Background: ASSURE‐CSU revealed differences in physician and patient reporting of angioedema. This post hoc analysis was conducted to evaluate the actual rate of angioedema in the study population and explore differences between patients with and without angioedema.Methods: This international observational study assessed 673 patients with inadequately controlled chronic spontaneous urticaria (CSU). Physicians abstracted angioedema data from medical records, which were compared with patient‐reported data. Patients in the Yes‐angioedema category had angioedema reported in the medical record and a patient‐reported source. For those in the No‐angioedema category, angioedema was reported in neither the medical record nor a patient‐reported source. Those in the Misaligned category had angioedema reported in only one source. Statistical comparisons between Yes‐angioedema and No‐angioedema categories were conducted for measures of CSU activity, health‐related quality of life (HRQoL), productivity and healthcare resource utilization (HCRU). Regression analyses explored the relationship between Dermatology Life Quality Index (DLQI) score and angioedema, adjusting for important covariates.Results: Among evaluable patients, 259 (40.3%), 173 (26.9%) and 211 (32.8%) were in the Yes‐angioedema, No‐angioedema and Misaligned category, respectively. CSU activity and impact on HRQoL, productivity, and HCRU was greater for Yes‐angioedema patients than No‐angioedema patients. After covariate adjustment, mean DLQI score was significantly higher (indicating worse HRQoL) for patients with angioedema versus no angioedema (9.88 vs 7.27, P < .001). The Misaligned category had similar results with Yes‐angioedema on all outcomes.Conclusions: Angioedema in CSU seems to be under‐reported but has significant negative impacts on HRQoL, daily activities, HCRU and work compared with no angioedema.

Background: Data on the prevalence and disease management of chronic urticaria (CU) and chronic spontaneous urticaria (CSU) in the pediatric population are scarce. This study assessed the prevalence of CU and CSU, and disease management among pediatric patients (0‐17 years).Methods: A physician‐based online survey was conducted in 5 European countries (United Kingdom, Germany, Italy, France, and Spain) assessing the annual diagnosed prevalence, disease characteristics, and treatment patterns in the target population. Results are based on physician responses and analyzed using descriptive statistics. Prevalence estimates were calculated based on the number of CU/CSU pediatric patients diagnosed, seen, and treated by the respondents and extrapolated to the total pediatric population from each country.Results: Across 5 European countries, the one‐year diagnosed prevalence of CU and CSU in pediatric patients was 1.38% (95% CI, 0.94‐1.86) and 0.75% (95% CI, 0.44‐1.08), respectively. Angioedema was reported in 6%‐14% of patients. A large proportion of CSU pediatric patients (40%‐60%) were treated with H1‐antihistamines at approved dose and 16%‐51% received H1‐antihistamines at higher doses. Approximately 1/3 of pediatric CSU patients remained uncontrolled with H1‐antihistamines at approved/higher doses. Other prescribed treatments were oral corticosteroids (10%‐28%) and topical creams (15%‐26%).Conclusions: This study revealed a prevalence of CSU among pediatric population comparable to adults and also suggested an unmet need for approved treatments for inadequately controlled pediatric CSU patients. It is truly of concern that harmful (oral steroids) or insufficient (topical creams) treatments were frequently used despite better and guideline‐recommended alternatives.

Introduction Chronic urticaria (CU), a proxy for chronic spontaneous urticaria, has been associated with a negative impact on health-related quality of life (HRQoL) and costs, but there is limited evidence on the burden of CU in Brazil. The objective of this study was to estimate the prevalence of CU and assess the burden of CU on HRQoL and healthcare resource utilization (HRU) among adults in Brazil. Methods This retrospective, cross-sectional study, pooled data from the 2011, 2012, and 2015 National Health and Wellness Survey in Brazil (n = 36,000). Respondents (aged ≥18 years) diagnosed with and treated for CU provided data on demographics, health history, HRQoL (mental and physical health status) on Short-Form SF-36v2, presence of psychological complaints, work impairment, activity impairment, and HRU. Generalized linear models, controlling for covariates, examined differences between those treated for CU and matched controls on the outcome variables. Results The prevalence of diagnosed CU was 0.41% (n = 249) and treated CU was 0.21% (n = 127). After adjustments, CU (currently treated for CU) was associated with worse mental functioning, physical functioning, and health utilities compared with controls (all p < 0.01). CU had over twice the odds of anxiety and sleep difficulties, over 1.5 times the work and activity impairment, twice the number of total physician visits, eight times the number of allergist visits, and twice the number of emergency room visits as controls (all p < 0.01). Conclusions Many CU patients using prescription treatment experienced anxiety and sleep disturbances, poorer HRQoL, significant work and activity impairment, and high HRU, compared with matched general population controls. Findings suggest an unmet need for more effective treatment and management of CU in Brazil. Funding Novartis Pharma AG and Genentech.

Background Non-alcoholic steatohepatitis (NASH) is a progressive form of non-alcoholic fatty liver disease characterized by excessive liver fat accumulation, inflammation, cell injury, and fibrosis. It is viewed as largely asymptomatic in its earlier (non-cirrhotic) stages, and information on the patient-perceived impact of NASH is scarce. Objective This study aimed to develop a NASH-specific patient-reported outcome (PRO) measure (NASH-CHECK) for use as a trial endpoint, using methods compliant with regulatory expectations. Methods A NASH conceptual model was developed based on the literature and clinical/patient expert review. The model guided concept elicitation (CE) interviews in patients with non-cirrhotic NASH recruited via a US tertiary care center. NASH-CHECK content was generated via thematic analysis of CE data and review by clinical/patient experts. Cognitive debriefing (CD) interviews with US patients evaluated content validity. Results The literature review confirmed that NASH impacts on functioning and health-related quality of life (HRQoL). Overall, 23 CE and 20 CD interviews were conducted. Key symptoms reported in CE interviews included pain in the upper-right abdomen (n = 14), fatigue (n = 18), poor sleep quality (n = 12), impaired memory (n = 13), and reduced focus (n = 11); key HRQoL impacts included impaired physical functioning, reduced ability to conduct daily living tasks, reduced quality of relationships, low mood, anxiety, and self-consciousness. The 52-item first-draft NASH-CHECK was reduced to 31 items based on patient feedback on item relevance, acceptability, and comprehension. Conclusions The interviews revealed key symptoms and broad HRQoL impacts of NASH. As a disease-specific PRO measure assessing symptoms and HRQoL, the NASH-CHECK is relevant, comprehensive, and acceptable to patients and clinicians.

Background: Chronic spontaneous urticaria (CSU) formerly known as chronic idiopathic urticaria (CIU) is a severe and distressing skin condition that remains uncontrolled in approximately one half of patients, despite the use of licensed, recommended doses of modern, second‐generation H₁‐antihistamines. So far, the humanistic, societal and economic burden of CSU/CIU has not been well quantified. Therefore it is important to broaden our understanding of how CSU/CIU impacts patients, society, and healthcare systems, by determining the disease burden of CSU/CIU and the associated unmet need; as well as to further guide the use of new treatments in an efficient and cost‐effective manner.Methods: ASSURE‐CSU is an observational, multicenter study being conducted in the UK, Germany, Canada, France, Italy, Spain, and The Netherlands. The study comprises a retrospective medical chart review in conjunction with patient surveys (including validated tools for assessment of disease impact) and an 8‐day patient diary. The primary objectives of the study are to describe patient demographics, medical history, treatments, and healthcare resource utilization based on medical‐record data and to assess the impact of disease, healthcare resource utilization, work days missed, and productivity loss based on patient‐reported data. Approximately 700 patients (aged ≥18 years) will be enrolled who have CSU/CIU despite currently receiving treatment, and have had persistent symptoms for at least 12 months. Data will be collected retrospectively for the 12 months (±1 month) prior to enrolment wherever possible, and prospectively for the week following enrolment.Discussion: ASSURE‐CSU will be the first study to examine the economic and humanistic burden of disease in patients diagnosed with CSU/CIU who are symptomatic despite treatment. By combining retrospective evaluation of medical records with prospective patient surveys and 8‐day diaries, across seven different countries, the ASSURE‐CSU study will contribute to a better understanding and acknowledgement of the burden of disease in patients with symptomatic chronic spontaneous urticaria.