title: Longitudinal assessment of health-related quality of life in an observational cohort of patients with cystic fibrosis.
abstract: BACKGROUND
  Patient-reported outcomes (PROs) are increasingly used to evaluate the efficacy of new treatments and the progression of chronic diseases. The Cystic Fibrosis Questionnaire-Revised (CFQ-R) is a disease-specific, PRO measure of health-related quality of life (HRQOL). We evaluated associations between changes in health status over time and HRQOL in a national CF database.
  
  
  METHODS
  Using the Epidemiologic Study of Cystic Fibrosis (ESCF) data, we identified participants who had completed age-appropriate CFQ-R assessments on two occasions separated by 9-15 months. Next, we developed multivariate regression models to test whether associations existed between (1) changes in respiratory signs/symptoms and changes in the respiratory health domains of the CFQ-R, (2) changes in nutritional health status and changes in the nutritional health domains of the CFQ-R, and (3) changes in treatment complexity and changes in the Treatment Burden scale of the CFQ-R.
  
  
  RESULTS
  We analyzed 1,947 pairs of assessments: 337 child (mean age 8.9, range 6-13 years), 581 parent (mean age of child 8.8, range 6-13 years), 398 adolescent (mean age 15.3, range 14-17 years), and 631 adult (mean age 26.9, range 18-73 years). On average, we found little change in both health status indicators and CFQ-R domain scores over 1 year. Significant associations over time, however, were found between increases in respiratory symptoms and worse CFQ-R Respiratory Symptom scores, declining weight and worsening scores on CFQ-R nutritional health domains, and increases in treatment complexity and worsening CFQ-R Treatment Burden scores for parent respondents.
  
  
  CONCLUSIONS
  Health status and HRQOL changes were small over a 1-year period in this CF population. However, changes in respiratory symptoms and weight were associated with significant changes on relevant CFQ-R scores, indicating that this PRO is sensitive to changes in health status over time.
authors.affiliation: Division of Respiratory Diseases, Children's Hospital Boston, Boston, Massachusetts 02115, USA. gregory.sawicki@childrens.harvard.edu
authors.forename: Gregory S
authors.initials: GS
authors.lastname: Sawicki
authors.forename: Lawrence
authors.initials: L
authors.lastname: Rasouliyan
authors.forename: Ann H
authors.initials: AH
authors.lastname: McMullen
authors.forename: Jeffrey S
authors.initials: JS
authors.lastname: Wagener
authors.forename: Susanna A
authors.initials: SA
authors.lastname: McColley
authors.forename: David J
authors.initials: DJ
authors.lastname: Pasta
authors.forename: Alexandra L
authors.initials: AL
authors.lastname: Quittner
mesh_terms: D000293:Adolescent; D000328:Adult; D000368:Aged; D002648:Child; D015331:Cohort Studies; D003550:Cystic Fibrosis / Q000150:complications / Q000453:epidemiology* / Q000523:psychology; D005260:Female; D006304:Health Status; D006306:Health Surveys / Q000706:statistics & numerical data; D006801:Humans; D008137:Longitudinal Studies; D008297:Male; D008875:Middle Aged; D011552:Pseudomonas Infections / Q000453:epidemiology / Q000209:etiology / Q000523:psychology; D011788:Quality of Life; D012135:Respiratory Sounds / Q000209:etiology; D011795:Surveys and Questionnaires; D016896:Treatment Outcome; D055815:Young Adult