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

Journal of Cystic Fibrosis : Official Journal of the European Cystic Fibrosis Society
Simon J NewsomeRuth H Keogh

Abstract

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. 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. 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 = ...Continue Reading

Citations

Sep 25, 2020·Pediatric Pulmonology·Adrienne P Savant, Susanna A McColley
May 14, 2020·International Journal of Molecular Sciences·Vincent D GiacaloneCamilla Margaroli
Jan 12, 2021·Expert Opinion on Drug Delivery·Douweh Leyla Gbian, Abdelwahab Omri
Aug 26, 2020·Journal of Cystic Fibrosis : Official Journal of the European Cystic Fibrosis Society·Edward F McKoneLaura Kirwan
Jan 19, 2022·Pediatric Pulmonology·Katerina ManikaJohn Tsanakas

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