Use of intravenous iron and risk of anaphylaxis: A multinational observational post-authorisation safety study in Europe.

Purpose: This post-authorisation safety study estimated the risk of anaphylaxis in patients receiving intravenous (IV) iron in Europe, with interest in iron dextran and iron non-dextrans. Studies conducted in the United States have reported risk of anaphylaxis to IV iron ranging from 2.0 to 6.

Comparison of remission and low disease activity states with DAPSA, MDA and VLDA in a clinical trial setting in psoriatic arthritis patients: 2-year results from the FUTURE 2 study.

Objectives: Remission (REM) or low disease activity (LDA) states were compared in a clinical trial setting of the FUTURE 2 study (NCT01752634) using Disease Activity Index for Psoriatic Arthritis (DAPSA) and Minimal Disease Activity (MDA) composite indices in secukinumab treated PsA patients.

Methods: The proportion of patients reaching DAPSA-REM (cut-off ≤4) or REM+LDA (≤14), and very low disease activity (VLDA; achieving 7/7 criteria) or MDA (≥5/7), were compared in the overall population, by prior use of anti-TNF therapy, and by time since diagnosis using as observed data. The proportion of patients who met individual core component and other variables of interest were also computed to assess residual disease activity in DAPSA-REM/REM+LDA states and VLDA/MDA responses.

Secukinumab efficacy on resolution of enthesitis in psoriatic arthritis: pooled analysis of two phase 3 studies.

Background: Enthesitis is one of the psoriatic arthritis (PsA) domains. Patients with enthesitis are associated with worse outcomes than those without enthesitis. The effect of secukinumab on the resolution of enthesitis in patients with PsA was explored using pooled data from the FUTURE 2 and 3 studies.

Patient-reported quality of life after tisagenlecleucel infusion in children and young adults with relapsed or refractory B-cell acute lymphoblastic leukaemia: a global, single-arm, phase 2 trial.

Background: The ELIANA trial showed that 61 (81%) of 75 paediatric and young adult patients with relapsed or refractory B-cell acute lymphoblastic leukaemia achieved overall remission after treatment with tisagenlecleucel, a chimeric antigen receptor targeted against the CD19 antigen. We aimed to evaluate patient-reported quality of life in these patients before and after tisagenlecleucel infusion.

Methods: ELIANA, a global, single-arm, open-label, phase 2 trial, was done in 25 hospitals across Australia, Austria, Belgium, Canada, France, Germany, Italy, Japan, Norway, Spain, and the USA.

Secukinumab provides sustained PASDAS-defined remission in psoriatic arthritis and improves health-related quality of life in patients achieving remission: 2-year results from the phase III FUTURE 2 study.

Background: Secukinumab has demonstrated sustained improvement in the signs and symptoms of psoriatic arthritis (PsA) over 2 years in the FUTURE 2 study (NCT01752634). This post hoc analysis assessed the ability of secukinumab to achieve Psoriatic Arthritis Disease Activity Score (PASDAS)-based remission or low disease activity (LDA) through 2 years among patients with PsA in the FUTURE 2 study.

Methods: PASDAS (cut-off scores: remission ≤ 1.

Early patient experiences with montelukast orally disintegrating tablets in Japan: a cross-sectional survey of treatment satisfaction in patients with asthma and/or allergic rhinitis.

Objective: Orally disintegrating tablets (ODTs) offer a valuable treatment option, particularly when swallowing solid tablets with water is difficult or inconvenient. Montelukast is an effective treatment for asthma and allergic rhinitis (AR), and an ODT formulation became available in Japan in 2015. This study investigated levels of satisfaction with this new formulation among adults with asthma and/or AR in Japan.

The real-world patient experience of fingolimod and dimethyl fumarate for multiple sclerosis.

Background: Oral disease-modifying therapies offer equivalent or superior efficacy and greater convenience versus injectable options.

Objectives: To compare patient-reported experiences of fingolimod and dimethyl fumarate.

Methods: Adult relapsing-remitting multiple sclerosis patients treated with fingolimod or dimethyl fumarate were recruited from an online patient community and completed an online survey about treatment side effects, discontinuation, and satisfaction.

Adding 3D automated breast ultrasound to mammography screening in women with heterogeneously and extremely dense breasts: Report from a hospital-based, high-volume, single-center breast cancer screening program.

Purpose: The aim of this study was to evaluate the impact of the 3D automated breast ultrasound (3D ABUS) when added to full field digital screening mammography (FFDSM), on breast cancer detection and recall rates in asymptomatic women with dense breasts examined in a high-volume breast cancer screening mammography center.

Methods And Material: 1668 asymptomatic women, age 40-74 years, with heterogeneously dense parenchyma (ACR3) or extremely dense breast (ACR4) were included in the study. FFDSM was performed using standard craniocaudal (CC) and mediolateral oblique (MLO) views followed by anteroposterior (AP); lateral (LAT) and medial (MED) acquisitions of 3D ABUS in both breasts.

Antibiotic treatment of signs and symptoms of pulmonary exacerbations: a comparison by care site.

Background: Antibiotic treatment of cystic fibrosis pulmonary exacerbations is inconsistent. Previous research has indicated that intravenous antibiotics are used more frequently at sites with better pulmonary function but it is not clear under what circumstances they are prescribed.

Method: Pediatric care sites enrolled in the Epidemiologic Study of Cystic Fibrosis were ranked by median FEV1 % predicted of children they followed.

Impact of asthma exacerbations and asthma triggers on asthma-related quality of life in patients with severe or difficult-to-treat asthma.

Background: Few data are available that evaluate the relationship among asthma exacerbations, asthma triggers, and asthma-related quality of life (QoL).

Objective: To evaluate the impact of asthma exacerbations and asthma triggers on QoL.

Methods: Patients with severe or difficult-to-treat asthma, ages ≥ 13 years (n = 2679) from the TENOR (The Epidemiology and Natural History of Asthma: Outcomes and Treatment Regimens) 3-year observational study were included.

Oral, inhaled, and intravenous antibiotic choice for treating pulmonary exacerbations in cystic fibrosis.

Rationale: Patients with cystic fibrosis (CF) experience frequent pulmonary exacerbations (PExs). Clinicians manage these episodes of worsening signs and symptoms in a variety of ways.

Objectives: To characterize the antibiotic management and associated change in lung function following PExs.

Risk factors for rate of decline in FEV1 in adults with cystic fibrosis.

Background: Previously we assessed risk factors for FEV(1) decline in children and adolescents using the Epidemiologic Study of Cystic Fibrosis (J Pediatr 2007;151:134-139); the current study assessed risk factors in adults.

Methods: Risk factors for FEV(1) decline over 3-5.5 years for ages 18-24 and ≥25 years were assessed using mixed-model regression.

Multiple antibiotic-resistant Pseudomonas aeruginosa and lung function decline in patients with cystic fibrosis.

Background: The goal of this study was to determine the association of multiple antibiotic-resistant Pseudomonas aeruginosa (MARPA) acquisition with lung function decline in patients with cystic fibrosis (CF).

Methods: Using data from Epidemiologic Study of Cystic Fibrosis (ESCF), we identified patients with spirometry data and MARPA, defined as PA (1) resistant to gentamicin and either tobramycin or amikacin, and (2) resistant to ≥1 antipseudomonal beta lactam. MARPA had to be detected in a respiratory culture after ≥2 years of PA-positive but MARPA-negative respiratory cultures.

Evaluation of the National Heart, Lung, and Blood Institute guidelines impairment domain for classifying asthma control and predicting asthma exacerbations.

Background: Accurate assessment of asthma control may help predict future asthma exacerbations.

Objective: To evaluate asthma guidelines impairment domain components as predictors of exacerbations in severe/difficult-to-treat asthma.

Methods: Children (aged 6-11 years; n = 289) and adolescents/adults (aged ≥ 12 years; n = 2,094) with complete baseline and 12-month data from The Epidemiology and Natural History of Asthma Outcomes and Treatment Regimens study were included.

Erratum to: Psychometric evaluation of the Cystic Fibrosis Questionnaire-Revised in a national, US sample.

Purpose: The psychometric properties of a health-related quality of life (HRQOL) instrument, the Cystic Fibrosis Questionnaire-Revised (CFQ-R), were evaluated in a national, US sample of patients with cystic fibrosis (CF). This is the first psychometric evaluation of the revised version of this instrument.

Methods: The Epidemiologic Study of CF is a national, US multicenter longitudinal cohort study containing CFQ-R and health outcomes data.

Practice patterns for oral corticosteroid burst therapy in the outpatient management of acute asthma exacerbations.

The use of a short course of oral corticosteroids (OCS), or "steroid burst," is standard practice in the outpatient management of acute severe exacerbations of asthma. Despite published guidelines, the actual practice patterns are unknown. A Web-based survey about typical patterns of OCS administration and total steroid burst dose was administered to pulmonologists (n = 150), allergists (n = 150), primary care physicians (n = 153), and pediatricians (n = 150).

Psychometric evaluation of the Cystic Fibrosis Questionnaire-Revised in a national sample.

Purpose: The psychometric properties of a health-related quality of life (HRQOL) instrument, the Cystic Fibrosis Questionnaire-Revised (CFQ-R), were evaluated in a national sample of patients with cystic fibrosis (CF).

Methods: The Epidemiologic Study of CF is a national, multicenter, longitudinal cohort study containing CFQ-R and health outcomes data. Developmentally appropriate versions of the CFQ-R were available from 7,330 patients aged 6-70 years and a proxy version from 2,728 parents of school-age children.

Longitudinal assessment of health-related quality of life in an observational cohort of patients with cystic fibrosis.

Background: Patient-reported outcomes (PROs) are increasingly used to evaluate the efficacy of new treatments and the progression of chronic diseases. The Cystic Fibrosis Questionnaire-Revised (CFQ-R) is a disease-specific, PRO measure of health-related quality of life (HRQOL). We evaluated associations between changes in health status over time and HRQOL in a national CF database.

Trends in the use of routine therapies in cystic fibrosis: 1995-2005.

Many therapies are used to treat manifestations of cystic fibrosis (CF). Trends in routine therapy use in Epidemiologic Study of Cystic Fibrosis patients were studied from 1995 to 2005. Patients (15,087) were assessed in 1995; 12,778 in 2005.

Impact of solifenacin on quality of life, medical care use, work productivity, and health utility in the elderly: an exploratory subgroup analysis.

Background: Overactive bladder (OAB) is a common problem among the elderly and a financial burden to society. The prevalence of OAB increases with age and affects > or = 25% of people aged > or = 65 years.

Objective: The goal of this exploratory subgroup analysis of the VESIcare Efficacy and Research Study US (VERSUS) was to assess changes in health-related quality of life (HRQoL), medical care resource utilization, work and activity impairment, and health utility among elderly patients with OAB who continued to have urgency symptoms with tolterodine and were willing to try solifenacin.

Impact of socioeconomic status, race, and ethnicity on quality of life in patients with cystic fibrosis in the United States.

Background: Patient-reported outcomes are increasingly used in clinical trials to assess the natural history of chronic diseases and the efficacy of new treatments. Understanding the effects of socioeconomic and minority status on health-related quality of life (HRQOL) will facilitate interpretation of the results of clinical trials and suggest targets for interventions to improve patient care and outcomes. The objective of this study was to examine the effects of socioeconomic and minority status on HRQOL in patients with cystic fibrosis (CF) from childhood through adulthood in a large, comprehensive database containing medical and HRQOL data for patients with CF.

Patient-reported outcomes among omalizumab and salmeterol/fluticasone combination therapy patients.

Background: Some asthma patients remain poorly controlled despite receiving care consistent with treatment guidelines.

Objective: This study compared the ability to sleep, work, and participate in leisure activities among subjects with immunoglobulin E-mediated (allergic) asthma initiating omalizumab (omalizumab start group) with subjects receiving moderate-to-high doses of salmeterol/fluticasone combination therapy, who continued on salmeterol/fluticasone combination therapy for at least a year without adding omalizumab (salmeterol/fluticasone combination continuation group).

Methods: Subjects completed an Internet-based screener and, if eligible, an Internet-based questionnaire.