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

Mirjam Stahl1,2,3, Mark O Wielpütz4,3,5, Isabell Ricklefs6,7

  • 11 Department of Translational Pulmonology.

American Journal of Respiratory and Critical Care Medicine
|November 10, 2018
PubMed

Insights

Preventive inhalation with hypertonic saline (HS) in infants with cystic fibrosis (CF) was safe and improved lung clearance index (LCI) and weight gain. This study supports HS as a potential early intervention for CF lung disease.

Area of Science:

  • Pediatric Pulmonology
  • Respiratory Medicine
  • Clinical Trials

Background:

  • Cystic fibrosis (CF) lung disease begins in infancy, necessitating early preventive strategies.
  • Lung clearance index (LCI) and chest MRI are emerging as sensitive measures for early CF lung disease.
  • Limited randomized controlled trials exist for preventive therapies in infants with CF.

Purpose of the Study:

  • To assess the feasibility, safety, and efficacy of inhaled hypertonic saline (HS) versus isotonic saline (IS) in infants with CF.
  • To evaluate LCI and MRI as outcome measures in this infant CF population.
  • To determine the impact of early HS inhalation on CF lung disease progression.

Main Methods:

  • A randomized, double-blind, controlled trial involving 42 infants with CF under 4 months of age.
  • Participants received twice-daily inhalation of 6% HS or 0.9% IS for 52 weeks.
  • LCI and chest MRI were primary outcome measures, alongside adverse events and weight gain.

Main Results:

  • Inhalation of HS and IS was well-tolerated, with no significant difference in adverse events between groups.
  • Infants receiving HS showed a greater improvement in LCI compared to IS.
  • Improved weight gain was observed in the HS group, while pulmonary exacerbations and MRI scores did not differ significantly.

Conclusions:

  • Early preventive inhalation of HS in infants with CF is safe, well-tolerated, and improves LCI and weight gain.
  • LCI is a feasible and sensitive endpoint for randomized controlled trials in infants with CF.
  • These findings support the use of HS as an early intervention to potentially modify CF lung disease progression.

Keywords:
cystic fibrosislung clearance indexlung diseasemagnetic resonance imagingpreventive therapy

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