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Patient registries have the potential to collect and analyze high-quality postauthorization data on new medicines.We used cystic fibrosis (CF) registry data to assess outcomes after the initiation of ivacaftor, a CF transmembrane conductance regulator (CFTR) potentiator approved for the treatment of CF with a defective gating CFTR mutation.Longitudinal trends were examined using mixed-effects regression analysis in 80 ivacaftor-treated patients with CF aged 6 to 56 years registered with the CF Registry of Ireland with at least 36 months of before and after commencement data. The effects of ivacaftor treatment on forced expiratory volume in 1 second (FEVIn the 36 months after ivacaftor initiation, FEVIn this study of real-world CF registry data, clinical outcomes improved and healthcare resource utilization decreased after commencing ivacaftor.

Interactive health literacy (HL) skills enable individuals to participate more fully in healthcare activities and play a role in improving their outcomes. We examine the associations between HL and cystic fibrosis (CF) outcomes and compare HL in a sample from both the Irish CF and general populations.A total of251 CF Registry participants aged 13-30 years completed the HLS-EU-Q16 survey and a disease-specific instrument for measuring quality of life (QoL) in CF. Health outcome and healthcare resource utilization (HCRU) data were sourced from the registry. CF patient outcomes were examined using generalized linear models (GLMs) with interactive HL categorization included as a factor. General population interactive HL data are from the 2011 European HL Survey (HLS-EU). Interactive HL in 180 age-sex matched CF and general population individuals was examined using a GLM with study population, sex and educational level included as factors and age as a covariate.Sufficient interactive HL (total sum score ≥13) was self-reported by 81.7% of individuals with CF. Sufficient HL was associated with fewer outpatient visits [7.02(SD = 6.7: 7.4) vs. 8.74(SD = 7.9: 9.6), P0.001], days hospitalized [10.25(SD = 9.8: 10.7) vs. 12.8(SD = 11.8: 13.9), P0.001], days on intravenous antibiotics [15.3(SD = 14.7: 15.8) vs. 19.7(18.5: 21.1), P0.001], days on oral antibiotics [27.4(SD = 26.7: 28.1) vs. 48.48(38.7: 42.4), P0.001] and better QoL [77.1(SD = 75.4-78.9) vs. 64.6(60.8-68.3), P0.001]. Mean HL scores in CF and general populations were sufficient, although higher among individuals with CF (14.3 vs. 13.1, P0.01).CF adolescents and young adults with sufficient levels of HL to obtain, understand, appraise and apply health information have better health-related outcomes.

Background Pulmonary infection is the main cause of death in cystic fibrosis (CF). Aspergillus fumigatus (AF) and Pseudomonas aeruginosa (PA) are the most prevalent fungal and bacterial pathogens isolated from the CF airway, respectively. Our aim was to determine the effect of different colonisation profiles of AF and PA on the clinical status of patients with CF. Methods A retrospective analysis of data from the Cystic Fibrosis Registry of Ireland from 2013 was performed to determine the effect of intermittent and persistent colonisation with AF or PA or co-colonisation with both microorganisms on clinical outcome measures in patients with CF. Key outcomes measured included forced expiratory volume in one second (FEV1), number of hospitalisations, respiratory exacerbations and antimicrobials prescribed, and complications of CF, including CF related diabetes (CFRD) and allergic bronchopulmonary aspergillosis (ABPA). Results The prevalence of AF and PA colonisation were 11% (5% persistent, 6% intermittent) and 31% (19% persistent, 12% intermittent) in the Irish CF population, respectively. Co-colonisation with both pathogens was associated with a 13.8% reduction in FEV1 (p = 0.016), higher levels of exacerbations (p = 0.042), hospitalisations (p = 0.023) and antimicrobial usage (p = 0.014) compared to non-colonised patients and these clinical outcomes were comparable to those persistently colonised with PA. Intermittent and persistent AF colonisation were not associated with poorer clinical outcomes or ABPA. Patients with persistent PA had a higher prevalence of CFRD diagnosis (p = 0.012). Conclusions CF patients co-colonised with AF and PA had poor clinical outcomes comparable to patients persistently colonised with PA, emphasising the clinical significance of co-colonisation with these microorganisms. Electronic supplementary material The online version of this article (doi:10.1186/s12890-017-0416-4) contains supplementary material, which is available to authorized users.

The aim of the study was to evaluate the impact of manual cleaning and manual cleaning followed by Ultraviolet-C disinfection on the colony forming units of bacteria retrievable from equipment and surfaces within clinic rooms following a CF outpatient encounter. While UV disinfection has proven to be effective within general healthcare settings, it has not been evaluated in a CF centre. Microbiological sampling was performed following outpatient encounters involving 11 adult patients with CF and chronic infection with P.aeruginosa, MRSA or E. coli ESBL. The results of this study suggest that manual cleaning followed by UV-C disinfection is more effective than manual cleaning alone at reducing environmental contamination within a CF clinic and that UV-C isinfection is likely to reduce the risk of fomite transmission in the CF outpatient setting.

BACKGROUND Interactive health literacy (HL) skills enable individuals to participate more fully in healthcare activities and play a role in improving their outcomes. We examine the associations between HL and cystic fibrosis (CF) outcomes and compare HL in a sample from both the Irish CF and general populations. METHODS A total of251 CF Registry participants aged 13-30 years completed the HLS-EU-Q16 survey and a disease-specific instrument for measuring quality of life (QoL) in CF. Health outcome and healthcare resource utilization (HCRU) data were sourced from the registry. CF patient outcomes were examined using generalized linear models (GLMs) with interactive HL categorization included as a factor. General population interactive HL data are from the 2011 European HL Survey (HLS-EU). Interactive HL in 180 age-sex matched CF and general population individuals was examined using a GLM with study population, sex and educational level included as factors and age as a covariate. RESULTS Sufficient interactive HL (total sum score ≥13) was self-reported by 81.7% of individuals with CF. Sufficient HL was associated with fewer outpatient visits [7.02(SD = 6.7: 7.4) vs. 8.74(SD = 7.9: 9.6), P < 0.001], days hospitalized [10.25(SD = 9.8: 10.7) vs. 12.8(SD = 11.8: 13.9), P < 0.001], days on intravenous antibiotics [15.3(SD = 14.7: 15.8) vs. 19.7(18.5: 21.1), P < 0.001], days on oral antibiotics [27.4(SD = 26.7: 28.1) vs. 48.48(38.7: 42.4), P < 0.001] and better QoL [77.1(SD = 75.4-78.9) vs. 64.6(60.8-68.3), P < 0.001]. Mean HL scores in CF and general populations were sufficient, although higher among individuals with CF (14.3 vs. 13.1, P < 0.01). CONCLUSION CF adolescents and young adults with sufficient levels of HL to obtain, understand, appraise and apply health information have better health-related outcomes.

Background: Real-world, post-approval studies contribute significantly to the evidence surrounding the impact of new treatments, including CFTR modulators, but can be complex undertakings. Elexacaftor-tezacaftor-ivacaftor (ETI) was approved by EMA sooner than expected in August 2020 during a global pandemic. Method: RECOVER, a multi-centre, post-approval study examining the impact of ETI, and conducted in 8 clinical sites in Ireland and the UK over 2 years, examines important outcomes in children and adults prescribed ETI. The study will be conducted in 2 phases in line with ETI approval: 12+ and 6–11. In addition to routine data collected as part of normal care, key RECOVER endpoints include lung clearance index (LCI), spirometry-controlled CT, treatment adherence, GI symptoms, inflammation, liver disease markers, nasal inflammation and nitric oxide metabolism. Results: To date, 96 participants (56% female) out of a target of 137 in people with CFaged 12 and above, have been recruited (predominantly 12–18yrs to date). Recruitment and sample collection has been impacted by the effect of COVID-19 on CF care and CF centre attendance. Key challenges have included: Sputum collection (risks of induction and non-sputum producing participants) and coordination of study activities with limited clinic attendance. Despite this, key baseline data, prior to commencing treatment, has been successfully collected on the majority of participants to date. For subjects recruited to date, 56% have F508del/F508del and 44% F508del/minimum function mutations, mean age is 16.1 years, mean FEV1 83.6% (23–111%), mean LCI 12.2 (6.9–24.3). Recruitment and data collection is ongoing. Conclusion: Despite the impact of accelerated approval and COVID-19, we have been able to proceed with study initiation, recruitment and sample collection. Data from RECOVER and other international post-approval studies is likely to add significantly to our understanding of the impact of ETI on people with CF.

Cystic fibrosis (CF) registries work by bringing patient data together from specialist CF centres and accumulating data on a relatively rare condition over patients' lifetimes. In this review, we examine the origin of national CF registries, the use of registries for monitoring the health of the population, the power of conducting longitudinal analysis of registry data, knowledge gleaned from changing demographics, the evolving area of international comparisons of registries, strategies for sustaining registries and CF registries of the future. Examples of research undertaken using registry data and routinely reported CF registry statistics from across the world are provided. The application, utility and challenges facing CF registries in the future are discussed.

Understanding the determinants of cost of cystic fibrosis (CF) care and health outcomes may be useful for financial planning for the delivery of CF services. Registries contain information otherwise unavailable to healthcare activity/cost monitoring systems. We estimated the direct medical cost of CF care using registry data and examined how cost was affected by patient characteristics and CF gene (CF Transmembrane Conductance Regulator [CFTR]) mutation. Healthcare resource utilisation data (2008–2012) were obtained for CF patients enrolled with the Irish CF Registry by 2013 from linked registry and national hospitalisation database records. Mean annual hospitalisation and medication per-patient costs were estimated by demographic profile, CFTR mutation, clinical status, and CF co-morbidity, and were presented in 2014 euro values. A mixed-effects regression model was used to examine the effect of demographic, CFTR mutation, and clinical outcomes on the log10 cost of direct medical CF care. Using 4261 observations from 1100 patients, we found that the median annual total cost per patient increased over the period 2008–2012 from €12,659 to €16,852, inpatient bed-day cost increased from €14,026 to €17,332, and medication cost increased from €5863 to €12,467. Homozygous F508-CFTR mutation (class II) cost was highest and milder mutation (class IV/V) cost was 49% lower. Baseline estimated cost in 2008 for a hypothetical underweight, homozygous F508del-CFTR 6-year-old female without chronic Pseudomonas aeruginosa/Staphylococcus aureus, CF-related diabetes (CFRD) or methicillin-resistant S. aureus (MRSA), and with a poor percent predicted forced expiratory volume in 1 s (ppFEV1) was €10,113, and was €21,082 in a 25-year-old with the same hypothetical profile. Chronic P. aeruginosa infection increased baseline cost by 39%, CF co-morbidity diabetes by 18%, and frequency of pulmonary exacerbation by 15%. Underweight, declining ppFEV1, chronic S. aureus colonisation, and time also influenced cost. CFTR mutation is an important factor influencing the cost of CF care. Costs differ among cohorts of CF patients eligible to access new and emerging CFTR repair therapies. These findings support the evaluation of outcome-associated cost in CFTR mutation-specific CF patient groups.

Background The current lifetable approach to survival estimation is favoured by CF registries. Recognising the limitation of this approach, we examined the utility of a parametric survival model to project birth cohort survival estimates beyond the follow-up period, where short duration of follow-up meant median survival estimates were indeterminable. Methods Parametric models were fitted to observed survivorship data from the US CF Foundation (CFF) Patient Registry 1980–1994 birth cohort. Model-predicted median survival was estimated. The best fitting model was applied to a Cystic Fibrosis Registry of Ireland dataset to allow an evaluation of the model's ability to estimate predicted median survival. This involved a comparison of birth cohort lifetable predicted and observed (Kaplan–Meier) median survival estimates. Results A Weibull model with main effects of gender and birth cohort was developed using a US CFF dataset (n=13 115) for which median survival was not directly estimable. Birth cohort lifetable predicted median survival for male and female patients born between 1985 and 1994 and surviving their first birthday was 50.9 and 42.4 years respectively. To evaluate the accuracy of a Weibull model in predicting median survival, a model was developed for the 1980–1984 Cystic Fibrosis Registry of Ireland birth cohort (n=243), which had an observed (Kaplan–Meier) median survival of 27.7 years. Model-predicted median survival estimates were calculated using data censored at different follow-up periods. The estimates converged to the true value as length of follow-up increased. Conclusions Accurate prognostic information that is clinically critical for care of patients affected by rare, life-limiting disorders can be provided by parametric survival models. Problems associated with short duration of follow-up for recent birth cohorts can be overcome using this approach, providing better opportunities to monitor survival and plan services locally.

Background Comparing international estimates of survival can be a useful way of highlighting differences in life expectancy between cystic fibrosis (CF) populations. In this study, we compared survival in two CF populations. Methods The current lifetable method takes age-specific mortality rates observed in a given year and applies them to a hypothetical population assuming those rates will remain the same in the future. This was used to compare median predicted survival in the United States (US) and the Republic of Ireland (RoI) (1986–2008). Median age at death among decedents was also examined. Results In both countries, median age at death was lower than median predicted survival. Successive increases in annual median predicted survival were not observed; rather an overall improvement was discerned over time. In the RoI, where absolute numbers of deaths were small, year-on-year fluctuations in age-specific mortality rates resulted in wide-ranging annual median predicted survival estimates. Conclusion Median age at death is not a good measure of CF survival. Though median predicted survival improved in each country over the study period, between-country comparison at a given time point may be misleading for rare disorders like CF. Longitudinal outcomes must be examined.

A study was performed to determine the utility of the 8-point recognition of stroke in the emergency room (ROSIER) instrument as a diagnostic tool in a large Irish emergency department.Fifty patients had ROSIER scores completed by doctors. Diagnoses achieved using the score were compared with discharge diagnoses. Relations between ROSIER score, impairment and length of stay were assessed.Forty-seven patients (94%) had a ROSIER Scoreor =1 indicating a stroke, 44 (94%) of these had stroke confirmed on investigation. Two patients with stroke were missed and three were wrongly identified. Positive predictive value for the ROSIER was 94%. ROSIER correlated with Scandinavian neurological stroke score. (r = -0.414, p = 0.003) and was associated with increasing length of stay in survivors (p = 0.16, one-way ANOVA. F = 3.116, df = 6).The ROSIER is a suitable and useful adjunct in the assessment of stroke patients in Ireland.

Objective Understanding the cost of CF care is important for evaluating cost effectiveness of new interventions and securing funding for patient services. We describe direct medical costs using a micro-costing approach, and examine clinical determinants associated with greater cost. Methods Resource use data for 2011 was sourced from Irish CF registry and hospitalisation datasets. Maintenance antibiotics, mucolytics, bronchodilators, pancreatic enzyme and hospitalisations costs were provided by the Irish healthcare service and NCPE Ireland. Costs were estimated for patients on an individual basis. Covariates associated with higher cost were examined using log-linear regression in children aged 6–17 years and in adults. Results Direct cost of care for 841 patients was an estimated €18 million. Annual average cost was €16,000 per child and €28,000 per adult. Patients with mild, moderate and severe disease had an annual average cost of €15,000, €31,000 and €53,000 respectively. Higher cost was associated with chronic P aeruginosa, pancreatic enzyme use and no. of respiratory exacerbations in both adults and children. Additionally, significant covariates in children included lower BMI Z-score, chronic S aureus and a lower socio-economic deprivation score, and in adults, lower FEV1%. Conclusions In deriving direct care costs for CF patients, we have provided evidence to confirm that these are substantial. The addition of indirect costs would likely increase the overall costs significantly. These findings underscore the need for an approach that ensures appropriate planning and resourcing of CF services into the future. Funding support from Vertex Pharmaceuticals, Inc.

Objective Type 2 diabetes has a long pre clinical asymptomatic phase. Early detection may delay or arrest disease progression. The Diabetes Mellitus and Vascular health initiative (DMVhi) was initiated as a prospective longitudinal cohort study on the prevalence of undiagnosed Type 2 diabetes and prediabetes, diabetes risk and cardiovascular risk in a cohort of Irish adults aged 45-75 years. Research Design and Methods Members of the largest Irish private health insurance provider aged 45 to 75 years were invited to participate in the study. Exclusion criteria: already diagnosed with diabetes or taking oral hypoglycaemic agents. Participants completed a detailed medical questionnaire, had weight, height, waist and hip circumference and blood pressure measured. Fasting blood samples were taken for fasting plasma glucose (FPG). Those with FPG in the impaired fasting glucose (IFG) range had a 75gm oral glucose tolerance test performed. Results 122,531 subjects were invited to participate. 29,144 (24%) completed the study. The prevalence of undiagnosed diabetes was 1.8%, of impaired fasting glucose (IFG) was 7.1% and of impaired glucose tolerance (IGT) was 2.9%. Dysglycaemia increased among those aged 45-54, 55-64 and 65-75 years in both males (10.6%, 18.5%, 21.7% respectively) and females (4.3%, 8.6%, 10.9% respectively). Undiagnosed T2D, IFG and IGT were all associated with gender, age, blood pressure, BMI, abdominal obesity, family history of diabetes and triglyceride levels. Using FPG as initial screening may underestimate the prevalence of T2D in the study population. Conclusions This study is the largest screening study for diabetes and prediabetes in the Irish population. Follow up of this cohort will provide data on progression to diabetes and on cardiovascular outcomes.