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Background In a 6-month clinical trial, TOBI Podhaler (TIP) has been shown to be safe and generally well tolerated compared to nebulised Tobramycin Inhalation Solution (TIS) in CF subjects [1]. However, there is little ‘real world' evidence for longer-term tolerance or likely adherence to TIP. Objectives To assess the longer term tolerance and likely adherence to TIP at our large regional UK adult CF centre. Methods We conducted a retrospective observational study of CF adults who completed a test dose of TIP in our centre between 1/1/12 and 1/1/13. Patient reported tolerance was documented and likely adherence was calculated by pharmacy prescription refill frequency. Results Of the 130 patients who completed a test dose of TIP (median 25.5 yrs, FEV 1 68% predicted), 58 patients (45%) continued to be prescribed TIP (median 29 months). In those discontinuing TIP (n = 72), the most common causes of discontinuation were ‘cough' (n = 17) and ‘tight chest' (n = 13). At test dose, the patients who subsequently discontinued TIP were older (median 27 yrs vs. 24 yrs), had lower FEV 1 (median 57% vs. 74%) and more frequently reported adverse events (AEs) (median 36% vs. 14%). Of the 72 patients who discontinued TIP, 56% were also intolerant of TIS. Prescription refill frequency in patients who have continued TIP was 65%. Conclusion ‘Real-world' tolerance to TIP in the longer term appears to be a significant challenge. Age, FEV 1 and AEs reported at trial may help to predict longer-term intolerance. In those continuing TIP, prescription refill frequency of 65% suggests that longer-term adherence was relatively well maintained.

Objectives In order to improve adherence, factors associated with compliance need to be identified. We investigated the relationships between the beliefs of CF adults about their treatment and reported adherence. Methods Patients attending the outpatient clinic in a regional adult CF centre were recruited over a three week period. Participants completed the Hospital Anxiety and Depression Scale and the Beliefs about Medicine Questionnaire. Adherence to pancreatic enzymes, vitamins, physiotherapy and exercise was recorded over two days using daily telephone diaries which use a day reconstruction method accompanied with a cued recall procedure. Demographics and spirometry results were recorded. Results 52 participants (25 male, mean age: 30 yrs) with median FEV 1 56.40% predicted were recruited (response rate: 94%). Multiple regression demonstrated that adherence was not influenced by participants' beliefs about their medications ( F (9,39) = 351, p = 0.951), with the model explaining 13.9% of the variance. However, weekday adherence and participants' beliefs about medication necessity were negatively correlated ( r = –0.305). Lung function ( r = –0.350) and anxiety ( r = –0.052) were negatively correlated with physiotherapy adherence. Conclusion This small-scale study highlights the impact patients' beliefs about medication-necessity, as well as disease severity and psychological status, have upon adherence. Further research utilising qualitative interviews with participants is under way to identify the main adherence-barriers and to develop interventions to improve treatment adherence in the CF patient population.

Background In the clinical trial setting, TOBI Podhaler (TIP) has been shown to be non-inferior to nebulised Tobramycin Inhalation Solution (TIS) in CF subjects in terms of effects on lung function, with increased adverse events but overall superior patient satisfaction. However, there is currently little ‘real world’ evidence of tolerance and effectiveness of TIP. Aim To assess the experience of CF adults receiving TIP at our large regional UK adult CF centre. Methods We report the preliminary results of a prospective observational study to evaluate changes in lung function, tolerability and antibiotic requirements of 73 CF adults that have received at least one dose of TIP at our centre to date. Patients also completed a visual analogue scale (VAS) score of tolerance, convenience, satisfaction and likelihood to continue (1 = very satisfied/not at all severe/very likely, 10 = not at all satisfied/very severe/not at all likely) at their 28-day review. Results Adverse events (AEs) were common following the test dose (most commonly cough in 24/73, 32.9%), although only 1 patient discontinued treatment, due to a 10% drop in FEV1% predicted. 27/72 patients so far have completed 28 days treatment, with median change in FEV1% predicted of –1.7% (IQR –6.1 to 2.4%). 12/27 patients (44.4%) reported AEs at their 28-day review; most commonly cough (10 patients, 37.0%), with median VAS score for severity of all AEs of 4/10. 12/27 patients (44.4%) were commenced on oral or IV antibiotics for pulmonary exacerbation at their 28-day review. Median VAS scores for overall satisfaction, ease of administration, and likelihood to continue were all 1/10. 12/72 patients to date have discontinued TIP before completion (10 patients) or at completion (2 patients) of 28 days treatment, 8 (66.7%) of whom were previously intolerant of TIS. The most common reason for discontinuation was cough (9/12 patients, 75.0%), with median VAS score for severity of all AEs 7/10. Conclusion Despite experiencing a high incidence of adverse events, patients commencing TIP report high levels of treatment satisfaction.

Background Meropenem is commonly used intravenously in CF patients infected with Pseudomonas aeruginosa, but there is limited evidence of its tolerability and effectiveness when used as a nebulised treatment. Aim To evaluate the use of nebulised meropenem in our large UK adult CF centre. Methods Medical records of patients who had trialled nebulised meropenem between 2008–2012 were reviewed for: demographics, lung function, BMI, sputum microbiology, indications and tolerability and number of exacerbations requiring IV antibiotics. Results 37 patients (18 male) trialled nebulised meropenem, with median age (IQR) 27 (20–37) years and FEV1% predicted 48% (35–64%). Patients received a 250 mg dose reconstituted with water for injection preceded by a bronchodilator. All patients were chronically infected with P. aeruginosa. The most common treatment indications were deteriorating condition or intolerance to other antibiotic nebulisers (21/37), with additional indications including treatment of Burkholderia cepacia complex (8/37) and non-tuberculous mycobacteria (8/37). 22/37 continue to tolerate the drug well. 10 of these 22 had previously been intolerant to Tobramycin Inhalation Solution (TIS) and 1 intolerant to nebulised amikacin. 4/37 discontinued due to no benefit or poor concordance. 11/37 did not tolerate nebulised meropenem, reporting adverse effects including chest tightness, increased cough, chest discomfort, nausea and lethargy. 6 of these 11 had previously been intolerant to TIS and 2 were intolerant to amikacin. There were no significant changes in lung function, BMI or requirement for IV antibiotics comparing the 12-month periods before and after commencing nebulised meropenem. Conclusion Nebulised meropenem is generally well tolerated in CF adults and offers an alternative antibiotic choice for people with CF unable to tolerate other treatments.

In common with other patients with chronic health conditions, adults with cystic fibrosis (CF) in the UK are eligible to claim for a variety of government-funded social security benefits. However, the UK government has recently announced planned changes to this system. In order to examine the

Patients with cystic fibrosis (CF) suffer recurrent bacterial pulmonary infections, but viral infections can also cause acute clinical deterioration.1 Certain patient groups suffer increased morbidity following pandemic (H1N1) 2009 influenza (swine flu),2 but there are few previous reports of outcomes in individuals with CF.3 4 The West Midlands, along with Greater London, has had the highest incidence of H1N1 influenza in the UK.5 We therefore examined the outcomes of patients diagnosed with H1N1 influenza at the West Midlands Adult CF Centre. From June 2009 to April 2010 all adults with CF at our regional centre with potential H1N1 influenza had nasopharyngeal swabs tested …

Home monitoring has the potential to detect early pulmonary exacerbations in people with cystic fibrosis (CF), with consequent improvements in health outcomes and healthcare associated costs. This study aims to assess the effects of home monitoring on hospital admissions, quality of life, antibiotic requirements, exacerbation frequency, lung function, nutritional outcomes, anxiety, depression, costs and health outcomes, as well as the qualitative effects on the patient experience. This randomised controlled mixed-methods trial aims to recruit 100 adults with CF cared for in one large regional CF centre. Participants are randomly allocated 1:1 to the intervention group (twice-weekly home monitoring of symptoms measured by the Cystic Fibrosis Respiratory Symptom Diary – Chronic Respiratory Infection Symptom Score (CFRSD-CRISS) and Forced Expiratory Volume in one second (FEV1)) or a control group (routine clinical care) for the 12-month study period. Measurements are recorded at study visits at baseline, 3, 6, 9 and 12 months. Spirometry, body weight, co-morbidities, medications, hospital inpatient days, courses of antibiotics (oral and intravenous), pulmonary exacerbations (defined by the modified Fuchs criteria) are recorded at each study visit. Health status, capability and health economics are measured at each study visit by the Hospital Anxiety and Depression Scale (HADS), the ICEpop CAPability measure for Adults (ICECAP-A), EuroQol 5 dimensions (EQ-5D-5L) questionnaire and an adapted resource use questionnaire. The patient experience is assessed by semi-structured qualitative interviews at baseline and 12 months. Results from this study will help to determine the effect of home monitoring on inpatient bed days and quality of life in adults with CF, as well as other relevant health and health economic outcomes. This study protocol is registered with Clinicaltrials.gov ( NCT02994706 ), date registered 16th July 2014

Background Increasing numbers of individuals with cystic fibrosis are becoming infected with the multidrug-resistant non-tuberculous mycobacterium (NTM) Mycobacterium abscessus, which causes progressive lung damage and is extremely challenging to treat. How this organism is acquired is not currently known, but there is growing concern that person-to-person transmission could occur. We aimed to define the mechanisms of acquisition of M abscessus in individuals with cystic fibrosis. Method Whole genome sequencing and antimicrobial susceptibility testing were done on 168 consecutive isolates of M abscessus from 31 patients attending an adult cystic fibrosis centre in the UK between 2007 and 2011. In parallel, we undertook detailed environmental testing for NTM and defined potential opportunities for transmission between patients both in and out of hospital using epidemiological data and social network analysis. Findings Phylogenetic analysis revealed two clustered outbreaks of near-identical isolates of the M abscessus subspecies massiliense (from 11 patients), differing by less than ten base pairs. This variation represents less diversity than that seen within isolates from a single individual, strongly indicating between-patient transmission. All patients within these clusters had numerous opportunities for within-hospital transmission from other individuals, while comprehensive environmental sampling, initiated during the outbreak, failed to detect any potential point source of NTM infection. The clusters of M abscessus subspecies massiliense showed evidence of transmission of mutations acquired during infection of an individual to other patients. Thus, isolates with constitutive resistance to amikacin and clarithromycin were isolated from several individuals never previously exposed to long-term macrolides or aminoglycosides, further indicating cross-infection. Interpretation Whole genome sequencing has revealed frequent transmission of multidrug resistant NTM between patients with cystic fibrosis despite conventional cross-infection measures. Although the exact transmission route is yet to be established, our epidemiological analysis suggests that it could be indirect.