Clinical and analytical considerations in the study of health status in device trials for heart failure

Journal of Cardiac Failure

Volumn 11, Issue 5 SPEC. ISS., 2005, Pages 396-403

  Normand, Sharon Lise T ^a,i   Rector, Thomas S ^b   Neaton, James D ^c   Piña, Ileana L ^d   Lazar, Ronald M ^e   Proestel, Scott E ^f   Fleischer, Dina J ^g   Cohn, Jay N ^g   Spertus, John A ^h  

^a HARVARD MEDICAL SCHOOL   (United States)

^b VA MEDICAL CENTER   (United States)

^c UNIVERSITY OF MINNESOTA   (United States)

^d CASE WESTERN RESERVE UNIVERSITY   (United States)

^e US Food and Drug Administration ^*  (United States)

^f CENTER FOR DRUG EVALUATION AND RESEARCH   (United States)

^g UNIVERSITY OF MINNESOTA MEDICAL SCHOOL   (United States)

^h UNIVERSITY OF MISSOURI KANSAS CITY   (United States)

ⁱ NONE   (United States)

Author keywords

Design; Endpoints; Quality of life; Regulatory

Indexed keywords

ARTICLE; FOOD AND DRUG ADMINISTRATION; HEALTH STATUS; HEART FAILURE; HUMAN; MEDICAL INSTRUMENTATION; OUTCOMES RESEARCH; PRIORITY JOURNAL; QUALITY OF LIFE; RELIABILITY; STATISTICAL ANALYSIS;

EID: 20444423366     PISSN: 10719164     EISSN: None     Source Type: Journal    
DOI: 10.1016/j.cardfail.2005.04.002     Document Type: Article

References (43)

1. K.E. Covinsky, A.W. Wu, C.S. Landefeld, A.F. Connors, R.S. Phillips, and J. Tsevat Health status versus quality of life in older patients: does the difference matter? Am J Med 106 1999 435 440
2. A. Leplege, and S. Hunt The problem of quality of life in medicine JAMA 278 1997 47 50
3. I.B. Wilson, and P.D. Cleary Linking clinical variables with health-related quality of life: a conceptual model of patient outcomes JAMA 273 1995 59 65
4. G.H. Guyatt, D.H. Feeny, and D.L. Patrick Measuring health-related quality of life Ann Intern Med 118 1993 622 629
5. M.A. Testa, and D.C. Simonson Assessment of quality-of-life outcomes N Engl J Med 334 1996 835 840
6. T.M. Gill, and A.R. Feinstein A critical appraisal of the quality of quality-of-life measurements JAMA 272 1994 619 626
7. G.H. Guyatt, B. Kirshner, and R. Jaeschke Measuring health status: what are the necessary measurement properties? J Clin Epidemiol 45 1992 1341 1345
8. L. Gorkin, N.K. Norvell, R.C. Rosen, E. Charles, S.A. Shumaker, and K.M. McIntyre Assessment of quality of life as observed from the baseline data of the Studies of Left Ventricular Dysfunction (SOLVD) trial quality-of-life substudy Am J Cardiol 71 1993 1069 1073
9. C.P. Green, C.B. Porter, D.R. Bresnahan, and J.A. Spertus Development and evaluation of the Kansas City Cardiomyopathy Questionnaire: a new health status measure for heart failure J Am Coll Cardiol 35 2000 1245 1255
10. S.J. Bennett, N.B. Oldridge, G.J. Eckert, J.L. Embree, S. Browning, and N. Hou Discriminant properties of commonly used quality of life measures in heart failure Qual Life Res 11 2002 349 359
11. T.S. Rector, S.H. Kubo, and J.N. Cohn Validity of the Minnesota Living with Heart Failure questionnaire as a measure of therapeutic response to enalapril or placebo Am J Cardiol 71 1993 1106 1110
12. G. Guyatt, S. Walter, and G. Norman Measuring change over time: assessing the usefulness of evaluative instruments J Chronic Dis 40 1987 171 178
13. G.H. Guyatt, S.J. Veldhuyzen Van Zanten, D.H. Feeny, and D.L. Patrick Measuring quality of life in clinical trials: a taxonomy and review Can Med Assoc J 140 1989 1441 1448
14. G.H. Guyatt, R.A. Deyo, M. Charlson, M.N. Levine, and A. Mitchell Responsiveness and validity in health status measurement: a clarification J Clin Epidemiol 42 1989 403 408
15. Knapp TR. The reliability of measuring instruments. Ed 2. Edgeworth Laboratory for Quantitative Educational and Behavioral Science Series. Vancouver, BC, Canada; 2002.
16. D.F. Alwin Problems in the estimation and interpretation of the reliability of survey data Qual Quant 23 1989 277 331
17. Linguistic validation manual for patient reported outcomes instruments 2004 MAPI Research Trust Lyon, France
18. K.W. Wyrwich, N.A. Nienaber, W.M. Tierney, and F.D. Wolinsky Linking clinical relevance and statistical significance in evaluating intra-individual changes in health-related quality of life Med Care 37 1999 469 478
19. R. Jaeschke, J. Singer, and G.H. Guyatt Measurement of health status. Ascertaining the minimal clinically important difference Contr Clin Trials 10 1989 407 415
20. N.C. Santanello, D. Baker, J.C. Cappelleri, K. Copley-Merriman, R. DeMarinis, and J.P. Gagnon Regulatory issues for health-related quality of life - PhRMA Health Outcomes Committee Workshop, 1999 Value Health 5 2002 14 25
21. T.S. Rector, and J.N. Cohn Assessment of patient outcome with the Minnesota Living with Heart Failure questionnaire: reliability and validity during a randomized, double-blind, placebo-controlled trial of pimobendan. Pimobendan Multicenter Research Group Am Heart J 124 1992 1017 1025
22. A.M. Feldman, K.L. Baughman, W.K. Lee, S.H. Gottlieb, J.L. Weiss, and L.C. Becker Usefulness of OPC-8212, a quinolinone derivative, for chronic congestive heart failure in patients with ischemic heart disease or idiopathic dilated cardiomyopathy Am J Cardiol 68 1991 1203 1210
23. M. Packer, W.S. Colucci, J.D. Sackner-Bernstein, V. Kinhal, S.S. Gottlieb, and J.J. Gregory Double-blind, placebo-controlled study of the effects of carvedilol in patients with moderate to severe heart failure. The PRECISE Trial. Prospective Randomized Evaluation of Carvedilol on Symptoms and Exercise Circulation 94 1996 2793 2799
24. B.M. Massie, M.R. Berk, S.C. Brozena, U. Elkayam, J.F. Plehn, and M.L. Kukin 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 88 1993 492 501
25. P. Giannuzzi, P.L. Temporelli, U. Corra, and L. Tavazzi Antiremodeling effect of long-term exercise training in patients with stable chronic heart failure: results of the Exercise in Left Ventricular Dysfunction and Chronic Heart Failure (ELVD-CHF) Trial Circulation 108 2003 554 559
26. A. Deswal, B. Bozkurt, Y. Seta, S. Parilti-Eiswirth, A. Hayes, and C. Blosch Safety and efficacy of a soluble P75 tumor necrosis factor receptor (Enbrel, etanercept) in patients with advanced heart failure Circulation 99 1999 3224 3226
27. T.S. Rector, G. Johnson, W.B. Dunkman, G. Daniels, L. Farrell, and A. Henrick Evaluation by patients with heart failure of the effects of enalapril compared with hydralazine plus isosorbide dinitrate on quality of life. V-HeFT II. The V-HeFT VA Cooperative Studies Group Circulation 87 1993 VI71 VI77
28. T.S. Rector, L.K. Tschumperlin, S.H. Kubo, A.J. Bank, G.S. Francis, and K.M. McDonald Use of the Living With Heart Failure questionnaire to ascertain patients' perspectives on improvement in quality of life versus risk of drug-induced death J Card Failure 1 1995 201 206
29. A.J. Baxter, A. Spensley, A. Hildreth, G. Karimova, J.E. O'Connell, and C.S. Gray Beta blockers in older persons with heart failure: tolerability and impact on quality of life Heart 88 2002 611 614
30. J.N. Cohn, S.O. Goldstein, B.H. Greenberg, B.L. Lorell, R.C. Bourge, and B.E. Jaski A dose-dependent increase in mortality with vesnarinone among patients with severe heart failure. Vesnarinone Trial Investigators N Engl J Med 339 1998 1810 1816
31. M.W. Conard, J.A. Spertus, P. Heidenreich, E. Peterson, W.S.C. Poston, and W. Weintraub Examining the concordance between patient and physician assessments of health status in heart failure patients J Am Coll Cardiol 39 Suppl 2 2002 461A
32. D.B. Rubin Multiple imputation after 18+ years J Am Stat Assoc 91 1996 473 489
33. J.W. Schafer, and J.W. Graham Misisng data. Our view of the state of the art Psychol Meth 7 2002 147 177
34. R.J. Little Pattern-mixture models for multivariate incomplete data J Am Stat Assoc 88 1993 125 134
35. D.L. Fairclough Methods of analysis for longitudinal studies of health-related quality of life M.J. Staquet R.D. Hayr P.M. Fayer Quality of life assessment in clinical trials: methods and practice 1998 Oxford University Press New York
36. R.J. Little, and Y. Wang Pattern-mixture models for multivariate incomplete data with covariates Biometrics 52 1996 98 111
37. G. Verbeke, and G. Molenberghs Linear mixed models for longitudinal data 2000 Springer New York
38. M.D. Schluchter, T. Greene, and G.J. Beck Analysis of change in the presence of informative censoring: application to a longitudinal trial of progressive renal disease Stat Med 20 2001 989 1007
39. G. Touloumi, S.J. Pocock, A.G. Babiker, and J. Darbyshire Estimation and comparison of rates of change in longitudinal studies with informative dropout Stat Med 18 1999 1215 1233
40. P.P. Glasziou, B.F. Cole, R.D. Belger, J. Hilden, and R.J. Simes Quality adjusted survival analysis with repeated quality of life measures Stat Med 17 1998 1215 1229
41. C. Wang, J. Douglas, and S. Anderson Item response models for the joint analysis of quality of life and survival Stat Med 21 2002 129 142
42. J.M. Lachin Worst-rank score analysis with informatively missing observations in clinical trials Control Clin Trials 20 1999 408 422
43. P.C. O'Brien Procedures for comparing samples with multiple endpoints Biometrics 40 1984 1079 1087