Initial evaluation of the Parent Cystic Fibrosis Questionnaire--Revised (CFQ-R) in infants and young children

Adrianne N Alpern; Lyndia C Brumback; Felix Ratjen; Margaret Rosenfeld; Stephanie D Davis; Alexandra L Quittner

doi:10.1016/j.jcf.2014.11.002

Initial evaluation of the Parent Cystic Fibrosis Questionnaire--Revised (CFQ-R) in infants and young children

J Cyst Fibros. 2015 May;14(3):403-11. doi: 10.1016/j.jcf.2014.11.002. Epub 2014 Nov 28.

Authors

Adrianne N Alpern¹, Lyndia C Brumback², Felix Ratjen³, Margaret Rosenfeld⁴, Stephanie D Davis⁵, Alexandra L Quittner⁶

Affiliations

¹ University of Miami, Department of Psychology, 5665 Ponce De Leon Blvd., Coral Gables, FL 33146, USA.
² University of Washington, Campus Box 354922, Seattle, WA, USA.
³ Division of Respiratory Medicine, Department of Pediatrics, Hospital for Sick Children, University of Toronto, Canada.
⁴ Seattle Children's Hospital and Department of Pediatrics, University of Washington, School of Medicine, Seattle, WA, USA.
⁵ Department of Pediatrics, Riley Hospital for Children, Indiana University, Indianapolis, IN 46202, USA.
⁶ University of Miami, Department of Psychology, 5665 Ponce De Leon Blvd., Coral Gables, FL 33146, USA. Electronic address: AQuittner@miami.edu.

PMID: 25443473
DOI: 10.1016/j.jcf.2014.11.002

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.

Keywords: Health-related quality of life; Infants; Patient reported outome; Psychometric evaluation; Treatment burden.

Publication types

Randomized Controlled Trial
Research Support, N.I.H., Extramural
Research Support, Non-U.S. Gov't

MeSH terms

Administration, Inhalation
Adolescent
Adult
Child
Child, Preschool
Cystic Fibrosis / diagnosis*
Cystic Fibrosis / therapy
Female
Health Status*
Humans
Infant
Male
Parents / psychology*
Psychometrics / methods
Quality of Life*
Saline Solution, Hypertonic / administration & dosage
Severity of Illness Index
Surveys and Questionnaires*
Young Adult

Substances

Saline Solution, Hypertonic

Abstract

Publication types

MeSH terms

Substances

Grants and funding