Inhaled hypertonic saline treatment feasible in young infants with cystic fibrosis
medwireNews: Preliminary findings from the phase II PRESIS trial suggest that preventative treatment with inhaled hypertonic saline (HS) is well tolerated and may improve lung clearance index (LCI) in young infants with cystic fibrosis.
Previous studies have demonstrated “that potentially reversible abnormalities such as airway mucus plugging, air trapping, neutrophilic inflammation and bronchial wall thickening are already present in young infants [with cystic fibrosis] in the first months of life”, suggesting that early preventative therapy may be a promising strategy to reduce lung damage, explain Mirjam Stahl (University of Heidelberg, Germany) and colleagues.
The PRESIS (Preventive Inhalation of Hypertonic Saline in Infants with Cystic Fibrosis Study) investigators randomly assigned 42 infants with cystic fibrosis aged less than 4 months to receive twice daily inhalations of HS (6% NaCl) or isotonic saline (IS; 0.9% NaCl) for 1 year. Participants inhaled the 4 mL solution using a jet nebuliser and an infant face mask.
Stahl and team report in the American Journal of Respiratory and Critical Care Medicine that inhalation of the study solutions “was generally well tolerated” over 1 year of treatment. All patients in the HS and IS groups experienced adverse events (AEs), the majority of which were mild (81.3%) or moderate (12.9%) in severity.
The most commonly reported AE in the HS group was rhinorrhoea, affecting 81.0% of HS-treated and 47.6% of IS-treated participants, followed by upper respiratory tract infection without fever (76.2 vs 81.0%) and cough (66.7 vs 57.1%). A corresponding 28.6% and 33.3% experienced serious AEs, none of which were considered by the investigators to be related to the study drug.
In the efficacy analysis, the researchers found that patients treated with HS experienced a “rapid and sustained” decline in average LCI, from 7.5 at baseline to 6.9 at the 1-year follow-up. By comparison, mean LCI was 7.2 at both timepoints for patients given IS, translating into a significant between-group difference in the change from baseline of 0.5.
Infants in the HS group also gained significantly more bodyweight on average over the study period compared with those given IS (4.8 vs 4.3 kg), but there were no significant differences between the groups in morphological abnormalities as measured with chest magnetic resonance imaging (MRI). Stahl and colleagues note that MRI subscores for mucus plugging, bronchial wall thickening/bronchiectasis and mosaic signal intensity “tended to increase” in both groups from baseline to 1 year, whereas those for pleural reaction and consolidation “tended to decrease”.
Taken together, these findings suggest that treatment with inhaled HS may have “therapeutic benefits on lung function and thriving in the first year of life”, say the researchers.
And they conclude that their results “support the conduct of future [randomised controlled trials] to evaluate safety and efficacy of preventive treatment strategies that have the potential to delay or even prevent irreversible lung damage in patients with [cystic fibrosis].”
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