Positive parental attitudes to participating in research involving newborn screened infants with CF

Abstract

Background: Information regarding recruitment of infants to research studies following the diagnosis of cystic fibrosis (CF) via newborn screening (NBS) is not currently available. This study aimed to assess parental attitudes and the feasibility of recruiting and retaining both NBS infants with CF and healthy control infants to a longitudinal, observational study. Methods: All infants underwent pulmonary function tests (PFTs) at~3 and~12 months of age. Infants with CF had additional combined chest high resolution computed tomography (HRCT), bronchoscopy and broncho-alveolar lavage (BAL) at~12 months of age. Parental attitude questionnaires (PAQs) were administered to all parents following the~3 month PFTs and to parents of infants with CF after completion of all tests at~12 months. Results: 86% (92/107) of families whose infant had CF consented to participate, of whom 92% had PFTs at~3 months of age with 99% of these having PFTs at~12 months of age. Recruitment of healthy controls was feasible but more challenging; 29% of those contacted agreed to participate; 73% of these had PFTs at~3 months of age; of whom 83% had repeated PFTs at~12 months of age. Completed PAQs were received from 71% of parents, (both of CF and healthy infants) at~3 months and from 58% parents of infants with CF at~12 months. Responses from the PAQs were generally positive, 95% of parents indicated they would recommend participation in such studies to other families. Discrepancies between responses at 3 and 12 months suggested that parental understanding of what the research entailed developed during the course of the study. Conclusions: The high recruitment and retention rates for newly diagnosed CF NBS infants to this observational study are encouraging. These findings will help inform future study design both in the field of CF and other conditions diagnosed by NBS.

Is chest CT useful in newborn screened infants with cystic fibrosis at 1 year of age?

“…The study was conducted between January 2009 and May 201217 18 25; chest CT scans at 1 year were performed in 65 NBS LCFC infants. Table 1 summarises clinical details of the infants.…”

“…Based on the incidence of bronchial dilatation detected by both scorers in this study, between 190 and 850 infants per group would be required if a randomised trial such as the recent Ivacaftor trial34 were to be extended to infants, in order to detect a reduction in bronchial dilatation of 50% with 90% power at a 5% significance level at 1 year of age; this number would rise further after accounting for those ineligible for such a trial or whose parents decline 35. Suggestions that such a study would be feasible with only 100/group were based on the incidence of bronchiectasis at 4, not 1 year of age 7.…”

Evolution of lung function during the first year of life in newborn screened cystic fibrosis infants

“…Attrition was minimal,12 with no bias between those who did and did not complete the study. Selection of various PFTs enabled different aspects of pathophysiology to be assessed 11.…”

“…Our primary hypothesis was that lung function would deteriorate further between 3 months and 1 year of age. We also aimed to investigate the determinants of lung function at 1 year, and to collect data to assess feasibility of recruiting NBS CF infants to invasive studies12 and inform future power calculations.…”

A novel method for infant multiple breath washout: First report in clinical practice

“…Following the MBW test, parents were asked to complete a questionnaire to determine the acceptability of the procedure. Although previous authors have described parental experiences of participating in similar research, no previously‐validated questionnaire directly applicable to the requirements of this study was identified. A questionnaire was therefore devised by the research team to determine the acceptability of the procedure.…”