Benralizumab in severe eosinophilic asthma by previous biologic use and key clinical subgroups: real-world XALOC-1 programme.

BACKGROUND: Pivotal Phase 3 trials and real-world studies have demonstrated benralizumab's overall efficacy and safety in severe eosinophilic asthma (SEA). Additional large-cohort data are needed to confirm its real-world effectiveness in SEA according to previous biologic use and key baseline characteristics important for treatment selection. METHODS: XALOC-1 is a large, multinational, retrospective, observational, real-world study programme of benralizumab in adults with SEA. This 48-week integrated analysis assessed annualised exacerbation rate (AER), maintenance oral corticosteroid (mOCS) use, asthma symptom control and lung function during a 12-month baseline period and up to 48 weeks after benralizumab initiation. Subgroup analyses were based on previous biologic use and key baseline clinical characteristics (mOCS use, blood eosinophil count, exacerbation history, age at asthma diagnosis, fractional exhaled nitric oxide level and presence of atopy and chronic rhinosinusitis with nasal polyps). RESULTS: Of 1002 patients analysed, 380 were biologic-experienced. At Week 48, 71.3% were exacerbation-free (versus 17.2% at baseline); relative reduction in AER was 82.7% overall and 72.9% in biologic-experienced patients; rates were maintained across all key clinical characteristic subgroups. Of patients using mOCS at baseline (n=274), 47.4% (130/274) eliminated their use by Week 48; the mean reduction from baseline in daily dose was 51.2% and, notably, 34.9% in biologic-experienced patients (n=115). Clinically significant improvements in asthma symptom control and lung function were observed. CONCLUSION: In this large, real-world programme, SEA patients treated with benralizumab had substantial improvements in clinical outcomes irrespective of previous biologic use and key clinical characteristics important to therapeutic decision-making in clinical practice.

Portuguese Heart Failure Prevalence Observational Study (PORTHOS) rationale and design - A population-based study.

INTRODUCTION AND OBJECTIVES: Current epidemiological data on heart failure (HF) in Portugal derives from studies conducted two decades ago. The main aim of this study is to determine HF prevalence in the Portuguese population. Using current standards, this manuscript aims to describe the methodology and research protocol applied. METHODS: The Portuguese Heart Failure Prevalence Observational Study (PORTHOS) is a large, three-stage, population-based, nationwide, cross-sectional study. Community-dwelling citizens aged 50 years and older will be randomly selected via stratified multistage sampling. Eligible participants will be invited to attend a screening visit at a mobile clinic for HF symptom assessment, anthropomorphic assessment, N-terminal pro-B-type natriuretic peptide (NT-proBNP) testing, one-lead electrocardiogram (ECG) and a sociodemographic and health-related quality of life questionnaire (EQ-5D). All subjects with NT-proBNP ≥125 pg/mL or with a prior history of HF will undergo a diagnostic confirmatory assessment at the mobile clinic composed of a 12-lead ECG, comprehensive echocardiography, HF questionnaire (KCCQ) and blood sampling. To validate the screening procedure, a control group will undergo the same diagnostic assessment. Echocardiography results will be centrally validated, and HF diagnosis will be established according to the European Society of Cardiology HF guidelines. A random subsample of patients with an equivocal HF with preserved ejection fraction diagnosis based on the application of the Heart Failure Association preserved ejection fraction diagnostic algorithm will be invited to undergo an exercise echocardiography. CONCLUSIONS: Through the application of current standards, appropriate methodologies, and a strong research protocol, the PORTHOS study will determine the prevalence of HF in mainland Portugal and enable a comprehensive characterization of HF patients, leading to a better understanding of their clinical profile and health-related quality of life.

Twenty years of real-world data to estimate chronic kidney disease prevalence and staging in an unselected population.

Chronic kidney disease (CKD) represents a global public health burden, but its true prevalence is not fully characterized in the majority of countries. We studied the CKD prevalence in adult users of the primary, secondary and tertiary healthcare units of an integrated health region in northern Portugal (n = 136 993; representing ∼90% of the region's adult population). Of these, 45 983 (33.6%) had at least two estimated glomerular filtration rate (eGFR) assessments and 30 534 (22.2%) had at least two urinary albumin:creatinine ratio (UACR) assessments separated by at least 3 months. CKD was defined according to the Kidney Disease: Improving Global Outcomes (KDIGO) guidelines as a persistent decrease in eGFR (<60 ml/min/1.73 m2) and/or an increase in UACR (≥30 mg/g). The estimated overall prevalence of CKD was 9.8% and was higher in females (5.5%) than males (4.2%). From these, it was possible to stratify 4.7% according to KDIGO guidelines. The prevalence of CKD was higher in older patients (especially in patients >70 years old) and in patients with comorbidities. This is the first real-world-based study to characterize CKD prevalence in a large, unselected Portuguese population. It probably provides the nearest estimate of the true CKD prevalence and may help healthcare providers to guide CKD-related policies and strategies focused on prevention and on the improvement of cardiovascular disease and other outcomes.

Cardiovascular Risk Profile and Lipid Management in the Population-Based Cohort Study LATINO: 20 Years of Real-World Data.

The rising prevalence of cardiovascular (CV) risk factors in Portugal has translated into more than 35,000 annual deaths due to CV diseases. We performed a multicenter observational cohort study encompassing clinical activities performed between 2000 and 2019 to characterize the CV risk profile and LDL-C management of patients in every CV risk category using electronic health records of a regional population in Portugal. We analyzed data from 14 health centers and 1 central hospital in the north of Portugal of patients between 40 and 80 years that had at least 1 family medicine appointment at these institutions. Living patients were characterized on 31 December 2019. CV risk assessment was computed according to the 2019 ESC/EAS Guidelines. Lipid-lowering therapy (LLT) and achievement of LDL-C targets were assessed. In total, the analysis included 78,459 patients. Patient proportions were 33%, 29%, 22%, and 17% for low, intermediate, high, and very high CV risk, respectively. Moderate-intensity statins were the most frequently used medication across all CV risk categories. High-intensity statins were used in 5% and 10% of high and very high CV risk patients, respectively. Ezetimibe was used in 6% and 10% of high and very high CV risk patients, respectively. LDL-C targets were achieved in 44%, 27%, 7%, and 3% of low, intermediate, high, and very high CV risk patients, respectively. For uncontrolled patients in the high and very high CV risk categories, a median LDL-C reduction of 44% and 53%, respectively, would be required to meet LDL-C targets. There are clear opportunities to optimize LDL-C management in routine clinical practice. The prescription of LLT according to CV risk represents an important missed treatment opportunity.

Prevalence and Characterisation of Patients with Asthma According to Disease Severity in Portugal: Findings from the EPI-ASTHMA Pilot Study.

Objective: To assess the feasibility of the procedures of EPI-ASTHMA. EPI-ASTHMA is a population-based multicentre stepwise study about the prevalence and characterisation of patients with asthma based on disease severity in Portugal. Methods: A pilot study of EPI-ASTHMA was conducted with adults from three primary care centres. We followed a stepwise approach comprising 4 stages: stage 0-invitation phone call (n ~1316); stage 1-telephone interview (n ~658); stage 2-clinical assessment with physical examination, diagnostic tests, and patient-reported outcome measures, to confirm the diagnosis of those with possible asthma at stage 1 (n ~160); stage 3-characterization of a subgroup of asthma patients by collecting data through a telephone interview, patient file review and CARATm app (n ~40), after 3-months. The frequency of asthma was calculated in relation to the entire study population (stage 1) and the frequency of difficult-to-treat/severe asthma in relation to the number of asthma patients (stage 3). Results: From 1305 adults invited, 892 (68%) accepted to participate (stage 0) and 574 (64%; 53[42-67] y; 43% male) were interviewed (stage 1). From those, 148 (26%; 60[46-68] y; 43% male) were assessed at stage 2, and 46 (31%; 51[39-67] y; 44% male) were diagnosed with asthma. Half of these patients (n = 23) accepted to install the app. Stage 3 was completed by 41 (93%) patients, of whom 31 (83%) had asthma confirmed by their file review. A total of 8% of participants had asthma, of those 17% had difficult-to-treat and 5% severe asthma. Conclusion: Attained recruitment rates and the quality of the results confirmed the feasibility of the EPI-ASTHMA stepwise approach. This pilot study provided insight into the improvement of the procedures to be generalized across the country.

EPI-ASTHMA study protocol: a population-based multicentre stepwise study on the prevalence and characterisation of patients with asthma according to disease severity in Portugal.

INTRODUCTION: In Portugal as in other countries, data on the epidemiology of asthma are mainly grounded in questionnaire studies. Additionally, the detailed characterisation of asthma in terms of disease severity, control and phenotypes remain scarce. Studies assessing the prevalence of asthma and its subgroups using accurate methods are needed. This study aims to determine the prevalence of asthma, difficult-to-treat asthma and severe asthma, and to evaluate sociodemographic and clinical characteristics of those patients, in mainland Portugal. METHODS AND ANALYSIS: A population-based nationwide study with a multicentre stepwise approach will be conducted between 2021 and 2023 in 38 primary care centres of the Portuguese National Health Service. The stepwise approach will comprise four stages: Stage 0-telephone call invitation to adult subjects (≥18 years) randomly selected (n~15 000); stage 1-telephone screening interview assessing the participants' respiratory symptoms (n~7500); stage 2-diagnostic visit, including physical examination, diagnostic tests (eg, spirometry, fraction of exhaled nitric oxide, blood eosinophil count) and patient-reported outcome measures for diagnostic confirmation of those identified with possible asthma at stage 1 (n~1800); stage 3-further evaluation of patients with asthma and of patients with difficult-to-treat asthma and severe asthma, after 3 months (n~460). At stage 3, data will be collected from a review of the patient's electronic health records, a follow-up telephone call and the CARATm (Caracteristicas Auto-reportadas de Asma em Tecnologias Móveis) app database. The prevalence of asthma, difficult-to-treat asthma and severe asthma will be determined as the percentage of patients with asthma confirmed from the overall population (stage 1). For the analysis of factors associated with asthma, difficult-to-treat asthma and severe asthma, logistic regression models will be explored. ETHICS AND DISSEMINATION: Ethical approvals for the study were obtained from the ethics committee of the local health unit of Matosinhos, Porto (38/CES/JAS), Alto Minho (38/2021/CES) and the regional health administration of Lisbon-Vale do Tejo (035/CES/INV/2021). Results will be published in peer-reviewed journals. TRIAL REGISTRATION NUMBER: NCT05169619.

Trends, geographical variation and factors associated with the use of anti-VEGF intravitreal injections in Portugal (2013-2018): a retrospective analysis of administrative data.

AIMS: The arrival of anti-vascular endothelial growth factor (anti-VEGF) therapies represented a treatment shift for several ophthalmological disorders and led to an increasing number of patients undergoing intravitreal injections. The aims of this observational study were to assess the expansion of anti-VEGF intravitreal injections in the Portuguese National Health System (NHS) and to identify factors correlated with geographical variations in episode rates. METHODS: Administrative database on discharge from Portuguese NHS hospitals was analysed for annual values and rates of intravitreal anti-VEGF injections at a national and regional level, between 2013 and 2018. RESULTS: The number of episodes of anti-VEGF treatment and patients treated increased 16% and 9% per year, respectively, between 2013 and 2018. During the study period around 72% of patients were treated in the Metropolitan areas of Lisbon and Porto and in the Central region. Intravitreal anti-VEGF treatment rates in 2018 were 560 per 100 000 population and presented high variability between municipalities. Higher anti-VEGF treatment rates at the municipality level were associated with shorter distances between their residence and the hospital. At the hospital level, higher ratio of ophthalmologists and higher organisational level were associated with higher anti-VEGF treatment rates. CONCLUSION: The number of episodes and patients treated with anti-VEGF injections has been growing in recent years. Proximity to healthcare, more access to ophthalmologists and hospitals with higher organisational levels are associated with higher anti-VEGF treatment rates. Improving access is crucial to reduce regional discrepancies and ensure optimal treatment frequency, which may improve health outcomes.