Pulmonary function deficits in newborn screened infants with cystic fibrosis managed with standard UK care are mild and transient

Abstract

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.

Elucidating progression of early cystic fibrosis lung disease

“…The results of the study by DAVIES et al [4] are encouraging for families and clinicians for children with CF. Overall impairments in lung function during infancy appear to be relatively mild and transient.…”

“…Overall, the data presented by DAVIES et al [4] provide important insights into the respiratory health of infants with CF in the era of newborn screening. The findings from the LCFC that newborn-screened infants being followed up regularly have relatively mild and transient lung function deficits in the first 2 years of life are encouraging for families and treating physicians.…”

“…There are some limitations to the study by DAVIES et al [4]. As with many other observational studies involving sedation and complex methodologies, data were only collected annually and, therefore, we do not have information regarding the natural variability or responses to treatment of these lung function indices in infants with CF.…”

“…In this issue of the European Respiratory Journal, DAVIES et al [4] from the London Cystic Fibrosis Collaboration (LCFC) extend their follow-up of a newborn-screened CF cohort and contemporaneous controls to 2 years of age. This prospective, longitudinal, observational study presents infant lung function outcomes from 62 infants with CF diagnosed following newborn screening and 34 healthy controls at the 2 year follow up visit.…”

Evolución de la función pulmonar en niños con fibrosis quística durante los primeros 3 años de vida

“…Las infecciones respiratorias bacterianas no se asociaron a la evolución de la función pulmonar. 20 Estas discrepancias entre las cohortes de pacientes con FQ australianos y londinenses seguidos con pruebas funcionales desde los primeros meses de la vida se podrían deber a diferencias técnicas y metodológicas con respecto a los estudios funcionales y controles utilizados. 5 Las publicaciones sobre el impacto de infecciones respiratorias bacterianas sobre la función pulmonar en los niños preescolares y escolares con FQ son más numerosas, aunque con resultados contradictorios.…”