Clinical change 2 years from start of elexacaftor-tezacaftor-ivacaftor in severe cystic fibrosis.

Rationale: Limited published research is available on the impact of elexacaftor/tezacaftor/ivacaftor (ETI) beyond the initial few months postdrug initiation, especially for those who initiated therapy via individual investigational new drug application. The experiences of patients with cystic fibrosis (CF) experiencing severe lung disease were reviewed for significant improvements in clinical symptoms and quality of life.

Objectives: To examine clinical outcomes 2 years post-ETI in patients with CF and advanced lung disease.

Impact of guideline adherence and race on asthma control in children.

Background: Asthma control in African Americans (AA) is considered more difficult to achieve than in Caucasian Americans (CA). The aim of this study was to compare asthma control over time among AA and CA children whose asthma is managed per NAEPP (EPR-3) guidelines.

Methods: This was a one-year prospective study of children referred by their primary care physicians for better asthma care in a specialty asthma clinic.

Impact of type of health care provider on long term asthma control.

Objective: Asthma prevalence is high and adherence to asthma guidelines is still less than adequate. The main objective of this study was to determine if there were significant differences in outcome measures if asthma care was provided per guidelines either by physicians (pediatric pulmonologists) or specialty trained advance practice nurses (APNs).

Methods: This was a three-year, prospective cohort study of children referred by their primary care providers to a tertiary care center for better asthma control.

Comparison of Two Therapies on Asthma Control in Children.

Childhood asthma carries significant morbidity. Aim of the study was to compare efficacy of 2 commonly used therapies for asthma control in children with asthma. This was a 1-year, prospective cohort study at a tertiary care children's hospital.

Impact of asthma severity on long-term asthma control.

Asthma is a common childhood disease with significant morbidity. Severe asthma accounts for just 4-6% of patients, but this group is more difficult to treat and is responsible for up to 40% of asthma expenses. The relationship between asthma severity and control is not well characterized.

Erratum to: Racial differences in pet ownership in families of children with asthma.

In the article by Sheikh SI, et al, "Racial differences in pet ownership in families of children with asthma" in World Journal of Pediatrics 2016;12(3):343-346 (doi: 10.1007/s12519-016-0027-9), the last author's name was incorrectly listed as "Don Hayes". His name should have read "Don Hayes Jr".

Age-Dependent Gender Disparities in Post Lung Transplant Survival Among Patients With Idiopathic Pulmonary Fibrosis.

Background: The influence of age and gender on survival after lung transplant in patients with idiopathic pulmonary fibrosis (IPF) is not well defined.

Methods: The United Network for Organ Sharing database was queried to identify IPF patients receiving lung transplant between 2005 and 2015.

Results: There were 6,677 patients receiving lung transplant between May 2015 and June 2015 who met the inclusion criteria, predominantly males (n = 4,769, 71%).

Role of CFTR mutation analysis in the diagnostic algorithm for cystic fibrosis.

Background: The cystic fibrosis transmembrane conductance regulator (CFTR) gene mutation identification is being used with increased frequency to aid in the diagnosis of cystic fibrosis (CF) in those suspected with CF. Aim of this study was to identify diagnostic outcomes when CFTR mutational analysis was used in CF diagnosis. CFTR mutational analysis results were also compared with sweat chloride results.

Improved quality-of-life of caregivers of children with asthma through guideline-based management.

Objective: The quality of life (QOL) of caregivers of children with asthma may be related to children's responses to asthma management.

Aim: To evaluate change in QOL over time of caregivers of children with asthma through guideline-based management.

Design: This was a 3-year prospective cohort study of children with asthma referred to our pediatric asthma center.

Pretransplant Panel Reactive Antibodies and Lung Transplant Outcomes in Children.

 Pre-lung transplant (LTx) panel reactive antibody (PRA) levels are associated with adverse outcomes in adult LTx recipients, but their impact in pediatric LTx recipients is unknown.  The United Network for Organ Sharing registry was queried from 2004 to 2013 to compare survival between pediatric LTx recipients with PRA class I and II levels = 0 versus > 0.  Overall, 333 pediatric LTx recipients had data on class I or II PRA and were included in the analysis.

Novel Computed Tomography Scoring System for Sinus Disease in Adults With Cystic Fibrosis.

Objective: There is no easy to use scoring system for computed tomography (CT) scans of the sinuses that is specific to cystic fibrosis (CF). We propose a simple and easily implemented scoring system to quantify severity of sinus disease in adults with CF.

Study Design: Case series with chart review.

Racial differences in pet ownership in families of children with asthma.

Background: Exposure to household domestic animals such as cats and dogs in early life may have some role in pathogenesis of asthma. Racial differences exist in the prevalence of asthma. We hypothesized that there may also be racial differences in pet ownership in families with asthma.

Influence of graft ischemic time on survival in children with cystic fibrosis after lung transplantation.

Background: The influence of graft ischemic time on survival after lung transplantation (LTx) in children with cystic fibrosis (CF) is not well studied.

Methods: The United Network for Organ Sharing (UNOS) database was queried from May 2005 to September 2013 to examine the impact of ischemic time of <4, 4-6, and >6 hr in pediatric LTx recipients with CF.

Results: One hundred and ninety-nine patients with CF under 18 years of age that were first-time LTx recipients from cadaveric donors were included in the analysis.

Environmental exposures and family history of asthma.

Background: Asthma pathogenesis is a complex interaction of genetic, ethnic, environmental and social/life style risk factors.

Aim: The goal of this study was to identify associations, if any, in children with asthma, between environmental risk factors (exposure to second-hand tobacco smoke (STS), pet ownership, race and a family history of asthma.

Methods: After IRB approval, from June 2011 to December 2014, 823 children with asthma were enrolled in this prospective cross sectional study.

Improving pediatric asthma care: A partnership between pediatric primary care clinics and a free-standing Children's Hospital.

Background: Asthma is a common chronic disease of childhood. Providers' adherence to asthma guidelines is still less than optimal.

Objectives: To determine if an Asthma Education Program aimed at primary care practices can improve asthma care within practices and if the results vary by duration of the program.

Screening high-risk children for asthma through a community intervention.

Objective: Many children, particularly those from inner city neighborhoods, have undiagnosed asthma. This study was done to evaluate the effectiveness of an asthma screening, referral and follow-up intervention in an inner city community setting in early identification of children at risk for undiagnosed asthma.

Methods: A descriptive longitudinal cohort design was used to assess children at baseline and at a 2-year follow-up.

Mortality Risk and Pulmonary Function in Adults With Cystic Fibrosis at Time of Wait Listing for Lung Transplantation.

Background: Lung transplantation (LTx) benefit for survival in cystic fibrosis (CF) patients placed on the wait list is not well studied.

Methods: To predict the relationship between initial forced expiratory volume in 1 second (FEV1) and forced vital capacity (FVC) and the hazard ratio (HR) associated with LTx in CF patients, the United Network for Organ Sharing database was queried from 2005 to 2006 for adult patients with CF. Survival was assessed from wait list entry time until death on wait list, death after LTx, or censoring.

Changes in Pulmonary Function and Controlled Ventilation-High Resolution CT of Chest After Antibiotic Therapy in Infants and Young Children with Cystic Fibrosis.

Background: Infants with cystic fibrosis (CF) develop early progressive lung disease which may be asymptomatic. Infant pulmonary function tests (IPFT) and controlled ventilation-high resolution computed tomography (CV-HRCT) of chest can detect early asymptomatic lung disease. It is not well established that these objective measures can detect changes in lung disease after clinical interventions.

Improvement of hepatic steatosis in cystic fibrosis with ivacaftor therapy.

Treatment of liver disease, including hepatic steatosis, in patients with cystic fibrosis (CF) is limited. With the development of ivacaftor, which corrects the gating defect of the CF transmembrane regulator channel, there is a potential new therapy available for this subgroup of the CF patient population. We present an adolescent with CF who had significant improvement in hepatic steatosis with ivacaftor treatment while hypothesizing on a mechanism of why it occurred.

Computed tomography correlates with improvement with ivacaftor in cystic fibrosis patients with G551D mutation.

Background: Ivacaftor corrects the cystic fibrosis transmembrane conductance regulator (CFTR) gating defect associated with G551D mutation and is quickly becoming an important treatment in patients with cystic fibrosis (CF) due to this genetic mutation.

Methods: A single-center study was performed in CF patients receiving ivacaftor to evaluate the usefulness of high resolution computed tomography (HRCT) of the chest as a way to gauge response to ivacaftor therapy.

Results: Ten patients with CF were enrolled for at least one year before and after starting ivacaftor.