Design, measurement, and analysis of clinical investigations

Textbook of Pediatric Rheumatology

Volumn , Issue , 2005, Pages 142-173

  Glannini, Edward H ^a  

^a NONE

Author keywords

[No Author keywords available]

Indexed keywords

EID: 78549245892     PISSN: None     EISSN: None     Source Type: Book    
DOI: 10.1016/B978-1-4160-0246-8.50012-7     Document Type: Chapter

References (115)

1. Evidence-Based Medicine Working Group Evidence-based medicine: a new approach to teaching the practice of medicine. JAMA 1992, 268:2420-2425.
2. Brunner HI, Giannini EH Evidence-based medicine in pediatric rheumatology. Clin Exp Rheumatol 2000, 18:407-414.
3. Levin A The Cochrane Collaboration. Ann Intern Med 2001, 135:309-312.
4. Laupacis A The Cochrane Collaboration: how is it progressing?. Stat Med 2002, 21:2815-2822.
5. Waters E Evidence-based paediatric practice and its relation to the Cochrane Collaboration. J Paediatr Child Health 2003, 39:137-138.
6. Churchill R Systematic reviews and the Cochrane Collaboration: improving the evidence-base for all psychiatric treatments. Epidemiol Psichiatr Soc 2003, 12:137-145.
7. Clarke M The Cochrane Collaboration: providing and obtaining the best evidence about the effects of health care. Eval Health Prof 2002, 25:8-11.
8. Guyatt GH, Rennie D Users' guides to the medical literature. JAMA 1993, 270:2096-2097.
9. Oxman AD, Sackett DL, Guyatt GH Users' guides to the medical literature. 1: How to get started. The Evidence-Based Medicine Working Group. JAMA 1993, 270:2093-2095.
10. Guyatt GH, Sackett DL, Cook DJ Users' guides to the medical literature. II: How to use an article about therapy or prevention. A: Are the results of the study valid? Evidence-Based Medicine Working Group. JAMA 1993, 270:2598-2601.
11. Guyatt GH, Sackett DL, Cook DJ Users' guides to the medical literature. II: How to use an article about therapy or prevention. B: What were the results and will they help me in caring for my patients? Evidence-Based Medicine Working Group. JAMA 1994, 271:59-63.
12. Jaeschke R, Guyatt G, Sackett DL Users' guides to the medical literature. III: How to use an article about a diagnostic test. A: Are the results of the study valid? Evidence-Based Medicine Working Group. JAMA 1994, 271:389-391.
13. Jaeschke R, Guyatt GH, Sackett DL Users' guides to the medical literature. III: How to use an article about a diagnostic test. B: What are the results and will they help me in caring for my patients? The Evidence-Based Medicine Working Group. JAMA 1994, 271:703-707.
14. Levine M, Walter S, Lee H, et al. Users' guides to the medical literature. IV: How to use an article about harm. Evidence-Based Medicine Working Group. JAMA 1994, 271:1615-1619.
15. Guyatt GH, Sackett DL, Sinclair JC, et al. Users' guides to the medical literature. IX: A method for grading health care recommendations. Evidence-Based Medicine Working Group. JAMA 1995, 274:1800-1804.
16. Laupacis A, Wells G, Richardson WS, Tugwell P Users' guides to the medical literature. V: How to use an article about prognosis. Evidence-Based Medicine Working Group. JAMA 1994, 272:234-237.
17. Oxman AD, Cook DJ, Guyatt GH Users' guides to the medical literature. VI: How to use an overview. Evidence-Based Medicine Working Group. JAMA 1994, 272:1367-1371.
18. Richardson WS, Detsky AS Users' guides to the medical literature. VII: How to use a clinical decision analysis. A: Are the results of the study valid? Evidence-Based Medicine Working Group. JAMA 1995, 273:1292-1295.
19. Richardson WS, Detsky AS Users' guides to the medical literature. VII: How to use a clinical decision analysis. B: What are the results and will they help me in caring for my patients? Evidence Based Medicine Working Group. JAMA 1995, 273:1610-1613.
20. Hayward RS, Wilson MC, Tunis SR, et al. Users' guides to the medical literature. VIII: How to use clinical practice guidelines. A: Are the recommendations valid? The Evidence-Based Medicine Working Group. JAMA 1995, 274:570-574.
21. Wilson MC, Hayward RS, Tunis SR, et al. Users' guides to the medical literature. VIII: How to use clinical practice guidelines. B: What are the recommendations and will they help you in caring for your patients? The Evidence-Based Medicine Working Group. JAMA 1995, 274:1630-1632.
22. Naylor CD, Guyatt GH Users' guides to the medical literature. X: How to use an article reporting variations in the outcomes of health services. The Evidence-Based Medicine Working Group. JAMA 1996, 275:554-558.
23. Naylor CD, Guyatt GH Users' guides to the medical literature. XI: How to use an article about a clinical utilization review. Evidence-Based Medicine Working Group. JAMA 1996, 275:1435-1439.
24. Guyatt GH, Naylor CD, Juniper E, et al. Users' guides to the medical literature. XII: How to use articles about health-related quality of life. Evidence-Based Medicine Working Group. JAMA 1997, 277:1232-1237.
25. Drummond MF, Richardson WS, O'Brien BJ, et al. Users' guides to the medical literature. XIII: How to use an article on economic analysis of clinical practice. A: Are the results of the study valid? Evidence-Based Medicine Working Group. JAMA 1997, 277:1552-1557.
26. O'Brien BJ, Heyland D, Richardson WS, et al. Users' guides to the medical literature. XIII: How to use an article on economic analysis of clinical practice. B: What are the results and will they help me in caring for my patients? Evidence-Based Medicine Working Group. JAMA 1997, 277:1802-1806.
27. Dans AL, Dans LF, Guyatt GH, Richardson S Users' guides to the medical literature. XIV: How to decide on the applicability of clinical trial results to your patient. Evidence-Based Medicine Working Group. JAMA 1998, 279:545-549.
28. McAlister FA, Laupacis A, Wells GA, Sackett DL Users' guides to the medical literature. XIX: Applying clinical trial results. B: Guidelines for determining whether a drug is exerting (more than) a class effect. Evidence-Based Medicine Working Group. JAMA 1999, 282:1371-1377.
29. Bucher HC, Guyatt GH, Cook DJ, et al. 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 1999, 282:771-778.
30. Richardson WS, Wilson MC, Guyatt GH, et al. Users' guides to the medical literature. XV: How to use an article about disease probability for differential diagnosis. Evidence-Based Medicine Working Group. JAMA 1999, 281:1214-1219.
31. Guyatt GH, Sinclair J, Cook DJ, Glasziou P Users' guides to the medical literature. XVI: How to use a treatment recommendation. Evidence-Based Medicine Working Group and the Cochrane Applicability Methods Working Group. JAMA 1999, 281:1836-1843.
32. Barratt A, Irwig L, Glasziou P, et al. Users' guides to the medical literature. XVII: How to use guidelines and recommendations about screening. Evidence-Based Medicine Working Group. JAMA 1999, 281:2029-2034.
33. Randolph AG, Haynes RB, Wyatt JC, et al. Users' guides to the medical literature. XVIII: How to use an article evaluating the clinical impact of a computer-based clinical decision support system. JAMA 1999, 282:67-74.
34. McAlister FA, Straus SE, Guyatt GH, Haynes RB Users' guides to the medical literature. XX: Integrating research evidence with the care of the individual patient. Evidence-Based Medicine Working Group. JAMA 2000, 283:2829-2836.
35. Hunt DL, Jaeschke R, McKibbon KA Users' guides to the medical literature. XXI: Using electronic health information resources in evidence-based practice. Evidence-Based Medicine Working Group. JAMA 2000, 283:1875-1879.
36. McGinn TG, Guyatt GH, Wyer PC, et al. Users' guides to the medical literature. XXII: How to use articles about clinical decision rules. Evidence-Based Medicine Working Group. JAMA 2000, 284:79-84.
37. Giacomini MK, Cook DJ Users' guides to the medical literature. XXIII: Qualitative research in health care. A: Are the results of the study valid? Evidence-Based Medicine Working Group. JAMA 2000, 284:357-362.
38. Giacomini MK, Cook DJ Users' guides to the medical literature. XXIII: Qualitative research in health care. B: What are the results and how do they help me care for my patients? Evidence-Based Medicine Working Group. JAMA 2000, 284:478-482.
39. Richardson WS, Wilson MC, Williams JW, et al. Users' guides to the medical literature. XXIV: How to use an article on the clinical manifestations of disease. Evidence-Based Medicine Working Group. JAMA 2000, 284:869-875.
40. Guyatt GH, Haynes RB, Jaeschke RZ, et al. Users' guides to the medical literature. XXV: Evidence-based medicine: principles for applying the Users' Guides to patient care. Evidence-Based Medicine Working Group. JAMA 2000, 284:1290-1296.
41. Sung NS, Crowley WF, Genel M, et al. Central challenges facing the national clinical research enterprise. JAMA 2003, 289:1278-1287.
42. Bloom FE Presidential address. Science as a way of life: perplexities of a physician-scientist. Science 2003, 300:1680-1685.
43. Petiti DM Meta-analysis, Decision Analysis, and Cost Effectiveness Analysis 1994, Oxford University Press, Oxford, UK.
44. Moses LE Statistical concepts fundamental to investigations. N Engl J Med 1985, 312:890-897.
45. Hill AB The environment and disease: association or causation?. Proc R Soc Med 1965, 58:295-300.
46. Noll RB, Kozlowski K, Gerhardt C, et al. Social, emotional, and behavioral functioning of children with juvenile rheumatoid arthritis. Arthritis Rheum 2000, 43:1387-1396.
47. Noll RB, Gartstein MA, Vannatta K, et al. Social, emotional, and behavioral functioning of children with cancer. Pediatrics 1999, 103:71-78.
48. Inman RD, Johnston ME, Hodge M, et al. Postdysenteric reactive arthritis: a clinical and immunogenetic study following an outbreak of salmonellosis. Arthritis Rheum 1988, 31:1377-1383.
49. Arguedas O, Fasth A, Andersson-Gare B A prospective population based study on outcome of juvenile chronic arthritis in Costa Rica. J Rheumatol 2002, 29:174-183.
50. Hashkes PJ, Balistreri WF, Bove KE, et al. The long-term effect of methotrexate therapy on the liver in patients with juvenile rheumatoid arthritis. Arthritis Rheum 1997, 40:2226-2234.
51. Ravelli A, Manzoni SM, Viola S, et al. Factors affecting the efficacy of intraarticular corticosteroid injection of knees in juvenile idiopathic arthritis. J Rheumatol 2001, 28:2100-2102.
52. Ravelli A, Martini A Early predictors of outcome in juvenile idiopathic arthritis. Clin Exp Rheumatol 2003, 21(5 Suppl 31):S89-S93.
53. Brunner HI, Lovell DJ, Finck BK, Giannini EH Preliminary definition of disease flare in juvenile rheumatoid arthritis. J Rheumatol 2002, 29:1058-1064.
54. Rider LG, Giannini EH, Harris-Love M, et al. Defining clinical improvement in adult and juvenile myositis. J Rheumatol 2003, 30:603-617.
55. Ruperto N, Ravelli A, Murray KJ, et al. Preliminary core sets of measures for disease activity and damage assessment in juvenile systemic lupus erythematosus and juvenile dermatomyositis. Rheumatology (Oxf) 2003, 42:1452-1459.
56. Wallace CA, Ruperto N, Giannini EH Childhood Arthritis and Rheumatology Research Alliance; Pediatric Rheumatology International Trials Organisation; Pediatric Rheumatology Collaborative Study Group: Preliminary criteria for clinical remission for select categories of juvenile idiopathic arthritis. J Rheumatol 2004, 11:2290-2294.
57. Care BA, Fasth A The natural history of juvenile chronic arthritis: a population based cohort study. II: Outcome. J Rheumatol 1995, 22:308-319.
58. Flato B, Lien G, Smerdel A, et al. Prognostic factors in juvenile rheumatoid arthritis: a case-control study revealing early predictors and outcome after 14.9 years. J Rheumatol 2003, 30:386-393.
59. Oen K Long-term outcomes and predictors of outcomes for patients with juvenile idiopathic arthritis. Best Pract Res Clin Rheumatol 2002, 16:347-360.
60. Oen K, Malleson PN, Cabral DA, et al. Disease course and outcome of juvenile rheumatoid arthritis in a multicenter cohort. J Rheumatol 2002, 29:1989-1999.
61. Oen K, Malleson PN, Cabral DA, et al. Early predictors of longterm outcome in patients with juvenile rheumatoid arthritis: subset-specific correlations. J Rheumatol 2003, 30:585-593.
62. Ruperto N, Levinson JE, Ravelli A, et al. Long-term health outcomes and quality of life in American and Italian inception cohorts of patients with juvenile rheumatoid arthritis. I: Outcome status. J Rheumatol 1997, 24:945-951.
63. Ruperto N, Ravelli A, Levinson JE, et al. Long-term health outcomes and quality of life in American and Italian inception cohorts of patients with juvenile rheumatoid arthritis. II: Early predictors of outcome. J Rheumatol 1997, 24:952-958.
64. Murray K, Thompson SD, Glass DN Pathogenesis of juvenile chronic arthritis: genetic and environmental factors. Arch Dis Child 1997, 77:530-534.
65. Glass DN, Giannini EH Juvenile rheumatoid arthritis as a complex genetic trait. Arthritis Rheum 1999, 42:2261-2268.
66. Jarvis JN, Dozmorov I, Jiang K, et al. Novel approaches to gene expression analysis of active polyarticular juvenile rheumatoid arthritis. Arthritis Res Ther 2004, 6:R15-R32.
67. Prahalad S, Ryan MH, Shear ES, et al. Juvenile rheumatoid arthritis: linkage to HLA demonstrated by allele sharing in affected sibpairs. Arthritis Rheum 2000, 43:2335-2338.
68. Savolainen A, Saila H, Kotaniemi K, et al. Magnitude of the genetic component in juvenile idiopathic arthritis. Ann Rheum Dis 2000, 59(12):1001.
69. Saila HM, Savolainen HA, Kotaniemi KM, et al. Juvenile idiopathic arthritis in multicase families. Clin Exp Rheumatol 2001, 19:218-220.
70. Forre O, Smerdel A Genetic epidemiology of juvenile idiopathic arthritis. Scand J Rheumatol 2002, 31:123-128.
71. Thomson W, Donn R Juvenile idiopathic arthritis genetics: what's new? What's next?. Arthritis Res 2002, 4:302-306.
72. Rosen P, Moroldo MB, Lovell DJ, et al. Discordant phenotypes for methotrexate response in juvenile rheumatoid arthritis. Arthritis Rheum 2002, 46(Suppl):S470.
73. Katz PP Introduction to special patient outcomes in rheumatology issue of Arthritis Care and Research. Arthritis Care Res 2003, 49(5 Suppl):S1-S14.
74. Hull RG Guidelines for management of childhood arthritis. Rheumatology (Oxf) 2001, 40:1309-1312.
75. Temple R Current definitions of phases of investigation and the role of the FDA in the conduct of clinical trials. Am Heart J 2000, 139:S133-S135.
76. Weiner DL, Yuh L Biovavailability studies. Statistics in the Pharmaceutical Industry 1994, 215-245. Marcel Dekker, New York. C.R. Buncher, J.-Y. Tsay (Eds.).
77. Lovell DJ, Giannini EH, Reiff A, et al. Etanercept in children with polyarticular juvenile rheumatoid arthritis. Pediatric Rheumatology Collaborative Study Group. N Engl J Med 2000, 342:763-769.
78. Honkanen VE, Siegel AF, Szalai JP, et al. A three-stage clinical trial design for rare disorders. Stat Med 2001, 20:3009-3021.
79. Giannini EH Can non-fundable trials be conducted anyway? The case for open, randomised, actively controlled trials in rheumatology. Ann Rheum Dis 1998, 57:128-130.
80. Shaikov AV, Maximov AA, Speransky AI, et al. Repetitive use of pulse therapy with methylprednisolone and cyclophosphamide in addition to oral methotrexate in children with systemic juvenile rheumatoid arthritis: preliminary results of a longterm study. J Rheumatol 1992, 19:612-616.
81. Ruperto N, Murray KJ, Gerloni V, et al. A randomized trial of parenteral methotrexate comparing an intermediate dose with a higher dose in children with juvenile idiopathic arthritis who failed to respond to standard doses of methotrexate. Arthritis Rheum 2004, 50:2191-2201.
82. Giannini EH The N of 1 trials design in the rheumatic diseases. Arthritis Care Res 1998, 1:109-115.
83. Temple R, Ellenberg SS Placebo-controlled trials and active-control trials in the evaluation of new treatments. Part 1: Ethical and scientific issues. Ann Intern Med 2000, 133:455-463.
84. Vickers AJ, de Craen AJ Why use placebos in clinical trials? A narrative review of the methodological literature. J Clin Epidemiol 2000, 53:157-161.
85. Emanuel EJ, Miller FG The ethics of placebo-controlled trials: a middle ground. N Engl J Med 2001, 345:915-919.
86. Freedman B, Glass KC, Weijer C Placebo orthodoxy in clinical research. II: Ethical, legal, and regulatory myths. J Law Med Ethics 1996, 24:252-259.
87. Freedman B Placebo-controlled trials and the logic of clinical purpose. IRB 1990, 12:1-6.
88. Freedman B, Weijer C, Glass KC Placebo orthodoxy in clinical research. I: Empirical and methodological myths. J Law Med Ethics 1996, 24:243-251.
89. Beaton DE, Bombardier C, Katz JN, Wright JG A taxonomy for responsiveness. J Clin Epidemiol 2001, 54:1204-1217.
90. Fortin PR, Stucki G, Katz JN Measuring relevant change: an emerging challenge in rheumatologic clinical trials. Arthritis Rheum 1995, 38:1027-1030.
91. Felson DT, Anderson JJ, Boers M, et al. American College of Rheumatology. Preliminary definition of improvement in rheumatoid arthritis. Arthritis Rheum 1995, 38:727-735.
92. van der Heijde, van 't Hof M, van Riel PL, van de Putte Development of a disease activity score based on judgment in clinical practice by rheumatologists. J Rheumatol 1993, 20:579-581.
93. Giannini EH, Ruperto N, Ravelli A, et al. Preliminary definition of improvement in juvenile arthritis. Arthritis Rheum 1997, 40:1202-1209.
94. Larsen A, Dale K, Eek M Radiographic evaluation of rheumatoid arthritis and related conditions by standard reference films. Acta Radiol Diagn (Stockh) 1977, 18:481-491.
95. Sharp JT, Young DY, Bluhm GB, et al. How many joints in the hands and wrists should be included in a score of radiologic abnormalities used to assess rheumatoid arthritis?. Arthritis Rheum 1985, 28:1326-1335.
96. Ware JE, Sherbourne CD The MOS 36-item short-form health survey (SF-36). I: Conceptual framework and item selection. Med Care 1992, 30:473-483.
97. Landgraf JM Measuring and monitoring quality of life in children and youth: a brief commentary. Soz Praventivmed 2001, 46:281-282.
98. Varni JW, Seid M, Kurtin PS PedsQL 4.0: reliability and validity of the Pediatric Quality of Life Inventory version 4.0 generic core scales in healthy and patient populations. Med Care 2001, 39:800-812.
99. Varni JW, Seid M, Smith Knight T, et al. The PedsQL in pediatric rheumatology: reliability, validity, and responsiveness of the Pediatric Quality of Life Inventory Generic Core Scales and Rheumatology Module. Arthritis Rheum 2002, 46:714-725.
100. Hays RD, Anderson R, Revicki D Psychometric considerations in evaluating health-related quality of life measures. Qual Life Res 1993, 2:441-449.
101. Brunner HI, Silverman ED, To T, et al. Risk factors for damage in childhood-onset systemic lupus erythematosus: cumulative disease activity and medication use predict disease damage. Arthritis Rheum 2002, 46:436-444.
102. Mantel N, Haenszel W Statistical aspects of the analysis of data from retrospective studies of disease. J Natl Cancer Inst 1959, 22:719-748.
103. Brophy JM, Joseph L Placing trials in context using Bayesian analysis: GUSTO revisited by Reverend Bayes. JAMA 1995, 273:871-875.
104. Blume J, Peipert JF What your statistician never told you about P-values. J Am Assoc Gynecol Laparosc 2003, 10:439-444.
105. Pounds S, Morris SW Estimating the occurrence of false positives and false negatives in microarray studies by approximating and partitioning the empirical distribution of p-values. Bioinformatics 2003, 19:1236-1242.
106. Gruvberger-Saal SK, Eden P, Ringner M, et al. Predicting continuous values of prognostic markers in breast cancer from microarray gene expression profiles. Mol Cancer Ther 2004, 3:161-168.
107. Hosmer DW, Lemeshow S Applied Logistic Regression 2000, John Wiley & Sons, New York. 2nd ed.
108. Giannini EH, Malagon CN, Van Kerckhove C, et al. Longitudinal analysis of HLA associated risks for iridocyclitis in juvenile rheumatoid arthritis. J Rheumatol 1991, 18:1394-1397.
109. Lee ET, Wang JW Statistical Methods for Survival Data Analysis 2003, John Wiley & Sons, New York.
110. Jekel JF, Elmore JG, Katz DL Epidemiology, Biostatistics, and Preventative Medicine 1996, WB Saunders, Philadelphia.
111. Portney LG, Watkins MP Foundations of Clinical Research. Applications and Practice 1993, 509-516. Appleton & Lange, Norwalk, CT.
112. Stevens JP Applied Multivariate Statistics for the Social Sciences 2001, Lawrence Erlbaum, Mahwah, NJ. 4th ed.
113. Moher D, Schulz KF, Altman D The CONSORT statement: revised recommendations for improving the quality of reports of parallel-group randomized trials. JAMA 2001, 285:1987-1991.
114. Moher D, Jones A, Lepage L Use of the CONSORT statement and quality of reports of randomized trials: a comparative before-and-after evaluation. JAMA 2001, 285:1992-1995.
115. Davidoff F, DeAngelis CD, Drazen JM, et al. Sponsorship, authorship, and accountability. N Engl J Med 2001, 345:825-826. discussion 826-827.