Objectives: Literature reviews have identified a lack of consensus about the definition of an exacerbation in CF. We sought to determine which exacerbation definitions are most commonly used in CF clinical trials, and which signs/symptoms are most commonly included in exacerbation definitions across all CF clinical trials. Method: Eligible randomised trials were those in people with CF aged over 6 years, where a sign/symptom-based definition was used to identify exacerbations as an outcome. Eligible trials were identified from reference lists of Cochrane Reviews in CF. Identified trials underwent citation tracking for further eligible trials. The number of times a definition was used across all trials was tallied. The number of times each symptom was included in any trial’s definition was also tallied. Results: The searches identified 36 trials that had clearly specified the exacerbation definition used. The most commonly used definitions were from the DNase trial by Fuchs (1994), used in 11 trials; the US CFF Conference (1994), used in 6 trials; and the EPIC study (2009), used in 3 trials. The symptoms most commonly used among all trials were: sputum (used in 36 trials), cough (35), spirometry (34), weight/appetite (33), chest sounds (29), CXR changes (29), fever (27) and dyspnoea (24). The remaining 13 signs/symptoms were each used in 18 or fewer trials. Conclusion: Researchers may use these results to guide selection of a definition for their research. Clinicians may use these results to consider whether the definition used in a particular trial includes signs and symptoms that are generally accepted as representing an exacerbation by most clinical trialists in CF.

Impaired mucociliary clearance and mucus retention contribute to the chronic cycle of airway inflammation, infection and damage in bronchiectasis. There is a strong rationale for the use of pharmacological strategies to aid airway clearance, often in combination with chest physiotherapy. Despite the availability of many candidate mucoactive agents, the evidence base for recommending these agents is currently limited. Recent research and trials have focused particularly on osmotic agents (hypertonic saline and mannitol), which increase airway hydration, and early studies appear promising for both of these agents. Dornase alfa is not effective in non-cystic fibrosis (CF) bronchiectasis, which underscores the importance of conducting high quality and adequately powered trials that specifically address the therapeutic options for non-CF bronchiectasis.

Aim: Measurement of chest excursion is used to evaluate lung expansion from three different thoracic regions.The aim of the study was to investigate the relationship between chest excursion and lung function, functional capacity and upper extremity muscle strength in clinicaly stable cystic fibrosis (CF) patients.Methods: Twenty-eight (18 males, 10 females) patients, the aged 7-21 years, with CF participated in the study.Pulmonary function test was performed using spirometry.Chest excursion was measured using a tape measure from axillar, subcostal, and epigastric regions.Functional capacity was evaluated with sixminute walk test (6MWT).Upper extremity muscle strength (shoulder flexion and abduction, elbow flexion, and hand grip strength) was evaluated using digital dynamometer.Results: The mean FEV1 was 85.80±23.44%.Chest excursion from axillar region was significantly correlated with FVC (r = 0.61), FEV1 (r = 0.66), PEF (r = 0.63), FEF25-75% (r = 0.58), 6MWT distance (r = 0.39), shoulder flexion (r = 0.40) and abduction (r = 0.41), elbow flexion (r = 0.54), and hand grip strength (r = 0.59, p < 0.05).Chest excursion from epigastric region was statistically related with FVC (r = 0.59), FEV1 (r = 0.58), PEF (r = 0.57), FEF25-75% (r = 0.48), 6MWT distance (r = 0.41), shoulder flexion (r = 0.44) and abduction (r = 0.42), elbow flexion (r = 0.49), and hand grip strength (r = 0.55, p < 0.05).Chest excursion from subcostal region was significantly correlated with FEV1 (r = 0.39), PEF (r = 0.38), and FEF25-75% (r = 0.39, p < 0.05).Conclusion: Chest excursion measurement from upper thorax was significantly correlated with lung function, functional capacity and upper extremity muscle strength.Increasing chest mobility was associated with increased functional capacity and upper extremity training.Therefore measurement chest mobility should be included in comprehensive evaluation of cystic fibrosis patients.

Introduction: Studies show Non-Invasive Ventilation (NIV) in Cystic Fibrosis (CF) has reduced respiratory failure and aided secretion clearance. Our aim was to examine the use of NIV in CF patients over 12 months (Jan–Dec 2008). Method: A retrospective observational study of adult CF inpatients treated with NIV was performed. Indication for NIV treatment, tolerance, complications and perceived benefit were recorded. Results: 20 patients were treated with NIV over 35 episodes, 9 male with a mean age of 31 years (SD 9). Median predicted lung function on admission was 23% (10−53%). 13 patients had 1 NIV episode; 7 patients had 2−6 episodes. NIV was used to aid sputum clearance in 5 patients (12 episodes) (34%), control hypercapnic symptoms in compensated type II respiratory failure in 10 patients (17 episodes) (49%) and to reverse acidosis in acute type II respiratory failure in 5 patients (6 episodes) (17%). NIV was tolerated well in 18 patients (33 episodes) (94%), with subjective benefit in all episodes. In patients with raised PaCO2, reduction of headache was reported in 9 patients (17 episodes) (74%), and decreased work of breathing in 3 patients (3 episodes) (13%). All 5 patients (12 episodes) started on NIV to aid sputum clearance and 1 patient with acute respiratory failure reported increased sputum clearance with NIV. 2 patients were unable to tolerate the mask and felt no subjective benefit. The most common complications reported by those patients who tolerated NIV were; aerophagia 4 (22%), pressure sores 3 (17%), difficulty sleeping 2 (11%) and dehydration 2 (11%). Conclusion: NIV was used successfully to control symptoms of hypercapnic respiratory failure and sputum retention and was generally well tolerated.

Purpose:The objective of this study is to provide a detailed account of the cost of paediatric CF to the NHS in the Eastern Region.Background: In a review of CF economic evaluation literature, Krauth, et al (2003) found five Cost of Illness (COI) studies based on patient data.The authors found that most studies underestimated healthcare costs.The studies also found that costs are dependent on patients' age, and grade of severity among other factors.They found no paediatric CF care studies carried out in the UK. Data collection methods:The analysis looks at the paediatric COI for the region in 1998 using a retrospective analysis on patient data collected in the Eastern Region CF Database.Costs are derived from national sources.Analysis: The viewpoint will be that of the NHS in the Eastern Region.Resource use and costs are reported separately.Given the variance in disease severity, it is appropriate to estimate costs of care at the individual level.An accumulation of costs per case method is applied.Results: The total cost of caring for 371 patients in the Eastern Region in 1998 was approximately € 3.3 million.The mean cost per patient is estimated to be € 8,900. Conclusion:This study provides an indication of the cost of care in the region and can help to inform decision makers when making decisions on CF care.This study can also provide a reference for future analysis of care in the region to determine the most cost-effective service.

Rationale: Recurrent pulmonary exacerbations are associated with progressive lung disease in cystic fibrosis (CF). Current definitions of an exacerbation, although not precisely defined, include new/ worsening symptoms, declining lung function, and/or changing radiologic appearance. Early diagnosis of exacerbations by rapid noninvasive means should expedite therapeutic intervention, thereby minimizing lung damage.Objectives: To identify biomarkers of lung exacerbation for point-of-care monitoring of CF lung disease progression.Methods: Saline-induced sputum was collected from adults with CF with an exacerbation and requiring hospitalization (FEV1 < 60%), a subset of these adults at hospital discharge, children with stable CF and preserved lung function (FEV1 > 70%), and control subjects (FEV1 > 80%). Sputum was arrayed by two-dimensional electrophoresis and differentially expressed proteins were identified by proteomic analysis.Measurements and Main Results: Sputum profiles from adults with CF with an exacerbation were characterized by extensive proteolytic degradation and influx of inflammation-related proteins, with some adults with CF approaching a "healthy" protein profile after hospitalization. Two children with CF showed profiles and biomarker expression resembling those of adults with an exacerbation. Levels of differentially expressed myeloperoxidase, cleaved alpha(1)-antitrypsin, IgG degradation, interleukin-8, and total protein concentration, together with their correlation to FEV1, were statistically significant. Statistical correlation analyses indicated that changes in myeloperoxidase expression and IgG degradation were the strongest predictors of FEV1.Conclusions: We identified extensive protein degradation and differentially expressed proteins as biomarkers of inflammation relating to pulmonary exacerbations. Prediction of exacerbation onset and more precise evaluation of the extent of resolution with treatment could be achieved by including biomarkers in standard assessment.

BACKGROUND:Colonisation with Burkholderia cepacia complex in patients with cystic fibrosis (CF) has been associated with adverse outcomes. The aim of the present study was to determine the actuarial survival of CF patients colonized with B. cepacia and to evaluate the efficacy of the Royal Prince Alfred Hospital segregation policy. A secondary aim was to characterize the specific genomovars and strains of B. cepacia isolated in an Australian clinic. METHODS:Retrospective review of spirometric and microbiological data on all patients colonized with B. cepacia. Each B. cepacia-colonized subject was matched with three case-control subjects. Phenotype and genomovar typing, random amplified polymorphic DNA strain type and B. cepacia epidemic strain marker analyses were performed. The effect of B. cepacia colonization on transplant-free survival was estimated by Cox's proportional hazards regression using the entire clinic population. RESULTS:Fifteen patients were colonized with B. cepacia, of whom six (40%) had died from CF-related disease by August 1998, compared with 30 of 173 (17.3%) of the entire clinic population. Cepacia status had a significant adverse effect on survival, with a hazard ratio of 2.16 (95% confidence interval 1.0-4.69; P = 0.05). The outcome was variable in subgroups of B. cepacia. DISCUSSION:Colonization with B. cepacia had a significant adverse effect on survival within the study population. Genomovar and strain typing contributed usefully in accessing the effectiveness of the hospital's segregation policy in preventing cross-colonization.