Quality of Life in Children and Adolescents with Cystic Fibrosis

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.

Factors influencing quality of life and disease severity in Hungarian children and young adults with cystic fibrosis

“…Quittner et al reported similar findings in observable dimensions in a large, multicentre, cohort study in the United States [25]. Previous publications identified the opposite trend between patient- and parent-proxy report [6,29]. In the present study, Respiratory symptoms were associated with lower HRQoL scores in CF patients; this is consistent with the findings of Szyndler et al [30].…”

“…Although parents know their children, they may not have insight into the child’s feelings and thoughts, which could explain their differing perspectives on the factors that influence HRQoL most. Additionally, children may not wish to discuss the true impact of CF on their lives, to avoid distressing their parents [6]. Consequently it is necessary to obtain information about HRQoL from both the child and his or her caregivers.…”

“…Health-related quality of life (HRQoL): The Cystic Fibrosis Questionnaire-Revised (CFQ-R) is a widely used, validated, disease-specific instrument for the measurement of HRQoL in patients with CF [5,6,9,11]. The Hungarian version of CFQ-R was used to evaluate HRQoL from both the patient’s perspective (patient-report) and their parent’s perspective (parent-proxy report).…”

Assessment of agreement between parents and children on health‐related quality of life in children with cystic fibrosis

“…1998; Levi & Drotar 1999; Britto et al . 2000; Eiser & Morse 2001; Abbott & Gee 2003; Sturms et al . 2003).…”

“…2003). However, there are strong arguments for obtaining information from both parents and children whenever possible (Eiser & Morse 2001; Abbott & Gee 2003), for example, to determine similarities and mismatches in the perception of the child and parent or to appreciate some of the complex family dynamics (Abbot & Gee 2003). In addition, it is important to look at the differences in HRQOL reports between parents and children in order to direct appropriate interventions if necessary (Henry et al .…”