Long-term benefits of inhaled tobramycin in children with cystic fibrosis: first clinical observations from Poland

Abstract

Background: Respiratory disease is the major cause of mortality in cystic fibrosis (CF) patients and inhaled antibiotic therapy may contribute to the stabilization of lung function.

Objectives: This is a small, open, uncontrolled, observational study of clinical experience obtained with 2 years of maintenance treatment with inhaled tobramycin in 12 children and adolescents with CF.

Methods: Twelve subjects aged 6-18 years infected by Pseudomonas aeruginosa were qualified for treatment with inhaled tobramycin. Pulmonary function, weight and height, clinical status, and chest X-ray were continually monitored. After an active 2-year treatment period, results of all measured parameters in our patients were compared with their previous results (2-year period before treatment with tobramycin).

Results: During 2 years before treatment with tobramycin solution for inhalation (TOBI), pulmonary function decline was observed, the median value of FEV(1) change was -7.6% (lower quartile -13.1, upper quartile -5.9). After 2 years of treatment, FEV(1) percent predicted value declined by 1.5% (lower quartile -11.1, upper quartile 3.7) from baseline; 2 years of TOBI therapy significantly reduced lung function decline (p = 0.049). There were no significant changes in thoracic gas volume and specific airway resistance before and after treatment. Two years of TOBI therapy significantly improved body mass index (p = 0.02). TOBI treatment significantly delayed progression of pulmonary X-ray changes assessed by Brasfield score (p = 0.02).

Conclusions: We found that patients with CF can gain substantial benefits from long-term TOBI treatment, including reduced pulmonary function decline, delayed progression of pulmonary X-ray changes and improved weight gain in growing children and adolescents.