Establishing New Standards in Hereditary Angioedema: Improving Outcomes Through Routine Prophylaxis

Abstract

Hereditary angioedema (HAE) is a rare but debilitating and potentially fatal disease that presents in various forms, and can be difficult to manage. Its underlying cause is mutations in a gene that, through its protein product, controls production of the tissue enzyme kallikrein and a peptide mediator, bradykinin. The resulting overproduction of bradykinin leads to increased vascular permeability and oedema. Patients experience episodes of swelling that are unpredictable in their timing, location, and severity, and significantly affect quality of life (QoL), both physically and psychologically.EMJ spoke to Sorena Kiani, Consultant Immunologist at the Barts Health NHS Trust (Barts), London, UK, who manages the UK's largest HAE patient cohort, ahead of a symposium held at the European Academy of Allergy and Clinical Immunology (EAACI) Hybrid Congress 2022 in Prague, Czechia. He explained how new specific medications to control and prevent HAE attacks, developed in the last decade, are giving clinicians and patients the opportunity to review treatment goals and raise expectations. Three prophylactic medications are now licenced for use and recommended in international treatment guidelines, which effectively prevent future HAE attacks.Kiani describes clinical and real-world data from UK clinics that demonstrate how berotralstat, the first orally-administered HAE prophylaxis to be licenced, can reduce the number, duration, and severity of HAE attacks, resulting in measurable improvements in patients' QoL. He also discusses the importance of shared decision-making in developing individualised treatment plans that take into account patient goals and expectations. Kiani is confident that with wider access to prophylaxis, and appropriate monitoring and support, many more HAE patients can expect to live attack-free, with a normalised QoL.