Duncan, Julie;
(2019)
The evolution of lung function in newborn screened preschool children with cystic fibrosis.
Doctoral thesis (M.D(Res)), UCL (University College London).
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Abstract
Most morbidity in cystic fibrosis (CF) is due to progressive pulmonary disease. Recently, small molecule therapies targeting the basic defect of the cystic fibrosis transmembrane conductance regulator protein have been developed, and newborn screening (NBS) for CF allows intervention before irreversible lung damage occurs. Unless the evolution of pulmonary function in young children is known, the optimal age of starting new therapies or enrolling CF NBS children into interventional trials is unclear. The London Cystic Fibrosis Collaboration has studied CF NBS infants longitudinally from diagnosis, as well as a contemporaneous group of matched healthy controls, and reported lung function was normal in the NBS CF group at two years of age. This thesis reports the preschool follow-up of the same infants, and describes the evolution of lung function to six years of age. The primary hypothesis was that lung function would remain within the normal range. Secondary aims were to compare preschool pulmonary function to that measured in children diagnosed with CF a decade earlier, and to describe which measures in NBS infants could predict preschool lung function. 67 preschool children with CF and 41 healthy controls underwent multiple breath washout (MBW), specific airway resistance and spirometry measurements. Lung Clearance Index (LCI), measured by MBW, was abnormal in CF NBS preschool children, but was better than children diagnosed with CF a decade earlier. The most significant predictor of abnormal preschool lung function was LCI at two years of age. Contrary to the hypothesis, compared to contemporaneous controls, lung function deteriorates at preschool follow-up in CF NBS children managed with standard UK care. As two year LCI was a significant predictor of later decline, implying that the root causes of decline were before this age, disease modifying therapies and interventional trials should be targeted before two years of age.
| Type: | Thesis (Doctoral) |
|---|---|
| Qualification: | M.D(Res) |
| Title: | The evolution of lung function in newborn screened preschool children with cystic fibrosis |
| Event: | UCL (University College London) |
| Open access status: | An open access version is available from UCL Discovery |
| Language: | English |
| Additional information: | Copyright © The Author 2019. Original content in this thesis is licensed under the terms of the Creative Commons Attribution 4.0 International (CC BY 4.0) Licence (https://creativecommons.org/licenses/by/4.0/). Any third-party copyright material present remains the property of its respective owner(s) and is licensed under its existing terms. |
| UCL classification: | UCL > Provost and Vice Provost Offices UCL > Provost and Vice Provost Offices > School of Life and Medical Sciences UCL > Provost and Vice Provost Offices > School of Life and Medical Sciences > Faculty of Population Health Sciences > UCL GOS Institute of Child Health |
| URI: | https://discovery.ucl.ac.uk/id/eprint/10084061 |
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