Search

Inhaled therapies are relatively simple and easy to be managed however ineffective use of aerosols when self-administered may occur. We described variation of the number of clinic visits, lung function and number of antibiotic courses performed over 12 months in participants with cystic fibrosis (CF), when supervised or not by physiotherapists (PTs) at home. Participants in 8 Italian CF centers with a prescription of dry-powder antibiotic choose whether to be supervised at home (PT-FU) or not (non-PT-FU), in adjunct to routine clinic visits. PTs assisted participants with their inhaled therapies regimen and reviewed the airway clearance program in use. Mixed-effect regression models were fitted to evaluate the variation of selected endpoints over time. A total of 163 participants were included. Lung function declined over time in both groups, at higher extent in the non-PT-FU group at 6 months (-1.8, 95%CI: -4.4 to 0.7 % predicted), without reaching statistical significance, whereas in the PT-FU group only, nearly one visit less was recorded (p=0.027). Regardless the type of supervision adopted, the number of antibiotic courses did not change compared to the previous year. We counted 19/90 (21.1%) drop-out in the PT-FU, double compared to the group followed up at the clinics (p=0.065). Participants under a course of an inhaled antibiotic therapy showed a 1-year decline in lung function, whereas only the group receiving home supervision counted nearly one visit less at the CF center, whose clinical relevance should be further discussed.

Reduced exercise tolerance and quality of life are commonly reported in chronic obstructive pulmonary disease (COPD) patients, and key elements of their pulmonary rehabilitation (PR) programmes are also represented from exercise training programmes that improve exercise tolerance (less fatigue and less dyspnoea) and quality of life of COPD patients [1] as well as reduction in healthcare use [3]. These well-proven and measurable effects have been demonstrated in stable COPD patients as well as in COPD during or after an acute exacerbation (AE) [4]. To date, publications attributable to such programmes unequivocally demonstrate the effectiveness of physical exercise in this patient population (Table 15.1). From these considerations and from the level of scientific publications, until now produced, exercise training programmes can be considered a cornerstone of PR in COPD. Exercise practice and prescription has nowadays different scopes justified from multiple complex and coexisting physiopathological backgrounds in COPD: cardiovascular and ventilatory limitation gas exchange dysfunctions, respiratory and peripheral muscle changes and not least a permanent oxidative stress status [2]. Furthermore comorbidities often present in COPD population (obesity, diabetes, neurological conditions) play a negative role in planning and then in reaching effective and long-term results [3]. Poor motivation anxiety and depression also can play an additional negative factor in the success of PR programmes. General accepted rules regarding the main outcomes for physical training in COPD patients (but also in healthy population) indicate that total training load must exceed the load encountered during daily life improving the aerobic capacity and muscle strength always reflecting the individual general features [5].

Background In cystic fibrosis (CF) the defective CF transmembrane conductance regulator protein may be responsible for the impaired transport of glutathione (GSH), the first line defense of the lung against oxidative stress. The aim of this single-blind, randomized, placebo-controlled trial was to evaluate the effect of inhaled GSH in patients with CF. Methods 54 adult and 51 pediatric patients were randomized to receive inhaled GSH or placebo twice daily for 12months. Results Twelve month treatment with inhaled GSH did not achieve our predetermined primary outcome measure of 15% improvement in FEV 1 %. Only in patients with moderate lung disease, 3, 6 and 9months therapy with GSH resulted in a statistically significant increase of FEV 1 values from the baseline. Moreover GSH therapy improved 6-minute walking test in pediatric population. GSH was well tolerated by all patients. Conclusions Inhaled GSH has slight positive effects in CF patients with moderate lung disease warranting further study. Trial registry ClinicalTrials.gov; No.: NCT01450267; URL: www.clinicaltrialsgov.

We previously reported that the combination of two safe proteostasis regulators, cysteamine and epigallocatechin gallate (EGCG), can be used to improve deficient expression of the cystic fibrosis transmembrane conductance regulator (CFTR) in patients homozygous for the CFTR Phe508del mutation. Here we provide the proof-of-concept that this combination treatment restored CFTR function and reduced lung inflammation (P

Inhaled hypertonic saline improves lung function and decreases pulmonary exacerbations in people with cystic fibrosis. However, side effects such as cough, narrowing of airways and saltiness cause intolerance of the therapy in 8% of patients. The aim of our study was to compare the effect of an inhaled solution of hyaluronic acid and hypertonic saline with hypertonic solution alone on safety and tolerability. A total of 20 patients with cystic fibrosis aged 6 years and over received a single treatment regimen of 7% hypertonic saline solution or hypertonic solution with 0.1% hyaluronate for 2 days nonconsecutively after a washout period in an open crossover study. Cough, throat irritation, and salty taste were evaluated by a modified ordinal score for assessing tolerability; “pleasantness” was evaluated by a five-level, Likert-type scale. Forced expiratory volume in 1 second was registered before and after the end of the saline inhalations. All 20 patients (nine males, 11 females, mean age 13 years, range 8.9–17.7) completed the study. The inhaled solution of 0.1% hyaluronic acid and hypertonic saline significantly improved tolerability and pleasantness compared to hypertonic saline alone. No major adverse effects were observed. No difference was documented in pulmonary function tests between the two treatments. Hyaluronic acid combined with hypertonic saline solution may contribute to improved adherence to hypertonic saline therapy. Further clinical trials are needed to confirm our findings. Considering the extraordinary versatility of hyaluronic acid in biological reactions, perspective studies could define its applicability to halting progression of lung disease in cystic fibrosis.

Meconium ileus has been detected as a risk factor for development of liver disease in cystic fibrosis, with influence on morbidity and mortality.To evaluate the effect of early treatment with ursodeoxycholic acid in patients with cystic fibrosis and meconium ileus to prevent chronic hepatic involvement and to explore the potential role of therapy on clinical outcomes.26 cystic fibrosis patients with meconium ileus (16 M, mean age 8,4 years, range 3,5-9) were assigned to two groups: group 1 (14 patients) treated early with ursodeoxycholic acid (UDCAe); group 2 (12 patients) treated with ursodeoxycholic acid at the onset of cystic fibrosis liver disease (UDCAd). Anthropometric data, pulmonary function tests, pancreatic status, complications such as diabetes, hepatic involvement and Pseudomonas aeruginosa colonisation were compared among groups.A higher prevalence of cystic fibrosis chronic liver disease was observed in the UDCAd group with a statistically significant difference at 9 years of age (p0.05). Chronic infection by P. aeruginosa was found in 7% of UDCAe and 33% of UDCAd (p0.05). No differences were observed in nutritional status and other complications.Early treatment with ursodeoxycholic acid may be beneficial in patients at risk of developing cystic fibrosis chronic liver disease such as those with meconium ileus. Multicentre studies should be encouraged to confirm these data.

The study evaluate the efficacy of the Positive Expiratory Pressure (PEP) mask as a method of chest physiotherapy, both on its own and in conjunction with other physiotherapy techniques, in patients with cystic fibrosis. The results of PEP therapy was evaluated with Ventilatory Lung Scintigraphy with 99mTc-DTPA (coll) aerosol

Objectives: Oxidative stress biomarkers as reactive oxygen species are induced by the sustained activation of neutrophils and other CF-derived defects in the lung of CF patients. Observed defects include an impaired glutathione (GSH) metabolism. Its supplementation may counterbalance the oxidative stress. A randomized, single blind controlled trial of inhaled GSH versus placebo (NCT01450267) is underway in order to evaluate the effect of GSH in cohort of CF patients. We report preliminary data on tolerability to GSH in a pediatric subset of enrolled patients. Methods: 48 CF patients (F 23, age M±DS 13.53 yrs), in regular follow up at the Regional Pediatric CF Center of Naples, were enrolled for RCT. The main inclusion criteria were: CF diagnosis by sweat test and/or two CF causing mutations, age of patients >6 yrs, FEV1% >40% of the predicted value, negative culture for Burkholderia cepacia. Spirometry was performed before and 10 and 60 minutes after GSH inhalation test (10mg/kg, maximum dosage 600mg/dose) in order to assess tolerability. Conclusions: No patients showed a decrease in FEV1% >15% after GSH inhalation as defined in the study design. A statistically significant increase was observed for FEF25−75% after 10 and 60 minutes from inhalation (FEF25−75 M±DS: T0 71.64±33.35 VS T10 76.37±36.73; p< 0.02 and T0 71.64±33.35 VS T60: 80.26±35.25; p< 0.0001) and for FEV1% after 60 minutes from inhalation (FEV1 M±DS: T0 97.90±21.03 VS T60 100.01±19.42; p< 0.01). No side effects were reported. On the basis of these preliminary results we are currently evaluating the efficacy of inhaled GSH on pulmonary function and inflammatory markers within a 12 months therapy.