Top

Pediatric Drugs

Published in:

01-01-2003 | Review Article

Quality of Life in Children and Adolescents with Cystic Fibrosis

Implications for Optimizing Treatments and Clinical Trial Design

Authors: Professor Janice Abbott, Louise Gee

Published in: Pediatric Drugs | Issue 1/2003

Abstract

Health related quality of life (QOL) as an outcome measure in clinical trials is becoming increasingly important. Trials should not only be able to demonstrate the pharmacologic activity of a therapy, but of equal importance, they should demonstrate clinical effectiveness that is of significant benefit to the patient. QOL measurement provides a way of incorporating the child/parent’s perspective of how cystic fibrosis (CF) and its therapies impact on their lives. Several validated generic instruments have been employed to measure QOL in adolescents and adults. QOL assessment is more difficult in children and, therefore, has been employed less often in children with CF.

Difficulties arise with the issue of whether children can report their own experiences directly, or whether a parent or clinician should report on behalf of the child. A child-centered approach is imperative since the literature indicates that children are able to report on their own QOL. An additional complication has been the use of adult measures with children. These are often inappropriate in their complexity, use of language, response scales, and time frame.

The evaluation of pharmacologic therapies can profit from QOL measurement. The effectiveness of a drug and any adverse effects that impact on daily life can be assessed from the child/parent’s viewpoint. Home therapy versus hospital therapy and drug delivery systems, are additional areas where QOL as an outcome measure is valuable. There have been relatively few appropriately powered trials in CF, and only a minority of these have evaluated QOL as an outcome measure.

This review highlights areas where QOL measurement is appropriate. It focuses on the pharmacologic trials that have employed QOL assessment for antibiotic, mucociliary clearance, anti-inflammatory, and nutritional therapies.

Methodological issues of incorporating QOL assessment into trials center on cross-cultural and data interpretation issues. QOL measurement in CF has been patchy and largely unreliable. The notion that improved symptoms equate with improved QOL is erroneous. Measurement of how symptoms impact on QOL is essential. Currently, the development and validation of CF specific measures (across the CF age range) provides optimism for appropriate QOL measurement in clinical trials, and for future meta-analysis and systematic reviews.

Kerem BS, Rommens JM, Buchanan JA, et al. Identification of the cystic fibrosis gene: genetic analysis. Science 1989; 245: 1073–80PubMedCrossRef

Welsh MJ. Electrolyte transport by airway epithelia. Physiol Rev 1990; 67: 1143–64

UK cystic fibrosis survey 1993 report. Bromley: The Cystic Fibrosis Trust, 1993

Dodge JV, Morison S, Lewis PA, et al. Incidence, population and survival of cystic fibrosis in the UK (1968–95). Arch Dis Child 1977; 77: 493–6CrossRef

Cystic fibrosis patients’ registry: 1995 annual data report. Bethesda (MD): Cystic Fibrosis Foundation, 1996

Ryan G, Mukhopadhyay S, Singh M. Nebulised anti-;pseudomonal antibiotics for cystic fibrosis (Cochrane Review). Available in The Cochrane Library [database on disk and CD ROM]. Updated quarterly. The Cochrane Collaboration; Issue 3. Oxford: Oxford Update Software, 2001

Kearney CE, Wallis CE. Deoxyribonuclease for cystic fibrosis (Cochrane Review). Available in The Cochrane Library [database on disk and CD ROM]. Updated quarterly. The Cochrane Collaboration; Issue 1. Oxford: Oxford Update Software, 2000

Wark PA, McDonald V. Nebulised hypertonic saline for cystic fibrosis (Cochrane Review). Available in The Cochrane Library [database on disk and CD ROM]. Updated quarterly. The Cochrane Collaboration; Issue 1. Oxford: Oxford Update Software, 2000

Gee L, Abbott J, Conway S, et al. Development of a disease specific health related quality of life measure for adults and adolescents with cystic fibrosis. Thorax 2000; 55: 946–54PubMedCrossRef

10.

Davis M, Modi A, Quittner AL, et al. Psychometric analysis of the cystic fibrosis questionnaire for parents of school-age children [abstract no. 329]. 24th European Cystic Fibrosis Conference; 2001 Jun 6–9; Vienna.

11.

Quittner AL, Davis M, Modi A, et al. Psychometric properties of a disease-specific quality of life measure for adolescents and adults with cystic fibrosis [abstract no. 330]. 24th European Cystic Fibrosis Conference; 2001 Jun 6–9; Vienna.

12.

Abbott J, Dodd M, Webb AK. Different perceptions of disease severity and self-care between patients with cystic fibrosis, their close companion, and physician. Thorax 1995; 50: 794–6PubMedCrossRef

13.

Abbott J, Webb AK, Dodd M. Quality of life in cystic fibrosis. J R Soc Med 1997; 90(31): 37–42PubMed

14.

Abbott J, Gee L. Contemporary psychosocial issues in cystic fibrosis: treatment adherence and quality of life. Disabil Rehabil 1998; 20: 262–71PubMedCrossRef

15.

Abbott J, Dodd M, Bilton D, et al. Treatment compliance in adults with cystic fibrosis. Thorax 1994; 49: 115–20PubMedCrossRef

16.

Conway S, Pond MN, Hamnett T, et al. Compliance with treatment in adult patients with cystic fibrosis. Thorax 1996; 51: 29–33PubMedCrossRef

17.

Kettler LJ, Winefield HR, Sawyer SM, et al. Determinants of adult adherence in cystic fibrosis. Thorax 2002; 57: 459–64PubMedCrossRef

18.

Abbott J, Conway S, Etherington C, et al. Perceived body image and eating behavior in young adults with cystic fibrosis and their healthy peers. J Behav Med 2000; 23, 6: 501–17PubMedCrossRef

19.

Tullis DE, Guyatt GH. Quality of life in cystic fibrosis. Pharmacoeconomics 1994; 8: 23–33CrossRef

20.

Quittner AL. Measurement of quality of life in cystic fibrosis. Curr Opin Pulm Med 1998; 4: 326–31PubMedCrossRef

21.

Orenstein D, Nixon P, Ross E, et al. The quality of well-being in cystic fibrosis. Chest 1989; 95: 344–7PubMedCrossRef

22.

Kaplan R, Anderson J, Wu A, et al. The quality of well-being scale. Med Care 1989; 27: S27–43PubMedCrossRef

23.

Czyzewski DI, Mariotto MJ, Bartholomew K, et al. Measurement of quality of well being in a child and adolescent cystic fibrosis population. Med Care 1994; 32: 965–72PubMedCrossRef

24.

Orenstein D, Pattishall E, Nixon P, et al. Quality of well-being before and after antibiotic treatment of pulmonary exacerbation in patients with cystic fibrosis. Chest 1990; 98: 1081–4PubMedCrossRef

25.

The EuroQol Group. EuroQol: a new facility for the measurement of health-related quality of life. Health Policy 1990; 16: 199–208

26.

Walters S, Warren RL. Assessment of quality of life in adults with CF in the United Kingdom using the EuroQol questionnaire [abstract]. 24th European Cystic Fibrosis Conference; 2001 Jun 6–9; Vienna.

27.

Hunt S, McEwen J, McKenna S. Measuring health status. London: Croom Helm, 1986

28.

Congleton J, Hodson M, Duncan-Skingle F. Quality of life in adults with cystic fibrosis. Thorax 1996; 51: 936–40PubMedCrossRef

29.

Yacoub MH, Banner NR, Khaghani A, et al. Heart-lung transplantation for cystic fibrosis and subsequent domino heart transplantation. J Heart Lung Transplant 1990; 9: 459–66

30.

Caine N, Sharples L, Smyth R, et al. Survival and quality of life of cystic fibrosis patients before and after heart-lung transplantation. Transplant Proc 1991; 23: 1203–4PubMed

31.

Dennis C, Caine N, Sharples L, et al. Heart-lung transplantation for end-stage respiratory disease in patients with cystic fibrosis at Papworth Hospital. J Heart Lung Transplant 1993; 12: 893–902PubMed

32.

Busschbach J, Horikx P, van der Bosch J, et al. Measuring the quality of life before and after bilateral lung transplantation in patients with cystic fibrosis. Chest 1994; 105: 911–7PubMedCrossRef

33.

Bergner M, Bobbitt R, Carter W, et al. The sickness impact profile: development and final version of a health status measure. Med Care 1981; 19: 787–805PubMedCrossRef

34.

de Jong W, Kaptein AA, van der Schans CP, et al. Quality of life in patients with cystic fibrosis. Pediatr Pulmonol 1997; 23: 95–100PubMedCrossRef

35.

Ware J, Sherbourne C. The Mos 36-item short-form health survey: conceptual framework and item selection. Med Care 1992; 30: 473–81PubMedCrossRef

36.

Goldbeck L, Shmitz T. Evaluation of HRQOL in adolescents and adults with cystic fibrosis [abstract no. PS10-4]. 22nd European Cystic Fibrosis Conference; 1998 Jun 13–19; Berlin.

37.

Gee L, Abbott J, Conway S, et al. Validation of the SF-36 for the assessment of quality of life in adolescents and adults with cystic fibrosis. J Cystic Fibrosis 2002; 1: 137–45CrossRef

38.

Barbers RG. Cystic fibrosis: bilateral living lobar vs cadaveric lung transplantation. Am J Med Sci 1998; 315(3): 155–60PubMedCrossRef

39.

Ranasinha C, Assoufi B, Shak S, et al. Efficacy and safety of short-term administration of aerosolised recombinant human Dnase I in adults with stable stage cystic fibrosis. Lancet 1993; 342: 199–202PubMedCrossRef

40.

Heijerman H, Rossem R, Bakker W. Effect of rhDNase on lung function and quality of life in adult cystic fibrosis patients. Neth J Med 1995; 46: 293–7PubMedCrossRef

41.

Wolter JM, Bowler SD, Nolan PJ, et al. Home intravenous therapy in cystic fibrosis: a prospective randomised trial examining clinical, quality of life and cost aspects. Eur Respir J 1997; 10: 896–900PubMed

42.

Guyatt G, Berman L, Townsend M, et al. A measure of quality of life for clinical trials in chronic lung disease. Thorax 1987; 42: 773–8PubMedCrossRef

43.

Bradley J, Dempster M, Wallace E, et al. The adaptation of a quality of life questionnaire for routine use in clinical practice: the CRQ in cystic fibrosis. Qual Life Res 1999; 8: 65–71PubMedCrossRef

44.

Jones PW. Quirk FH, Littlejohns CM, et al. A self-complete measure for chronic airflow limitation: the St. Georges respiratory questionnaire. Am Rev Respir Dis 1992; 145: 1321–7PubMed

45.

O’Boyle C, Browne J, Hickey A, et al. Schedule for the Evaluation of Individual Quality of Life (SEIQoL): a Direct Weighting Procedure for Quality of Life Domains (SEIQoL-DW). Administration manual. Dublin: Royal College of Surgeons in Ireland, Department of Psychology, 1995: 1–9

46.

Eiser C, Morse R. Quality of life measures in chronic diseases of childhood. Health Technol Assess 2001; 5(4): 1–157PubMed

47.

Munzenberger PJ, Van Wagnen CA, Abdulhamid I, et al. Quality of life as a treatment outcome in patients with cystic fibrosis. Pharmacotherapy 1999; 19(4): 393–8PubMedCrossRef

48.

Landgraf J. The Child Health Questionnaire (CHQ): a users manual. Boston (MA): The Health Institute, New England Medical Centre, 1996

49.

Varni JW, Seid M, Rode CA. The PedsQL: measurement model for the pediatric quality of life inventory. Med Care 1999; 37(2): 126–39PubMedCrossRef

50.

Aussage P, Henry B, Grosskopf C, et al. A new instrument for assessing quality of life in CF pediatric patients: the CFQ child C [abstract]. Pediatr Pulmonol 2000; Suppl. 20: 333

51.

Henry B, Grosskopf C, Aussage P, et al. Construction of a disease specific quality of life questionnaire for cystic fibrosis [abstract]. Pediatr Pulmonol 1996; Suppl. 13: 337–8

52.

Henry B, Staab D, Prados C, et al. How to measure quality of life in cystic fibrosis across countries and cultures: the cystic fibrosis questionnaire [abstract]. Pediatr Pulmonol 1998; Suppl. 17: 392–3

53.

Quittner AL, Sweeny S, Watrous M, et al. Translation and linguistic validation of a disease-specific quality of life measure for cystic fibrosis. J Pediatr Psychol 2000; 25(6): 403–14PubMedCrossRef

54.

Wenninger K, Aussage P, Wahn U, et al. The revised German cystic fibrosis questionnaire: validation of a disease-specific health-related quality of life instrument. Qual Life Res. In press

55.

Quittner AL, Buu A, Watrous M, et al. Cystic fibrosis questionnaire: a health-related quality of life measure — user manual. English version 1. Gainesville (FL): North American Cystic Fibrosis Foundation, 2000

56.

Schmitz TG, Goldbeck L. Validation of a new CF-specific quality of life module (FLZ-CF) for adolescent and adult patients [abstract no. 327]. 24th European Cystic Fibrosis Conference; 2001 Jun 6–9; Vienna.

57.

Connolly M, Johnson JA, Brown N, et al. Development of a disease-specific health-related quality of life instrument for people with cystic fibrosis (working paper 00-11). Legal deposit 2000. Ottawa (ON): National Library Canada, 2000

58.

Tullis ED, Gilljam M, Corey M. Development of a CF-specific quality of life instrument for adults with cystic fibrosis [abstract]. Pediatr Pulmonol 1999; Suppl. 19: 327

59.

Idler EL, Benyammini Y. Self-rated health and mortality: a review of twenty-seven community studies. J Health Soc Behav 1997; 38: 21–37PubMedCrossRef

60.

Aitken ML, Burke W, McDonald G, et al. Recombinant human DNase inhalation in subjects and patients with cystic fibrosis: a phase 1 study. JAMA 1992; 267: 1947–51PubMedCrossRef

61.

Czyzewski DI, Bartholomew LK. Quality of life outcomes in children and adolescents with cystic fibrosis. In: Drotar D, editor. Measuring health-related quality of life in children and adolescents: implications for research and practice. Mahwah (NJ): Lawrence Erlbaum Associates, 1998

62.

Perrin EC, Perrin JM. Clinicians’ assessments of children’ understanding of illness. Am J Dis Child 1983; 137: 874–8PubMed

63.

Perrin EC, Gerrity PS. There’s a demon in your belly: children’s understanding of illness. Pediatrics 1981; 67: 841–9PubMed

64.

Bibace R, Walsh ME. Children’s conceptions of illness. In: Bibace R, Walsh ME, editors. Children’s conceptions of health, illness and bodily functions. San Francisco (CA): Jossey Bass, 1981

65.

Siegal M, Patty J, Eiser C. A re-examination of children’s conceptions of contagion. Psychol Health 1990; 4: 159–65CrossRef

66.

Juniper EF, Guyatt GH, Feeny DH, et al. Measuring quality of life in children with asthma. Qual Life Res 1996; 5: 35–46PubMedCrossRef

67.

French DJ, Christie MJ, Snowden AJ. The reproducibility of the childhood asthma questionnaires: measures of quality of life for children with asthma aged 4–16 years. Qual Life Res 1994; 3: 215–24PubMedCrossRef

68.

Theunissen NCM, Vogels TGC, Koopman HM, et al. The proxy problem: child report versus parent report in health-related quality of life research. Qual Life Res 1998; 7: 387–97PubMedCrossRef

69.

Guyatt GH, Juniper EF, Griffith LE, et al. Children and adult perceptions of childhood asthma. Pediatrics 1997; 99: 165–8PubMedCrossRef

70.

Achenbach TM. The child behaviour profi1e: an empirically based system for assessing children’s behaviour problems and competences. Int J Mental Health 1979; 7: 24–42

71.

Jessop DJ, Riessman CK, Stein RE. Chronic childhood illness and maternal mental health. J Dev Behav Pediatr 1988; 9: 147–56PubMedCrossRef

72.

Britto MT, Kotagal UR, Chenier T, et al. Adolescent vs parental report of health related quality of life in cystic fibrosis [abstract]. Pediatr Pulmonol 2000; Suppl. 20: 333

73.

Quittner AL, Espelage DL, Davis MA. National validation of a health related quality of life measure [abstract]. Pediatr Pulmonol 2000; Suppl. 20: 334–5

74.

Thompson RJ, Gustafson KE, Hamlett KW. Psychological adjustment of children with cystic fibrosis: the role of the child cognitive processes and maternal adjustment. J Pediatr Psychol 1992; 17: 741–55PubMedCrossRef

75.

Christie MJ, French D, Snowden A, et al. Development of child-centered disease-specific questionnaires for living with asthma. Psychosom Med 1993; 55: 541–8PubMed

76.

Duffy CM, Arsenault L, Duffy KNW. Levels of agreement between parents and children in rating dysfunction in juvenile rheumatoid arthritis and juvenile spondyloarthrides. J Rheumatol 1993; 20: 1234–9

77.

Apajasalo M, Rautonen J, Holmberg C, et al. Quality of life in pre-adolescence: a 17-dimensional health-related measure. Qual Life Res 1996; 5: 532–8PubMedCrossRef

78.

Ridgeway D, Waters E, Kuczaj SA. Acquisition of emotion-descriptive language: receptive and productive vocabulary norms for ages 18 months to 6 years. Dev Psychol 1985; 21: 901–8CrossRef

79.

The Cochrane Library [database on disk and CD ROM]. Updated quarterly. The Cochrane Collaboration; Issue 1. Oxford: Oxford Update Software, 2000

80.

Quittner AL, Buu A. Effects of tobramycin solution for inhalation on global ratings of quality of life in patients with cystic fibrosis and Pseudomonas aeruginosa infection. Pediatr Pulmonol 2002; 33: 269–76PubMedCrossRef

81.

Ramström H, Erwander I, Mared L, et al. Pharmaceutical intervention in the care of cystic fibrosis patients. J Clin Pharm Ther 2000; 25(6): 427–34PubMedCrossRef

82.

Ramsey BW, Astley SJ, Aitken ML, et al. Efficacy and safety of short-term administration of aerosolised recombinant human deoxyribonuclease in patients with cystic fibrosis. Am Rev Respir Dis 1993; 148: 145–51PubMed

83.

Eisenberg JD, Aitken ML, Dorkin HL, et al. Safety of repeated intermittent courses of aerosolized recombinant human deoxyribonuclease in patients with cystic fibrosis. J Pediatr 1997; 131(1): 118–24PubMedCrossRef

84.

Fuchs H, Borowitz DS, Christiansen DH, et al. Effects of aerosolised recombinant human DNase on exacerbations of respiratory symptoms and on pulmonary function in patients with cystic fibrosis. N Engl J Med 1994; 331: 637–42PubMedCrossRef

85.

Munzenberger P, Van Wagnen CA, Abdulhamid I, et al. Impact of dornase alfa (Pulmozyme) on quality of life in patients with mild cystic fibrosis [abstract]. Pharmacotherapy 1996; 16: 515

86.

Mellis CJ. Effect of rhDNase on quality of life and spirometry in patients with cystic fibrosis [abstract]. Australian rhDNase Phase 111b Study Group. Aust NZ J Med 1997; 27: 257CrossRef

87.

Eng PA, Morton J, Douglass JA, et al. Short-term efficacy of ultrasonically nebulised hypertonic saline in cystic fibrosis. Pediatr Pulmonol 1996; 21: 77–83PubMedCrossRef

88.

Riedler J, Reade T, Button B, et al. Inhaled hypertonic saline increases sputum expectoration in cystic fibrosis. J Paediatr Child Health 1996; 32: 48–50PubMedCrossRef

89.

Balfour-Lynn I, Klein NJ, Dinwiddie R. Randomised controlled trial of inhaled corticosteroids (fluticasone propionate) in cystic fibrosis. Arch Dis Child 1997; 77(2): 124–30PubMedCrossRef

90.

Britto MT, Hornung RW, Kotagal UR, et al. Factors associated with changes in health related quality of life in patients with cystic fibrosis [abstract]. Pediatr Pulmonol 2001; Suppl. 22: 347–8

91.

Southern KW, Barker PM, Solis A. Macrolide antibiotics for cystic fibrosis (Cochrane Review). Available in The Cochrane Library [database on disk and CD ROM]. Updated quarterly. The Cochrane Collaboration; Issue 1. Oxford: Oxford Update Software, 2001

92.

Mukhopadhyay S, Singh M, Cater JI, et al. Nebulised antipseudomonal antibiotic therapy in cystic fibrosis: a meta-analysis of benefits and risks. Thorax 1996; 51: 364–8PubMedCrossRef

93.

Gilbert J, Robinson T, Littlewood JM. Home intravenous antibiotic treatment in cystic fibrosis. Arch Dis Child 1988; 63: 512–7PubMedCrossRef

94.

Winter RJD, George RID, Deacock SJ, et al. Self-administered home intravenous antibiotic therapy in bronchiectasis and cystic fibrosis. Lancet 1984; 23(3): 1338–9CrossRef

95.

Strandvik B, Hjelte L, Malmborg AS. Home intravenous treatment of patients with cystic fibrosis. Acta Paediatr Scand 1992; 81: 340–4CrossRef

96.

van Aalderen WM, Mannes GP, Bosma ES, et al. Home care in cystic fibrosis patients. Eur Respir J 1995; 8(1): 172–5PubMedCrossRef

97.

Bakker W, Vinks AA, Mouton JW, et al. Continuous intravenous home treatment of airway infections using ceftazidime administration via portable pump in patients with cystic fibrosis: a multicenter study. Ned Tijdschr Geneeskd 1993; 137(48): 2486–9PubMed

98.

Pond MN, Newport M, Jones D, et al. Home versus hospital intravenous antibiotic therapy in treatment of young adults with cystic fibrosis. Eur Respir J 1994; 7: 1640–4PubMedCrossRef

99.

Donati MA, Guenette G, Auerbaech H. Prospective controlled study of home and hospital therapy of cystic fibrosis pulmonary disease. J Pediatr 1987; 111: 28–33PubMedCrossRef

100.

Marco T, Asensio O, Bosque M, et al. Home intravenous antibiotics for cystic fibrosis (Cochrane Review). Available in The Cochrane Library [database on disk and CD ROM]. Updated quarterly. The Cochrane Collaboration; Issue 1. Oxford: Oxford Update Software, 2001

101.

Lieberman J. Dornase aerosol effect on sputum viscosity in cases of cystic fibrosis. JAMA 1968; 205: 312–3PubMedCrossRef

102.

Shak S, Capon DJ, Hellmiss R, et al. Recombinant human DNase 1 reduces the viscosity of cystic fibrosis sputum. Proc Natl Acad Sci USA 1990; 87: 9188–92PubMedCrossRef

103.

Hubbard RC, McElvaney NG, Birrer P, et al. A preliminary study of aerosolized recombinant human deoxyribonuclease I in the treatment of cystic fibrosis. N Engl J Med 1992; 326: 812–5PubMedCrossRef

104.

Abbott J, Baumann U, Conway S, et al. Cross-cultural differences in health related quality of life in adolescents with cystic fibrosis. Disabil Rehabil 2001; 22(18): 837–44

105.

Goa KL, Lamb H. Dornase alfa: a review of pharmacoeconomic and quality-of-life aspects of its use in cystic fibrosis. Pharmacoeconomics 1997; 12(3): 409–22PubMedCrossRef

106.

Dasgupta B, Tomkiewicz RP, Brown NE, et al. Combined effects of hypertonic saline and rhDNase on cystic fibrosis sputum in-vitro. Pediatr Pulmonol 1995; 20 Suppl. 12: A201–36

107.

Kahn TZ, Wagner JS, Bost T, et al. Early pulmonary inflammation in infants with cystic fibrosis. Am J Respir Crit Care Med 1995; 151: 1975–82

108.

Eigen H, Rosenstein BJ, Fitzsimmons S, et al. A multicentre study of alternate day prednisone therapy in patients with cystic fibrosis. Cystic Fibrosis Foundation Prednisone Trial Group. J Pediatr 1995; 126: 515–23PubMedCrossRef

109.

Schmidt J, Davidson AGF, Seear M, et al. Is the acquisition of pseudomonads in cystic fibrosis patients increased by use of inhaled corticosteroids: unexpected results from a double blind placebo controlled study. Pediatr Pulmonol 1997; Suppl. 14: 293–4

110.

Dezateux C, Walters S, Balfour-Lynn I. Inhaled corticosteroids for cystic fibrosis (Cochrane Review). Available in The Cochrane Library [database on disk and CD ROM]. Updated quarterly. The Cochrane Collaboration; Issue 3. Oxford: Oxford Update Software, 2001

111.

Cystic fibrosis patients registry: 1997 annual data report. Bethesda (MD): Cystic Fibrosis Foundation, 1998

112.

Macdonald A. Nutritional management of cystic fibrosis. Arch Dis Child 1996; 74: 81–7PubMedCrossRef

113.

Smyth R, Walters S. Oral calorie supplements for cystic fibrosis (Cochrane Review). Available in The Cochrane Library [database on disk and CD ROM]. Updated quarterly. The Cochrane Collaboration; Issue 3. Oxford: Oxford Update Software, 2001

114.

Hanning RM, Blimkie CJR, Bar-Or O, et al. Relationships among nutritional status and skeletal and respiratory muscle function in cystic fibrosis: does early dietary supplementation make a difference. Am J Clin Nutr 1993; 57: 580–7PubMed

115.

Anderson RT, Aaronson NK, Wilkin D. Critical review of the international assessments of health-related quality of life. Qual Life Res 1993; 2: 369–95PubMedCrossRef

116.

Collings JA. International differences in psychosocial well-being: a comparative study of adults with epilepsy in three countries. Seizure 1994; 3: 183–90PubMedCrossRef

117.

Baker GA, Gagnon D, McNulty P. The relationship between seizure frequency, seizure type and quality of life: findings from three European countries. Epilepsy Res 1998; 30: 231–40PubMedCrossRef

118.

Erbil P, Razavi D, Farvacques C, et al. Cancer patients’ psychological adjustment and perception of illness: cultural differences between Belgium and Turkey. Support Care Cancer 1996; 4: 455–61PubMedCrossRef

119.

de Haes JCJM, Olschewski M. Quality of life assessment in a cross-cultural context: use of the Rotterdam symptom checklist in a multinational randomised trial comparing CMF and Zoladex (Goserlin) treatment in early breast cancer. Ann Oncol 1998; 9: 745–50PubMedCrossRef

120.

Lydick E, Epstein RS. Interpretation of quality of life changes. Qual Life Res 1993; 2: 221–6PubMedCrossRef

121.

Anastasi A, Urbina S. Psychological testing. 7th ed. Upper Saddle River (NJ): Prentice-Hall, 1997

122.

Kazis LE, Anderson JJ, Meenan RF. Effect sizes for interpreting changes in health status. Med Care 1989; 27 Suppl. 3: S178–89PubMedCrossRef

123.

McHorney CA, Tarlov AR. Individual patient monitoring in clinical practice: are available health status surveys adequate. Qual Life Res 1995; 4: 293–307PubMedCrossRef

124.

Nunnally JC, Bernstein IH. Psychometric theory. New York (NY): McGraw Hill, 1994

125.

Juniper EF, Guyatt GH, Willan A, et al. Determining the minimal important change in a disease-specific quality of life instrument. J Clin Epidemiol 1994; 47: 81–7PubMedCrossRef

126.

Jaeschke R, Singer J, Guyatt GH. Measurement in health status: ascertaining the minimal clinically important difference. Control Clin Trials 1989; 10: 407–15PubMedCrossRef

127.

Redelmeier DA, Guyatt GH, Goldstein RS. Assessing the minimal important difference in symptoms: a comparison of two techniques. J Clin Epidemiol 1996; 49: 1215–9PubMedCrossRef

128.

Wyrwich KW, Nienaber NA, Tierney WM, et al. Linking clinical relevance and statistical significance in evaluating intra-individual changes in health related quality of life. Med Care 1999; 37: 469–78PubMedCrossRef

129.

Wyrwich KW, Tierney WM, Wolinsky FD. Further evidence supporting an SEM-based criterion for identifying meaningful intra-individual changes in health related quality of life. J Clin Epidemiol 1999; 52: 861–73PubMedCrossRef

130.

Weinstein MC, Stasson WB. Foundations of cost-effectiveness analysis for health and medical practices. N Engl J Med 1977; 296: 716–21PubMedCrossRef

131.

Torrance GW. Measurement of health state utilities for economic appraisal: a review. J Health Econ 1986; 5: 1–30PubMedCrossRef

132.

Deverill M, Brazier J, Green C, et al. The use of QALY and non-QALY measures of health related quality of life. Pharmacoeconomics 1998; 13: 411–20PubMedCrossRef

133.

Epstein AM, Hall JA, Tognetti J, et al. Using proxies to evaluate quality of life: can they provide valid information about patients’ health status and satisfaction with medical care. Med Care 1989; 27 Suppl. 3: S91–8PubMedCrossRef

134.

Torrance GW. Social preference for health states: an empirical evaluation of three measurement techniques. Socioecon Plann Sci 1976; 10: 129–36CrossRef

135.

Thompson MS. Willingness to pay and accept risks to cure chronic disease. Am J Public Health 1986; 76: 392–6PubMedCrossRef

136.

Read JL, Quinn RJ, Berwick DM, et al. Preferences for health outcomes: comparisons of assessment methods. Med Decis Making 1984; 4: 315–29PubMedCrossRef

137.

Bakker C, van der Linden S. Health related utility measurement: an introduction. J Rheumatol 1995; 22: 1197–9PubMed

138.

Sockrider MM, Swank PR, Seilheimer DK, et al. Measuring clinical status in cystic fibrosis: internal validity and reliability of a modified NIH score. Pediatr Pulmonol 1994; 17: 86–96PubMedCrossRef

139.

Kanga J, Kuhn R, Craigmyle L, et al. Cystic fibrosis clinical score: a new scoring system to evaluate acute pulmonary exacerbation. Clin Ther 1999; 21: 1343–56PubMedCrossRef

Title: Quality of Life in Children and Adolescents with Cystic Fibrosis
Implications for Optimizing Treatments and Clinical Trial Design
Authors: Professor Janice Abbott
Louise Gee
Publication date: 01-01-2003
Publisher: Springer International Publishing
Published in: Pediatric Drugs / Issue 1/2003
Print ISSN: 1174-5878
Electronic ISSN: 1179-2019
DOI: https://doi.org/10.2165/00128072-200305010-00004

At a glance: The STEP trials

Springer Medicine

Quality of Life in Children and Adolescents with Cystic Fibrosis

Implications for Optimizing Treatments and Clinical Trial Design

Abstract

At a glance: The STEP trials

Springer Medicine

Abstract

Please log in to get access to this content

Other articles of this Issue 1/2003

Lansoprazole

Spasticity Associated with Cerebral Palsy in Children

The Use of Proton Pump Inhibitors in Children

Adolescent Smoking

Formoterol Delivered by Turbuhaler®

Lansoprazole in Children