Validation of the Italian version of the Cystic Fibrosis Quality of Life Questionnaire (CFQoL), a disease specific measure for adults and adolescents with cystic fibrosis.

BACKGROUND: Disease specific, health-related quality of life (HRQoL) measurement is important in cystic fibrosis (CF). This work aimed to translate the original English Cystic Fibrosis Quality of Life Questionnaire (CFQoL) into Italian, evaluate the linguistic translation and to psychometrically evaluate the Italian version of the CFQoL. METHODS: The linguistic translation followed the international guidelines of forward and backward translation. Psychometric evaluation of the Italian CFQoL involved the assessment of construct validity, internal reliability, concurrent validity, known groups validity and test-retest reliability. RESULTS: The instrument was acceptable to adolescents and adults with CF and demonstrated robust psychometric properties. Principle components analysis indicated that the factorial structure was essentially similar to the original, and the internal reliability of each domain was good (Cronbach alpha coefficients 0.73 to 0.91). Appropriate domains of the CFQoL and SF-36 correlated well indicating good concurrent validity (r=0.68-0.80). Consistent with theoretical expectations some domains were able to discriminate between disease severity groups. Test-retest reliability, assessed by intraclass correlation coefficients, was found to be excellent (ICC 0.83 to 0.98). CONCLUSIONS: The Italian CFQoL is a valid and reliable measure. Its use in individual patient monitoring and research should complement traditional clinical outcome measures.

Efficacy, safety, and local pharmacokinetics of highly concentrated nebulized tobramycin in patients with cystic fibrosis colonized with Pseudomonas aeruginosa.

BACKGROUND AND AIM: Progressive respiratory failure due to Pseudomonas aeruginosa colonization is the most significant morbidity in patients with cystic fibrosis (CF). This trial was designed to investigate the efficacy and safety of a highly concentrated (300mg/4mL) tobramycin solution for inhalation (TSI) [Bramitob] in patients with CF and P. aeruginosa infection. METHODS: Fifty-nine patients were randomized to receive a 4-week treatment with tobramycin or placebo administered twice daily via the Pari LC Plus nebulizer and Pari TurboBoy compressor, followed by a 4-week run-out phase. Pulmonary function (forced expiratory volume in 1 second [FEV(1)], forced vital capacity [FVC], and forced expiratory flow at the midportion of vital capacity [FEF(25-75%)]), P. aeruginosa susceptibility, microbiologic results, and in vitro minimum inhibitory concentration for 90% of strains (MIC(90)) were the efficacy outcome measures, while safety was monitored by the recording of adverse events, audiometry (bone conduction at 250-8,000Hz frequency), laboratory tests, physical examination and general health condition. The concentration of tobramycin attained in sputum was measured in a cohort of 21 patients. RESULTS: FEV(1) significantly increased from baseline in the tobramycin group compared with no change in the placebo group: the absolute difference between groups (intent-to-treat population) of predicted normal was 13.2% at week 2 (p = 0.002) and 13.3% at week 4 (p = 0.003). Significant differences in favor of the tobramycin group were also observed for FVC and FEF(25-75%). The microbiologic results at the end of the treatment period (P. aeruginosa-negative culture, persistence, superinfection) showed a significantly better outcome in the tobramycin group compared with placebo (p = 0.033). The effects of tobramycin on pulmonary function and microbiology were not maintained at the end of the run-out phase. Mean sputum concentrations of tobramycin after the first dose (695.6 +/- 817.0 microg/mL) were similar to those measured after the last dose (716.9 +/- 799 microg/mL) and were superior to the detected specific MIC(90). The proportion of patients with drug-related adverse events was lower in the tobramycin group and no signs of renal or auditory toxicity were observed. CONCLUSIONS: The 4-week administration of a highly concentrated TSI significantly improved pulmonary function and microbiologic outcome compared with placebo and was well tolerated. The results of this study should be confirmed in further long-term trials in larger populations.