Effectiveness of Berotralstat▼ for Hereditary Angioedema Prophylaxis

Hereditary angioedema (HAE) attacks can be debilitating and potentially life-threatening, particularly when they affect the abdomen or airways. Recently targeted prophylactic medications have been developed and licensed and treatment practices have evolved. Long-term prophylaxis (LTP) is recommended in international guidelines to reduce attack frequency and improve quality of life (QoL) for patients. EMJ spoke to HAE experts Isabelle Boccon-Gibod and Sorena Kiani about their clinical experience of treating patients with HAE and with first-line prophylaxis, in particular oral berotralstat. In clinical trials, berotralstat has been shown to reduce attack rates by over 90% and improve patient-reported outcomes, including reduced symptom severity. Real-world data further supports its long-term effectiveness and tolerability, although some patients discontinue medication due to adverse effects or insufficient efficacy. Boccon-Gibod and Kiani emphasised the importance of shared decision making between healthcare professionals and patients with HAE when choosing LTP treatments, and explained that patients are more likely to continue with their medication and be able to normalise their lives when they feel supported and are well-informed about medications and potential side-effects. Regular follow-ups and education, along with addressing potential side effects, are crucial to ensuring treatment success.