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Investigating the effects of long-term dornase alfa use on lung function using registry data

Newsome, S.J., Daniel, Rhian M. ORCID:, Carr, S.B., Bilton, D. and Keogh, R.H. 2019. Investigating the effects of long-term dornase alfa use on lung function using registry data. Journal of Cystic Fibrosis 18 (18) , pp. 110-117. 10.1016/j.jcf.2018.08.004

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Background Dornase alfa (DNase) is one of the commonest cystic fibrosis (CF) treatments and is often used for many years. However, studies have not evaluated the effectiveness of its long-term use. We aimed to use UK CF Registry data to investigate the effects of one-, two-, three-, four- and five-years of DNase use on lung function to see if the benefits of short-term treatment use are sustained long term. Methods We analysed data from 4,198 people in the UK CF Registry from 2007 to 2015 using g-estimation. By controlling for time-dependent confounding we estimated the effects of long-term DNase use on percent predicted FEV1 (ppFEV1) and investigated whether the effect differed by ppFEV1 at treatment initiation or by age. Results Considering the population as a whole, there was no significant effect of one-year's use of DNase; change in ppFEV1 over one year was −0.1% in the treated compared to the untreated (p = 0.51) and this did not change with long-term use. However, treatment was estimated to be more beneficial in people with lower lung function (p < 0.001); those with ppFEV1 < 70% at treatment initiation, showed an increase in lung function over one year that was sustained out to five years. The estimated effect of DNase did not depend on age (p = 0.35). Conclusions DNase improved lung function in individuals with reduced lung function, bringing a step-change in lung function, but no change in the slope of decline. There was no evidence for a benefit in lung function in those initiating treatment with ppFEV1 > 70%.

Item Type: Article
Date Type: Publication
Status: Published
Schools: Medicine
Publisher: Elsevier
ISSN: 1569-1993
Date of First Compliant Deposit: 7 September 2018
Date of Acceptance: 2 August 2018
Last Modified: 05 May 2023 17:14

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