Lebensqualität bei pulmonal arterieller und chronisch thromboembolischer pulmonaler Hypertonie

Zusammenfassung

Die Lebensqualität (Quality of life, QoL) im Allgemeinen und die gesundheitsbezogene Lebensqualität („Health-related QoL; HRQOL) im Besonderen rücken aktuell immer mehr in den Fokus des Interesse bei chronischen Krankheiten und somit auch für Patienten mit einer pulmonal-arteriellen Hypertonie (PAH) oder chronisch thromboembolischen pulmonalen Hypertonie (CTEPH). Es gibt Überlegungen, diese als primäre oder ko-primäre Outcome-Parameter in Studien einzusetzen. Die Erfassung der QoL/HRQOL und die Objektivierung klinisch relevanter Veränderungen dieser ist allerdings nicht trivial. Diese Übersichtsarbeit beleuchtet sowohl die allgemeinen Aspekte der Evaluation der HRQOL als auch die Vor- und Nachteile der hierbei aktuell verwendeten Instrumente.

Abstract

Assessments of general quality of life (QoL) and health-related quality of life (HRQOL) are increasingly important in the care of patients with chronic diseases including pulmonary arterial and chronic thromboembolic pulmonary hypertension and are under consideration as important endpoint of clinical trials and drug registration. The assessment of quality of life is not trivial. This review outlines the assets and pitfalls of general aspects of HRQOL and instruments used in PH.

Schlüsselwörter

gesundheitsbezogene Lebensqualität - Outcome-Parameter - HRQOL-Instrumente

Keywords

health-related quality of life - outcome parameters - HRQOL instruments

• Literatur

• 1 Hoeper MM, Bogaard HJ, Condliffe R et al. Definitions and diagnosis of pulmonary hypertension. J Am Coll of Cardiol 2013; 62 (Suppl. 25) D42-50
• 2 Galie N, Manes A, Negro L et al. A meta-analysis of randomized controlled trials in pulmonary arterial hypertension. Eur Heart J 2009; 30: 394-403
• 3 McLaughlin VV, Badesch DB, Delcroix M et al. End points and clinical trial design in pulmonary arterial hypertension. J Am Coll Cardiol 2009; 54: S97-107
• 4 Peacock A, Keogh A, Humbert M. Endpoints in pulmonary arterial hypertension: the role of clinical worsening. Curr Opin Pulm Med 2010; 16 (Suppl. 01) S1-9
• 5 Epstein AM. The outcomes movement – will it get us where we want to go?. NEJM 1990; 323: 266-70
• 6 Guyatt GH, Feeny DH, Patrick DL. Measuring health-related quality of life. Ann Intern Med 1993; 118: 622-9
• 7 Guillevin L, Armstrong I, Aldrighetti R et al. Understanding the impact of pulmonary arterial hypertension on patients' and carers' lives. Eur Respir Rev 2013; 22: 535-42
• 8 Gihl A. Health-related quality of life in pulmonary arterial hypertension. Adv Pulm Hypertens 2013; 8: 215-22
• 9 Hwang B, Howie-Esquivel J, Fleischmann KE et al. Family caregiving in pulmonary arterial hypertension. Heart Lung 2012; 41: 26-34
• 10 Lowe B, Grafe K, Ufer C et al. Anxiety and depression in patients with pulmonary hypertension. Psychosom Med 2004; 66: 831-6
• 11 Harzheim D, Klose H, Pinado FP et al. Anxiety and depression disorders in patients with pulmonary arterial hypertension and chronic thromboembolic pulmonary hypertension. Respir Res 2013; 14: 104
• 12 Halank M, Einsle F, Lehman S et al. Exercise capacity affects quality of life in patients with pulmonary hypertension. Lung 2013; 191: 337-43
• 13 White J, Hopkins RO, Glissmeyer EW et al. Cognitive, emotional, and quality of life outcomes in patients with pulmonary arterial hypertension. Respir Res 2006; 7: 55
• 14 Mullen MP, Andrus J, Labella MH et al. Quality of life and parental adjustment in pediatric pulmonary hypertension. Chest 2014; 145: 237-44
• 15 Taichman DB, Shin J, Hud L et al. Health-related quality of life in patients with pulmonary arterial hypertension. Respir Res 2005; 6: 92
• 16 WHO. Preamble to the Constitution of the World Health Organization as adopted by the International Health Conference: Official Records of the World Health Organization. 1948: 100
• 17 The WHOQOL Group. What quality of life?. World Health Forum 1996; 17: 354-6
• 18 Lindstrom B. Quality of life: a model for evaluating health for all. Conceptual considerations and policy implications. Soz Praventivmed 1992; 37: 301-6
• 19 Radoschewski M. Gesundheitsbezogene Lebensqualität – Konzepte und Maße. Entwicklungen und Stand im Überblick. Bundesgesundheitsblatt Gesundheitsforschung Gesundheitsschutz 2000; 43: 165-89
• 20 Ware Jr JE. Standards for validating health measures: definition and content. J Chronic Dis 1987; 40: 473-80
• 21 Patrick DL, Deyo RA. Generic and disease-specific measures in assessing health status and quality of life. Med Care 1989; 27: S217-32
• 22 Coons SJ, Rao S, Keininger DL et al. A comparative review of generic quality-of-life instruments. Pharmacoeconomics 2000; 17: 13-35
• 23 McDowell I. Measuring health. A guide to rating scales and questionnaires. General health status and quality of life. 3rd. ed. Oxford University Press; 2006: 520-710
• 24 McDowell I. Measuring health. A guide to rating scales and questionnaires. Glossary of technical terms. 3rd. ed. Oxford University Press; 2006: 711-7
• 25 Terwee CB, Bot SD, de Boer MR et al. Quality criteria were proposed for measurement properties of health status questionnaires. J Clin Epidemiol 2007; 60: 34-42
• 26 Frei A, Williams K, Vetsch A et al. A comprehensive systematic review of the development process of 104 patient-reported outcomes (PROs) for physical activity in chronically ill and elderly people. Health Qual Life Outcomes 2011; 9: 116
• 27 Cronbach LJ. Coefficient alpha and the internal structures of tests. Psychometrika 1951; 16: 297-334
• 28 Paiva CE, Barroso EM, Carneseca EC et al. A critical analysis of test-retest reliability in instrument validation studies of cancer patients under palliative care: a systematic review. BMC Med Res Methodol 2014; 14: 8
• 29 Terwee CB, Dekker FW, Wiersinga WM et al. On assessing responsiveness of health-related quality of life instruments: guidelines for instrument evaluation. Qual Life Res 2003; 12: 349-62
• 30 Liang MH. Longitudinal construct validity: establishment of clinical meaning in patient evaluative instruments. Med Care 2000; 38: II84-90
• 31 Cohen J. Statistical power analysis for the behavioral sciences. 2nd. Ed. Hillsdale, NJ: Lawrence Erlbaum; 1988
• 32 Kazis LE, Anderson JJ, Meenan RF. Effect sizes for interpreting changes in health status. Med Care 1989; 27: S178-89
• 33 Crosby RD, Kolotkin RL, Williams GR. Defining clinically meaningful change in health-related quality of life. J Clin Epidemiol 2003; 56: 395-407
• 34 Jaeschke R, Singer J, Guyatt GH. Measurement of health status. Ascertaining the minimal clinically important difference. Control Clin Trials 1989; 10: 407-15
• 35 Sprangers MA, Moinpour CM, Moynihan TJ et al. Assessing meaningful change in quality of life over time: a users' guide for clinicians. Mayo Clin Proc 2002; 77: 561-71
• 36 Revicki D, Hays RD, Cella D et al. Recommended methods for determining responsiveness and minimally important differences for patient-reported outcomes. J Clin Epidemiol 2008; 61: 102-9
• 37 Juniper EF, Guyatt GH, Willan A et al. Determining a minimal important change in a disease-specific Quality of Life Questionnaire. J Clin Epidemiol 1994; 47: 81-7
• 38 Norman GR, Sridhar FG, Guyatt GH et al. Relation of distribution- and anchor-based approaches in interpretation of changes in health-related quality of life. Med Care 2001; 39: 1039-47
• 39 Whately-Smith C, Watkins C, Mann H et al. Utility values in health technology assessments: a statistician's perspective. Pharm Stat 2014;
• 40 Puhan MA, Schunemann HJ, Wong E et al. The standard gamble showed better construct validity than the time trade-off. J Clin Epidemiol 2007; 60: 1029-33
• 41 Ware Jr JE, Sherbourne CD. The MOS 36-item short-form health survey (SF-36). I. Conceptual framework and item selection. Med Care 1992; 30: 473-83
• 42 Ware J, Kosinski M, Keller S. A 12-Item Short-Form Health Survey: construction of scales and preliminary tests of reliability and validity. Med Care 1996; 34: 220-33
• 43 Bullinger M. German translation and psychometric testing of the SF-36 Health Survey: preliminary results from the IQOLA Project. International Quality of Life Assessment. Soc Sci Med 1995; 41: 1359-66
• 44 Hunt SM, McKenna SP, McEwen J et al. The Nottingham Health Profile: subjective health status and medical consultations. Soc Sci Med A 1981; 15: 221-9
• 45 Kohlmann T, Bullinger M, Raspe HH. Die deutsche Form des Nottingham Health Profiles (NHP): Skalenstruktur, Reliabilität und Validität. Soz Praventivmed 1997; 42: 175-85
• 46 The EuroQol Group. EuroQol: a new facility for the measurement of health-related quality of life. Health Policy 1990; 16: 199-208
• 47 Greiner W, Claes C, Busschbach JJ et al. Validating the EQ-5D with time trade off for the German population. Eur J Health Econ 2005; 6: 124-30
• 48 Chen H, De Marco T, Kobashigawa EA et al. Comparison of cardiac and pulmonary-specific quality-of-life measures in pulmonary arterial hypertension. Eur Respir J 2011; 38: 608-16
• 49 Twiss J, McKenna S, Ganderton L et al. Psychometric performance of the CAMPHOR and SF-36 in pulmonary hypertension. BMC Pulm Med 2013; 13: 45
• 50 Wilkens H, Grimminger F, Hoeper M et al. Burden of pulmonary arterial hypertension in Germany. Respir Med 2010; 104: 902-10
• 51 Chua R, Keogh AM, Byth K et al. Comparison and validation of three measures of quality of life in patients with pulmonary hypertension. Intern Med J 2006; 36: 705-10
• 52 Strange G, Keogh AM, Williams TJ et al. Bosentan therapy in patients with pulmonary arterial hypertension: the relationship between improvements in 6 minute walk distance and quality of life. Respirology 2008; 13: 674-82
• 53 Gilbert C, Brown MC, Cappelleri JC et al. Estimating a minimally important difference in pulmonary arterial hypertension following treatment with sildenafil. Chest 2009; 135: 137-42
• 54 Wilson D, Parsons J, Tucker G. The SF-36 summary scales: problems and solutions. Soz Praventivmed 2000; 45: 239-46
• 55 Simon GE, Revicki DA, Grothaus L et al. SF-36 summary scores: are physical and mental health truly distinct?. Med Care 1998; 36: 567-72
• 56 Taft C, Karlsson J, Sullivan M. Do SF-36 summary component scores accurately summarize subscale scores?. Qual Life Res 2001; 10: 395-404
• 57 Brazier J, Roberts J, Deverill M. The estimation of a preference-based measure of health from the SF-36. J Health Econ 2002; 21: 271-92
• 58 Shafazand S, Goldstein MK, Doyle RL et al. Health-related quality of life in patients with pulmonary arterial hypertension. Chest 2004; 126: 1452-9
• 59 McKenna S, Doughty N, Meads D et al. The Cambridge Pulmonary Hypertension Outcome Review (CAMPHOR): A measure of health-related quality of life and quality of life for patients with pulmonary hypertension. Qual Life Res 2006; 15: 103-15
• 60 Cima K, Twiss J, Speich R et al. The German adaptation of the Cambridge Pulmonary Hypertension Outcome Review (CAMPHOR). Health Qual Life Outcomes 2012; 10: 110
• 61 Brooks R. EuroQol: the current state of play. Health Policy 1996; 37: 53-72
• 62 Myers C, Wilks D. Comparison of Euroqol EQ-5D and SF-36 in patients with chronic fatigue syndrome. Qual Life Res 1999; 8: 9-16
• 63 Roman A, Barbera JA, Castillo MJ et al. Health-related quality of life in a national cohort of patients with pulmonary arterial hypertension or chronic thromboembolic pulmonary hypertension. Arch Broncho-pneumol 2013; 49: 181-8
• 64 McKenna SP, Ratcliffe J, Meads DM et al. Development and validation of a preference based measure derived from the Cambridge Pulmonary Hypertension Outcome Review (CAMPHOR) for use in cost utility analyses. Health Qual Life Outcomes 2008; 6: 65
• 65 Rector TS, Kubo SH, Cohn JN. Patient self-assessment of their congestive heart failure part 2: content, reliability and validity of a new measure the Minnesota Living with Heart Failure Questionnaire. Heart Fail 1987; 3: 198-209
• 66 Cenedese E, Speich R, Dorschner L et al. Measurement of Quality of Life in Pulmonary Hypertension and its Significance. Eur Respir J 2006; 28: 808-15
• 67 Bonner N, Abetz L, Meunier J et al. Development and validation of the living with pulmonary hypertension questionnaire in pulmonary arterial hypertension patients. Health Qual Life Outcomes 2013; 11: 161
• 68 Swetz KM, Shanafelt TD, Drozdowicz LB et al. Symptom burden, quality of life, and attitudes toward palliative care in patients with pulmonary arterial hypertension: results from a cross-sectional patient survey. J Heart Lung Transplant 2012; 31: 1102-8
• 69 Gomberg-Maitland M, Thenappan T, Rizvi K et al. United States validation of the Cambridge Pulmonary Hypertension Outcome Review (CAMPHOR). J Heart Lung Transplant 2008; 27: 124-30
• 70 Small M, Piercy J, Pike J et al. Incremental burden of disease in patients diagnosed with pulmonary arterial hypertension receiving monotherapy and combination vasodilator therapy. Adv Ther 2014; 31: 168-79
• 71 McCabe C, Bennett M, Doughty N et al. Patient-reported outcomes assessed by the CAMPHOR questionnaire predict clinical deterioration in idiopathic pulmonary arterial hypertension and chronic thromboembolic pulmonary hypertension. Chest 2013; 144: 522-30
• 72 Chen H, Rosenzweig EB, Gotzkowsky SK et al. Treatment satisfaction is associated with improved quality of life in patients treated with inhaled treprostinil for pulmonary arterial hypertension. Health Qual Life Outcomes 2013; 11: 31
• 73 Batal O, Khatib OF, Bair N et al. Sleep quality, depression, and quality of life in patients with pulmonary hypertension. Lung 2011; 189: 141-9
• 74 Meads DM, McKenna SP, Doughty N et al. The responsiveness and validity of the CAMPHOR Utility Index. Eur Respir J 2008; 32: 1513-9
• 75 Yorke J, Corris P, Gaine S et al. emPHasis-10: development of a health-related quality of life measure in pulmonary hypertension. Eur Respir J 2014; 43: 1106-13
• 76 Rival G, Lacasse Y, Martin S et al. Effect of Pulmonary Arterial Hypertension Specific Therapies on Health-Related Quality of Life: A Systematic Review. Chest 2014;
• 77 Pepke-Zaba J, Beardsworth A, Chan M et al. Tadalafil therapy and health-related quality of life in pulmonary arterial hypertension. Curr Med Res Opin 2009; 25: 2479-85
• 78 Duffels MG, Vis JC, van Loon RL et al. Effect of bosentan on exercise capacity and quality of life in adults with pulmonary arterial hypertension associated with congenital heart disease with and without Down's syndrome. Am J Cardiol 2009; 103: 1309-15
• 79 Vis JC, Duffels MG, Mulder P et al. Prolonged beneficial effect of bosentan treatment and 4-year survival rates in adult patients with pulmonary arterial hypertension associated with congenital heart disease. Int J Cardiol 2013; 164: 64-9
• 80 Denton CP, Humbert M, Rubin L et al. Bosentan treatment for pulmonary arterial hypertension related to connective tissue disease: a subgroup analysis of the pivotal clinical trials and their open-label extensions. Ann Rheum Dis 2006; 65: 1336-40
• 81 Denton CP, Pope JE, Peter HH et al. Long-term effects of bosentan on quality of life, survival, safety and tolerability in pulmonary arterial hypertension related to connective tissue diseases. Ann Rheum Dis 2008; 67: 1222-8
• 82 Mereles D, Ehlken N, Kreuscher S et al. Exercise and respiratory training improve exercise capacity and quality of life in patients with severe chronic pulmonary hypertension. Circulation 2006; 114: 1482-9
• 83 Nagel C, Prange F, Guth S et al. Exercise training improves exercise capacity and quality of life in patients with inoperable or residual chronic thromboembolic pulmonary hypertension. PLoS One 2012; 7: e41603
• 84 Sitbon O, Gressin V, Speich R et al. Bosentan for the treatment of human immunodeficiency virus-associated pulmonary arterial hypertension. Am J Respir Crit Care Med 2004; 170: 1212-7
• 85 Gnanasakthy A, Lewis S, Clark M et al. Potential of patient-reported outcomes as nonprimary endpoints in clinical trials. Health Qual Life Outcomes 2013; 11: 83
• 86 Mordin M, Lewis S, Gnanasakthy A et al. Patient-reported outcomes as mentioned in product development guidance. Value Health 2010; 13: A17-A8
• 87 US Department of Health and Human Services, Food and Drug Administration. Guidance for industry: Patient-reported outcome measures: use in medical product development to support labeling claims. http://www.fda.gov/downloads/Drugs/GuidanceComplianceRegulatoryInformation/Guidances/UCM193282.pdf Accessed May 4, 2014
• 88 Reeve BB, Wyrwich KW, Wu AW et al. ISOQOL recommends minimum standards for patient-reported outcome measures used in patient-centered outcomes and comparative effectiveness research. Qual Life Res 2013; 22: 1889-905
• 89 Brettschneider C, Lühmann D, Raspe H. Der Stellenwert von Patient-Reported Outcomes (PRO) im Kontext von Health Technology Assessment (HTA). Informative value of Patient-Reported Outcomes (PRO) in Health Technology Assessment (HTA). Schriftenreihe Health Technology Assessment Bd. 109. Köln: Deutsches Institut für Medizinische Dokumentation und Information (DIMDI); 2011
• 90 DeMuro C, Clark M, Mordin M et al. Reasons for rejection of patient-reported outcome label claims: a compilation based on a review of patient-reported outcome use among new molecular entities and biologic license applications, 2006-2010. Value Health 2012; 15: 443-8
• 91 http://www.eventbrite.com/e/pulmonary-arterial-hypertension-public-meeting-on-patient-focused-drug-development-registration-9798737287 Accessed May 4, 2014.