D1.333 - Digital clinical registry pilot for hereditary angioedema: standardized monthly real-world monitoring of C1 inhibitor deficiency in Serbia

Hereditary angioedema due to C1 inhibitor deficiency (C1-INH-HAE) is a rare autosomal dominant disorder characterized by an unpredictable disease course. In routine clinical practice, infrequent assessments may fail to capture temporal variability in attack burden, treatment use, and quality of life. Digital clinical registries may facilitate standardized longitudinal data collection and improve care continuity across centers.

Objective: To describe the implementation of the Digital Clinical HAE Registry Pilot (D-E-E-P) enabling continuous real-world monitoring of patients with C1-INH-HAE (HAE type I and type II) in Serbia and to report early operational outcomes.

The Digital Clinical HAE Registry Pilot is a web-based registry using predefined electronic case report forms to capture structured data during standardized monthly physician–patient interactions. Monitoring includes monthly assessment of attack frequency, localization, severity, and treatment utilization. Health-related quality of life is assessed using the Angioedema Quality of Life Questionnaire (AE-QoL) twice yearly and the Angioedema Control Test (AECT) four times yearly, integrated into routine follow-up. Individual patient reports are generated at each visit, with aggregated anonymized reports compiled periodically for evaluation of disease course. Access to individual patient data is restricted to the supervising physician. Participation is voluntary and based on written informed consent.

The registry was initiated in January 2025 and incorporates retrospective data from a structured patient support program conducted between September 2020 and 2024. To date, 73 patients with C1-INH-HAE are enrolled, monitored by 13 physicians from 8 healthcare institutions across Serbia, ensuring broad geographic representation. Across the combined program and registry period, 64 months of observation have been accrued, comprising 3,045 monthly physician–patient interactions, 456 AE-QoL questionnaires, and 912 AECT assessments.

This digital registry pilot establishes a scalable national framework for continuous, standardized real-world monitoring of C1-INH-HAE. It enables longitudinal assessment of disease burden and supports evidence-based clinical decision-making, timely follow-up, and continuity of care across multiple centers.