Development of the preschool cystic fibrosis questionnaire-revised (CFQ-R)

Abstract

AIMS: Patient-reported outcomes (PROs) are critical to evaluating the effects of chronic disease. The Cystic Fibrosis Questionnaire- Revised (CFQ-R; Quittner et al., 2005) is the best validated PRO for cystic fibrosis (CF), with strong reliability and validity in patients with CF ages 6 through 70 (Quittner et al., 2014). However, currently there is no validated PRO to measure HRQoL in patients under age 6. As new treatment guidelines for young children with CF are implemented, it will be important to assess their effects. Thus, this study aimed to develop a pictorial adaptation of the CFQ-R for children ages 3-6 as an outcome measure for routine care, disease progression and efficacy of newly introduced treatments. METHODS: Children (n = 12, mean age = 4.51 years, SD = .84) completed open-ended, qualitative interviews to assess how CF affects their daily functioning. Recorded interviews were transcribed and coded in Atlas.ti to verify content saturation and generate preliminary items. Cognitive testing with forced-choice responses measured the clarity and comprehensiveness of the measure and rating scales. Psychometric validation was then conducted with preschool-age children in the United States and Australia (n = 248, mean age = 4.21 years, SD = .93) to evaluate item and scale level reliability and convergent validity. RESULTS: Saturation of content was achieved. Most children could answer questions about their physical and emotional functioning, respiratory and digestive symptoms, energy level, body image and treatments. The preliminary child instrument consisted of 35 items; however we deleted items assessing social functioning (α = .53), which were not well understood, and revised pictures for the Treatment Burden Scale (α = .21; see Fig. 1). Other items were modified or deleted based on internal consistencies and item-to-total correlations for each scale. The revised instrument consists of 28 items. Adequate to strong reliabilities were obtained for all scales in this revised version (α = .68-.80). CONCLUSIONS: Young children were able to reliably report on their daily symptoms and functioning. With further validation, this PRO may be used in clinical trials of medications for young children with CF ages 3-6. Future research will identify other psychometric properties of the Preschool CFQ-R and determine the minimal clinically important difference score (MCID).L6235262952018-08-22 <br />