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

• DOI: 10.1164/rccm.201807-1203oc
• Publication Date: 2018-11-09T08:03:34Z
• Authors: Mirjam Stahl; Mark O. Wielpütz; Isabell Ricklefs; Christian Dopfer; Sandra Barth; Anne Schlegtendal; Simon Y. Graeber; Olaf Sommerburg; Gesa Diekmann; Johannes Hüsing; Cordula Koerner-R...; Lutz Nährlich; Anna-Maria Dittrich; Matthias V. Kopp; Marcus A. Mall
• Keywords: Hypertonic saline

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