Initial evaluation of the Parent Cystic Fibrosis Questionnaire--Revised (CFQ-R) in infants and young children
Adrianne N Alpern1, Lyndia C Brumback2, Felix Ratjen3
1University of Miami, Department of Psychology, 5665 Ponce De Leon Blvd., Coral Gables, FL 33146, USA.
Insights
Parents can reliably report on symptoms and daily functioning in young children with cystic fibrosis (CF). Patient-reported outcomes (PROs) show promise for evaluating CF treatments in this age group.
Area of Science:
- Pediatric Pulmonology
- Clinical Trial Endpoints
- Patient-Reported Outcomes
Background:
- Evaluating treatments for young children with cystic fibrosis (CF) is challenging due to a lack of practical endpoints.
- Patient-reported outcomes (PROs) may offer a reliable solution for this age group.
- The modified Parent Cystic Fibrosis Questionnaire--Revised (CFQ-R) was assessed in children aged 4-60 months.
Purpose of the Study:
- To assess the psychometric properties of the modified Parent CFQ-R in young children with CF.
- To characterize symptom presentation and daily functioning patterns in children aged 4-60 months.
- To inform the development of future outcome measures for pediatric CF research.
Main Methods:
- Parents (N=314) completed the CFQ-R and Treatment Adherence Questionnaire (TAQ) quarterly.
- A weekly Parent Symptom Diary was also utilized.
- Psychometric properties, including construct validity and internal consistency, were evaluated.
Main Results:
- The Parent CFQ-R demonstrated good construct validity and adequate internal consistency (α = .58–.75).
- Associations were found between the CFQ-R, age, TAQ, and the Parent Symptom Diary.
- The Treatment Burden scale showed responsiveness to change.
Conclusions:
- Parents serve as reliable observers of symptoms and daily functioning in young children with CF.
- PROs, like the Parent CFQ-R, show potential as reliable endpoints for this pediatric population.
- Further research is needed to identify key CF symptoms in infants and develop parent proxy PROs aligned with regulatory guidelines.
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.
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