Francesca Lucca

Hyaluronic Acid Improves the Tolerability of Hypertonic Saline in the Chronic Treatment of Cystic Fibrosis Patients: A Multicenter, Randomized, Controlled Clinical Trial

UNLABELLED TRIAL DESIGN AND METHODS: Between December 2009 and July 2011, four cystic fibrosis (CF) centers in Italy participated in a randomized, double-blind, controlled clinical trial to test whether 7% hypertonic saline (HS) administered together with 0.1% hyaluronic acid (HA) was better tolerated by patients who previously did not tolerate HS well on its own. Participants were CF patients at least 8 years old, in clinically stable conditions, with forced expiratory volume in 1 sec (FEV1) at least 50% predicted. Forty patients were recruited and randomized to receive either HS or HS plus HA (5 mL to be inhaled over 15 min, twice daily for 28 days). Primary endpoints were cough, throat irritation, salty taste, and overall acceptability, as assessed by each patient on a semiquantitative scale on a diary card. Secondary endpoint was FEV1 change at the end of treatment. Patients were randomized into randomly permuted blocks. The first and last doses were administered in hospital. In between, patients were treated at home. Patients, all caregivers, and the statistician who conducted the analysis (different from the one who generated the random list) were blinded to group assignment. RESULTS Severity of cough, throat irritation, and saltiness were more severe in patients treated with HS alone, both after the first inhalation and over the entire treatment period. Overall pleasantness was rated higher by patients treated with the combination product. All differences were highly significant. There were no changes in FEV1 between the first and last administrations. Five patients did not complete the study. Four patients (two from each group) withdrew because of cough or throat irritation. One more patient from the HS group withdrew because of a respiratory exacerbation at week 3. CONCLUSIONS HS is currently a cornerstone in the treatment of CF patients. The addition of HA to HS reduces the prevalence and severity of cough, throat irritation, and saltiness and may improve compliance in patients who previously did not tolerate HS well on its own. Longer-term studies could further assess the benefit of chronic treatment.

Detection and monitoring of lung inflammation in cystic fibrosis during respiratory tract exacerbation using diffusion-weighted magnetic resonance imaging

The aim was to investigate whether diffusion-weighted magnetic resonance imaging (DWI) detects and monitors inflammatory and lung function changes during respiratory tract exacerbations (RTE) treatment in patients with cystic fibrosis (CF). 29 patients with RTE underwent DWI pre- and post-antibiotic treatment. A control group of 27 stable patients, matched for age and sex, underwent DWI with the same time gap as those undergoing RTE treatment. Clinical status and lung function were assessed at each DWI time point. The CF-MRI scoring system was used to assess structural lung changes in both CF groups. Significant reduction in the DWI score over the course of antibiotic treatment (p<0.0001) was observed in patients with RTE, but not in the control group. DWI score had a strong inverse correlation with clinical status (r=−0.504, p<0.0001) and lung function (r=−0.635, p<0.0001) in patients with RTE. Interestingly, there were persistent significant differences in the CF-MRI score between the RTE and control group at both baseline and follow-up (p<0.001), while the differences in DWI score were only observed at baseline (p<0.001). DWI is a promising imaging method for noninvasive detection of pulmonary inflammation during RTE, and may be used to monitor treatment efficacy of anti-inflammatory treatment. DWI is a promising imaging method to detect and monitor the severity of inflammation during RTE in CF patients http://ow.ly/Ulcc30c0ebU

Antibiotic resistance evolution of Pseudomonas aeruginosa in cystic fibrosis patients (2010-2013)

Pseudomonas aeruginosa is the predominant pathogen responsible of chronic colonization of the airways in cystic fibrosis (CF) patients. There are few European data about antibiotic susceptibility evolution of P aeruginosa in CF patients.

Asymmetric dimethylarginine and related metabolites in exhaled breath condensate of children with cystic fibrosis

Asymmetric dimethylarginine (ADMA) competitively inhibits nitric oxide synthase (NOS). Its levels in specimens from murine models and asthmatic patients are related to inflammation and oxidative stress. Patients with cystic fibrosis(CF) reportedly have higher arginase activity, lower NO production and NOS expression than healthy controls.

181 Spinal deformities (SD) in young CF patients: two years experience of screening and follow up

Background: Increased survival in CF patients (pts) requires assessment of extrapulmonary morbidities, as SD. Scoliosis (S) is more frequent in CF than in general population; it causes functional spinal disorders and restrictive pulmonary disease and may be influenced by CF musculoskeletal involvement. Aims: To describe: • SD distribution in a group of CF pts • SD and clinical status relationship • 2 years follow up in a subgroup Methods: Respiratory therapist measured the angle of trunk rotation (ATR) with scoliometer in the Adams forward bending test position in CF pts, 6−19 years old (yrs). Results: 58 pts (M 46.55%, aged 12.45±3.61 yrs, median %ile for weight 39.22 and for height 49.12, mean FEV1 91.79±16.94%pred) were enrolled. No difference at baseline existed between males and females. Pts were divided in: (1) Negative group (N): ATR 0−4o (n = 48); (2) Positive group (P): ATR 5o (n = 10). Pts in P were older (p = 0.001); N had greater FVC%pred (p = 0.008). 6 thoracic (60%), 3 lumbar (30%) and 1 thoracolumbar (10%) curves (70% with right convexity) were found. Specialized physician prescribed 10 spine x-ray diagnosing S (>10o Cobb) in 3 pts (5.17%) with indication of brace. ATR correlated with height %ile (r = 0.7; p = 0.02) in P; no relationship with lung function was found. 36 pts were reassessed 2 years after the 1st evaluation. ATR was the same in 52.78% of pts, while it increased in 25%. Changes in ATR didn’t correlate with respiratory and nutritional status. Conclusion: Our data justify SD early screening and at least yearly reassessment because of their possible progression and lack of correlation with clinical data. A study to assess S and back pain prevalence in adult CF pts is ongoing. 183 Mycobacterium abscessus: stand alone hospital admission for agressive physiotherapy and nutritional review can extend periods between toxic IV antibiotic treatments J. Tame1, A. Turnbull1, J. Forton1,2. 1Noah’s Ark Children’s Hospital for Wales, University Hospital of Wales, Department of Paediatric Respiratory Medicine, Cardiff, United Kingdom; 2Cardiff University, Department of Child Health, Cardiff, United Kingdom