title: Initial evaluation of the Parent Cystic Fibrosis Questionnaire--Revised (CFQ-R) in infants and young children.
abstract: BACKGROUND
  There is an urgent need to evaluate treatments for young children with cystic fibrosis (CF); however, efforts have been hampered by a lack of reliable, practical endpoints. To examine whether a patient-reported outcome could be reliable in children 4 to 60 months of age, we assessed the psychometric properties of the modified Parent Cystic Fibrosis Questionnaire--Revised (CFQ-R) using data from the Infant Study of Inhaled Saline (ISIS). We also characterized patterns of symptom presentation and daily functioning in children in this age range to inform future measure development.
  
  
  METHODS
  Parents (N=314) completed the CFQ-R and Treatment Adherence Questionnaire (TAQ) at five quarterly visits, as well as a weekly Parent Symptom Diary.
  
  
  RESULTS
  The Parent CFQ-R demonstrated good construct validity and adequate internal consistency (α's .58-.75). Associations with age, TAQ, and Parent Symptom Diary were observed. The Treatment Burden scale demonstrated responsiveness to change.
  
  
  CONCLUSIONS
  Parents were reliable observers of young children's symptoms and daily functioning, and PROs show promise for this age group. Research is needed to identify key symptoms in infants and young children with CF, and to develop a parent proxy PRO according to FDA/EMA guidelines.
authors.affiliation: University of Miami, Department of Psychology, 5665 Ponce De Leon Blvd., Coral Gables, FL 33146, USA.
authors.forename: Adrianne N
authors.initials: AN
authors.lastname: Alpern
authors.affiliation: University of Washington, Campus Box 354922, Seattle, WA, USA.
authors.forename: Lyndia C
authors.initials: LC
authors.lastname: Brumback
authors.affiliation: Division of Respiratory Medicine, Department of Pediatrics, Hospital for Sick Children, University of Toronto, Canada.
authors.forename: Felix
authors.initials: F
authors.lastname: Ratjen
authors.affiliation: Seattle Children's Hospital and Department of Pediatrics, University of Washington, School of Medicine, Seattle, WA, USA.
authors.forename: Margaret
authors.initials: M
authors.lastname: Rosenfeld
authors.affiliation: Department of Pediatrics, Riley Hospital for Children, Indiana University, Indianapolis, IN 46202, USA.
authors.forename: Stephanie D
authors.initials: SD
authors.lastname: Davis
authors.affiliation: University of Miami, Department of Psychology, 5665 Ponce De Leon Blvd., Coral Gables, FL 33146, USA. Electronic address: AQuittner@miami.edu.
authors.forename: Alexandra L
authors.initials: AL
authors.lastname: Quittner
mesh_terms: D000280:Administration, Inhalation; D000293:Adolescent; D000328:Adult; D002648:Child; D002675:Child, Preschool; D003550:Cystic Fibrosis / Q000175:diagnosis* / Q000628:therapy; D005260:Female; D006304:Health Status; D006801:Humans; D007223:Infant; D008297:Male; D010290:Parents / Q000523:psychology*; D011594:Psychometrics / Q000379:methods; D011788:Quality of Life; D012462:Saline Solution, Hypertonic / Q000008:administration & dosage; D012720:Severity of Illness Index; D011795:Surveys and Questionnaires; D055815:Young Adult
chemical_list: D012462:Saline Solution, Hypertonic
keywords: Health-related quality of life; Infants; Patient reported outome; Psychometric evaluation; Treatment burden