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Am J Respir Crit Care Med. 2019 May 15;199(10):1238-1248. doi: 10.1164/rccm.201807-1203OC.

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

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1 Department of Translational Pulmonology.
2 Division of Pediatric Pulmonology and Allergy and Cystic Fibrosis Center, Department of Pediatrics, and.
3 Translational Lung Research Center Heidelberg, German Center for Lung Research, Heidelberg, Germany.
4 Department of Diagnostic and Interventional Radiology, University of Heidelberg, Heidelberg, Germany.
5 Department of Radiology, German Cancer Research Center, Heidelberg, Germany.
6 Division of Pediatric Allergology and Pneumology, Department of Pediatrics, Medical University of Lübeck, Lübeck, Germany.
7 Airway Research Center North, German Center for Lung Research, Lübeck, Germany.
8 Department of Pediatric Pneumology, Allergology and Neonatology, Hannover Medical School, Hannover, Germany.
9 Biomedical Research in Endstage and Obstructive Lung Disease, German Center for Lung Research, Hannover, Germany.
10 Department of Pediatrics, Justus-Liebig-University Giessen, Giessen, Germany.
11 Universities Giessen and Marburg Lung Center, German Center for Lung Research, Giessen, Germany.
12 Department of Pediatric Pulmonology, University Children's Hospital of Ruhr University Bochum at St. Josef-Hospital, Bochum, Germany.
13 Department of Pediatric Pulmonology, Immunology and Intensive Care Medicine, Charité-Universitätsmedizin Berlin, Berlin, Germany.
14 Berlin Institute of Health, Berlin, Germany; and.
15 Coordination Center for Clinical Trials, Heidelberg University Hospital, Heidelberg, Germany.


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 (-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 (NCT01619657).


cystic fibrosis; lung clearance index; lung disease; magnetic resonance imaging; preventive therapy


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