Characteristics of individuals with cystic fibrosis in the United States ineligible for ivacaftor and elexacaftor/tezacaftor/ivacaftor.

Background: We characterized people with cystic fibrosis (CF) ineligible by genotype (not age) for currently approved CFTR modulator therapy using data from the US CF Foundation Patient Registry (CFFPR).

Methods: We summarized clinical characteristics using CFFPR data from 2017 to 2022. Annual rate of change in percent predicted of forced expiratory volume in one second (ppFEV) was estimated using generalized estimating equations.

Complications and Practice Variation in the Use of Peripherally Inserted Central Venous Catheters in People With Cystic Fibrosis: The Prospective Study of Peripherally Inserted Venous Catheters in People With Cystic Fibrosis Study.

Background: Peripherally inserted central catheters (PICCs) are used commonly to administer antibiotics to people with cystic fibrosis (CF), but their use can be complicated by venous thrombosis and catheter occlusion.

Research Question: Which participant-, catheter-, and catheter management-level attributes are associated with increased risk of complications of PICCs among people with CF?

Study Design And Methods: This was a prospective observational study of adults and children with CF who received PICCs at 10 CF care centers in the United States. The primary end point was defined as occlusion of the catheter resulting in unplanned removal, symptomatic venous thrombosis in the extremity containing the catheter, or both.

Financial impacts of the COVID-19 pandemic on cystic fibrosis care: lessons for the future.

Background: Chronic care delivery models faced unprecedented financial pressures, with a reduction of in-person visits and adoption of telehealth during the COVID-19 pandemic. We sought to understand the reported financial impact of pandemic-related changes to the cystic fibrosis (CF) care model.

Methods: The U.

Impact of Socioeconomic Position on Access to the U.S. Lung Transplant Waiting List in a Matched Cystic Fibrosis Cohort.

Referrals for lung transplant and transplant rates in the United States are lower than in Canada for patients with advanced cystic fibrosis (CF) lung disease. Further study of factors limiting access are needed to optimize referral and transplant for this population. To determine the effect of socioeconomic position, while accounting for disease severity, on the likelihood of wait-listing for lung transplant in the United States.

Rapid lung function decline in adults with early-stage cystic fibrosis lung disease.

Rationale: The prevalence of adults living with cystic fibrosis (CF) who have early-stage lung disease is increasing.

Objectives: Describe the prevalence and evaluate spirometric risk factors associated with the subgroup of patients with early-stage lung disease and FEV decline of ≥5% predicted/year.

Methods: Retrospective cohort study of patients ≥18 years with FEV% predicted ≥80% included in the US CF Foundation Patient Registry from 2010-2013.

Effect of Including Important Clinical Variables on Accuracy of the Lung Allocation Score for Cystic Fibrosis and Chronic Obstructive Pulmonary Disease.

Clinical variables associated with shortened survival in patients with advanced-stage cystic fibrosis (CF) are not included in the lung allocation score (LAS). To identify variables associated with wait-list and post-transplant mortality for CF lung transplant candidates using a novel database and to analyze the impact of including new CF-specific variables in the LAS system. A deterministic matching algorithm identified patients from the Scientific Registry of Transplant Recipients and the Cystic Fibrosis Foundation Patient Registry.

Eradication of persistent methicillin-resistant Staphylococcus aureus infection in cystic fibrosis.

Background: The prevalence of methicillin-resistant Staphylococcus aureus (MRSA) in individuals with cystic fibrosis (CF) has increased significantly. While studies demonstrate that persistent MRSA infection in CF is associated with poor clinical outcomes, there are no randomized controlled studies informing management.

Methods: The Persistent MRSA Eradication Protocol was a double-blind, randomized, placebo-controlled study investigating a comprehensive 28-day treatment regimen with or without inhaled vancomycin for eradication of MRSA.

Cystic fibrosis patient registries: A valuable source for clinical research.

Cystic Fibrosis (CF) patient registries are valuable data sources for researchers studying the natural history, treatment paradigms, and long-term health outcomes of individuals with CF. In this review, we discuss the role of CF patient registries in facilitating comparative effectiveness research, particularly evaluating therapies and variation in health care delivery. We also discuss the limitations of registry-based research, particularly indication bias, as well as statistical methods that can be used to address these issues.

Standardized Treatment of Pulmonary Exacerbations (STOP) study: Physician treatment practices and outcomes for individuals with cystic fibrosis with pulmonary Exacerbations.

Background: Pulmonary Exacerbations (PEx) are associated with increased morbidity and mortality in individuals with CF. PEx management practices vary widely, and optimization through interventional trials could potentially improve outcomes. The object of this analysis was to evaluate current physician treatment practices and patient outcomes for PEx.

Preliminary comparison of normalized T1 and non-contrast perfusion MRI assessments of regional lung disease in cystic fibrosis patients.

Background: Previous studies have shown that Magnetic Resonance Imaging (MRI) techniques can be used to non-invasively assess lung disease in CF patients. In this study, we compare the sensitivity of normalized T1 (nT1) and non-contrast perfusion MRI techniques to detect regional lung disease in CF patients.

Materials And Methods: MRI data were obtained for eight adult CF patients without overt pulmonary exacerbation (FEV1=45-127%) and six healthy volunteers on a Siemens Espree 1.

Impact of lung allocation score on survival in cystic fibrosis lung transplant recipients.

Background: The lung allocation score (LAS) has changed organ allocation for lung transplantation in the United States. Previous investigations of transplant recipients reported an association between high LAS and an increased risk of death after lung transplantation. We hypothesize that a high LAS predicts survival in lung transplant recipients with cystic fibrosis (CF) in the United Network for Organ Sharing Scientific Registry of Transplant Recipients database.

Rate of Uptake of Ivacaftor Use after U.S. Food and Drug Administration Approval among Patients Enrolled in the U.S. Cystic Fibrosis Foundation Patient Registry.

Rationale: Chronic cystic fibrosis (CF) therapies have variable rates of prescribed use, and therapies are rarely prescribed to more than 80% of eligible patients. Ivacaftor was approved in the United States in January 2012 for patients ages 6 years and older with a G551D mutation in their CF gene.

Objectives: To examine the rate of uptake and patterns of documented ivacaftor use among U.

Utilization of antibiotics for methicillin-resistant Staphylococcus aureus infection in cystic fibrosis.

Objectives: The purpose of this study was to characterize the utilization of antibiotics for chronic methicillin-resistant Staphylococcus aureus (MRSA) infection in cystic fibrosis (CF) patients with acute pulmonary exacerbations (PEx).

Methods: An anonymous national cross-sectional survey of CF Foundation accredited care programs was performed using an electronic survey tool.

Results: Fifty-eight percent (152/261) CF Foundation accredited programs completed the survey.

Association between the introduction of a new cystic fibrosis inhaled antibiotic class and change in prevalence of patients receiving multiple inhaled antibiotic classes.

Background: In 2010, aztreonam for inhalation solution joined aminoglycosides and colistimethate as a new cystic fibrosis (CF) chronic inhaled antimicrobial therapy. We studied how the introduction of this new inhaled antibiotic class changed the management of US CF patients.

Methods: The use of inhaled aminoglycosides, colistimethate, and aztreonam among patients followed in the CF Foundation Patient Registry was analyzed by age group, lung disease stage, and microbiologic status both annually, and at individual visits between 2009 and 2012.

Antibiotic management of lung infections in cystic fibrosis. II. Nontuberculous mycobacteria, anaerobic bacteria, and fungi.

Airway infections are a key component of cystic fibrosis (CF) lung disease. Whereas the approach to common pathogens such as Pseudomonas aeruginosa is guided by a significant body of evidence, other infections often pose a considerable challenge to treating physicians. In Part I of this series on the antibiotic management of difficult lung infections, we discussed bacterial organisms including methicillin-resistant Staphylococcus aureus, gram-negative bacterial infections, and treatment of multiple bacterial pathogens.

Antibiotic management of lung infections in cystic fibrosis. I. The microbiome, methicillin-resistant Staphylococcus aureus, gram-negative bacteria, and multiple infections.

Despite significant advances in treatment strategies targeting the underlying defect in cystic fibrosis (CF), airway infection remains an important cause of lung disease. In this two-part series, we review recent evidence related to the complexity of CF airway infection, explore data suggesting the relevance of individual microbial species, and discuss current and future treatment options. In Part I, the evidence with respect to the spectrum of bacteria present in the CF airway, known as the lung microbiome is discussed.

Eradication strategy for persistent methicillin-resistant Staphylococcus aureus infection in individuals with cystic fibrosis--the PMEP trial: study protocol for a randomized controlled trial.

Background: The prevalence of methicillin-resistant Staphylococcus aureus (MRSA) respiratory infection in cystic fibrosis (CF) has increased dramatically over the last decade, and is now affecting approximately 25% of patients. Epidemiologic evidence suggests that persistent infection with MRSA results in an increased rate of decline in FEV1 and shortened survival. Currently, there are no conclusive studies demonstrating an effective and safe treatment protocol for persistent MRSA respiratory infection in CF.

Normalized T1 magnetic resonance imaging for assessment of regional lung function in adult cystic fibrosis patients--a cross-sectional study.

Background: Cystic fibrosis (CF) patients would benefit from a safe and effective tool to detect early-stage, regional lung disease to allow for early intervention. Magnetic Resonance Imaging (MRI) is a safe, non-invasive procedure capable of providing quantitative assessments of disease without ionizing radiation. We developed a rapid normalized T1 MRI technique to detect regional lung disease in early-stage CF patients.

Isoflurane and ketamine anesthesia have different effects on ventilatory pattern variability in rats.

We hypothesize that isoflurane and ketamine impact ventilatory pattern variability (VPV) differently. Adult Sprague-Dawley rats were recorded in a whole-body plethysmograph before, during and after deep anesthesia. VPV was quantified from 60-s epochs using a complementary set of analytic techniques that included constructing surrogate data sets that preserved the linear structure but disrupted nonlinear deterministic properties of the original data.

Update on methicillin-resistant Staphylococcus aureus in cystic fibrosis.

Purpose Of Review: Respiratory infection is a major contributor to morbidity and mortality in cystic fibrosis (CF). One infection the CF community is particularly concerned about is methicillin-resistant Staphylococcus aureus (MRSA). Worldwide, the prevalence of MRSA has been rising and the impact on clinical outcomes and optimal prevention and treatment strategies are unclear.

Higher PEEP in patients with acute lung injury: a systematic review and meta-analysis.

Background: Studies of ventilation strategies that included higher PEEP in patients with acute lung injury (ALI) or acute respiratory distress syndrome (ARDS) have yielded conflicting results.

Objective: To determine whether higher PEEP during volume-limited and pressure-limited ventilation is associated with 28-day mortality or barotrauma rates in patients with ALI/ARDS.

Methods: We searched MEDLINE, CENTRAL, EMBASE, CINAHL, Web of Science, and the bibliographies of retrieved papers to identify randomized controlled trials that compared higher and lower PEEP in adult patients with ALI/ARDS who were already receiving volume-limited or pressure-limited ventilation.