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

JAMA

Volumn 282, Issue 8, 1999, Pages 771-778

  Bucher, Heiner C ^a   Guyatt, Gordon H ^b,d   Cook, Deborah J ^b   Holbrook, Anne ^b   McAlister, Finlay A ^c  

^a KANTONSSPITAL BASEL   (Switzerland)

^b MCMASTER UNIVERSITY   (Canada)

^c UNIVERSITY OF ALBERTA HOSPITAL   (Canada)

^d MCMASTER UNIVERSITY MEDICAL CENTRE   (Canada)

Author keywords

[No Author keywords available]

Indexed keywords

ABACAVIR; ALENDRONIC ACID; ANTIHYPERTENSIVE AGENT; ANTILIPEMIC AGENT; ANTIVIRUS AGENT; ATORVASTATIN; BENZOTHIOPHENE DERIVATIVE; BEZAFIBRATE; BISPHOSPHONIC ACID DERIVATIVE; CALCIUM ANTAGONIST; DIHYDROPYRIDINE DERIVATIVE; NELFINAVIR; RALOXIFENE; THIAZIDE DIURETIC AGENT;

ACQUIRED IMMUNE DEFICIENCY SYNDROME; AGED; BONE DENSITY; CASE REPORT; COST BENEFIT ANALYSIS; DRUG INDUCED DISEASE; DYSPEPSIA; FEMALE; HUMAN; HYPERLIPIDEMIA; HYPERTENSION; MEDICAL LITERATURE; MEDICAL RESEARCH; OSTEOPOROSIS; OUTCOMES RESEARCH; POSTMENOPAUSE OSTEOPOROSIS; PRIORITY JOURNAL; REVIEW; TREATMENT OUTCOME;

EID: 0033603794     PISSN: 00987484     EISSN: None     Source Type: Journal    
DOI: 10.1001/jama.282.8.771     Document Type: Review

References (83)

1. Delmas PD, Bjarnason NH, Mitlak BH, et al. Effects of raloxifene on bone mineral density, serum cholesterol concentrations, and uterine endometrium in postmenopausal women. N Engl J Med. 1997;337: 1641-1647.
2. Temple RJ. A regulatory authority's opinion about surrogate endpoints. In: Nimmo WS, Tucker GT, eds. Clinical Measurement in Drug Evaluation. New York, NY: John Wiley & Sons Inc; 1995:57.
3. Hammer SM, Katzenstein DA, Hughes MD, et al. A trial comparing nucleoside monotherapy with combination therapy in HIV-infected adults with CD4 cell counts from 200 to 500 per cubic millimeter. N Engl J Med. 1996;335:1081-1090.
4. Delta Coordinating Committee. Delta: a randomised double-blind controlled trial comparing combinations of zidovudine plus didanosine or zalcitabine with zidovudine alone in HIV-infected individuals. Lancet 1996;348:283-291.
5. Saravolatz LD, Winslow DL, Collins G, et al. Zidovudine alone or in combination with didanosine or zalcitabine in HIV-infected patients with the acquired immunodeficiency syndrome or fewer than 200 CD4 cells per cubic millimeter. N Engl J Med. 1996; 335:1099-1106.
6. Hammer SM, Squires KE, Hughes MD, et al. A controlled trial of two nucleoside analogues plus indinavir in persons with human immunodeficiency virus infection and CD4 cell counts of 200 per cubic millimeter or less. N Engl J Med 1997;337:725-733.
7. Niebauer J, Coats AJ. Treating chronic heart failure: time to take stock. Lancet. 1997;349:966-967.
8. Massie BM, Berk MR, Brozena SC, et al. Can further benefit be achieved by adding flosequinan to patients with congestive heart failure who remain symptomatic on diuretic, digoxin, and an angiotensin converting enzyme inhibitor? results of the flosequinan-ACE inhibitor trial (FACET). Circulation. 1993;88:492-501.
9. How to read clinical journals, IV: to determine etiology or causation. CMAJ. 1981;124:985-990.
10. Oxman AD, Sackett DL, Guyatt GH. Users' guides to the medical literature, I: how to get started. JAMA. 1993;270:2093-2095.
11. Guyatt G, Walter S, Shannon H, Cook D, Jaeschke R, Heddle N. Basic statistics for clinicians, IV: correlation and regression. CMAJ. 1995;152:497-504.
12. Guyatt GH, Thompson PJ, Berman LB, et al. How should we measure function in patients with chronic heart and lung disease? J Chronic Dis. 1985;38:517-524.
13. Mahler DA, Weinberg DH, Wells CK, Feinstein AR. The measurement of dyspnea: contents, interobserver agreement, and physiologic correlates of two new clinical indexes. Chest. 1984;85:751-758.
14. Verschuren WM, Jacobs DR, Bloemberg BP, et al. Serum total cholesterol and long-term coronary heart disease mortality in different cultures. JAMA. 1995; 274:131-136.
15. Mellors JW, Rinaldo CR Jr, Gupta P, et al. Prognosis in HIV-1 infection predicted by the quantity of virus in plasma. Science. 1996;272:1167-1170.
16. Mellors JW, Kingsley LA, Rinaldo CR, et al. Quantitation of HIV-1 RNA in plasma predicts outcome after seroconversion. Ann Intern Med. 1995;122:573-579.
17. Ruiz L, Romeu J, Clotet B, et al. Quantitative HIV-1 RNA as a marker of clinical stability and survival in a cohort of 302 patients with a mean CD4 cell count of 300 × 10(6)/1. AIDS. 1996;10:F39-F44.
18. O'Brien TR, Blattner WA, Waters D, et al. Serum HIV-1 RNA levels and time to development of AIDS in the Multicenter Hemophilia Cohort Study. JAMA. 1996;276:105-110.
19. Yerly S, Perneger TV, Hirschel B, et al. A critical assessment of the prognostic value of HIV-1 RNA levels and CD4+ cell counts in HIV-infected patients. Arch Intern Med. 1998;158:247-252.
20. Ho DD. Viral counts in HIV infection. Science. 1996;272:1124-1125.
21. Cummings SR, Nevitt MC, Browner WS, et al. Risk factors for hip fracture in white women. N Engl J Med. 1995;332:767-773.
22. Marshall D, Johnell O, Wedel H. Meta-analysis of how well measures of bone mineral density predict occurrence of osteoporotic fractures. BMJ. 1996; 312:1254-1259.
23. Huang C, Ross PD, Wasnich RD. Short-term and long-term fracture prediction by bone mass measurements. J Bone Miner Res. 1998;13:107-113.
24. Bigger JT Jr, Fleiss JL, Kleiger R, et al. The relationships among ventricular arrhythmias, left ventricular dysfunction, and mortality in the 2 years after myocardial infarction. Circulation. 1984;69:250-258.
25. McAlister FA, Teo KK. Antiarrhythmic therapies for the prevention of sudden cardiac death. Drugs. 1997;54:235-252.
26. Echt DS, Liebson PR, Mitchell LB, et al, and the Cardiac Arrhythmia Suppression Trial. Mortality and morbidity in patients receiving encainide, flecainide, or placebo. N Engl J Med. 1991;324:781-788.
27. Moore TJ. Deadly Medicine. New York, NY: Simon & Schuster; 1995.
28. Drexler H, Banhardt U, Meinertz T, et al. Contrasting peripheral short-term and long-term effects of converting enzyme inhibition in patients with congestive heart failure: a double-blind, placebo-controlled trial. Circulation. 1989;79:491-502.
29. Lewis CR. Comparison of lisinopril versus placebo for congestive heart failure. Am J Cardiol. 1989; 63:12D-16D.
30. Giles TD, Fisher MB, Rush JE. Lisinopril and captopril in the treatment of heart failure in older patients. Am J Med. 1988;85:44-47.
31. Riegger GA. Effects of quinapril on exercise tolerance in patients with mild to moderate heart failure. Eur Heart J. 1991;12:705-711.
32. Garg R, Yusuf S. Overview of randomized trials of angiotensin-converting enzyme inhibitors on mortality and morbidity in patients with heart failure. JAMA. 1995;273:1450-1456.
33. Di Bianco R, Shabetai R, Kostuk W, et al. A comparison of oral milrinone, digoxin, and their combination in the treatment of patients with chronic heart failure. N Engl J Med. 1989;320:677-683.
34. Sueta CA, Gheorghiade M, Adams KF, et al, and the Epoprostenol Multicenter Research Group. Safety and efficacy of epoprostenol in patients with severe congestive heart failure. Am J Cardiol. 1995;75:34A-43A.
35. Packer M, Carver JR, Rodeheffer RJ, et al, for the Promise Study Research Group. Effect of oral milrinone on mortality in severe chronic heart failure. N Engl J Med. 1991;325:1468-1475.
36. Califf RM, Adams KF, McKenna WJ, et al. A randomized controlled trial of epoprostenol therapy for severe congestive heart failure. Am Heart J. 1997; 134:44-54.
37. Yee KM, Struthers AD. Can drug effects on mortality in heart failure be predicted by any surrogate measure? Eur Heart J. 1997;18:1860-1864.
38. Hampton JR, van Veldhuisen DJ, Kleber FX, et al. Randomised study of effect of ibopamine on survival in patients with advanced severe heart failure. Lancet. 1997;349:971-977.
39. Cameron DW, Heath-Chiozzi M, Danner S, et al, and the Advanced HIV Disease Ritonavir Study Group. Randomised placebo-controlled trial of ritonavir in advanced HIV-1 disease. Lancet. 1997;351:543-549.
40. Watts NB, Harris ST, Genant HK, et al. Intermittent cyclical etidronate treatment of postmenopausal osteoporosis. N Engl J Med. 1990;323:73-79.
41. Storm T, Thamsborg G, Steiniche T, Genant HK, Sorensen OH. Effect of intermittent cydical etidronate therapy on bone mass and fracture rate in women with postmenopausal osteoporosis. N Engl J Med. 1990;322:1265-1271.
42. Liberman UA, Weiss SR, Broll J, et al, and the Alendronate Phase III Osteoporosis Treatment Study Group. Effect of oral alendronate on bone mineral density and the incidence of fractures in postmenopausal osteoporosis. N Engl J Med. 1995;333:1437-1443.
43. Riggs BL, Hodgson SF, O'Fallon WM, et al. Effect of fluoride treatment on the fracture rate in postmenopausal women with osteoporosis. N Engl J Med. 1990;322:802-809.
44. Meunier PJ, Sebert J-L, Reginster J-Y, et al. Fluoride salts are no better at preventing new vertebral fractures than calcium-vitamin D in postmenopausal osteoporosis. Osteoporos Int. 1998;8:4-12.
45. Dawson-Hughes B, Harris SS, Krall EA, Dallal GE. Effect of calcium and vitamin D supplementation on bone density in men and women 65 years of age and older. N Engl J Med. 1977;337:670-676.
46. McAlister FA, Straus S, Sackett DL. Randomized clinical trials of antihypertensive drugs: all that glitters is not gold. CMAJ. 1998;159:488-490.
47. Staessen JA, Fagard R, Thijs L, et al. Randomised double-blind comparison of placebo and active treatment for older patients with isolated systolic hypertension. Lancet 1997;350:757-764.
48. Psaty BM, Siscovick DS, Weiss NS, et al. Hypertension and outcomes research: from clinical trials to clinical epidemiology. Am J Hypertens. 1996;9:178-183.
49. Borhani NO, Mercuri M, Borhani PA, et al. Final outcome results of the multicenter isradipine diuretic atherosclerosis study (MIDAS): a randomized controlled trial. JAMA. 1996;276:785-791.
50. Tatti P, Pahor M, Byington RP, et al. Outcome results of the fosinopril versus amlodipine cardiovascular events randomized trial (FACET) in patients with hypertension and NIDDM. Diabetes Care. 1998;21:597-603.
51. Estacio RO, Jeffers BW, Hiatt WR, Biggerstaff SL, Gifford N, Schrier RW. The effect of nisoldipine as compared with enalapril on cardiovascular outcomes in patients with non-insulin-dependent diabetes and hypertension. N Engl J Med. 1998;338:645-652.
52. McAlister FA, Laupacis A, Wells GA, Sackett DL, for the Evidence-Based Medicine Working Group. Users' guide to the medical literature, XIX: applying clinical trial results part B: guidelines for determining whether a drug is exerting (more than) a class effect. JAMA. In press.
53. Bucher HC, Griffith LE, Guyatt GH. Systematic review on risk and benefit of different cholesterol lowering interventions. Arterioscler Thromb Vasc Biol. 1999;19:187-195.
54. Muldoon MF, Manuck SB, Matthews KA. Lowering cholesterol concentration and mortality. BMJ. 1990;301:309-314.
55. Smith GD, Song F, Sheldon TA. Cholesterol lowering and mortality. BMJ. 1993;306:1367-1373.
56. Prentice RL. Surrogate endpoints in clinical trials. Stat Med. 1989;8:431-440.
57. Fleming TR. Surrogate markers in AIDS and cancer trials. Staf Med. 1994;13:1423-1435.
58. Cooper DA, Gatell JM, Kroon S, et al, and the European-Australian Collaborative Group. Zidovudine in persons with asymptomatic HIV infection and CD4+ cell counts greater than 400 per cubic millimeter. N Engl J Med. 1993;329:297-303.
59. Concorde Coordinating Committee. Concorde: MRC/ANRS randomised double-blind controlled trial of immediate and deferred zidovudine in symptom-free HIV infection. Lancet. 1994;343:871-881.
60. Guyatt GH, Sackett DL, Cook DJ, for the Evidence-Based Medicine Working Group. Users' guides to the medical literature, II: how to use an article about therapy or prevention A: are the results of the study valid? JAMA. 1993;270:2598-2601.
61. Dans AL, Dans LF, Guyatt GH, Richardson S, for the Evidence-Based Medicine Working Group. Users' guides to the medical literature, XIV: how to decide on the applicability of clinical trial results to your patient. JAMA. 1998;279:545-549.
62. Ettinger B, Black D, Cummings S, et al. Raloxifene reduces the risk of incident vertebral fractures: 24 month interim analyses. Osteoporos Int. 1998;8 (suppl 3):11.
63. Clendeneninn N, Quart B, Anderson R, Knowles M, Chang Y. Analysis of long-term virologic data from the viracept (nelfinavir) 511 protocol using 3 HIV-RNA assays. In: Abstracts from the 5th Conference on Retrovirusesand Opportunistic Infections; Chicago, III; 1998.
64. Brun-Vezinet F, Boucher C, Loveday C, et al, and the Delta Virology Working Group and Coordinating Committee. HIV-1 viral load, phenotype, and resistance in a subset of drug-naive participants from the Delta trial: the national virology groups. Lancet. 1997; 350:983-990.
65. Montaner JS, Reiss P, Cooper D, et al. A randomized, double-blind trial comparing combinations of nevirapine, didanosine, and zidovudine for HIV-infected patients: the Netherlands, Canada, and Australia (INCAS) study. JAMA. 1998;279:930-937.
66. CEASAR Coordinating Committee. Randomised trial of addition of lamivudine or lamivudine plus loviride to zidovudine-containing regimens for patients with HIV-1 infection: the CAESAR trial. Lancet. 1997;349:1413-1421.
67. Fischl M, Greenberg S, Clumeck N, et al. Safety and activity of abacavir (1592, ABC) with 3TC/ZDV in antiretroviral naive subjects. In: Abstracts from the 12th World AIDS Conference: Geneva, Switzerland; June 28-July 3, 1998.
68. Katzenstein DA, Hammer SM, Hughes MD, et al, and the AIDS Clinical Triais Group Study 175 Virology Study Team. The relation of virologic and immunologic markers to clinical outcomes after nucleoside therapy in HIV-infected adults with 200 to 500 CD4 cells per cubic millimeter. N Engl J Med. 1996;335:1091-1098.
69. Collins R, Peto R, MacMahon S, et al. Blood pressure, stroke, and coronary heart disease, part 2: short-term reductions in blood pressure; overview of randomised drug trials in their epidemiological context. Lancet. 1990;335:827-838.
70. MacMahon S, Peto R, Cutler J; et al. Blood pressure, stroke, and coronary heart disease, part 1: prolonged differences in blood pressure; prospective observational studies corrected for the regression dilution bias. Lancet. 1990;335:765-774.
71. Psaty BM, Smith NL, Siscovick DS, et al. Health outcomes associated with antihypertensive therapies used as first-line agents: a systematic review and metaanalysis. JAMA. 1997;277:739-745.
72. Heinonen TM, Stein E, Weiss SR, et al. The lipid-lowering effects of atorvastatin, a new HMG-coA reductase inhibitor: results of a randomized, double-masked study. Clin Ther. 1996;18:853-863.
73. Bakker-Arkema RG, Davidson MH, Goldstein RJ, et al. Efficacy and safety of a new HMG-coA reductase inhibitor, atorvastatin, in patients with hypertriglyceridemia. JAMA. 1996;275:128-133.
74. Law MR, Wald NJ, Thompson SG. By how much and how quickly does reduction in serum cholesterol concentration lower risk of ischaemic heart disease? BMJ. 1994;308:367-372.
75. Winocour PH, Durrington PN, Bhatagnar D, et al. The effect of bezafibrate on very low density lipoprotein (VLDL), intermediate density lipoprotein (IDL), and low density lipoprotein (LDL) composition in type 1 diabetesassociated with hypercholesterolaemia or combined hyperlipidaemia. Atherosclerosis. 1992;93:83-94.
76. Jones IR, Swai A, Taylor R, Miller M, Laker MF, Alberti KG. Lowering of plasma glucose concentrations with bezafibrate in patients with moderately controlled NIDDM. Diabetes Care. 1990;13:855-863.
77. Jones P, Kafonek S, Laurora I, Hunninghake D. Comparative dose efficacy study of atorvastatin versus simvastatin, pravastatin, lovastatin, and fluvastatin in patients with hypercholesterolemia (the CURVES study). Am J Cardiol. 1998;81:582-587.
78. Downs JR, Clearfield M, Weis S, et al. Primary prevention of acute coronary events with lovastatin in men and women with average cholesterol levels. JAMA. 1998;279:1615-1622.
79. Shepherd J, Cobbe SM, Ford I, et al. Prevention of coronary heart disease with pravastatin in men with hypercholesterolemia. N Engl J Med. 1995;333:1301-1307.
80. Karpf DB, Shapiro DR, Seeman E, et al. Prevention of nonvertebral fractures by alendronate: a metaanalysis. JAMA. 1997;277:1159-1164.
81. Black DM, Cummings SR, Karpf DB, et al, and the Fracture Intervention Trial Research Group. Randomised trial of effect of alendronate on risk of fracture in women with existing vertebral fractures. Lancet. 1996;348:1535-1541.
82. Cummings SR, Eckert S, Krueger KA, et al. The effect of raloxifene on risk of breast cancer in postmenopausal women. JAMA. 1999;281:2189-2197.
83. Ettinger B, Black DM, Mitlak BH, et al. Reduction of vertebral fracture risk in postmenopausal women with osteoporosis treated with raloxifene: results from a 3-year randomized trial. JAMA. 1999;282:637-645.