Background - Intermittent or chronic pulmonary infections caused by Pseudo - monas aeruginosa (Pa) deteriorate clinical status and worsen lung function in patients with cystic fibrosis (CF). The prognosis of the disease and life expectancy of patients are substantially dependent on lung infections and inflammation; therefore the primary goal of the treatment is the early termination of the infection. Patients and Methods - Efficacy of tobramycin (TOBI® 300 mg/5 mL solution for inhalation, henceforth TOBI®) inhalation in Pa pulmonary infections was studied in a non-interventional, observational, open-label, single-arm trial in subjects with CF. Fifty-three patients aged six to 31 years (averaged 15.8 years) were enrolled into the study. Three treatment cycles of TOBI® inhalation (28 days on drug, 28 days off drug) plus 6 months observational period were evaluated. Primary endpoint was changing in the predictive values of forced expiratory volume in 1 sec (FEV1) compared to the initial values after three cycles of the treatment. Secondary endpoints were changing in the FEV1 predictive values at the end of the complete study compared to the initial values; ratios of patients with decreased density of originally Pa-positive result of sputum culture; as well as safety and tolerability of the TOBI® treatment. Results - FEV1 and FEV1% results were unchanged comparing to the initial values at the end of the treatment and after the observational period in the whole study population. However, sputum cultures became negative in 47.2% of all subjects as a result of the treatment, and the FEV1 values were gradually increased in these patients: after the third treatment cycle plus 160 mL, and at the end of the study plus 110 mL comparing to the initial values. Similar considerable increase was detected in the predictive FEV1% in this sub-group: after the first cycle plus 5.2%, at the end of third cycle plus 7%, after the observational period above 3.8% considering the starting results. Conclusions - TOBI® inhalation proved to be effective in improvement of lung function results and eradication of infection in our patients with Pa positive CF. The treatment was well tolerated and safe.
|Number of pages||6|
|Journal||Lege Artis Medicinae|
|Publication status||Published - Jan 1 2015|
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