Pulmonary function deficits in newborn screened infants with cystic fibrosis managed with standard UK care are mild and transient.
Gwyneth DaviesJanet StocksLena P ThiaAh-Fong HooAndrew BushPaul AuroraLucy BrennanSimon LeeSooky LumPhilippa CottamJoanne MilesJane ChudleighJane KirkbyIan M Balfour-LynnSiobhán B CarrColin WallisHilary WyattAngie Wadenull nullPublished in: The European respiratory journal (2017)
With the advent of novel designer molecules for cystic fibrosis (CF) treatment, there is huge need for early-life clinical trial outcomes, such as infant lung function (ILF). We investigated the degree and tracking of ILF abnormality during the first 2 years of life in CF newborn screened infants.Forced expiratory volume in 0.5 s (FEV0.5), lung clearance index (LCI) and plethysmographic functional residual capacity were measured at ∼3 months, 1 year and 2 years in 62 infants with CF and 34 controls.By 2 years there was no significant difference in FEV0.5 z-score between CF and controls, whereas mean LCI z-score was 0.81 (95% CI 0.45-1.17) higher in CF. However, there was no significant association between LCI z-score at 2 years with either 3-month or 1-year results. Despite minimal average group changes in any ILF outcome during the second year of life, marked within-subject changes occurred. No child had abnormal LCI or FEV0.5 on all test occasions, precluding the ability to identify "high-risk" infants in early life.In conclusion, changes in lung function are mild and transient during the first 2 years of life in newborn screened infants with CF when managed according to a standardised UK treatment protocol. Their potential role in tracking disease to later childhood will be ascertained by ongoing follow-up.
Keyphrases
- cystic fibrosis
- lung function
- early life
- pseudomonas aeruginosa
- clinical trial
- chronic obstructive pulmonary disease
- healthcare
- palliative care
- air pollution
- randomized controlled trial
- type diabetes
- mass spectrometry
- young adults
- mental health
- cerebral ischemia
- intensive care unit
- chronic pain
- study protocol
- blood brain barrier
- pain management
- insulin resistance
- phase iii
- finite element