Acute Treatment Assessment in Hereditary Angioedema

Hereditary Angioedema (HAE) is a rare genetic disorder characterized by recurrent episodes of swelling in the skin and mucosal tissues, often due to a deficiency or dysfunction of the C1 inhibitor protein. While the condition is rare, its unpredictable and potentially life-threatening nature-particularly when attacks affect the upper airways-emphasizes the necessity of prompt and effective treatment. Even patients receiving prophylactic therapy require access to acute interventions to minimize attack severity, frequency and the overall burden of the disease. However, evaluating the efficacy of these acute treatments remains a significant challenge due to the variability and complexity of HAE attacks. Evaluating acute treatment efficacy in HAE is inherently difficult, given the wide variation in the manifestation of attacks. Differences in attack location, severity, duration and symptoms both across individuals and within the same patient over time further complicate assessments. While tools like the Angioedema Activity Score (AAS), Angioedema Control Test (ACT) and Angioedema Quality of Life Questionnaire (AE-QoL) have standardized assessments for overall disease burden and prophylactic therapy, similar standardized metrics are lacking for acute treatment evaluation.

Author(s): Danny Jean

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