Assessment of long-term safety and efficacy of dupilumab in children with asthma (LIBERTY ASTHMA EXCURSION): an open-label extension study.

BACKGROUND: Dupilumab efficacy and safety in children aged 6-11 years with uncontrolled, moderate-to-severe asthma were shown in the VOYAGE study-a 52-week, multinational, multicentre, phase 3 randomised, double-blind, placebo-controlled trial. We aimed to evaluate the long-term safety and efficacy of dupilumab in children with moderate-to-severe asthma who previously participated in the VOYAGE study. METHODS: 365 of 408 children with moderate-to-severe asthma from VOYAGE enrolled in EXCURSION, a 52 week, open-label extension study conducted at 70 centres across 17 countries. 240 children continued with add-on dupilumab (dosed according to bodyweight: 100 mg for those weighing ≤30 kg and 200 mg for those weighing more than 30 kg at EXCURSION baseline) once every 2 weeks administered by subcutaneous injection (dupilumab/dupilumab group) and 125 children on placebo during VOYAGE initiated dupilumab (100 or 200 mg, according to bodyweight), once every 2 weeks administered by subcutaneous injection (placebo/dupilumab group). Following a protocol amendment, for a subset of children weighing 30 kg or less, the dose was changed to 300 mg once every 4 weeks. The primary endpoint for the open-label extension study was the number and proportion of patients with any treatment-emergent adverse event (TEAE) during the 52-week study period in the overall population (defined as children aged 6-11 years old with moderate-to-severe asthma who previously completed VOYAGE). Statistical analyses were descriptive. This study is registered with ClinicalTrials.gov (NCT03560466; EXCURSION). FINDINGS: Children who completed VOYAGE were eligible to enrol in EXCURSION between June 21, 2018 and Aug 18, 2020. During EXCURSION, the safety profile and proportion of patients reporting TEAEs were consistent with those observed during the parent study (VOYAGE). In the overall population, 232 (63·6%) of 365 patients experienced at least one TEAE (dupilumab/dupilumab: 147 [61·3%]; placebo/dupilumab: 85 [68·0%]). The most frequently reported TEAEs were nasopharyngitis, pharyngitis, and upper respiratory tract infections. INTERPRETATION: In EXCURSION, long-term treatment with dupilumab was well tolerated with an acceptable safety profile. FUNDING: Sanofi and Regeneron Pharmaceuticals.

Children with uncontrolled asthma from economically disadvantaged neighborhoods: Needs assessment and the development of a school-based telehealth and electronic inhaler monitoring system.

BACKGROUND: Children from economically disadvantaged communities often encounter healthcare access barriers, increasing risk for poorly controlled asthma and subsequent healthcare utilization. This highlights the need to identify novel intervention strategies for these families. OBJECTIVE: To better understand the needs and treatment preferences for asthma management in children from economically disadvantaged communities and to develop a novel asthma management intervention based on an initial needs assessment and stakeholder feedback. METHODS: Semistructured interviews and focus groups were conducted with 19 children (10-17 years old) with uncontrolled asthma and their caregivers, 14 school nurses, 8 primary care physicians, and three school resource coordinators from economically disadvantaged communities. Interviews and focus groups were audio-taped and transcribed verbatim and then analyzed thematically to inform intervention development. Using stakeholder input, an intervention was developed for children with uncontrolled asthma and presented to participants for feedback to fully develop a novel intervention. RESULTS: The needs assessment resulted in five themes: (1) barriers to quality asthma care, (2) poor communication across care providers, (3) problems identifying and managing symptoms and triggers among families, (4) difficulties with adherence, and (5) stigma. A proposed video-based telehealth intervention was proposed to stakeholders who provided favorable and informative feedback for the final development of the intervention for children with uncontrolled asthma. CONCLUSIONS: Stakeholder input and feedback provided information critical to the development of a multicomponent (medical and behavioral) intervention in a school setting that uses technology to facilitate care, collaboration, and communication among key stakeholders to improve asthma management for children from economically disadvantaged neighborhoods.

Examination of the uses, needs, and preferences for health technology use in adolescents with asthma.

Objective: To examine the health technology uses and preferences of adolescents with asthma using a qualitative descriptive individual interview approach. Methods: Twenty adolescents were recruited from regularly scheduled asthma clinic appointments from February to July 2016. Patients were interviewed about their technology use and ways in which health technology could improve their asthma management using an open-ended semi-structured interview format. Interviews were audio recorded, transcribed, and coded into themes. Results: Social media (e.g. Snapchat, Instagram) and general communication (e.g. messaging) were the most common uses of technology while medical reminders (e.g. appointment, refill, medication) were the most common use of health technology. Adolescents identified ways in which health technology could improve their asthma management including (1) tracking symptoms and medication, (2) medical reminders, and (3) asthma and self-management knowledge specifically related to medications and individual action plans. Other themes that emerged included a desire to customize health technology to fit with individual schedules and medical routines and use of health technology data with medical providers. Conclusions: Adolescents and parents experience a number of challenges related to managing asthma, and health technology interventions should focus on ways to improve adherence and self-management. Future research considerations and potential interventions including ways to integrate adolescent preferences with evidence-based interventions are discussed.

Heterogeneity in asthma care in a statewide collaborative: the Ohio Pediatric Asthma Repository.

BACKGROUND AND OBJECTIVE: Asthma heterogeneity causes difficulty in studying and treating the disease. We built a comprehensive statewide repository linking questionnaire and medical record data with health outcomes to characterize the variability of clinical practices at Ohio children's hospitals for the treatment of hospitalized asthma. METHODS: Children hospitalized at 6 participating Ohio children's hospitals for asthma exacerbation or reactive airway disease aged 2 to 17 were eligible. Medical, social, and environmental histories and past asthma admissions were collected from questionnaires and the medical record. RESULTS: From December 2012 to September 2013, 1012 children were enrolled. There were significant differences in the population served, emergency department and inpatient practices, intensive care unit usage, discharge criteria, and length of stay across the sites (all P < .0001, total n = 1012). Public insurance was highest in Cleveland and Cincinnati (72 and 65%). In the emergency department, Cincinnati and Akron had the highest intravenous magnesium sulfate use (37% and 33%); Columbus administered the most intramuscular epinephrine (15%). Cleveland and Columbus had the highest intensive care unit admittance (44% and 41%) and proportion of long-stay patients (95% and 85%). Moderate/severe asthma severity classification was associated with discharge prescription for inhaled corticosteroids (odds ratio = 2.7; 95% confidence interval: 1.6-4.5; P = .004) but not stay length. CONCLUSIONS: These data highlight the need for standardization of treatment practices for inpatient asthma care. There is considerable opportunity for personalized care plans that incorporate a patient's asthma impairment, risk, and treatment response history into hospital practices for asthma exacerbation treatment. The Ohio Pediatric Asthma Repository is a unique statewide resource in which to conduct observational, comparative effectiveness, and ultimately intervention studies for pediatric asthma.

Cost-effectiveness of initiating extrafine- or standard size-particle inhaled corticosteroid for asthma in two health-care systems: a retrospective matched cohort study.

BACKGROUND: Real-life studies are needed to determine the cost-effectiveness of asthma therapies in clinical practice. AIM: To compare the cost-effectiveness of extrafine-particle inhaled corticosteroid (ICS) with standard size-particle ICS in the United Kingdom (UK) and United States (US). METHODS: These retrospective matched cohort analyses used large electronic databases to study asthma-related outcomes for patients in the UK (12-60 years old; n=1730) and US (12-80 years; n=10,312) prescribed extrafine beclomethasone or fluticasone as their first ICS therapy for asthma. Patients were matched on demographic characteristics and asthma severity during 1 baseline year, and asthma control and asthma-related costs were compared during 1 outcome year. RESULTS: In both the UK and US, adjusted odds of risk-domain asthma control were similar, whereas the odds of overall control (no hospitalisation or oral steroids for asthma, no antibiotics for lower respiratory infection, limited reliever use) were greater for extrafine ICS in both countries (UK odds ratio, 1.23; 95% confidence interval (CI), 1.01-1.50). Asthma-related annual costs, adjusted for baseline, were significantly lower for extrafine-particle ICS cohorts in both countries (UK difference, -£66 (95% CI,-93 to -37)). Cost-effectiveness analyses using the two measures of asthma control found 92 and 98% probabilities of extrafine-particle ICS being the preferred treatment strategy (less costly and more effective than standard size-particle ICS) in the UK, and 84 and 100% probabilities in the US. CONCLUSIONS: Initiating ICS therapy for asthma as extrafine-particle ICS seems the dominant treatment option (less costly and more effective) compared with standard size-particle ICS in both the UK and the US.

The theory and application of UW ehealth-PHINEX, a clinical electronic health record-public health information exchange.

BACKGROUND: Electronic health records (EHRs) hold the promise of improving clinical quality and population health while reducing health care costs. However, it is not clear how these goals can be achieved in practice. METHODS: Clinician-led teams developed EHR data extracts to support chronic disease use cases. EHRs were linked with community-level data to describe disease prevalence and health care quality at the patient, health care system, and community risk factor levels. Software was developed and statistical modeling included multivariate, mixed-model, longitudinal, data mining, and geographic information system (GIS)/spatial regression approaches. RESULTS: A HIPAA-compliant limited data set was created on 192,201 patients seen in University of Wisconsin Family Medicine clinics throughout Wisconsin in 2007-2009. It was linked to a commercially available database of approximately 6000 variables describing community-level risk factors at the census block group. Areas of increased asthma and diabetes prevalence have been mapped, identified, and compared to economic hardship. CONCLUSIONS: A comprehensive framework has been developed for clinical-public health data exchange to develop new evidence and apply it to clinical practice and health policy. EHR data at the neighborhood level can be used for future population studies and may enhance understanding of community-level patterns of illness and care.

The burden of asthma in the United States: level and distribution are dependent on interpretation of the national asthma education and prevention program guidelines.

Asthma imposes a growing burden on society in terms of morbidity, quality of life, and healthcare costs. Although federally sponsored national surveys provide estimates of asthma prevalence, these surveys are not designed to characterize the burden of asthma by self-reported disease activity. We sought to characterize asthma burden in the United States. This study was based on a cross-sectional random-digit-dial household telephone survey designed to identify adult patients and parents of children with current asthma. Global asthma burden was comprised of three components: short-term symptom burden (4-week recall), long-term symptom burden (past year), and functional impact (activity limitation). Using this construct, only 10.7% of individuals were classified as having a global asthma burden consistent with mild intermittent disease, and 77.3% had moderate to severe persistent disease. These results suggest that a majority of the United States population with asthma experiences persistent rather than intermittent asthma burden. In addition, the discordance in type and distribution of asthma symptoms reported by individual subjects suggests that the exact estimate of the burden of asthma is related to how the National Asthma Education and Prevention Program classification is operationalized. Inquiry into recent day or nighttime symptoms alone underestimates the burden of asthma and may lead to inadequate treatment of asthma based on national guideline recommendations.