Management of hereditary angioedema in the UK: a comparison with international guidelines.

Abstract

Hereditary angioedema (HAE) is a rare genetic disorder characterized by recurrent episodes of swelling, which can potentially be life-threatening, resulting in substantial physical, psychological, educational, and occupational burden. Treatment of HAE is divided into on-demand treatment for acute attacks and long-term prophylaxis for prevention of attacks, and there are now several effective therapies available. However, in the UK, use of on-demand therapy is intended for attacks defined as clinically significant, and access to long-term prophylaxis is determined primarily by frequency-based criteria. In this policy-focused review, we compare UK access criteria with international guidelines on best practice. We conclude that UK access criteria result in a group of HAE patients with persisting unmet need, and this may have a greater impact in children and young people. In view of this, we recommend greater flexibility in access criteria, allowing clinical judgement, shared decision-making, and broader measures of disease burden to inform treatment eligibility, so we can individualize care and fully meet the needs of these patients.