• Am. J. Respir. Crit. Care Med. · May 2019

    Comparative Study Pragmatic Clinical Trial

    Preventive Inhalation of Hypertonic Saline in Infants with Cystic Fibrosis (PRESIS): A Randomized, Double-Blind, Controlled Study.

    • Mirjam Stahl, Mark O Wielpütz, Isabell Ricklefs, Christian Dopfer, Sandra Barth, Anne Schlegtendal, Simon Y Graeber, Olaf Sommerburg, Gesa Diekmann, Johannes Hüsing, Cordula Koerner-Rettberg, Lutz Nährlich, Anna-Maria Dittrich, Matthias V Kopp, and Marcus A Mall.
    • 1 Department of Translational Pulmonology.
    • Am. J. Respir. Crit. Care Med. 2019 May 15; 199 (10): 1238-1248.

    AbstractRationale: Cystic fibrosis (CF) lung disease starts in early infancy, suggesting that preventive treatment may be most beneficial. Lung clearance index (LCI) and chest magnetic resonance imaging (MRI) have emerged as promising endpoints of early CF lung disease; however, randomized controlled trials testing the safety and efficacy of preventive therapies in infants with CF are lacking. Objectives: To determine the feasibility, safety, and efficacy of preventive inhalation with hypertonic saline (HS) compared with isotonic saline (IS) in infants with CF, including LCI and MRI as outcome measures. Methods: In this randomized, double-blind, controlled trial, 42 infants with CF less than 4 months of age were randomized across five sites to twice-daily inhalation of 6% HS (n = 21) or 0.9% IS (n = 21) for 52 weeks. Measurements and Main Results: Inhalation of HS and IS was generally well tolerated by infants with CF, and the number of adverse events did not differ between groups (P = 0.49). The change in LCI from baseline to Week 52 was larger in infants with CF treated with HS (-0.6) than in those treated with IS (-0.1; P < 0.05). In addition, weight gain was improved in infants with CF treated with HS (P < 0.05), whereas pulmonary exacerbations and chest MRI scores did not differ in the HS group versus the IS group. Conclusions: Preventive inhalation with HS initiated in the first months of life was safe and well tolerated and resulted in improvements in LCI and weight gain in infants with CF. Our results support the feasibility of LCI as an endpoint in randomized controlled trials in infants with CF. Clinical trial registered with www.clinicaltrials.gov (NCT01619657).

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