. Schatzkin, M. Gail, and L. Freedman The promise and peril of surrogate endpoints in cancer research T. Burzykowski, G. Molenberghs, M. Buyse, The evaluation of surrogate endpoints 2005 Springer New York, NY 349 366
    • (2005) The Evaluation of Surrogate Endpoints , pp. 349-366
    • Schatzkin, A.1    Gail, M.2    Freedman, L.3
  • 36
    • 33748746412 scopus 로고    scopus 로고
    • The history of surrogate endpoint validation
    • T. Burzykowski, G. Molenberghs, M. Buyse, Springer New York, NY
    • G. Molenbergh, M. Buyse, and T. Burzykowski The history of surrogate endpoint validation T. Burzykowski, G. Molenberghs, M. Buyse, The evaluation of surrogate endpoints 2005 Springer New York, NY 67 82
    • (2005) The Evaluation of Surrogate Endpoints , pp. 67-82
    • Molenbergh, G.1    Buyse, M.2    Burzykowski, T.3
  • 37
    • 0034802767 scopus 로고    scopus 로고
    • Considerations in the evaluation of surrogate endpoints in clinical trials: Summary of a National Institutes of Health workshop
    • V.G. De Gruttola, P. Clax, D.L. DeMets, G.J. Downing, S.S. Ellenberg, and L. Friedman Considerations in the evaluation of surrogate endpoints in clinical trials: summary of a National Institutes of Health workshop Control Clin Trials 22 2001 485 502
    • (2001) Control Clin Trials , vol.22 , pp. 485-502
    • De Gruttola, V.G.1    Clax, P.2    Demets, D.L.3    Downing, G.J.4    Ellenberg, S.S.5    Friedman, L.6
  • 38
    • 79960559978 scopus 로고    scopus 로고
    • Sufficient conditions for concluding surrogacy based on observed data
    • Z. Wu, P. He, and G. Zhi Sufficient conditions for concluding surrogacy based on observed data Stat Med 30 2010 2422 2434
    • (2010) Stat Med , vol.30 , pp. 2422-2434
    • Wu, Z.1    He, P.2    Zhi, G.3
  • 39
    • 33748751897 scopus 로고    scopus 로고
    • Surrogate threshold effect: An alternative measure for meta-analytic surrogate endpoint validation
    • T. Burzykowski, and M. Buyse Surrogate threshold effect: an alternative measure for meta-analytic surrogate endpoint validation Pharm Stat 5 2006 1973 1986
    • (2006) Pharm Stat , vol.5 , pp. 1973-1986
    • Burzykowski, T.1    Buyse, M.2
  • 40
    • 84858865962 scopus 로고    scopus 로고
    • Predicting treatment effect from surrogate endpoints and historical trials: An extrapolation involving probabilities of a binary outcome or survival to a specific time
    • 10.1111/j.1541-0420.2011.01646.x
    • S.G. Baker, D.J. Sargent, M. Buyse, and T. Burzykowski Predicting treatment effect from surrogate endpoints and historical trials: an extrapolation involving probabilities of a binary outcome or survival to a specific time Biometrics 68 2012 248 257 10.1111/j.1541-0420.2011.01646.x
    • (2012) Biometrics , vol.68 , pp. 248-257
    • Baker, S.G.1    Sargent, D.J.2    Buyse, M.3    Burzykowski, T.4
  • 41
    • 0033895537 scopus 로고    scopus 로고
    • Intermediate markers as surrogate endpoints in cancer research
    • A. Schatzkin Intermediate markers as surrogate endpoints in cancer research Hematol Oncol Clin North Am 14 2000 887 905
    • (2000) Hematol Oncol Clin North Am , vol.14 , pp. 887-905
    • Schatzkin, A.1


* 이 정보는 Elsevier사의 SCOPUS DB에서 KISTI가 분석하여 추출한 것입니다.