Are all outcomes in chronic heart failure rated equally? An argument for a patient-centred approach to outcome assessment

Heart Failure Reviews

Volumn 19, Issue 2, 2014, Pages 153-162

  Chang, Sungwon ^a,b   Newton, Phillip J ^c   Inglis, Sally ^c   Luckett, Tim ^c,d   Krum, Henry ^e   Macdonald, Peter ^f   Davidson, Patricia M ^c,d  

^a CURTIN UNIVERSITY   (Australia)

^b UNIVERSITY OF WESTERN SYDNEY   (Australia)

^c UNIVERSITY OF TECHNOLOGY SYDNEY   (Australia)

^d UNIVERSITY OF NEW SOUTH WALES   (Australia)

^e MONASH UNIVERSITY   (Australia)

^f ST VINCENT S HOSPITAL   (Australia)

Author keywords

Chronic heart failure; Composite endpoints; Outcome assessment; Outcome measurement; Patient important outcome; Patient centred

Indexed keywords

ADVERSE DRUG REACTION; ARTICLE; EVENT FREE SURVIVAL; FUNCTIONAL STATUS; HEALTH CARE COST; HEALTH CARE DISPARITY; HEALTH CARE QUALITY; HEALTH CARE UTILIZATION; HEART FAILURE; HOSPITAL READMISSION; HOSPITALIZATION; HUMAN; MORTALITY; OUTCOME ASSESSMENT; PATIENT CARE; PATIENT COMPLIANCE; PATIENT SAFETY; PATIENT SATISFACTION; PROGNOSIS; QUALITY OF LIFE; SELF CARE;

CHRONIC DISEASE; HEART FAILURE; HUMANS; OUTCOME ASSESSMENT (HEALTH CARE); PATIENT-CENTERED CARE; QUALITY OF LIFE; TREATMENT OUTCOME;

EID: 84896728104     PISSN: 13824147     EISSN: 15737322     Source Type: Journal    
DOI: 10.1007/s10741-012-9369-0     Document Type: Article

References (79)

1. Clark RA, McLennan S, Dawson A, Wilkinson D, Stewart S (2004) Uncovering a hidden epidemic: a study of the current burden of heart failure in Australia. Heart Lung Circ 13:266-273
2. Krum H, Abraham WT (2009) Heart failure. Lancet 373:941-955
3. Stewart AL, Greenfield S, Hays RD, Wells K, Rogers WH et al (1989) Functional status and well-being of patients with chronic conditions: results from the medical outcomes study. JAMA 262:907-913
4. Jeon Y-H, Kraus S, Jowsey T, Glasgow N (2010) The experience of living with chronic heart failure: a narrative review of qualitative studies. BMC Health Serv Res 10:77
5. Pattenden JF, Roberts H, Lewin RJP (2007) Living with heart failure; patient and carer perspectives. Eur J Cardiovasc Nurs 6:273-279
6. Weinberger M, Oddone EZ, Henderson WG (1996) Does increased access to primary care reduce hospital readmissions? N Engl J Med 334:1441-1447
7. Normand C (2009) Measuring outcomes in palliative care: Limitations of QALYs and the road to PalYs. J Pain Symptom Manage 38:27-31
8. Davidson P, Cockburn J, Daly J, Fisher R (2004) Patient-centred needs assessment: rationale for a psychometric measure for assessing needs in heart failure. J Cardiovasc Nurs 19:162-170
9. Allen LA, Hernandez AF, O'Connor CM, Felker GM (2009) End points for clinical trials in acute heart failure syndromes. J Am Coll Cardiol 53:2248-2258
10. Anand IS, Florea VG (2010) End points in chronic heart failure clinical trials. Dialogues Cardiovasc Med 15:81-102
11. Neaton JD, Gray G, Zuckerman BD, Konstam MA (2005) Key issues in end point selection for heart failure trials: composite end points. J Cardiac Fail 11:567-575
12. Zannad F, Stough WG, Pitt B, Cleland JGF, Adams KF et al (2008) Heart failure as an endpoint in heart failure and non-heart failure cardiovascular clinical trials: the need for a consensus definition. Eur Heart J 29:413-421
13. Kleinpell RM (1997) Whose outcomes, patients, providers, or payers? Nurs Clin N Am 32:513-520
14. Doward LC, Gnanasakthy A, Baker MG (2010) Patient reported outcomes: looking beyond the label claim. Health Qual Life Outcomes 8(1):89
15. Gabriel SE, Normand S-LT (2012) Getting the methods right - the foundation of patient-centered outcomes research. N Engl J Med 367:787-790
16. Guyatt G, Rennie D, Meade MO, Cook D (2008) Users' guides to the medical literature: essentials of evidence-based clinical practice New York. McGraw-Hill Professional Publishing, NY
17. Brennan TA, Leape LL, Laird NM, Hebert L, Localio AR et al (1991) Incidence of adverse events and negligence in hospitalized patients. N Engl J Med 324:370-376
18. Lee WC, Chavez YE, Baker T, Luce BR (2004) Economic burden of heart failure: a summary of recent literature. Heart Lung 33:362-371
19. Radford M (2005) ACC/AHA key data elements and definitions for measuring the clinical management and outcomes of patients with chronic heart failure: a report of the American College of Cardiology/American Heart Association task force on clinical data standards (Writing Committee to Develop Heart Failure Clinical Data Standards). J Am Coll Cardiol 46:1179-1207
20. Lohr KN (1988) Outcome measurement: concepts and questions. Inquiry 25:37-50
21. Cohn J, Cleland JGF, Lubsen J, Borer JS, Steg PG et al (2009) Unconventional end points in cardiovascular clinical trials: should we be moving away from morbidity and mortality? J Cardiac Fail 15:199-205
22. Yusuf S, Negassa A (2002) Choice of clinical outcomes in randomized trials of heart failure therapies: disease-specific or overall outcomes? Am Heart J 143:22-28
23. Walsh MN, Bove AA, Cross RR, Ferdinand KC, Forman DE et al (2012) ACCF 2012 health policy statement on patient-centered care in cardiovascular medicine: a report of the American College of Cardiology Foundation Clinical Quality Committee. J Am Coll Cardiol 59:2125-2143
24. Patrick D (2003) Patient reported outcomes: an organising tool for concepts, measures, and applications. Qual Life Newsl 31:1-5
25. Rector TS, Tschumperlin LK, Kubo SH, Bank AJ, Francis GS et al (1995) 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 Cardiac Fail 1:201-206
26. United States Department of Health and Human Services Food and Drug Administration Guidance for Industry (2009) Patient-reported outcome measures: use in medical product development to support labeling claims. Available at: www.fda.gov/downloads/Drugs/GuidanceComplianceRegulatoryInformation/Guidances/ UCM193282.pdf
27. Chang S, Gholizadeh L, Salamonson Y, Digiacomo M, Betihavas V et al (2011) Health span or life span: the role of patient-reported outcomes in informing health policy. Health Policy 100:96-104
28. Erickson P (2004) A health outcomes framework for assessing health status and quality of life: enhanced data for decision making. JNCI Monogr 2004(33):168-177
29. Greenhalgh J, Long AF, Flynn R (2005) The use of patient reported outcome measures in routine clinical practice: lack of impact or lack of theory? Soc Sci Med 60:833-843
30. World Health Organization (2003) Adherence to long-term therapies: evidence for action. World Health Organization, Geneva
31. Suarez-Almazor ME, Conner-Spady B, Kendall CJ, Russell AS, Skeith K (2001) Lack of congruence in the ratings of patients' health status by patients and their physicians. Med Decis Making 21:113
32. Willke RJ, Burke LB, Erickson P (2004) Measuring treatment impact: a review of patient-reported outcomes and other efficacy endpoints in approved product labels. Control Clin Trials 25:535-552
33. Spertus JA (2008) Evolving applications for patient-centered health status measures. Circulation 118:2103-2110
34. Rose EA, Gelijns AC, Moskowitz AJ, Heitjan DF, Stevenson LW et al (2001) Long-term use of a left ventricular assist device for end-stage heart failure. N Engl J Med 345:1435-1443
35. Chang S, Davidson PM, Newton PJ, Krum H, Salamonson Y, Macdonald P (2012) What is the methodological and reporting quality of health related quality of life in chronic heart failure clinical trials? Int J Cardiol. doi: 10.1016/j.ijcard.2012.01.019
36. Leidy N, Vernon M (2008) Perspectives on patient-reported outcomes: content validity and qualitative research in a changing clinical trial environment. Pharmacoeconomics 26:363-370
37. Luo X, Cappelleri JC (2008) A practical guide on incorporating and evaluating patient-reported outcomes in clinical trials. Clin Res Regul Aff 25:197-211
38. Chassany O, Sagnier P, Marquis P, Fullerton S, Aaronson N (2002) Patient-reported outcomes: the example of health-related quality of life - a European guidance document for the improved integration of health-related quality of life assessment in the drug regulatory process. Drug Inf J 36:209-238
39. Hakamies-Blomqvist L, Luoma ML, Sjöström J, Pluzanska A, Sjödin M et al (2001) Timing of quality of life (QoL) assessments as a source of error in oncological trials. J Adv Nurs 35:709-716
40. Revicki DA, Osoba D, Fairclough D, Barofsky I, Berzon R et al (2000) Recommendations on health-related quality of life research to support labeling and promotional claims in the United States. Qual Life Res 9:887-900
41. Kvien TK, Heiberg T, Hagen KB (2007) Minimal clinically important improvement/difference (MCII/MCID) and patient acceptable symptom state (PASS): what do these concepts mean? Ann Rheum Dis 66:iii40-iii41
42. Spertus JA, Conard MW, Rinaldi J, Tsuyuki R, Krumholz H et al (2002) The Kansas City cardiomyopathy questionnaire is sensitive to clinical change in congestive heart failure. J Am Coll Cardiol 39(Supplement 2):460
43. Caughey GE, Roughead EE, Shakib S, Vitry AI, Gilbert AL (2011) Co-morbidity and potential treatment conflicts in elderly heart failure patients: a retrospective, cross-sectional study of administrative claims data. Drugs Aging 28:575-581
44. Jennings BM, Staggers N, Brosch LR (1999) A classification scheme for outcome indicators. J Nurs Scholarsh 31:381-388
45. Opasich C, Gualco A, De Feo S, Barbieri M, Cioffi G et al (2008) Physical and emotional symptom burden of patients with end-stage heart failure: what to measure, how and why. J cardiovasc Med 9:1104-1108
46. Liao L, Allen LA, Whellan DJ (2008) Economic burden of heart failure in the elderly. Pharmacoeconomics 26:447-462
47. Bleumink GS, Knetsch AM, Sturkenboom MCJM, Straus SMJM, Hofman A et al (2004) Quantifying the heart failure epidemic: prevalence, incidence rate, lifetime risk and prognosis of heart failure - the Rotterdam Study. Eur Heart J 25:1614-1619
48. Yusuf S, Reddy S, Ounpuu S, Anand S (2001) Global burden of cardiovascular diseases. Circulation 104:2855-2864
49. Buzney EA, Kimball AB (2008) A critical assessment of composite and coprimary endpoints: a complex problem. J Am Acad Dermatol 59:890-896
50. Moyé LA (2003) Introduction to composite endpoint multiple analyses in clinical trials: fundamentals for investigators. Springer, New York, pp 219-238
51. Chi GYH (2005) Some issues with composite endpoints in clinical trials. Fundam Clin Pharmacol 19:609-619
52. Freemantle N, Calvert M, Wood J, Eastaugh J, Griffin C (2003) Composite outcomes in randomized trials: greater precision but with greater uncertainty? JAMA 289:2554-2559
53. Bethel MA, Holman R, Haffner SM, Califf RM, Huntsman-Labed A et al (2008) Determining the most appropriate components for a composite clinical trial outcome. Am Heart J 156:633-640
54. Packer M (2001) Proposal for a new clinical end point to evaluate the efficacy of drugs and devices in the treatment of chronic heart failure. J Cardiac Fail 7:176-182
55. Cleland JGF (2002) How to assess new treatments for the management of heart failure: composite scoring systems to assess the patients' clinical journey. Eur J Heart Fail 4:243-247
56. Braunwald E, Cannon CP, McCabe CH (1992) An approach to evaluating thrombolytic therapy in acute myocardial infarction: the 'unsatisfactory outcome' end point. Circulation 86:683-687
57. Taylor AL, Ziesche S, Yancy C, Carson P, D'Agostino R Jr. et al (2004) Combination of isosorbide dinitrate and hydralazine in blacks with heart failure. N Engl J Med 351:2049-2057
58. Felker GM, Anstrom KJ, Rogers JG (2008) A global ranking approach to end points in trials of mechanical circulatory support devices. J Cardiac Fail 14:368-372
59. Tukey JW (1979) Methodology, and the statistician's responsibility for BOTH accuracy AND relevance. J Am Stat Assoc 74:786-793
60. Long AF, Dixon P (1996) Monitoring outcomes in routine practice: defining appropriate measurement criteria. J Eval Clin Pract 2:71-78
61. Davies HTO, Crombie IK (1997) Interpreting health outcomes. J Eval Clin Pract 3:187-199
62. Epping-Jordan JE, Pruitt SD, Bengoa R, Wagner EH (2004) Improving the quality of health care for chronic conditions. Qual Saf Health Care 13:299-305
63. Sears SF, Sowell LV, Kuhl EA, Handberg EM, Kron J et al (2006) Quality of death: implantable cardioverter defibrillators and proactive care. Pacing Clin Electrophysiol 29:637-642
64. Long AF (1997) Key issues in outcomes measurement. Int J STD AIDS 8:663-667
65. Cowley AJ, Skene AM, Enoximone Investigators (1994) Treatment of severe heart failure: quantity or quality of life? A trial of enoximone. Br Heart J 72:226-230
66. Clifford S, Barber N, Elliott R, Hartley E, Horne R (2006) Patient-centred advice is effective in improving adherence to medicines. Pharm World Sci 28:165-170
67. Ganz PAMD (2002) What outcomes matter to patients: a physician-researcher point of view. Med Care 40(6):11-19
68. Boyd CM, Darer J, Boult C, Fried LP, Boult L et al (2005) Clinical practice guidelines and quality of care for older patients with multiple comorbid diseases. JAMA 294:716-724
69. Zanolla L, Zardini P (2003) Selection of endpoints for heart failure clinical trials. Eur J Heart Fail 5:717-723
70. Kind P, Lafata JE, Matuszewski K, Raisch D (2009) The use of QALYs in clinical and patient decision-making: issues and prospects. Value Health 12:S27-S30
71. Carr-Hill RA (1991) Allocating resources to health care: is the QALY (Quality Adjusted Life Year) a technical solution to a political problem? Int J Health Serv 21:351-363
72. Cote I, Gregoire JP, Moisan J (2000) Health-related quality-of-life measurement in hypertension: a review of randomised controlled drug trials. Pharmacoeconomics 18:435-450
73. Gohler A, Conrads-Frank A, Worrell SS, Geisler BP, Halpern EF et al (2008) Decision-analytic evaluation of the clinical effectiveness and cost-effectiveness of management programmes in chronic heart failure. Eur J Heart Fail 10:1026-1032
74. Stewart S, Jenkins A, Buchan S, McGuire A, Capewell S et al (2002) The current cost of heart failure to the National Health Service in the UK. Eur J Heart Fail 4:361-371
75. Krumholz HM (2004) The year in health care delivery and outcomes research. J Am Coll Cardiol 44:1130-1136
76. Van den Block L, Deschepper R, Drieskens K, Bauwens S, Bilsen J et al (2007) Hospitalisations at the end of life: using a sentinel surveillance network to study hospital use and associated patient, disease and healthcare factors. BMC Health Serv Res 7:69-78
77. Kozma C, Reeder C, Schulz R (1993) Economic, clinical, and humanistic outcomes: a planning model for pharmacoeconomic research. Clin Ther 15:1121-1132
78. Drummond MF, Sculpher MJ, Torrance GW, O'Brien B, Stoddard GL (2005) Methods for the economic evaluation of health care programmes. Oxford University Press, Oxford
79. Ferreira-González I, Permanyer-Miralda G, Busse JW, Bryant DM, Montori VM et al (2007) Methodologic discussions for using and interpreting composite endpoints are limited, but still identify major concerns. J Clin Epidemiol 60:651-657