Eosinophilic Granulomatosis With Polyangiitis (EGPA)
Conditions
Keywords
Eosinophilic Granulomatosis With Polyangiitis (Churg-Strauss), EGPA, Real-world study, Benralizumab, Vasculitis
Brief summary
This is a multicenter, retro-prospective observational study evaluating the effectiveness and safety of benralizumab in adult patients with eosinophilic granulomatosis with polyangiitis (EGPA) in a real-world setting. The study will include patients treated with benralizumab 30 mg every 4 weeks at EGPA referral centers participating in the European EGPA Study Group. Clinical, laboratory, lung function, treatment, relapse, and safety data will be collected from medical charts at baseline and during follow-up up to 24 months.
Detailed description
BELONG-EGPA is a multicenter, retro-prospective observational study conducted within centers belonging to the European EGPA Study Group. The study is designed to evaluate the long-term real-world effectiveness and safety of benralizumab in adult patients with eosinophilic granulomatosis with polyangiitis (EGPA). The study population will include adult patients with EGPA who meet the 2022 American College of Rheumatology/European Alliance of Associations for Rheumatology classification criteria for EGPA or the criteria proposed in the MIRRA trial, and who receive benralizumab 30 mg every 4 weeks. Only patients with at least 3 months of available follow-up after starting benralizumab will be included. Demographic, clinical, biological, lung function, and treatment-related data will be collected from medical charts through a standardized electronic case report form. Data will be collected at the start of benralizumab treatment and at 3, 6, 12, and 24 months of follow-up. The primary effectiveness outcomes include achievement of complete response and partial response, changes in lung function measured by pre-bronchodilator FEV1, and disease relapses. Complete response is defined as no disease activity, with Birmingham Vasculitis Activity Score equal to 0, and an oral corticosteroid dose of 4.0 mg/day or less of prednisone, prednisolone, or equivalent. Partial response is defined as no disease activity with an oral corticosteroid dose greater than 4.0 mg/day. Secondary outcomes include treatment persistence, oral corticosteroid tapering and discontinuation, effectiveness and safety in first-line patients compared with patients previously exposed to mepolizumab, outcomes after benralizumab dosage switching, and adverse events and serious adverse events during treatment. Additional outcomes include changes in organ manifestations, discontinuation of disease-modifying antirheumatic drugs, changes in ANCA status, and variation in eosinophil count. All data will be anonymized and stored in a centralized database.
Interventions
BIOLOGICALBenralizumab 30 mg/ml
Benralizumab administered at a dose of 30 mg every 4 weeks in adult patients with eosinophilic granulomatosis with polyangiitis (EGPA) in a real-world setting. Patients may receive benralizumab as first-line biologic therapy or after previous exposure to mepolizumab. Treatment use, persistence, dosage changes, effectiveness outcomes, and safety events will be assessed during follow-up.
Sponsors
European EGPA Study Group
Study design
Observational model
COHORT
Time perspective
OTHER
Eligibility
Sex/Gender
ALL
Age
18 Years to No maximum
Healthy volunteers
No
Inclusion criteria
* Age 18 years or older. * Diagnosis of eosinophilic granulomatosis with polyangiitis (EGPA) according to the 2022 American College of Rheumatology/European Alliance of Associations for Rheumatology classification criteria or the criteria proposed in the MIRRA trial. * Treatment with benralizumab 30 mg every 4 weeks. * At least 3 months of available follow-up data after starting benralizumab. * Written informed consent for study participation and data collection.
Exclusion criteria
* Age younger than 18 years. * No available follow-up data after starting benralizumab. * Follow-up after benralizumab initiation shorter than 3 months. * Lack of written informed consent, where required by local regulations.
Design outcomes
Primary
| Measure | Time frame | Description |
|---|---|---|
| Complete Response | Baseline and up to 24 months (at 3, 6, 12, 24 months) | Proportion of patients achieving complete response during follow-up. Complete response is defined as no disease activity (BVAS=0) and oral corticosteroid dose ≤4.0 mg/day. |
| Change in Lung Function | Baseline, 3, 6, 12, and 24 months | Change in pre-bronchodilator forced expiratory volume in 1 second (FEV1) from baseline to each follow-up time point. Unit of Measure: Milliliters |
| Disease relapse | 3, 6, 12, and 24 months | Proportion of patients experiencing disease relapse after achieving complete response. Relapse is defined as active vasculitis, BVAS \>0, and/or worsening asthma or ear-nose-throat manifestations leading to an increase in oral corticosteroid dose to \>4.0 mg/day, initiation of a new immunosuppressive therapy, or hospitalization. |
| Partial response | Baseline and up to 24 months (at 3, 6, 12, 24 months) | Proportion of patients achieving complete response during follow-up. Partial response is defined as no disease activity (BVAS=0) and oral corticosteroid dose \>4.0 mg/day. |
Secondary
| Measure | Time frame | Description |
|---|---|---|
| Treatment persistence | 3, 6, 12, and 24 months | Proportion of patients who continue benralizumab treatment, switch benralizumab dosage, or discontinue benralizumab for any reason. |
| Change in oral corticosteroid dose expressed as prednisone-equivalent dose | Baseline, 3, 6, 12, and 24 months | The daily dose of each oral corticosteroid will be converted to prednisone-equivalent mg/day using standardized glucocorticoid equivalence factors. Unit of Measure: mg/day of prednisone equivalent |
| Oral corticosteroid discontinuation | 3, 6, 12, and 24 months | Proportion of patients who discontinue oral corticosteroid therapy during follow-up. |
| Overall clinical benefit in first-line versus post-mepolizumab patients | Baseline, 3, 6, 12, and 24 months | A comparison of the overall clinical benefit in patients treated with benralizumab as first-line therapy versus patients switching to benralizumab following prior exposure to mepolizumab. Unit of Measure: Percentage of participants |
| Effectiveness after benralizumab dosage switching | 3 months after dosage switch | Assessment of complete response, partial response, and changes in lung function in patients switching benralizumab dosage from 30 mg every 4 weeks to 30 mg every 8 weeks or from 30 mg every 8 weeks to 30 mg every 4 weeks. |
| Adverse event rate | 3, 6, 12, and 24 months | Proportion of patients experiencing at least one adverse event during benralizumab treatment, regardless of causal association with treatment. |
Countries
Italy
Contacts
CONTACTGiacomo Emmi, MD/PhD
CONTACTIrene Mattioli, PharmD/PhD
PRINCIPAL_INVESTIGATORGiacomo Emmi, MD/PhD
University of Trieste, Italy
Outcome results
None listed