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

Abstract

IntroductionIn 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 analysisA 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 disseminationEthical 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 numberNCT05169619.

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

“…EPI-ASTHMA used a stepwise approach, sequentially comprising 4 stages, that has been described in detail elsewhere. 15 A brief summary of each stage is provided below and Table 1 specifies the data collected at each stage.…”

“…The minimum initial sample size estimation for this pilot study was found to be 1316 participants based on an estimated prevalence described elsewhere. 15 The characteristics of participants were described with mean and standard deviation (SD) for normally distributed variables; for skewed distributions we used median and interquartile range (IQR; range from 25th to 75th percentile). Categorical variables were described with absolute frequencies, proportions and 95% confidence intervals (95% CI).…”

“…To provide a more complete picture of the prevalence of asthma, difficult-to-treat and severe asthma in Portugal and to better understand patient’s characteristics, treatment profile and healthcare resources use, we designed EPI-ASTHMA, a population-based multicentre stepwise study. 15 The stepwise approach, divided in 4 stages, aimed to simulate the clinical practice diagnostic pathway, which commonly initiates with the evaluation of symptom patterns and clinical features, and then evolves to a clinical decision by using different diagnostic tests. EPI-ASTHMA combines different data collection methods, including screening interviews, self-reported questionnaires, diagnostic tests, clinical interview with physical examination and access to the patient’s electronic health record (EHR).…”