Standards for the care of people with cystic fibrosis (CF); Planning for a longer life.

This is the final of four papers updating standards for the care of people with CF. That this paper "Planning a longer life" was considered necessary, highlights how much CF care has progressed over the past decade. Several factors underpin this progress, notably increased numbers of people with CF with access to CFTR modulator therapy.

Standards for the care of people with cystic fibrosis; establishing and maintaining health.

This is the second in a series of four papers updating the European Cystic Fibrosis Society (ECFS) standards for the care of people with CF. This paper focuses on establishing and maintaining health. The guidance is produced using an evidence-based framework and with wide stakeholder engagement, including people from the CF community.

Gaining an Understanding of Pneumocystosis in Wales.

pneumonia (PcP) is a serious complication of many significant immunocompromising conditions. Prior incidence estimates in Wales are based on PcP's presentation in the HIV and transplant populations. The objectives were to describe the incidence of PcP in Wales using laboratory reporting measures and assess the impact of underlying immunosuppression cause on mortality.

Weight a minute: Exploring the effect on weight and body composition after the initiation of elexacaftor/tezacaftor/ivacaftor in adults with CF.

Background: Life expectancy for people with CF (PwCF) continues to increase. However, a trend of overweight and obesity is emerging along with concern of developing comorbitities. Body composition (BC) is associated with several health indices.

Cefiderocol to manage chronic, multi-drug-resistant complex infection in a patient with cystic fibrosis: a case report.

In cystic fibrosis (CF) patients, Gram-negative complex (Bcc) infections are associated with recurrent pulmonary exacerbations. Bcc organisms are innately resistant to many antibiotics, and infection with is a contraindication to lung transplantation. We report a CF patient with severe lung disease, colonized with Bcc, with a history of around nine exacerbations per year for over 10 years, for whom antibiotic regimens (including targeted and broad-spectrum antibiotics) had not cleared infection or extended the interval between exacerbations.

A pilot study investigating the effects of a manuka honey sinus rinse compared to a standard sinus rinse on sino-nasal outcome test scores in cystic fibrosis patients.

Background: People with cystic fibrosis (CF) are prone to bacterial respiratory infections; these are often antibiotic resistant, are difficult to treat, and impact on the quality of life and lung function. The upper respiratory tract can act as a reservoir for these pathogens, and as part of clinical care, sinus rinses are used to alleviate symptoms in the upper airway. We have developed a sinus rinse containing manuka honey, to identify whether it can help improve symptoms or reduce the bacterial load.

The Exeter Activity Unlimited statement on physical activity and exercise for cystic fibrosis: methodology and results of an international, multidisciplinary, evidence-driven expert consensus.

Background: The roles of physical activity (PA) and exercise within the management of cystic fibrosis (CF) are recognised by their inclusion in numerous standards of care and treatment guidelines. However, information is brief, and both PA and exercise as multi-faceted behaviours require extensive stakeholder input when developing and promoting such guidelines.

Method: On 30 June and 1 July 2021, 39 stakeholders from 11 countries, including researchers, healthcare professionals and patients participated in a virtual conference to agree an evidence-based and informed expert consensus about PA and exercise for people with CF.

British Thoracic Society survey of the career intentions of respiratory medicine specialty trainees in the UK.

There were respiratory consultant post vacancies in 82% of surveyed UK hospitals in 2021. Understanding respiratory trainees' career intentions is vital to plan and train a future respiratory workforce. In 2020, the British Thoracic Society surveyed trainee members (n=144) to assess career plans and perceived barriers and facilitators when applying for consultant posts.

The Presence of in the Respiratory Tract of Cystic Fibrosis Patients Accelerates Lung Function Decline: A Retrospective Review of Lung Function.

Exophiala dermatitidis is increasingly isolated from cystic fibrosis (CF) respiratory samples. The decision to treat is hampered by limited evidence demonstrating the clinical significance of isolating E. dermatitidis.

Efficacy and safety of elexacaftor plus tezacaftor plus ivacaftor versus tezacaftor plus ivacaftor in people with cystic fibrosis homozygous for F508del-CFTR: a 24-week, multicentre, randomised, double-blind, active-controlled, phase 3b trial.

Background: Elexacaftor plus tezacaftor plus ivacaftor is a triple-combination cystic fibrosis transmembrane conductance regulator (CFTR) modulator regimen shown to be generally safe and efficacious in people with cystic fibrosis aged 12 years or older with at least one F508del-CFTR allele. We aimed to assess the magnitude and durability of the clinical effects of this triple combination regimen in people with cystic fibrosis homozygous for the F508del-CFTR mutation.

Methods: We conducted a multicentre, randomised, double-blind, active-controlled, phase 3b trial of elexacaftor plus tezacaftor plus ivacaftor at 35 medical centres in Australia, Belgium, Germany, and the UK.

Inflammation biomarkers in sputum for clinical trials in cystic fibrosis: current understanding and gaps in knowledge.

Rationale: Sputum biomarkers hold promise as a direct measure of inflammation within the cystic fibrosis (CF) lung, but variability in study design and sampling methodology have limited their use. A full evaluation of the reliability, validity and clinical relevance of individual biomarkers is required to optimise their use within CF clinical research.

Objectives: A biomarker Special Interest Working Group was established within the European Cystic Fibrosis Society-Clinical Trials Network Standardisation Committee, to perform a review of the evidence regarding sputum biomarkers in CF.

Real-World Outcomes of Ivacaftor Treatment in People with Cystic Fibrosis: A Systematic Review.

Cystic fibrosis (CF) is a rare, progressive, multi-organ genetic disease. Ivacaftor, a small-molecule CF transmembrane conductance regulator modulator, was the first medication to treat the underlying cause of CF. Since its approval, real-world clinical experience on the use of ivacaftor has been documented in large registries and smaller studies.

Are We Missing the Opportunity to Measure Muscle Mass on Computed Tomography Thorax?

Hand grip strength (HGS) and fat free mass index (FFMI) are important indicators of skeletal muscle mass and correlate with prognosis in patients with respiratory diseases. It is also possible to estimate muscle mass by measuring muscle density and volume on cross sectional imaging. We reviewed all patients of the All Wales Cystic Fibrosis Centre who had a computed tomography thorax as part of routine clinical care between 2013 and 2017.

Association between HbA and the development of cystic fibrosis-related diabetes.

Aims: To examine HbA as a predictor of risk for future development of cystic fibrosis-related diabetes and to assess the association with the development of retinopathy in people with cystic fibrosis-related diabetes.

Methods: A 7-year retrospective longitudinal study was conducted in 50 adults with cystic fibrosis, comparing oral glucose tolerance test results with HbA values in predicting the development of cystic fibrosis-related diabetes. Retinal screening data were also compared with HbA measurements to assess microvascular outcome.

A treatment evaluator tool to monitor the real-world effectiveness of inhaled aztreonam lysine in cystic fibrosis.

Background: Studies are required that evaluate real-world outcomes of inhaled aztreonam lysine in patients with cystic fibrosis (CF).

Methods: Our treatment-evaluator tool assessed the effectiveness of inhaled aztreonam in routine practice in 117 CF patients across four time periods (6-12 (P2) and 0-6months (P1) pre-initiation, and 0-6 (T1) and 6-12months (T2) post-initiation). Outcomes were: changes in %-predicted forced expiratory volume in 1s (FEV), body-mass index (BMI), hospitalisation days and intravenous antibiotic usage.

Lifestyle treatments in cystic fibrosis: The NHS should pay.

With the NHS under increasing financial pressure and healthcare costs soaring year on year, it is perhaps not surprising that assessment agencies focus on cost-effectiveness analysis when assessing new therapies. Such an approach does not however, always take sufficient account of treatment burden, lifestyle and patient choice and therefore new equally effective but perhaps "easier to take" formulations and faster delivery systems for current therapies do not always take precedence in current treatment guidelines. In arguing that the NHS should pay for so-called lifestyle treatments in cystic fibrosis the counterintuitive nature of some of the current decision making is discussed and a more holistic approach to improve NHS efficiency is presented.

Hyperpharmacotherapy in ageing cystic fibrosis patients: The first report of an atypical hip fracture.

Cystic fibrosis (CF) is a common autosomal recessive disorder in Caucasian populations with respiratory, gastrointestinal and endocrine manifestations. Thanks to recent advances in medical therapies and infection control, life expectancy of a patient with CF has significantly increased from less than 5 years in the mid-1900s to almost 50 years nowadays. However, as CF patients are living longer, multimorbidity and Hyperpharmacotherapy are becoming more common.

Retinal screening of patients with cystic fibrosis-related diabetes in Wales -- a real eye opener.

Background: The National Screening Committee recommends annual retinal screening for all diabetic patients over the age of twelve years. Currently, patients on insulin therapy for Cystic Fibrosis-Related Diabetes Mellitus (CFRD) at the All Wales Adult Cystic Fibrosis Service, UK are invited for annual screening.

Methods: Age, sex, FEV(1)%, BMI, HbA1c, duration of diabetes, duration of insulin therapy, presence of retinopathy and attendance at retinal screening in 2012 were examined for patients on insulin therapy for CFRD.

Branch retinal vein occlusion in an asymptomatic adult with cystic fibrosis.

Purpose: This article aims to describe a case of asymptomatic branch retinal vein occlusion (BRVO) in a patient with cystic fibrosis (CF) and discuss the possible link between the two.

Case Report: A young adult (aged 35 years) with CF who presented for routine ocular examination was found to have a superior temporal BRVO in the left eye. Visual acuity was unaffected, measuring -0.