Determining Adherence to Inhaled Corticosteroids From the Epic Electronic Medical Record.

Objective: Often we call the patient's pharmacy to obtain a refill history to assess inhaled corticosteroid (ICS) adherence. The purpose of this project was to determine the accuracy of refill histories for ICS (with or without long-acting beta agonist) listed in Epic's Medication Dispense History.

Methods: We evaluated 61 patients and used data from 38 who met the following criteria: 1) under the care of the UF Pediatric Severe Asthma Clinic; 2) taking the same dose of the same ICS product for 6 months before the patient's last clinic visit; and 3) having data available from the pharmacy where the last ICS prescription was electronically sent.

Legal Needs, Health, and Health Care Utilization Among Patients Participating in the Delaware Medical Legal Partnership.

Context: Unmet legal needs can exacerbate health disparities and contribute to a lack of adherence to treatment plans and medical recommendations for care. Medical legal partnerships (MLPs) are integrated health care and legal aid interventions offered by many health systems in the United States. Although much research has been published regarding the success of MLPs with specific patient groups, there is a gap in literature regarding the nature of MLPs in a more general, at-risk patient population.

Sleep and asthma management in youth with poorly-controlled asthma and their caregivers: a qualitative approach.

Objective: Youth with poorly-controlled asthma are at increased risk for sleep disturbances caused by nocturnal symptoms like coughing. Asthma-related sleep disturbances can have downstream consequences for youth with asthma and their families. This study aims to describe (1) sleep disturbances in adolescents with poorly-controlled asthma and their caregivers and (2) the relationship between sleep and asthma management.

Arnold-Chiari malformation: an uncommon etiology for a brief resolved, unexplained event in an infant.

Joseph N, Rajderkar D, Baker D, Prabhakaran S. Arnold-Chiari malformation: an uncommon etiology for a brief resolved, unexplained event in an infant. .

Continuous Albuterol With Benzalkonium in Children Hospitalized With Severe Asthma.

Background And Objectives: The albuterol dropper bottle used to prepare solutions for continuous nebulization contains the preservative benzalkonium chloride (BAC). BAC, by itself, has been shown to cause bronchospasm. We hypothesized that BAC would decrease the therapeutic efficacy of albuterol in patients with acute asthma exacerbations.

Asthma Management Responsibility, Control, and Quality of Life Among Emerging Adolescents.

Objective: To examine how asthma control is related to the association between the division of responsibility for asthma management and asthma-related quality of life among early adolescents.

Methods: Forty-nine youth aged 10-15 years (Mage = 12.25, 57.

Direct Observed Therapy of Inhaled Corticosteroids for Asthma at School or Daycare.

Poor adherence with inhaled corticosteroid (ICS) medication is common in the pediatric population and can result in poor asthma control with increased frequency of asthma-related complications. The purpose of this study was to determine whether or not the initiation of ICS administration twice per day at school/daycare in patients with poor medication adherence at home improves asthma health care outcomes. We retrospectively selected patients followed by our Pediatric Pulmonology Clinic who had poorly controlled asthma and had been assigned to receive ICS twice daily at school/daycare due to poor adherence with ICS therapy.

A Behavioral Family Intervention for Children with Overweight and Asthma.

Objective: The Childhood Health and Asthma Management Program (CHAMP) is a behavioral family lifestyle intervention for youth with overweight or obesity (OV/OB) and asthma. This pilot randomized controlled trial examined the feasibility, acceptability, and preliminary efficacy of CHAMP.

Methods: A sample of 24 children ( = 8.

Identification of Pharmacodynamic Transcript Biomarkers in Response to FGFR Inhibition by AZD4547.

The challenge of developing effective pharmacodynamic biomarkers for preclinical and clinical testing of FGFR signaling inhibition is significant. Assays that rely on the measurement of phospho-protein epitopes can be limited by the availability of effective antibody detection reagents. Transcript profiling enables accurate quantification of many biomarkers and provides a broader representation of pathway modulation.

Characterization of FGFR1 Locus in sqNSCLC Reveals a Broad and Heterogeneous Amplicon.

FGFR1 amplification occurs in ~20% of sqNSCLC and trials with FGFR inhibitors have selected FGFR1 amplified patients by FISH. Lung cancer cell lines were profiled for sensitivity to AZD4547, a potent, selective inhibitor of FGFRs 1-3. Sensitivity to FGFR inhibition was associated with but not wholly predicted by increased FGFR1 gene copy number.

Assessment of repeatability of hyperpolarized gas MR ventilation functional imaging in cystic fibrosis.

Rationale And Objectives: Hyperpolarized (HP) gas magnetic resonance imaging (MRI) is an advanced imaging technique that provides high-resolution regional information on lung function without using ionizing radiation. Before this modality can be considered for assessing clinical or investigational interventions, baseline repeatability needs to be established. We assessed repeatability of lung function measurement using HP helium-3 MRI (HP (3)He MRI) in a small cohort of patients with cystic fibrosis (CF).

The effect of VEGF-targeted therapy on biomarker expression in sequential tissue from patients with metastatic clear cell renal cancer.

Purpose: To investigate how biologically relevant markers change in response to antiangiogenic therapy in metastatic clear cell renal cancer (mRCC) and correlate these changes with outcome.

Experimental Design: The study used sequential tumor tissue and functional imaging (taken at baseline and 12-16 weeks) obtained from three similar phase II studies. All three studies investigated the role of VEGF tyrosine kinase inhibitors (TKI) before planned nephrectomy in untreated mRCC (n = 85).

Tumor stromal architecture can define the intrinsic tumor response to VEGF-targeted therapy.

Purpose: The aim of the study was to investigate the vascular and stromal architecture of preclinical tumor models and patient tumor specimens from malignancies with known clinical outcomes to VEGFi treatment, to gain insight into potential determinants of intrinsic sensitivity and resistance.

Experimental Design: The tumor stroma architecture of preclinical and clinical tumor samples were analyzed by staining for CD31 and α-smooth muscle actin (α-SMA). Tumor models representative of each phenotype were then tested for sensitivity to the VEGFR2-blocking antibody DC101.

Evolution of pancreatic function during the first year in infants with cystic fibrosis.

Objective: To describe pancreatic function during the first year of life in infants diagnosed with cystic fibrosis (CF) using serial fecal elastase measurements.

Study Design: This was a longitudinal study of 82 infants diagnosed with CF through newborn screening. Monthly stool samples were sent to a central laboratory for fecal elastase measurements.

MEDI0639: a novel therapeutic antibody targeting Dll4 modulates endothelial cell function and angiogenesis in vivo.

The Notch signaling pathway has been implicated in cell fate determination and differentiation in many tissues. Accumulating evidence points toward a pivotal role in blood vessel formation, and the importance of the Delta-like ligand (Dll) 4-Notch1 ligand-receptor interaction has been shown in both physiological and tumor angiogenesis. Disruption of this interaction leads to a reduction in tumor growth as a result of an increase in nonfunctional vasculature leading to poor perfusion of the tumor.

AZD4547: an orally bioavailable, potent, and selective inhibitor of the fibroblast growth factor receptor tyrosine kinase family.

The fibroblast growth factor (FGF) signaling axis is increasingly implicated in tumorigenesis and chemoresistance. Several small-molecule FGF receptor (FGFR) kinase inhibitors are currently in clinical development; however, the predominant activity of the most advanced of these agents is against the kinase insert domain receptor (KDR), which compromises the FGFR selectivity. Here, we report the pharmacologic profile of AZD4547, a novel and selective inhibitor of the FGFR1, 2, and 3 tyrosine kinases.

Using hyperpolarized 3He MRI to evaluate treatment efficacy in cystic fibrosis patients.

Purpose: To use hyperpolarized (HP) (3)He MR imaging to assess functional lung ventilation in subjects with cystic fibrosis (CF) before and after treatment.

Materials And Methods: We performed HP (3)He static ventilation MRI scans on three subjects, using a Philips 3.0 Tesla (T) Achieva MRI scanner, before and after 11 days of in-patient treatment with combined intravenous and inhaled therapies for pulmonary exacerbations of CF.

Vandetanib inhibits both VEGFR-2 and EGFR signalling at clinically relevant drug levels in preclinical models of human cancer.

Vandetanib is a multi-targeted receptor tyrosine kinase inhibitor that is in clinical development for the treatment of solid tumours. This preclinical study examined the inhibition of two key signalling pathways (VEGFR-2, EGFR) at drug concentrations similar to those achieved in the clinic, and their contribution to direct and indirect antitumour effects of vandetanib. For in vitro studies, receptor phosphorylation was assessed by Western blotting and ELISA, cell proliferation was assessed using a cell viability endpoint, and effects on cell cycle determined using flow cytometry.

Vascular endothelial growth factor receptors VEGFR-2 and VEGFR-3 are localized primarily to the vasculature in human primary solid cancers.

Purpose: Vascular endothelial growth factor (VEGF) signaling is key to tumor angiogenesis and is an important target in the development of anticancer drugs. However, VEGF receptor (VEGFR) expression in human cancers, particularly the relative expression of VEGFR-2 and VEGFR-3 in tumor vasculature versus tumor cells, is poorly defined.

Experimental Design: VEGFR-2- and VEGFR-3-specific antibodies were identified and used in the immunohistochemical analysis of human primary cancers and normal tissue.

Empathy and enduring depersonalization: the role of self-related processes.

Empathy has two key components: affective and cognitive. It relies on "embodied" processes such as the generation, representation and perception of feeling states. People diagnosed with Depersonalization Disorder (DPD) report disturbances in affective experience, such as emotional numbing, alongside aberrations in "body image" such as increased self-focus and feelings of "disembodiment".

Emotional memory and perception of emotional faces in patients suffering from depersonalization disorder.

Previous work has shown that patients with depersonalization disorder (DPD) have reduced physiological responses to emotional stimuli, which may be related to subjective emotional numbing. This study investigated two aspects of affective processing in 13 patients with DPD according to the DSM-IV criteria and healthy controls: the perception of emotional facial expressions (anger, disgust, fear, happiness, sadness, and surprise) and memory for emotional stimuli. Results revealed a specific lack of sensitivity to facial expression of anger in patients, but normal enhancement of memory for peripheral aspects of arousing emotional material.

Phase I trial of intramuscular injection of a recombinant adeno-associated virus serotype 2 alphal-antitrypsin (AAT) vector in AAT-deficient adults.

A phase I trial of intramuscular injection of a recombinant adeno-associated virus serotype 2 (rAAV2) alpha1-antitrypsin (AAT) vector was performed in 12 AAT-deficient adults, 10 of whom were male. All subjects were either homozygous for the most common AAT mutation (a missense mutation designated PI*Z) or compound heterozygous for PI*Z and another mutation known to cause disease. There were four dose cohorts, ranging from 2.

Lamotrigine as an add-on treatment for depersonalization disorder: a retrospective study of 32 cases.

Objectives: Depersonalization disorder (DPD) is a chronic condition characterized by the persistent subjective experience of unreality and detachment from the self. To date, there is no known treatment. Lamotrigine as sole agent was not found to be effective in a previous small double-blind, randomized crossover trial.