Inhaled hypertension therapy has been shown to be safe and effective in improving respiration in children with cystic fibrosis (CF) in German studies.
The changes in lung clearance index (LCI) from baseline to week 52 were greater in infants treated with isotonic saline at -0.6 compared to -0.1 in infants treated with isotonic saline.blood<0.05), Dr. Mirjam Stahl of the University of Heidelberg, Germany.
Inhalation twice daily of 6.0% high salt solution and 0.9% isotonic saline was generally well tolerated and the frequency of side effects was not different between the two groupsblood= 0.49), they Respiratory and Critical Care Medicine Journal.
High-grade salinity has been shown to be beneficial for older babies and children with CF, but randomized controlled trials have shown that treatment with a sterile solution of high-quality saline inhaled in spray form for the purpose of diluting mucus in airways . – In infants with progressive, hereditary pulmonary disease. Previous studies of mice and CF patients have suggested treatments to reduce mucous plugging that can cause airway obstruction and infection.
This is the first study to show that two scales of quantitative results (LCI and chest MRI) are possible in clinical trials in very young infants with CF.
According to the statement, "In a newborn screening test for infants and preschool children identified as a newborn screening test, CF lung disease starts only one month after birth and there is limited opportunity for preventive treatment," Stahl said in a statement.
She noted that delaying the onset and progression of pulmonary disease associated with CF could be a promising treatment strategy for infants with high-saline infectious disease.
In a study of PRESIS (a high-density saline injection in infants with CF), the researchers found that CF treated in one of the five sites in Germany had 42 infants (<4 months) receiving twice the hypertension menstrual twice daily at 6.0% (N = 21) or 0.9% concentration of isotonic saline (n = 21).
In addition to assessing treatment efficacy, the researchers also investigated whether breast T-MRI used to determine LCI and efficacy is safely performed in infants.
"The clinical trial testing the safety and efficacy of prophylactic treatment starting from the first month of infants diagnosed with CF until now has been challenging because of the lack of quantitative results of this age-old lung disease measure," says Stahl. I went through.
In a recent series of studies, they suggested that LCI, a measure of ventilation homogeneity due to multiple breathing cleansing, could be a useful measure of lung function, disease progression, and response to infant care. Likewise, chest MR imaging is sensitive to early pulmonary abnormalities in infants with CF.
Other key findings from PRESIS:
- Weight gain was improved in newborns treated with high-salt salineblood& Lt; 0.05)
- Lung deterioration and breast MRI scores were not different between the two groups
"PRESIS is the first randomized controlled trial to test the feasibility, safety and initial efficacy of lung disease initiated within the first month of CF patients using LCI and MRI as a quantitative outcome measure for early pulmonary disease." This study demonstrates that a randomized controlled trial, beginning with early infancy, including 12 months of regular treatment, including inhalation solutions and regular LCI and MRI measurements, is available and well received by the parent. "
The results of this study show that early intervention has a positive effect on pulmonary function and prosperity among infants with CF, and LCI is more sensitive to treatment response than MRI morphological score or lung deterioration in infants with CF Scale. .
"This initial randomized controlled trial supports inhaled hypertensive physiological saline from the first month of life that preventive treatment is safe and has a therapeutic effect on pulmonary function and the prosperity of infants receiving treatment," concludes Stahl. "This data supports future randomized controlled trials to determine the safety and efficacy of prophylactic treatment strategies that can delay or prevent progressive lung injury in CF patients."
The study was funded by the Dietmar Hopp-Foundation and the German Federal Ministry of Education and Research. The solutions and inhalation devices used in this study were provided by PARI Pharma.
2018-09-11T00: 00: 00-0400