This is a national research programme which addresses key outcomes in the recently
established Cystic Fibrosis (CF) Newborn Screening programme (NBS), introduced in
2011. The Irish Comparative Outcome Study (ICOS) of CF was established and in
Part I studied children with CF (CwCF) to age 3-5 years. This study aims to continue
this research programme in ICOS Part 2, which will follow CwCF to age 8-11. The
hypothesis is that screen-detected CwCF will continue to show the benefits of
screening. The study aim is to evaluate important clinical and economic early
parameters of the screening programme by comparing screen-detected CF with
symptomatic presentation in terms of the following: (i) clinical outcomes, including
growth, lung function, infection rates, pancreatic function, number/duration of hospital
admissions; (ii) comparative timing of signs of early respiratory complications in
CwCF through chest x-ray grading and thoracic CT; (iii) the effect of sex and
presence of DF508 gene mutation on early disease development; (iv) comparison of
early clinical outcomes in screen-detected CF patients diagnosed and treated in
Ireland with comparable data from the European Cystic Fibrosis Registry; (v) a costcomparison
study addressing health costs incurred by parents and by the health
services following diagnosis, including travel, time off work, hospital visits, tests,
hospital admissions and (vi) comparative carer burden and parental quality of life.
The timing of this research allows for assessment of the effect of new CFTR
modulators Orkambi and Kalydeco.
The proposed timely research on the CF NBS will utilise both data collected by the
National Cystic Fibrosis Registry and complementary additional clinical data required.
Costs will be determined by parental questionnaires and chart review. A new
Challenge of Living with CF-Short Form will be used to assess parental carer burden.
The results will provide further timely feedback on policy and management for all
stakeholders.