Safety and efficacy of the cystic fibrosis transmembrane conductance regulator potentiator icenticaftor (QBW251).

Background: This is the first-in-human study of icenticaftor, an oral potentiator of the cystic fibrosis (CF) transmembrane conductance regulator (CFTR) channel. Restoration of CFTR activity has shown significant clinical benefits, but more studies are needed to address all CFTR mutations.

Methods: Safety, pharmacodynamics/pharmacokinetics of icenticaftor were evaluated in a randomized, double-blind, placebo-controlled study in healthy volunteers.

Osteoclastogenesis and sphingosine-1-phosphate secretion from human osteoclast precursor monocytes are modulated by the cystic fibrosis transmembrane conductance regulator.

Osteopenia and increased fracture rates are well-recognized in patients with cystic fibrosis (CF) disease. In CF pathology, F508del is the most common CFTR mutation, with more than 85% of patients carrying it on at least one allele. The underlying molecular defect in CFTR caused by the F508del-CFTR mutation in osteoclastogenesis, i.

Effectiveness of a plasma treatment device on microbial air quality in a hospital ward, monitored by culture.

This study analysed the effectiveness of plasma treatment on airborne bacteria and surface counts during a 14-day intervention within a four-bedded bay in an adult respiratory ward at Cork University Hospital, Ireland. One-hundred-litre air samples were collected twice daily every weekday for 4 weeks, with settle plates and surface swabs. The plasma treatment did not have an effect on airborne bacteria and fungi that was detectable by culture.

The global impact of SARS-CoV-2 in 181 people with cystic fibrosis.

With the growing SARS-CoV-2 pandemic, we need to better understand its impact in specific patient groups like those with Cystic Fibrosis (CF). We report on 181 people with CF (32 post-transplant) from 19 countries diagnosed with SARS-CoV-2 prior to 13 June 2020. Infection with SARS-CoV-2 appears to exhibit a similar spectrum of outcomes to that seen in the general population, with 11 people admitted to intensive care (7 post-transplant), and 7 deaths (3 post-transplant).

The virtual CF clinic: Implications for sputum microbiology.

The COVID19 pandemic has shifted the paradigm of how outpatient clinics are delivered within CF care, resulting in a significant reduction of patient visits to CF centres. One consequence of this has been a reduction in the number of sputa/cough swabs that patients submit for routine analysis. This report examines why it is important to maintain optimal sputum microbiology and explores (i).

New strategies of physical activity assessment in cystic fibrosis: a pilot study.

Background: Regular physical activity (PA) is a valued part of cystic fibrosis (CF) care. Although the accelerometer, SenseWear Armband (SWA), accurately measures habitual PA in CF, it is mostly used for research purposes. For the first time, we analyzed different methods of measuring PA in daily life by the use of smartphones and other electronic devices such as smartwatch and Fitbit.

Cohort study of sleep quality in adult patients with acute pulmonary exacerbations of cystic fibrosis.

Background: The impact of an acute pulmonary exacerbation of cystic fibrosis (CF) on sleep quality has not been established. Patients have greater burden of symptoms, higher intensity of therapy and are often admitted to hospital outside of their usual sleeping environment.

Aims: To evaluate the prevalence of, and factors associated with, poor sleep quality in adult patients admitted to hospital with an acute exacerbation of CF lung disease.

Pulmonary Exacerbations in Adults With Cystic Fibrosis: A Grown-up Issue in a Changing Cystic Fibrosis Landscape.

Pulmonary exacerbations (PExs) are significant life events in people with cystic fibrosis (CF), associated with declining lung function, reduced quality of life, hospitalizations, and decreased survival. The adult CF population is increasing worldwide, with many patients surviving prolonged periods with severe multimorbid disease. In many countries, the number of adults with CF exceeds the number of children, and PExs are particularly burdensome for adults as they tend to require longer courses and more IV treatment than children.

Monitoring early stage lung disease in cystic fibrosis.

Purpose Of Review: Early stage lung disease has long been synonymous with infancy and childhood. As diagnosis happens earlier and conventional management improves, we are seeing larger proportions of people with cystic fibrosis (CF) in adolescence and even adulthood with well preserved lung health. The availability of highly effective cystic fibrosis transmembrane conductance regulator modulator drugs for a large proportion of the CF population will impact even further.

Noninvasive Ventilation in Cystic Fibrosis: Clinical Indications and Outcomes in a Large UK Adult Cystic Fibrosis Center.

Background: Noninvasive ventilation (NIV) is routinely used to treat patients with cystic fibrosis and respiratory failure. However, evidence on its use is limited, with no data on its role in disease progression and outcomes. The aim of this study was to assess the indications of NIV use and to describe the outcomes associated with NIV in adults with cystic fibrosis in a large adult tertiary center.

Vitamin E supplementation in people with cystic fibrosis.

Background: People with cystic fibrosis are at an increased risk of fat-soluble vitamin deficiency, including vitamin E. Vitamin E deficiency can cause a host of conditions such as haemolytic anaemia, cerebellar ataxia and cognitive difficulties. Vitamin E supplementation is widely recommended for people with cystic fibrosis and aims to ameliorate this deficiency.

Concerns regarding the safety of azithromycin in pregnancy - relevance for women with cystic fibrosis.

Chronic oral azithromycin therapy improves clinical outcomes in people with cystic fibrosis (CF), and is recommended for treatment of CF lung disease. Azithromycin is categorized as pregnancy class B. The data for risk of congenital malformations associated with use of azithromycin during pregnancy ranges from no risk to a small increased risk.

Delafloxacin--A novel fluoroquinolone for the treatment of ciprofloxacin-resistant Pseudomonas aeruginosa in patients with cystic fibrosis.

Introduction: Fluoroquinolone antibiotics, namely ciprofloxacin and levofloxacin, play an important role in treating infection in cystic fibrosis (CF) and ciprofloxacin remains the last widely used and orally available antipseudomonal agent. Recently, a new fluoroquinolone, delafloxacin, has been approved by the FDA for the indication of Acute Bacterial Skin and Skin Structure Infections (ABSSSI). This antibiotic is a novel dual-targeting anionic fluoroquinolone and differs from previous agents in its class, as it lacks a protonatable substituent.

COVID-19 in a complex obstetric patient with cystic fibrosis.

We report the first case of COVID-19 in a pregnant patient with cystic fibrosis. We describe the diagnosis, clinical course and management of the patient and their family with regards to clinical, social and infection control measures around delivery. This case highlights the importance of the cooperation of multidisciplinary teams to achieve good clinical outcomes in complex patients with COVID-19.

Mindfulness moderates the relationship between emotional eating and body mass index in a sample of people with cystic fibrosis.

Purpose: Self-regulation in eating is significant for enhancing life expectancy of people with cystic fibrosis (CF), but research with this population is scarce.

Methods: In a cross-sectional study, adults with CF completed a number of psychometric scales exploring typical eating behaviours that may increase calorific intake including motivations to eat palatable foods and scales that may be associated with decreased calorific intake: mindfulness, mindful eating and self-compassion.

Results: Findings suggested that motivations to eat palatable foods and eating behaviours correlate with higher BMI, while mindfulness, mindful eating and self-compassion did not reach significance.

Nebuliser hygiene in cystic fibrosis: evidence-based recommendations.

Unlabelled: Nebulised therapies are extensively used in the daily therapeutic management of cystic fibrosis both for mucociliary clearance and for the management of chronic infections. Extensive developments have been made in relation to nebulised drug delivery mechanisms and drug formulations, and guidelines have been prepared that have addressed the appropriate use of such therapies. However, due to these developments, a plethora of nebuliser devices and drug chambers exist, and frequently, the limited guidance provided in relation to nebuliser hygiene is to follow manufacturers' instructions.

The outcome of pregnancy in women with cystic fibrosis: a UK population-based descriptive study.

Objective: To estimate the incidence of cystic fibrosis in pregnancy and to explore obstetric and neonatal outcomes.

Design: A population-based descriptive study using the methodology of the UK Obstetric Surveillance System (UKOSS).

Setting: All consultant-led maternity units in the UK.

Duplex real-time PCR assay for the simultaneous detection of and spp.

Several members of the Gram-negative environmental bacterial genus are associated with serious infections, with being the most common. Despite their pathogenic potential, little is understood about these intrinsically drug-resistant bacteria and their role in disease, leading to suboptimal diagnosis and management. Here, we performed comparative genomics for 158 spp.