Advertisement

Dornase alfa and rate of lung function decline in European patients with cystic fibrosis: A retrospective registry cohort study

Published:August 21, 2020DOI:https://doi.org/10.1016/j.jcf.2020.08.004
      Dornase alfa, a treatment that hydrolyses DNA, is approved as a therapy for patients with Cystic Fibrosis (CF). This mucolytic effect was shown in a pivotal clinical trial to improve lung function and reduce the frequency of CF pulmonary exacerbations [
      • Fuchs H.J.
      • Borowitz D.S.
      • Christiansen D.H.
      • Morris E.M.
      • Nash M.L.
      • Ramsey B.W.
      • et al.
      Effect of aerosolized recombinant human DNase on exacerbations of respiratory symptoms and on pulmonary function in patients with cystic fibrosis. The Pulmozyme Study Group.
      ]. Subsequent longitudinal studies of the impact of dornase alfa on lung function in United States’ and United Kingdom cohorts have shown additional long-term benefits including reductions in lung function decline, although these findings vary owing to differences in study design [
      • Hodson M.E.
      • McKenzie S.
      • Harms H.K.
      • Koch C.
      • Mastella G.
      • Navarro J.
      • et al.
      Dornase alfa in the treatment of cystic fibrosis in Europe: a report from the epidemiologic registry of cystic fibrosis.
      ,
      • Konstan M.W.
      • Wagener J.S.
      • Pasta D.J.
      • Millar S.J.
      • Jacobs J.R.
      • Yegin A.
      • et al.
      Clinical use of dornase alpha is associated with a slower rate of FEV1 decline in cystic fibrosis.
      ,
      • Newsome S.J.
      • Daniel R.M.
      • Carr S.B.
      • Bilton D.
      • Keogh R.H.
      Investigating the effects of long-term dornase alfa use on lung function using registry data.
      ]. In this study, we examined whether lung function decline is impacted by chronic dornase alfa treatment in subjects across Europe enrolled in the European Cystic Fibrosis Society Patient Registry (ECFSPR).
      To read this article in full you will need to make a payment

      Purchase one-time access:

      Academic & Personal: 24 hour online accessCorporate R&D Professionals: 24 hour online access
      One-time access price info
      • For academic or personal research use, select 'Academic and Personal'
      • For corporate R&D use, select 'Corporate R&D Professionals'

      Subscribe:

      Subscribe to Journal of Cystic Fibrosis
      Already a print subscriber? Claim online access
      Already an online subscriber? Sign in
      Institutional Access: Sign in to ScienceDirect

      References

        • Fuchs H.J.
        • Borowitz D.S.
        • Christiansen D.H.
        • Morris E.M.
        • Nash M.L.
        • Ramsey B.W.
        • et al.
        Effect of aerosolized recombinant human DNase on exacerbations of respiratory symptoms and on pulmonary function in patients with cystic fibrosis. The Pulmozyme Study Group.
        N Engl J Med. 1994; 331: 637-642
        • Hodson M.E.
        • McKenzie S.
        • Harms H.K.
        • Koch C.
        • Mastella G.
        • Navarro J.
        • et al.
        Dornase alfa in the treatment of cystic fibrosis in Europe: a report from the epidemiologic registry of cystic fibrosis.
        Pediatr Pulmonol. 2003; 36: 427-432
        • Konstan M.W.
        • Wagener J.S.
        • Pasta D.J.
        • Millar S.J.
        • Jacobs J.R.
        • Yegin A.
        • et al.
        Clinical use of dornase alpha is associated with a slower rate of FEV1 decline in cystic fibrosis.
        Pediatr Pulmonol. 2011; 46: 545-553
        • Newsome S.J.
        • Daniel R.M.
        • Carr S.B.
        • Bilton D.
        • Keogh R.H.
        Investigating the effects of long-term dornase alfa use on lung function using registry data.
        J Cyst Fibros. 2019; 18: 110-117