Effect of rhDNase on lung function and quality of life in adult cystic fibrosis patients

Heijerman, H.G.M.

doi:10.1016/0300-2977(95)00022-f

Cited by 26 publications

(9 citation statements)

References 10 publications

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“…The most appropriate measure to employ will depend upon the purpose of the study. [39,40] Problems then arise with scoring systems, the interpretation of the data with regard to comparability across studies, and the scales' validity, reliability and sensitivity. Utility measures may also be described as generic.…”

Section: The Measurement Of Qol In Cfmentioning

confidence: 99%

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Quality of Life in Children and Adolescents with Cystic Fibrosis

Abbott

Gee

2003

Pediatric Drugs

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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.

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Section: The Measurement Of Qol In Cfmentioning

confidence: 99%

“…The magnitude of dyspnea was also rated on a visual analog scale. [84] Heijerman et al [40] examined the effect of DNase on lung function and QOL. Ranasinha et al [39] reported an improvement in lung function in both adolescents and adults following 10 days of DNase therapy (2.5mg twice daily).…”

Section: Dornase Alfa (Dnase)mentioning

confidence: 99%

Quality of Life in Children and Adolescents with Cystic Fibrosis

Abbott

Gee

2003

Pediatric Drugs

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“…To date, no disease-specific quality of life instrument exists for cystic fibrosis; however, using general instruments or limited scales representing specific domains, several studies incorporated quality of life as an outcome in these patients. [4][5][6][7][8][9][10][11] The Quality of Well-Being 12 (QWB) scale, an interview-administered, general quality of life instrument, was investigated in several studies. A significant relationship was seen between the QWB scale and pulmonary function tests and exercise performance in 44 patients with cystic fibrosis ranging in age from 7-36 years.…”

Section: (Pharmacotherapy 1999;19(4):393-398)mentioning

confidence: 99%

Quality of Life as a Treatment Outcome in Patients with Cystic Fibrosis

et al. 1999

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We attempted to determine the responsiveness and validity of the Quality of Well-Being (QWB) scale in 20 consecutive children and adolescents with cystic fibrosis. The QWB score was determined for 6-day periods immediately before and after hospital admission, and at 6- and 12-month follow-up. With the instrument's scale of zero-1, responsiveness was indicated by significant changes in QWB score (0.09), physical (0.019), social (0.021), and symptom-problem complexes (0.04) domains, and all pulmonary function tests from before to after treatment of an acute exacerbation. Only the symptom-problem complex domain significantly changed from after treatment to 6- and 12-month follow-up. Validity was shown by significant correlations between before and after QWB scores and forced vital capacity (r=0.476), residual volume total lung capacity ratio (r=0.452), forced expiratory volume in 1 second (r=0.358), and forced expiratory flow between 25% and 75% of vital capacity (r=0.35).

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“…5 The value of these outcomes to patients and families is not quantifiable, but is likely to be significant. But what financier ever gambled on subjective evidence?…”

Section: What Is Cost Effectiveness In Cystic Fibrosis?mentioning

confidence: 99%

Recombinant human DNase (rhDNase) in cystic fibrosis: is it cost effective?

Conway

1997

Archives of Disease in Childhood

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Effect of rhDNase on lung function and quality of life in adult cystic fibrosis patients

Cited by 26 publications

References 10 publications

Quality of Life in Children and Adolescents with Cystic Fibrosis

Quality of Life in Children and Adolescents with Cystic Fibrosis

Quality of Life as a Treatment Outcome in Patients with Cystic Fibrosis

Recombinant human DNase (rhDNase) in cystic fibrosis: is it cost effective?

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