Are the results of randomized trials influenced by preference effects? Part I. Findings from a systematic review

Optimizing Health: Improving the Value of Healthcare Delivery

Volumn , Issue , 2006, Pages 265-291

  Stengel, Dirk ^a   Sehouli, Jalid ^b   Porzsolt, Franz ^c  

^a Clinical Epidemiology Division   (Germany)

^b CHARITÉ UNIVERSITÄTSMEDIZIN BERLIN   (Germany)

^c UNIVERSITY HOSPITAL ULM   (Germany)

Author keywords

[No Author keywords available]

Indexed keywords

EID: 58149386142     PISSN: None     EISSN: None     Source Type: Book    
DOI: 10.1007/978-0-387-33921-4_27     Document Type: Chapter

References (90)

1. Andrews, G. (1999). Randomized controlled trials in psychiatry: important but poorly accepted. British Medical Journal, 319, 562-564.
2. Antman, K., Suit, H., Amato, D., Corson, J., Wood, W., Proppe, K., et al. (1984). Preliminary results of a randomized trial of adjuvant doxorubicin for sarcomas: lack of apparent difference between treatment groups. Journal of Clinical Oncology, 2, 601-608.
3. Antman, K., Amato, D., Wood, W., Carson, J., Suit, H., Proppe, K., et al. (1985). Selection bias in clinical trials. Journal of Clinical Oncology, 3, 1142-1147.
4. Ashok, P.W., Kidd, A., Flett, G.M., Fitzmaurice, A., Graham, W., Templeton, A. (2002). A randomized comparison of medical abortion and surgical vacuum aspiration at 10-13 weeks gestation. Human Reproduction, 17, 92-98.
5. Bain, C., Cooper, K.G., Parkin, D.E. (2001). A partially randomized patient preference trial of microwave endometrial ablation using local anaesthesia and intravenous sedation or general anaesthesia: a pilot study. Gynaecological Endoscopy, 10, 223-228.
6. Bakker, A., van Dyck, R., Spinhoven, P., van Balkom, A.J.L.M. (1999). Paroxetine, clomipramine, and cognitive therapy in the treatment of panic disorder. Journal of Clinical Psychiatry, 60, 831-838.
7. Bakker, A., Spinhoven, P., van Balkom, A.J., Vleugel, L., van Dyck, R. (2000). Cognitive therapy by allocation versus cognitive therapy by preference in the treatment of panic disorder. Psychotherapy and Psychosomatics, 69, 240-243.
8. BARI Investigators (1996). Comparison of coronary bypass surgery with angioplasty in patients with multivessel disease. New England Journal of Medicine, 335, 217-225.
9. Bedi, N., Chilvers, C., Churchill, R., Dewey, M., Duggan, C., Fielding, K., et al. (2000). Assessing effectiveness of treatment of depression in primary care: partially randomized preference trial. British Journal of Psychiatry, 177, 312-318.
10. Benson, K., Hartz, A.J. (2000). A comparison of observational studies and randomized, controlled trials. New England Journal of Medicine, 342, 1878-1886.
11. Bijker, N., Peterse, J.L., Fentiman, I.S., Julien, J.P., Hart, A.A., Avril, A., et al. (2002). Effects of patient selection on the applicability of results from a randomized clinical trial (EORTC 10853) investigating breast-conserving therapy for DCIS. British Journal of Cancer, 87, 615-620.
12. Bower, P., Byford, S., Sibbald, B., Ward, E., King, M., Lloyd, M., et al. (2000). Randomized controlled trial of non-directive counselling, cognitive-behaviour therapy, and usual general practitioner care for patients with depression. II. Cost effectiveness. British Medical Journal, 321, 1389-1392.
13. Brewin, C.R., Bradley, C. (1989). Patient preferences and randomized clinical trials. British Medical Journal, 299, 313-315.
14. Brooks, M.M., Jones, R.H., Bach, R.G., Chaitman, B.R., Kern, M.J., Orszulak, T.A., et al. (2000). Predictors of mortality and mortality from cardiac causes in the Bypass Angioplasty Revascularization Investigation (BARI) randomized trial and registry: for the BARI Investigators. Circulation, 101, 2682-2689.
15. CASS Principal Investigators and Their Associates (1984). Coronary artery surgery study (CASS): a randomized trial of coronary artery bypass surgery; comparability of entry characteristics and survival in randomized patients and nonrandomized patients meeting randomization criteria. Journal of the American College of Cardiology, 3, 114-128.
16. Chard, J.A., Lilford, R. J. (1998). The use of equipoise in clinical trials. Social Science and Medicine, 47, 891-898.
17. Chaitman, B.R., Rosen, A.D., Williams, D.O., Bourassa, M.G., Aguirre, F.V., Pitt, B., et al. (1997). Myocardial infarction and cardiac mortality in the Bypass Angioplasty Revascularization Investigation (BARI) randomized trial. Circulation, 96, 2162-2170.
18. Chilvers, C., Dewey, M., Fielding, K., Gretton, V., Miller, P., Palmer, B., et al. (2001). Antidepressant drugs and generic counselling for treatment of major depression in primary care: randomized trial with patient preference arms. British Medical Journal, 322, 772-775.
19. Concato, J., Shah, N., Horwitz, R.I. (2000). Randomized, controlled trials, observational studies, and the hierarchy of research designs. New England Journal of Medicine, 342, 1887-1892.
20. Cooper, K.G., Grant, A.M., Garratt, A.M. (1997a). The impact of using a partially randomized patient preference design when evaluating alternative managements for heavy menstrual bleeding. British Journal of Obstetrics and Gynaecology, 104, 1367-1373.
21. Cooper, K.G., Parkin, D.E., Garratt, A.M., Grant, A.M. (1997b). A randomized comparison of medical and hysteroscopic management in women consulting a gynaecologist for treatment of heavy menstrual loss. British Journal of Obstetrics and Gynaecology, 104, 1360-1366.
22. Coward, D.D. (2002). Partial randomization design in a support group intervention study. Western Journal of Nursing Research, 24, 406-421.
23. Coward, D.D. (2003). Facilitation of self-transcendence in a breast cancer support group. II. Oncology Nursing Forum, 30, 291-300.
24. De C Williams, A.C., Nicholas, M.K., Richardson, P.H., Pither, C.E., Fernandes, J. (1999). Generalizing from a controlled trial: the effects of patient preference versus randomization on the outcome of inpatient versus outpatient chronic pain management. Pain, 83, 57-5.
25. Detre, K.M., Guo, P., Holubkov, R., Califf, R.M., Sopko, G., Bach, R., et al. (1999). Coronary revascularization in diabetic patients: a comparison of the randomized and observational components of the Bypass Angioplasty Revascularization Investigation (BARI). Circulation, 99, 633-640.
26. Feit, F., Brooks, M.M., Sopko, G., Keller, N.M., Rosen, A., Krone, R., et al. (2000). Longterm clinical outcome in the Bypass Angioplasty Revascularization Investigation Registry: comparison with the randomized trial; BARI Investigators. Circulation, 101, 2795-2802.
27. Gossop, M., Johns, A., Green, L. (1986). Opiate withdrawal: inpatient versus outpatient programmes and preferred versus random assignment to treatment. British Medical Journal, 293, 103-104.
28. Halpern, S.D. (2003). Evaluating preference effects in partially unblinded, randomized clinical trials. Journal of Clinical Epidemiology, 56, 109-115.
29. Helsing, M., Bergman, B., Thaning, L., Hero, U. (1998). Quality of life and survival in patients with advanced non-small cell lung cancer receiving supportive care plus chemotherapy with carboplatin and etoposide or supportive care only: a multicentre randomized phase III trial; Joint Lung Cancer Study Group. European Journal of Cancer, 34, 1036-1044.
30. Henshaw, R.C., Naji, S.A., Russell, I.T., Templeton, A.A. (1993). Comparison of medical abortion with surgical vacuum aspiration: women's preferences and acceptability of treatment. British Medical Journal, 307, 714-717.
31. Howie, F.L., Henshaw, R.C., Naji, S.A., Russell, I.T., Templeton, A.A. (1997). Medical abortion or vacuum aspiration? Two year follow up of a patient preference trial. British Journal of Obstetrics and Gynaecology, 104, 829-833.
32. Jack, W.J.L., Cherry, U., Rodger, A. (1990). Recruitment to a prospective breast conservation trial: why are so few patients randomized? British Medical Journal, 301, 83-85.
33. Jansen, S.J.T., Stiggelbout, A.M., Nooij, M., Kievit, J. (2000). The effect of individually assessed preference weights on the relationship between holistic utilities and nonpreference- based assessment. Quality of Life Research, 9, 541-557.
34. Julien, J.P., Bijker, N., Fentiman, I.S., Peterse, J.L., Delledonne, V., Rouanet, P., et al. (2000). Radiotherapy in breast-conserving treatment for ductal carcinoma in situ: first results of the EORTC randomized phase III trial 10853; EORTC Breast Cancer Cooperative Group and EORTC Radiotherapy Group. Lancet, 355, 528-533.
35. Kaptchuk, T.J. (2001). The double-blind, randomized, placebo-controlled trial: gold standard or golden calf? Journal of Clinical Epidemiology, 54, 541-549.
36. Kassirer, J.P. (1994). Incorporating patients' preferences into medical decisions. New England Journal of Medicine, 330, 1895-1896.
37. Kendrick, D., Fielding, K., Bentley, E., Miller, P., Kerslake, R., Pringle, M. (2001). The role of radiography in primary care patients with low back pain of at least 6 weeks duration: a randomized (unblinded) controlled trial. Health Technology Assessment, 5, 1-69.
38. Kennedy, W.A., Laurier, C., Malo, J.L., Ghezzo, H., L'Archeveque, J., Contandriopoulos, A. P. (2003). Does clinical trial subject selection restrict the ability to generalize use and cost of health services to "real life" subjects? International Journal of Technology Assessment in Health Care, 19, 8-16.
39. Kerry, S., Hilton, S., Patel, S., Dundas, D., Rink, E., Lord, J. (2000). Routine referral for radiography of patients presenting with low back pain: is patients' outcome influenced by GPś referral for plain radiography? Health Technology Assessment, 40(20), 1-121.
40. King, M., Sibbald, B., Ward, E., Bower, P., Lloyd, M., Gabbay, M., et al. (2000). Randomized controlled trial of non-directive counselling, cognitive-behaviour therapy and usual general practitioner care in the management of depression as well as mixed anxiety and depression in primary care. Health Technology Assessment, 4(19), 1-83.
41. King, M., Nazareth, I., Lampe, F., Bower, P., Chandler, M., Morou, M., et al. (2005). Impact of participant and physician intervention preferences on randomized trials: a systematic review. Journal of the American Medical Association, 293, 1089-1099.
42. Kitchener, H.C., Burns, S., Nelson, L., Myers, A.J., Fletcher, I., Desai, M., et al. (2004). A randomized controlled trial of cytological surveillance versus patient choice between surveillance and colposcopy in managing mildly abnormal cervical smears. BJOG, 111, 63-70.
43. Kotwall, C.A., Mahoney, L.J., Myers, R.E., Decoste, L. (1992). Reasons for non-entry in randomized clinical trials for breast cancer: a single institutional study. Journal of Surgical Oncology, 50, 125-129.
44. Kramer, M.S., Shapiro, S.H. (1984). Scientific challenges in the application of randomized trials. Journal of the American Medical Association, 252, 2739-2745.
45. Lambert, M.F., Wood, J. (2000). Incorporating patient preferences into randomized trials. Journal of Clinical Epidemiology, 53, 163-166.
46. Lilford, R.J. (2003). Ethics of clinical trials from a Bayesian and decision analytic perspective: whose equipoise is it anyway? British Medical Journal, 326, 980-981.
47. MacLehose, R.R., Reeves, B.C., Harvey, I.M., Sheldon, T.A., Russell, I.T., Black, A.M. (2000). A systematic review of comparisons of effect sizes derived from randomized and non-randomized studies. Health Technology Assessment, 4(34), 1-154.
48. Mattila, P.S., Joki-Erkkila, V.P., Kilpi, T., Jokinen, J., Herva, E., Puhakka, H. (2003). Prevention of otitis media by adenoidectomy in children younger than 2 years. Archives of Otolaryngology Head and Neck Surgery, 129, 163-168.
49. McCormack, J., Greenhalgh, T. (2000). Seeing what you want to see in randomized controlled trials: versions and perversions of UKPDS data. British Medical Journal, 320, 1720-1723.
50. McCulloch, P., Taylor, I., Sasako, M., Lovett, B., Griffin, D. (2002). Randomized trials in surgery: problems and possible solutions. British Medical Journal, 324, 1448-1451.
51. McKay, J.R., Alterman, A.I., McLellan, A.T., Snider, E.C., O'Brien, C.P. (1995). Effect of random versus nonrandom assignment in a comparison of inpatient and day hospital rehabilitation for male alcoholics. Journal of Consulting and Clinical Psychology, 63, 70-78.
52. McKay, J.R., Alterman, A.I., McLellan, A.T., Boardman, C.R., Mulvaney, F.D., O'Brien, C. P. (1998). Effect of random versus nonrandom assignment in the evaluation of treatment for cocaine abusers. Journal of Consulting and Clinical Psychology, 66, 697-701.
53. McPherson, K. (1994). The best and the enemy of the good: randomized controlled trials, uncertainty, and assessing the role of patients choice in medical decision making. Journal of Epidemiology and Community Health, 48, 6-15.
54. McPherson, K., Chalmers, I. (1998). Incorporating patient preferences into clinical trials: information about patientś preference must be obtained first. British Medical Journal, 317, 78.
55. McPherson, K., Britton, A.R., Wennberg, J.E. (1997). Are randomized controlled trials controlled? Patient preferences and unblind trials. Journal of the Royal Society of Medicine, 90, 652-656.
56. Melchart, D., Steger, H. G., Linde, K., Makarian, K., Hatahet, Z., Brenke, R., et al. (2002). Integrating patient preferences in clinical trials: a pilot study of acupuncture versus midazolam for gastroscopy. Journal of Alternative Complementary Medicine, 8, 265-274.
57. Miller, P., Chilvers, C., Dewey, M., Fielding, K., Gretton, V., Palmer, B., et al. (2003). Counseling versus antidepressant therapy for the treatment of mild to moderate depression in primary care: economic analysis. International Journal of Technology Assessment in Health Care, 19, 80-90.
58. Moher, D., Schulz, K.F., Altman, D.G., for the CONSORT Group (2001). The CONSORT statement: revised recommendations for improving the quality of reports of parallelgroup randomized trials. Lancet, 357, 1191-1194.
59. Morrison, D.A., Sethi, G., Sacks, J., Grover, F., Sedlis, S., Esposito, R., et al. (1999). A multicenter, randomized trial of percutaneous coronary intervention versus bypass surgery in high-risk unstable angina patients: the AWESOME (Veterans Affairs Cooperative Study #385, angina with extremely serious operative mortality evaluation) investigators from the Cooperative Studies Program of the Department of Veterans Affairs. Controlled Clinical Trials, 20, 601-619.
60. Morrison, D.A., Sethi, G., Sacks, J., Henderson, W., Grover, F., Sedlis, S., et al. (2001). Percutaneous coronary intervention versus coronary artery bypass graft surgery for patients with medically refractory myocardial ischemia and risk factors for adverse outcomes with bypass: a multicenter, randomized trial; Investigators of the Department of Veterans Affairs Cooperative Study #385, the Angina With Extremely Serious Operative Mortality Evaluation (AWESOME). Journal of the American College of Cardiology, 38, 143-149.
61. Morrison, D.A., Sethi, G., Sacks, J., Henderson, W., Grover, F., Sedlis, S., et al. (2002a). Percutaneous coronary intervention versus coronary bypass graft surgery for patients with medically refractory myocardial ischemia and risk factors for adverse outcomes with bypass: the VA AWESOME multicenter registry: comparison with the randomized clinical trial. Journal of the American College of Cardiology, 39, 266-273.
62. Morrison, D.A., Sethi, G., Sacks, J., Henderson, W. G., Grover, F., Sedlis, S., et al. (2002b). Percutaneous coronary intervention versus repeat bypass surgery for patients with medically refractory myocardial ischemia: AWESOME randomized trial and registry experience with post-CABG patients. Journal of the American College of Cardiology, 40, 1951-1954.
63. Mosekilde, L., Beck-Nielsen, H., Sørensen, O.H., Nielsen, S.P., Charles, P., Vestergaard, P., et al. (2000). Hormonal replacement therapy reduces forearm fracture incidence in recent postmenopausal women - results of the Danish Osteoporosis Prevention Study. Maturitas, 36, 181-193.
64. Mosekilde, L., Beck-Nielsen, H., Sørensen, O.H., Nielsen, S.P., Charles, P., Vestergaard, P., et al. (2001). Behandling med hormonsubstitutionsterapi nedsoetter risikoen for underarmsbrud hos postmenopausale kvinder: Resultater fra Danish Osteoporosis Prevention Study. [Hormone replacement therapy reduces the risk of forearm fracture in postmenopausal women: results of the Danish Osteoporosis Prevention Study]. Ugeskr Loeger, 163, 7064-7069.
65. Nicolaides, K., Brizot, M.L., de Patel, F., Snijders, R. (1994). Comparison of chorionic villus sampling and amniocentesis for fetal karyotyping at 10-13 weeks gestation. Lancet, 344, 435-439.
66. Noel, P.H., Larme, A.C., Meyer, J., Marsh, G., Correa, A., Pugh, J.A. (1998). Patient choice in diabetes education curriculum: nutritional versus standard content for type 2 diabetes. Diabetes Care, 21, 896-901.
67. Olschewski, M., Scheurlen, H. (1985). Comprehensive cohort study: an alternative to randomized consent design in a breast preservation trial. Methods of Information in Medicine, 24, 131-134.
68. Paradise, J.L., Bluestone, C.D., Bachman, R.Z., Colborn, D.K., Bernard, B.S., Taylor, F.H., et al. (1984). Efficacy of tonsillectomy for recurrent throat infection in severely affected children: results of parallel randomized and nonrandomized clinical trials. New England Journal of Medicine, 310, 674-683.
69. Paradise, J.L., Bluestone, C.D., Rogers, K.D., Taylor, F.H., Colborn, D.K., Bachman, R.Z., et al. (1990). Efficacy of adenoidectomy for recurrent otitis media in children previously treated with tympanostomy-tube placement: results of parallel randomized and nonrandomized trials. Journal of the American Medical Association, 263, 2066-2073.
70. Peppercorn, J.M., Weeks, J.C., Cook, E.F., Joffe, S. (2004). Comparison of outcomes in cancer patients treated within and outside clinical trials: conceptual framework and structured review. Lancet, 363, 263-270.
71. Plaisier, P.W., Berger, M.Y., van der Hul, R.L., Nijs, H.G.T., den Toom, R., Terpstra, O. T., et al. (1994). Unexpected difficulties in randomizing patients in a surgical trial: a prospective study comparing extracorporeal shock wave lithotripsy with open cholecystectomy. World Journal of Surgery, 18, 769-773.
72. Prescott, R.J., Counsell, C.E., Gillespie, W.J., Grant, A.M., Russell, I.T., Kiauka, S., et al. (1999). Factors that limit the quality, number and progress of randomized controlled trials. Health Technology Assessment, 3(20), 1-143.
73. Rauschecker, H.F., Sauerbrei, W., Gatzemeier, W., Sauer, R., Schauer, A., Schmoor, C., et al. (1998). Eight-year results of a prospective non-randomized study on therapy of small breast cancer: the German Breast Cancer Study Group (GBSG). European Journal of Cancer, 34, 315-323.
74. Reddihough, D.S., King, J., Coleman, G., Catanese, T. (1998). Efficacy of programmes based on conductive education for young children with cerebral palsy. Developmental and Medicine Child Neurology, 40, 763-770.
75. Renjilian, D.A., Perri, M.G., Nezu, A.M., McKelvey, W.F., Shermer, R.L., Anton, S.D. (2001). Individual versus group therapy for obesity: effects of matching participants to their treatment preferences. Journal of Consulting and Clinical Psychology, 69, 717-721.
76. Ross, S., Grant, A., Counsell, C., Gillespie, W., Russell, I., Prescott, R. (1999). Barriers to participation in randomized controlled trials: a systematic review. Journal of Clinical Epidemiology, 52, 1143-1156.
77. Rücker, G. (1989). A two-stage trial design for testing treatment, self-selection and treatment preference effects. Statistics in Medicine, 8, 477-485.
78. Sauerbrei, W., Bastert, G., Bojar, H., Beyerle, C., Neumann, R.L., Schmoor, C., et al. (2000). Randomized 2 × 2 trial evaluating hormonal treatment and the duration of chemotherapy in node-positive breast cancer patients: an update based on 10 years' follow- up; German Breast Cancer Study Group. Journal of Clinical Oncology, 18, 94-101.
79. Schmoor, C., Olschewski, M., Schumacher, M. (1996). Randomized and non-randomized patients in clinical trials: experiences with comprehensive cohort studies. Statistics in Medicine, 15, 263-271.
80. Schmoor, C., Sauerbrei, W., Bastert, G., Schumacher, M. (2000). Role of isolated locoregional recurrence of breast cancer: Results of four prospective studies. Journal of Clinical Oncology, 18, 1696-1708.
81. Schmoor, C., Olschewski, M., Sauerbrei, W., Schumacher, M. (2002). Long-term followup of patients in four prospective studies of the German Breast Cancer Study Group (GBSG): a summary of key results. Onkologie, 25, 143-150.
82. Schwartz, L.M., Woloshin, S., Baczek, L. (2002). Media coverage of scientific meetings: too much, too soon? Journal of the American Medical Association, 287, 2859-2863.
83. Sedlis, S.P., Morrison, D.A., Lorin, J.D., Esposito, R., Sethi, G., Sacks, J., et al. (2002). Percutaneous coronary intervention versus coronary bypass graft surgery for diabetic patients with unstable angina and risk factors for adverse outcomes with bypass: outcome of diabetic patients in the AWESOME randomized trial and registry. Journal of the American College of Cardiology, 40, 1555-1566.
84. Sedlis, S.P., Ramanathan, K.B., Morrison, D.A., Sethi, G., Sacks, J., Henderson, W. (2004). Department of Veterans Affairs Cooperative Study #385, Angina With Extremely Serious Operative Mortality Evaluation (AWESOME) Investigators: outcome of percutaneous coronary intervention versus coronary bypass grafting for patients with low left ventricular ejection fractions, unstable angina pectoris, and risk factors for adverse outcomes with bypass (the AWESOME Randomized Trial and Registry). American Journal of Cardiology, 94, 118-120.
85. Silverman, W.A., Altman, D.G. (1996). Patient's preferences and randomized trials. Lancet, 347, 171-174.
86. Stiggelbout, A.M., de Haes, J.C.J.M. (2001). Patient preference for cancer therapy: an overview of measurement approaches. Journal of Clinical Oncology, 19, 220-230.
87. Wallage, S., Cooper, K.G., Graham, W.J., Parkin, D.E. (2003). A randomized trial comparing local versus general anaesthesia for microwave endometrial ablation. BJOG, 110, 799-807.
88. Ward, E., King, M., Lloyd, M., Bower, P., Sibbald, B., Farrelly, S., et al. (2000). Randomized controlled trial of non-directive counselling, cognitive-behaviour therapy, and usual general practitioner care for patients with depression. I. Clinical effectiveness. British Medical Journal, 321, 1383-1388.
89. Woodward, J., Kelly, S.M. (2004). A pilot study for a randomized controlled trial of water birth versus land birth. BJOG, 111, 537-545.
90. Zelen, M. (1979) A new design for randomized clinical trials. New England Journal of Medicine, 300, 1242-1245.