Inhaled hypertonic saline solution safe and effective in infants with CF

A study in Germany found that treatment with inhaled hypertonic saline was both safe and effective in improving the respiration of infants with cystic fibrosis (CF).

The change in the pulmonary clearance index (LCI) from baseline to week 52 was greater in infants treated with hypertonic saline, at -0.6 compared to -0.1 in infants treated with an isotonic saline solution (P<0.05), Mirjam Stahl, MD, from the University of Heidelberg, Germany, and colleagues reported.

Inhalation twice daily of hypertonic saline solution at 6.0% and 0.9% isotonic saline solution were generally well tolerated, and the number of adverse events n & # 39; 39 did not differ between the two groups (P= 0.49), they wrote in the American Journal of Respiratory and Critical Care Medicine.

Hypertonic saline has been shown to be beneficial to older infants and children with cystic fibrosis, but the randomized controlled trial is the first to look at treatment, which is a sterilized solution of inhaled hypertonic saline in nebulized form. in order to thin the mucus in the airways. – in infants with progressive hereditary lung disease. Previous studies in mice and children with cystic fibrosis have shown that treatment reduces clogging of mucous membranes that can lead to airway obstruction and infections.

The study is also the first to show that two quantitative outcome measures – ICV and thoracic magnetic resonance imaging (MRI) – can be used in clinical trials involving very young infants with cystic fibrosis.

"Several studies conducted worldwide on infants and preschool children with cystic fibrosis identified by neonatal screening revealed that fibrocystic lung disease began in the first months of life, leaving only There is little room for maneuver for preventive therapeutic interventions, "Stahl said in a statement.

She noted that by delaying the onset and progression of CF-badociated lung disease, hypertonic saline could be a promising therapeutic strategy for infants and children with the disease.

As part of the PRESIS study (preventive inhalation of hypertonic saline solution in infants with cystic fibrosis), the researchers randomized 42 infants (aged less than 4 months) with CF on the disease. one of five German sites to receive hypertonic saline twice a day at a concentration of 6.0% salt. (n = 21) or isotonic saline at a concentration of 0.9% (n = 21).

In addition to evaluating the effectiveness of the treatment, the researchers examined whether the ICM and thorax MRI (used to determine efficacy) could be performed safely on infants.

"Until now, tests testing the safety and effectiveness of preventive therapies starting in the first months of life in infants with cystic fibrosis were hampered by the lack of quantitative measures of the results of lung disease in this difficult age group, "wrote Stahl and his colleagues.

They added that a series of recent studies suggested that ICV, which is a measure of the homogeneity of ventilation derived from multiple breath washing, could be a useful measure of lung function, disease progression and response to treatment in infants. Similarly, it has been demonstrated that thoracic MRI is sensitive to the detection of early abnormalities of lung structure in infants with cystic fibrosis.

Other key findings of PRESIS include:

• Weight gain was improved in infants treated with hypertonic saline (P<0.05)
• Pulmonary exacerbations and thoracic MRI scores did not differ between the two groups

"PRESIS is the first randomized trial of controlled trials investigating the feasibility, safety and initial effectiveness of a preventive treatment of early pulmonary disease during the first months of life. in infants with cystic fibrosis using ICM and MRI as quantitative measures of early lung disease, "the researchers wrote. . "This study demonstrates that randomized controlled trials starting in early childhood, including regular treatment with inhalation solutions and repeated measures of LCI and MRI over a 12-month period, are feasible and well accepted by the parents."

The results also indicate that early intervention had beneficial effects on lung function and development in cystic fibrosis infants, and the study suggests that ICV is a more sensitive measure of treatment response than morphology scores. MRI or pulmonary exacerbations in infants with CF, the researchers wrote.

"This initial randomized controlled trial confirms that preventive treatment with hypertonic saline solution that is inhaled in the first months of life is safe and offers therapeutic benefits for lung function and the development of infants with cystic fibrosis," concluded Stahl. colleagues. "These data support the conduct of future randomized controlled trials designed to determine the safety and effectiveness of preventive treatment strategies that may delay or prevent progressive lung injury in CF patients."

2018-09-11T00: 00: 00-0400

Last update 09/11/2018