TY - JOUR
T1 - Pulmonary function deficits in newborn screened infants with cystic fibrosis managed with standard UK care are mild and transient
AU - Davies, Gwyneth
AU - Stocks, Janet
AU - Thia, Lena P.
AU - Hoo, Ah Fong
AU - Bush, Andrew
AU - Aurora, Paul
AU - Brennan, Lucy
AU - Lee, Simon
AU - Lum, Sooky
AU - Cottam, Philippa
AU - Miles, Joanne
AU - Chudleigh, Jane
AU - Kirkby, Jane
AU - Balfour-Lynn, Ian M.
AU - Carr, Siobhán B.
AU - Wallis, Colin
AU - Wyatt, Hilary
AU - Wade, Angie
AU - Prasad, Ammani
AU - Shankar, Anu
AU - Owens, Catherine
AU - Pao, Caroline
AU - Ahmed, Deeba
AU - Ruiz, Gary
AU - Davies, Jane
AU - Price, John
AU - Rosenthal, Mark
AU - Suri, Ranjan
AU - Chavasse, Richard
AU - Nguyen, The Thanh Diem
AU - Gustafsson, Per
PY - 2017/11/1
Y1 - 2017/11/1
N2 - 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.
AB - 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.
UR - http://www.scopus.com/inward/record.url?scp=85034961008&partnerID=8YFLogxK
U2 - 10.1183/13993003.00326-2017
DO - 10.1183/13993003.00326-2017
M3 - Article
AN - SCOPUS:85034961008
SN - 0903-1936
VL - 50
JO - European Respiratory Journal
JF - European Respiratory Journal
IS - 5
M1 - 1700326
ER -