ABSTRACT
AIMS: Respiratory complications in Cystic Fibrosis (CF) are still the leading cause of death nowadays in these patients. High-Resolution Computed Tomography is the gold standard method for staging lung disease in CF. In this study we assessed lung ultrasound findings in asymptomatic preschool patients affected by CF. METHODS: This is a case-control study with a total of 70 enrolled patients (20 patients affected by CF, 50 healthy controls) aged from 31 to 6 years. All included patients were without intercurrent lung problems and without antibiotic therapy in the last 30 days. For each patient a lung Point of Care Ultrasound (POCUS) of lung was performed. RESULTS: B lines < 3 and sub-pleural consolidations < 1 cm were statistically more frequent in CF patients, both in terms of number of affected patients (p 0.02 and p 0.0001 respectively) and frequency (p 0.0181 and p 0.0001 respectively); the prevalence of B lines < 3 in control group was high (47.73%) however the prevalence of sub-pleural consolidations was very low (2.27%). In both groups coalescent B lines affected a greater number of infants and were in higher number of findings than patients aged between 2 and 6 years. CONCLUSIONS: The presence of multiple subpleural pulmonary consolidations < 1 cm in asymptomatic preschool children could be a ultrasound markers of subclinical pulmonary disease such as CF. POCUS of lung is confirmed as a useful tool for the clinician as confirmation of a clinical suspicion, help reduce the use of ionizing radiation.
Subject(s)
Cystic Fibrosis , Lung , Point-of-Care Systems , Ultrasonography , Humans , Cystic Fibrosis/diagnostic imaging , Cystic Fibrosis/complications , Ultrasonography/methods , Child, Preschool , Male , Case-Control Studies , Female , Lung/diagnostic imaging , Pilot Projects , Prospective Studies , Child , AdultABSTRACT
BACKGROUND: Ivacaftor is an innovative treatment for CF. Ivacaftor monotherapy in a phase III trial for patients with F508del and a residual function (RF) mutation showed improvement in lung function. We evaluated the effectiveness and safety of ivacaftor in patients with severe CF carrying RF mutations. METHODS: Data were collected from Italian CF centers with patients enrolled in an ivacaftor compassionate use program. Data were collected 1 year before and 1 year after commencement of ivacaftor. RESULTS: Twenty-six patients received ivacaftor. The mean [standard deviation (SD)] percent predicted FEV1 significantly increased from 33.9% (8.3) before treatment to 44.0% (10.7) after 12 months of treatment (p < 0.00001). The mean distance in the 6-min walking-test significantly improved from 458.2 (110.5) m at baseline to 524.8 (91.9) m after 12 months (p < 0.00001). The overall number of days of antibiotic therapy decreased from 1693 during the year before ivacaftor to 714 in the year following ivacaftor, and the number of days of intravenous antibiotic treatment dropped from 714 to 88; both results were statistically significant (p < 0.00001). Patients needing intravenous therapy decreased from 23 to 5 of 26. The mean (SD) sweat chloride level decreased from a baseline of 79 (22.3) mmol/L to 65 (30.6) mmol/L, but this variation was not significant (p = 0.26). No safety concerns were registered. CONCLUSIONS: In patients with CFTR mutations that confer RF with severe lung disease, treatment with Ivacaftor is safe and results in a clinically significant improvement that was evident at 1 month and maintained at 12 months.
Subject(s)
Aminophenols/therapeutic use , Cystic Fibrosis/drug therapy , Cystic Fibrosis/physiopathology , Lung Diseases/drug therapy , Lung Diseases/etiology , Lung/physiopathology , Mutation , Quinolones/therapeutic use , Respiratory Function Tests , Adult , Aminophenols/pharmacology , Cystic Fibrosis/complications , Cystic Fibrosis/genetics , Cystic Fibrosis Transmembrane Conductance Regulator/genetics , Female , Humans , Lung Diseases/physiopathology , Male , Middle Aged , Quinolones/pharmacology , Retrospective Studies , Treatment Outcome , Young AdultABSTRACT
Off-label use is very common among pediatricians, and the main cause is attributable to the lack of drugs specifically designed and marketed for children in different age groups. In this study, we evaluated, through a questionnaire, the knowledge of off-label drugs in pediatrics. Furthermore, we made a directory of 28 off-label drugs most commonly used by pediatricians in agreement with data obtained from Italian Agency for drugs (AIFA) referred to the law no. 648/1996; 180 pediatricians referred to the Italian Society of Pediatrics Calabrian section were asked to complete an online anonymous questionnaire. Eighty five (47.3%) of these completed the anonymous questionnaire, 40% revealed that they used off-label drugs "sometimes"; generally, drugs were used off-label for age and to treat respiratory diseases. For 75 pediatricians (88%) the information about the risk/benefit of off-label drugs is inadequate and 63 pediatricians (74%) did not have a good knowledge about this practice. In conclusion, the knowledge of off-label drugs is very low in pediatricians; more information about off-label drugs could be useful in order to improve the appropriateness of drugs' prescription and to reduce the development of side effects and improving drug safety.
