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Inhaled hypertonic saline solution is safe, effective for infants with CF


A German study has shown that treatment with inhaled hypertonic saline is both safe and effective to improve children's cystic fibrosis (CF).

The change in lung clearance index (LCI) from baseline to week 52 was higher in infants treated with hypertonic saline -0.6 versus -0.1 in isotonic saline in infants (P<0.05), Mirjam Stahl, MD from the University of Heidelberg in Germany, and reported by a colleague.

A twice-daily inhalation of 6.4% hypertonic saline and inhalation of 0.9% isotonic saline was well tolerated and the number of adverse reactions in both groups (P= 0.49), they wrote American Journal of Respiratory and Critical Care Medicine.

Although the hypertonic saline solution has been shown to benefit both older children and CF children, a randomized, controlled study is the first to check the treatment – which includes the inhalation of a sterilized hypertonic saline solution that is inhaled in the nebulized form to reduce mucus in the respiratory tract – – in infants with advanced, congenital lung disease. Previous studies in mice and children with CF showed treatment to reduce the mucosal stopper, which may lead to airway obstruction and infections.

This study is also the first to show that two quantitative results – LCI and chest magnetic resonance imaging (MRI) – are useful in clinical trials with very small infants with CF.

"Several studies from the world's teams of infants and preschoolers with CF, discovered by newborn screening, revealed that CF lung disease begins in the first months of life, leaving only a limited possibility for prophylactic therapeutic intervention," Stahl said in a statement.

She noted that by inhibiting the onset and progression of CF-related lung diseases, the hypertonic saline solution could be a promising treatment strategy for infants and children with this disease.

In a study PRESIS (Hypertensive inhalation for infant prevention with CF), 42 new infants (<4 months of age) were randomized to receive KF in one of five places in Germany to receive hypertonic saline twice daily at 6.0% salt concentration (n = 21) or an isotonic saline solution at a concentration of 0.9% (n = 21).

In addition to assessing the efficacy of the treatment, the researchers tested whether LCI and thoracic MR (used to measure efficacy) can be safely performed in infants.

"So far, studies in which the safety and efficacy of prophylactic treatments have been tested, starting with the first months of life in infants with CF, was hampered by the lack of quantitative outcomes of lung disease in this challenging age group," writes Stahl and colleagues.

They added that a series of recent studies have shown that LCI, which is a measure of ventilation uniformity derived from multi-alive milking, can be useful for lung function, disease progression and response to infant therapy. It has also been shown that the thoracic MR is sensitive to detecting early changes in the pulmonary structure of infants with CF.

Among the other key findings of PRESIS:

  • In hypertonic saline treated infants, body weight gain improved (P<0.05)
  • Lung excitements and chest MRI did not differ between the two groups

"PRESIS is the first, randomized controlled trial, safety and initial efficacy of prophylactic lung disease initiated during the first months of life in infants with CF with the use of LCI and MRI as quantified early lung disease indicators," researchers write. "This study shows that parents are possible and well-accepted randomized controlled trials starting early in their childhood, including regular inhalational treatments and repeated 12-month LCI and MRI tests."

The findings also suggest that early intervention had a positive effect on lung function and thriving in infants with CF, and the study showed that LCI is a more sensitive treatment response than MR morphology or pulmonary exacerbation in infants with KF, according to researchers.

"This initial, randomized, controlled study confirms that prophylaxis with an inhaled hypertonic saline solution that begins in the first months of life is safe and has therapeutic benefits for lung function and palatable CF infants," concluded Stahl and colleagues. "These data support further randomized controlled trials to determine the safety and efficacy of preventive treatment strategies that can prevent or prevent progressive lung damage in patients with CF."

The research was supported by Dietmar Hopp-Fonda and the German Federal Ministry of Education and Research. The solutions used in the study and inhalation devices were provided by PARI Pharma.

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

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