Heterogeneity in Reported Side Effects Following Initiation of Elexacaftor-Tezacaftor-Ivacaftor: Experiences at a Quaternary CF Care Center.

Background: The benefits of Elexecaftor-Tezacaftor-Ivacaftor (ETI) therapy on the health and wellbeing of people with CF (pwCF) are well documented. Since approval, however, a growing number of potential side effects have emerged in reports from clinical practice. With current understanding of ETI tolerability limited to data from clinical trials, the prevalence of side effects and their impact on care decision making remains poorly categorized.

Continuous glucose monitoring and advanced glycation endproducts for prediction of clinical outcomes and development of cystic fibrosis-related diabetes in adults with CF.

Introductions: Cystic fibrosis-related diabetes (CFRD) is associated with pulmonary decline, compromised nutritional status, and earlier mortality. Onset is often insidious, so screening for early detection of glycemic abnormalities is important. Continuous glucose monitoring (CGM) has been validated in people with CF and has been shown to detect early glycemic variability otherwise missed on 2-hour oral glucose tolerance testing (OGTT).

Supplemental Oxygen Alters the Airway Microbiome in Cystic Fibrosis.

Features of the airway microbiome in persons with cystic fibrosis (pwCF) are correlated with disease progression. Microbes have traditionally been classified for their ability to tolerate oxygen. It is unknown whether supplemental oxygen, a common medical intervention, affects the airway microbiome of pwCF.

Continuous Glucose Monitoring and HbA1c in Cystic Fibrosis: Clinical Correlations and Implications for CFRD Diagnosis.

Context: The clinical utility and implications of continuous glucose monitoring (CGM) in cystic fibrosis (CF) are unclear.

Objective: We examined the correlation between CGM measures and clinical outcomes in adults with CF, investigated the relationship between hemoglobin A1c (HbA1c) and CGM-derived average glucose (AG), and explored CGM measures that distinguish cystic fibrosis-related diabetes (CFRD) from normal and abnormal glucose tolerance.

Methods: This prospective observational study included 77 adults with CF who had CGM and HbA1c measured at 2 to 3 time points 3 months apart.

The Effects of Ivacaftor on Bone Density and Microarchitecture in Children and Adults with Cystic Fibrosis.

Context: Cystic fibrosis (CF) transmembrane conductance (CFTR) dysfunction may play a role in CF-related bone disease (CFBD). Ivacaftor is a CFTR potentiator effective in improving pulmonary and nutritional outcomes in patients with the G551D-CFTR mutation. The effects of ivacaftor on bone health are unknown.

Effects of a primary palliative care intervention on quality of life and mental health in cystic fibrosis.

Background: Despite the significant impact of chronic symptoms on quality of life with cystic fibrosis (CF), the role of palliative care in management of this disease is not well defined. The coping, goal assessment, and relief from evolving CF symptoms (CF-CARES) model is a primary palliative care intervention designed to provide chronic symptom management at all stages of the disease. The goal of this pilot study was to estimate the effectiveness of the CF-CARES intervention on improving chronic symptoms and quality of life for people living with CF.

Celiac Disease in Patients with Cystic Fibrosis-Related Bone Disease.

Both cystic fibrosis (CF) and celiac disease can cause low bone mineral density (BMD) and fractures. Celiac disease may occur at a higher frequency in patients with CF than the general population, and symptoms of these conditions may overlap. We report on two patients presenting with CF-related bone disease in the past year who were subsequently found to have concurrent celiac disease.

Impact of CFTR Modulation on Intestinal pH, Motility, and Clinical Outcomes in Patients With Cystic Fibrosis and the G551D Mutation.

Objectives: A defect in bicarbonate secretion contributes to the pathophysiology of gastrointestinal complications in patients with cystic fibrosis (CF). We measured gastrointestinal pH, clinical outcomes, and intestinal transit profiles in patients with the G551D mutation before and after treatment with ivacaftor, a CF transmembrane regulator channel (CFTR) potentiator.

Methods: Observational studies of ivacaftor effectiveness were conducted in the United States and Canada.

Young adults with cystic fibrosis have altered trabecular microstructure by ITS-based morphological analysis.

Unlabelled: Young adults with cystic fibrosis have compromised plate-like trabecular microstructure, altered axial alignment of trabeculae, and reduced connectivity between trabeculae that may contribute to the reduced bone strength and increased fracture risk observed in this patient population.

Introduction: The risk of fracture is increased in patients with cystic fibrosis (CF). Individual trabecular segmentation (ITS)-based morphological analysis of high-resolution peripheral quantitative computed tomography (HR-pQCT) images segments trabecular bone into individual plates and rods of different alignment and connectivity, which are important determinants of trabecular bone strength.

Development and evaluation of a palliative care curriculum for cystic fibrosis healthcare providers.

Background: Primary palliative care refers to basic skills that all healthcare providers can employ to improve quality of life for patients at any stage of disease. Training in these core skills is not commonly provided to clinicians caring for cystic fibrosis (CF) patients. The objective of this study was to assess change in comfort with core skills among care team members after participation in CF-specific palliative care training focused on management of burdensome symptoms and difficult conversations.

Trends in bone mineral density in young adults with cystic fibrosis over a 15 year period.

Background: Improvements in clinical care have led to increased life expectancy in patients with cystic fibrosis (CF) over the past several decades. Whether these improvements have had significant effects on bone health in patients with CF is unclear.

Methods: This is a cross-sectional study comparing clinical characteristics and bone mineral density (BMD) measured by dual energy X-ray absorptiometry (DXA) in adults with CF evaluated in 1995-1999 to age-, race-, and gender-matched patients with CF evaluated in 2011-2013 at the same center on calibrated DXA machines.

Compromised bone microarchitecture and estimated bone strength in young adults with cystic fibrosis.

Context: Young adults with cystic fibrosis (CF) are at risk for low bone density and fractures, but the underlying alterations in bone microarchitecture that may contribute to their increased fracture risk are currently unknown.

Objective: The main goal of this study was to use high-resolution peripheral quantitative computed tomography (HR-pQCT) to characterize the bone microarchitecture, volumetric bone mineral density (vBMD), and estimated strength of the radius and tibia in young adults with CF compared with healthy volunteers.

Design And Setting: This was a cross-sectional study at an outpatient clinical research center within a tertiary academic medical center.

Generation of multipotent lung and airway progenitors from mouse ESCs and patient-specific cystic fibrosis iPSCs.

Deriving lung progenitors from patient-specific pluripotent cells is a key step in producing differentiated lung epithelium for disease modeling and transplantation. By mimicking the signaling events that occur during mouse lung development, we generated murine lung progenitors in a series of discrete steps. Definitive endoderm derived from mouse embryonic stem cells (ESCs) was converted into foregut endoderm, then into replicating Nkx2.

An official American Thoracic Society Policy statement: managing conflict of interest in professional societies.

Background: Competing interests occur frequently in health care. This results in the potential for conflict of interest (COI). COI can lead to biased generation or assessment of evidence and misinform healthcare decision makers.

Ethical issues in the chronically critically ill patient.

The chronically critically ill are a challenging population of patients. Their mortality rate is high and expected functional status is low. The physician responsible for the care of these patients often is conflicted because the gains experienced by these patients may be small or absent whereas the pressure by society to use medical resources better is great.

Long-term care, rehabilitation, and legal and ethical considerations in the management of neuromuscular disease with respiratory dysfunction.

Neuromuscular disease, while for the most part progressive, allows for ample time to discuss options for long-term care. Frank discussions among clinicians, the patient, and family will allow for an ethical decision process. With the clinician's awareness of the inherent difficulties in judging life satisfaction, an early discussion of first noninvasive measures, then tracheostomy with positive pressure ventilation, will allow for a rational, moral, and continuing treatment plan.

Lung function, breathing pattern, and gas exchange in interstitial lung disease.

Background: The aim of this study was to determine the relation between the severity of abnormalities in ventilatory function tests and tidal breathing pattern and gas exchange indices in interstitial lung disease.

Methods: Pulmonary function, ventilation, carbon dioxide production, oxygen consumption, arterial blood gas tensions, and pH were measured during resting steady state conditions in 60 patients with proved interstitial lung disease. Patients were categorised by forced vital capacity (FVC) (percentage of predicted values) as having a mild, moderate, or severe restrictive defect with means (SD) of 71% (4%), 57% (4%), and 41% (7%) of predicted values, respectively.

Chronic eosinophilic pneumonia. A report of 19 cases and a review of the literature.

Clinical, roentgenographic and pathologic findings in patients with chronic eosinophilic pneumonia, including 19 additional cases, have been reviewed and summarized. Most patients present with subacute respiratory and constitutional symptoms and have failed to respond to therapy for presumptive pneumonia. A previous history of atopy, most often asthma, will be obtained in one-half.

Congenital bronchial atresia. A report of 4 cases and a review of the literature.

The clinical, radiographic, and pathologic findings in 82 patients with congenital bronchial atresia (CBA) have been reviewed, and we have discussed 4 additional cases. Most patients are asymptomatic and come to attention because of abnormal radiographic findings of a round or lobulated perihilar, solid, or cystic mass--the mucoid impaction sign. Typically, the region distal to the mass is hyperinflated.

Intravascular bronchioloalveolar tumor (IV-BAT).

Intravascular bronchioloalveolar tumor (IV-BAT) is a recently described, histologically distinct, malignant, primary lung neoplasm. Originally it was believed to arise from type II alveolar cells which invade blood vessels within the lung, and hence its name. More recent ultrastructural studies suggest an origin from mesenchymal cells, and therefore the tumor may be more appropriately considered a sarcoma.

Gallium 67 citrate scanning and serum angiotensin converting enzyme levels in sarcoidosis.

Gallium 67 citrate scans and serum angiotensin converting enzyme (ACE) levels were obtained in 54 patients with sarcoidosis and analyzed in relation to clinical manifestations. 67Ga scans were abnormal in 97% of patients with clinically active disease (n = 30) and in 71% of patients with inactive disease (n = 24). Serum ACE levels were abnormally high (2 standard deviations above the control mean) in 73% of patients with clinically active disease and in 54% of patients with inactive disease.

Angiotensin-converting enzyme in serum and in bronchoalveolar lavage in sarcoidosis.

Angiotensin-converting enzyme (ACE) determinations were made in serum and in bronchoalveolar lavage fluid in 20 controls and in 28 patients with sarcoidosis. Serum ACE was significantly higher in patients with active sarcoidosis (54.3 +/- 19.