PRESIS: Hypertonic saline safe, effective in infants with cystic fibrosis
A clinical trial including lung clearance index and MRI as measures for early lung disease was feasible and showed that preventive inhalation hypertonic saline in infants with cystic fibrosis was safe and effective in improving lung clearance index and weight gain.
“Cystic brosis lung disease starts in early infancy, suggesting that preventive therapy may be most effective,” Mirjam Stahl, MD, pediatric pulmonologist in the department of translational pulmonology at the Translational Lung Research Center Heidelberg in Germany, and colleagues wrote in the American Journal of Respiratory and Critical Care Medicine. “Implementation of [cystic fibrosis] newborn screening has created a unique window of opportunity to test this concept in clinical trials, and recent studies indicate that the lung clearance index and chest MRI may be promising outcome measures of early [cystic fibrosis] lung disease.”
Stahl and colleagues conducted the randomized, double-blind, controlled PRESIS trial, which enrolled 40 infants (mean age, 0.26 years) with cystic fibrosis.
From June 2012 to November 2015, the researchers randomly assigned infants receiving treatment across ve sites to twice-daily inhalation of 6% hypertonic saline (n = 20) or 0.9% isotonic saline (n = 20) for 52 weeks.
According to the medication diary completed quarterly by parents, treatment adherence was high and comparable between the two arms, with 71.8% of those in the isotonic saline arm vs. 77.2% of those in the hypertonic saline arm adhering to treatment (P = .863).
Results showed both treatments were safe, with six adverse reactions (28.6%) observed among infants in the hypertonic saline arm vs. seven adverse reactions in the isotonic saline arm (33.3%; P = .49).
Additionally, no differences were observed in pulmonary exacerbations and chest MRI scores between the two arms.
However, changes in lung clearance index favored the hypertonic saline arm compared with the isotonic saline arm (–0.6 vs. –0.1; P < .05), and improvement in weight gain was associated with hypertonic saline treatment (P < .05).
“These data support the conduct of future [randomized controlled trials] to determine safety and efcacy of preventive treatment strategies that have the potential to delay or prevent progressive lung damage in infants with [cystic fibrosis],” Stahl and colleagues wrote. – by Jennifer Southall
Disclosures: The authors report no relevant financial disclosures.