Patient-Reported Outcomes of Benralizumab in Real-World Use in Severe Eosinophilic Asthma Patients in Taiwan (BEAT)

Study Overview
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Brief Summary
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An open-label, single-arm, non-interventional, prospective, multicenter study involving primary data collection within real-world settings for patients who receive benralizumab for treatment of severe uncontrolled eosinophilic asthma

Detailed Description
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Benralizumab (Fasenra®) is a respiratory biologic agent targeting interleukin-5 (IL-5), an important member of the inflammatory cascade responsible for the pathogenesis of severe asthma. In 2019, Taiwan Food and Drug Administration (TFDA) approved benralizumab for the treatment of severe eosinophilic asthma (SEA). Since March 2020, benralizumab has been reimbursed by Taiwan National Health Insurance (NHI). This prospective study (BEAT) aims to understand the use, effectiveness, and patient reported outcomes (PRO) of reimbursed benralizumab treatment in a real-world setting in Taiwan.

Official Title
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Patient-Reported Outcomes of Benralizumab in Real-World Use in Severe Eosinophilic Asthma Patients in Taiwan

Conditions
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Asthma

Participation Criteria
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Eligibility Criteria
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Inclusion Criteria: Subjects eligible for enrolment in the study and treated with benralizumab according to Taiwan label and reimbursement criteria must meet ALL the following criteria: Male or female patients ≥ 18 years of age (or ≥ 20 years of age for patients enrolled before January 1st, 2023, according to age of majority as defined by Taiwan regulations), with physician's confirmed diagnosis of severe uncontrolled asthma Asthma requiring medium- or high-dose inhaled corticosteroid plus long-acting β-adrenoceptor agonist as maintenance treatment Patients who have been prescribed but not yet initiated* treatment with reimbursed benralizumab (Fasenra®) according to the SmPC, prior to signed informed consent, and for whom the decision to prescribe this therapy is clearly separated from the physician's decision to include the patient in the current study. *Note: Treatment may be initiated (administration of first injection) at or after enrolment. Benralizumab Taiwan reimbursement criteria: ≥ 2 acute exacerbations in the last 12 months, including at least 1 associated with emergency department (ED) visit or hospitalization; AND At least 6 months of maintenance OCS use at ≥ 5 mg/day of prednisolone or equivalent dose; AND Peripheral blood eosinophil ≥ 300 cells/μL in the last 12 months within the year before the initiation of benralizumab. Provision of signed written informed consent form (ICF) indicating that they understand the purpose of the study and procedures required for participation Patients must be able and willing to read, comprehend written instructions, and complete the paper questionnaires required by the protocol (ACQ-5, PGI-C, and PGI-S). In case a patient does not own a smartphone or is not willing to perform the home spirometer, enrolment into the study is up to the physicians' discretion. We anticipate that 90% of patients will participate the home spirometer assessment. Exclusion Criteria: Subject must not meet ANY exclusion criteria: Documented active lung diseases other than asthma and not within reimbursed label Currently enrolled in an interventional clinical study in parallel, except: Patients being in parallel documented in a national asthma registry Patients having completed any other clinical trials including those with biologic treatment. An acute or chronic condition that, in the investigator's opinion, would limit the patients' ability to complete questionnaires, participate in this study, or impact the interpretations of results. Concurrent biologics for asthma are not allowed. Acceptable wash-out periods for other asthma biologics: ≥ 30 days from last dose of previous biologics. Patients already started benralizumab treatment are not allowed. If the patients had received benralizumab treatment before, there should be an interval of ≥ 6 months from the last dose of prior benralizumab course to the newly initiated benralizumab treatment.
Sexes Eligible for Study
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All

Accepts Healthy Volunteers
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No

Study Plan
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How is the study designed?
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What is the study measuring?
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Primary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| To evaluate the change in asthma control after initiation of benralizumab in a real-world Taiwan setting | Primary outcome measure: Changes from baseline in Asthma Control Questionnaire, five-question version (ACQ-5) scored from 0 (totally controlled) to 6 (severely uncontrolled) | after 8 weeks of benralizumab treatment | 
| To evaluate the change in asthma control after initiation of benralizumab in a real-world Taiwan setting | Secondary outcome measure: Changes from baseline in ACQ-5 scored from 0 (totally controlled) to 6 (severely uncontrolled) | after 1, 2, 3, 4, 24, and 56 weeks of benralizumab treatment | 
| To evaluate the change in asthma control after initiation of benralizumab in a real-world Taiwan setting | Secondary outcome measure: Percentage of patients with an improvement of ≥ 0.5 points (minimal clinically importance differences [MCID]) in ACQ-5 scored from 0 (totally controlled) to 6 (severely uncontrolled) | at 1, 2, 3, 4, 8, 24, and 56 weeks compared to baseline | 
| To evaluate the change in asthma control after initiation of benralizumab in a real-world Taiwan setting | Percentage of patients with well-controlled asthma (ACQ-5 ≤ 0.75), partly controlled asthma (ACQ-5 between > 0.75 and < 1.5) and not well-controlled asthma (ACQ-5 ≥ 1.5) scored from 0 (totally controlled) to 6 (severely uncontrolled) | at 1, 2, 3, 4, 8, 24, and 56 weeks of benralizumab treatment | 

Secondary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| To assess change in overall asthma status and disease severity after initiation of benralizumab | Patient Global Impression of Change (PGI-C) response and PGI-C responder endpoint (a little better, moderately better, much better) The PGI-C is a single item designed to capture the participant's perception in change in overall disease status since the first dose of benralizumab using a 7-point scale (1- much better to 7- much worse) | at Week 1, 2, 3, 4, 8, 24, and 56 in asthma | 
| To assess change in overall asthma status and disease severity after initiation of benralizumab | Patient Global Impression of Severity (PGI-S) in asthma The PGI-S is a single question designed to capture patient's perception of overall symptom severity on a scale of no symptom to very severe | Changes from baseline after 1, 2, 3, 4, 8, 24, and 56 weeks | 
| To determine the change on lung function after treatment with benralizumab | Pre-bronchodilator changes in FEV1 and FVC assessed by standard hospital spirometry (if available) | after 24 and 56 weeks of treatment with benralizumab | 
| To assess the rate and change of acute exacerbations after initiation of benralizumab in a real-world Taiwan setting | Annualized acute exacerbation rate and changes | from baseline at Week 24 and 56 | 
| To assess the rate and change of acute exacerbations after initiation of benralizumab in a real-world Taiwan setting | Proportion of patients with 0, 1, and ≥ 2 acute exacerbations | at Week 24 and 56 | 
| To assess the rate and change of acute exacerbations after initiation of benralizumab in a real-world Taiwan setting | Severity of exacerbation (use or temporary increase of systemic corticosteroids, emergency department visit, or hospitalization) | at Week 24 and 56 | 
| To assess the ability to reduce OCS dose after initiation of benralizumab in a real-world Taiwan setting | Mean and median OCS daily dose reductions | at Week 4, 8, 24, and 56 | 
| To assess the ability to reduce OCS dose after initiation of benralizumab in a real-world Taiwan setting | Proportion of patients with a ≥ 25%, ≥ 50%, ≥ 75%, and 100% OCS daily dose reduction | at Week 4, 8, 24, and 56 | 
| To assess the ability to reduce OCS dose after initiation of benralizumab in a real-world Taiwan setting | Changes from baseline in cumulated OCS dose | at Week 4, 8, 24, and 56 | 
| To describe characteristics of patients with benralizumab treatment in a real-world Taiwan setting | Baseline asthma disease history and commodities | known at the time of baseline data collection | 
| To describe characteristics of patients with benralizumab treatment in a real-world Taiwan setting | Background medication | at baseline and at Week 4, 8, 24, and 56 | 
| To describe characteristics of patients with benralizumab treatment in a real-world Taiwan setting | Biomarker status (blood eosinophil and serum IgE level, if available) | at baseline and Week 4, 8, 24, and 56 | 
| To describe characteristics of patients with benralizumab treatment in a real-world Taiwan setting | Adherence to benralizumab scheduled dose during study period and investigator-chosen reasons for discontinuation | through study completion, up to 56 weeks | 
| To describe characteristics of patients with benralizumab treatment in a real-world Taiwan setting | Most recent pre-bronchodilator FEV1 and FVC assessed by standard hospital spirometry | in the past 12 months |