Journal article

Publication year: 2019

Pages: 1238-1248

Journal: American Journal of Respiratory and Critical Care Medicine

Volume number: 199

Issue number: 10

ISSN: 1073-449X

eISSN: 1535-4970

DOI Link: https://doi.org/10.1164/rccm.201807-1203OC

Publisher: American Thoracic Society

Abstract: 

Rationale: 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 (20.6) than in those treated with IS (20.1; 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.

Harvard Citation style: Stahl, M., Wielpuetz, M., Ricklefs, I., Dopfer, C., Barth, S., Schlegtendal, A., et al. (2019) Preventive Inhalation of Hypertonic Saline in Infants with Cystic Fibrosis (PRESIS) A Randomized, Double-Blind, Controlled Study, American Journal of Respiratory and Critical Care Medicine, 199(10), pp. 1238-1248. https://doi.org/10.1164/rccm.201807-1203OC

APA Citation style: Stahl, M., Wielpuetz, M., Ricklefs, I., Dopfer, C., Barth, S., Schlegtendal, A., Graeber, S., Sommerburg, O., Diekmann, G., Huesing, J., Koerner-Rettberg, C., Naehrlich, L., Dittrich, A., Kopp, M., & Mall, M. (2019). Preventive Inhalation of Hypertonic Saline in Infants with Cystic Fibrosis (PRESIS) A Randomized, Double-Blind, Controlled Study. American Journal of Respiratory and Critical Care Medicine. 199(10), 1238-1248. https://doi.org/10.1164/rccm.201807-1203OC