Current status of the management of hereditary angioedema in China: a patient-based, cross-sectional survey

Background Hereditary angioedema (HAE) is a rare, inherited disorder characterized by unpredictable and recurrent cutaneous and mucosal oedema. Objectives This study aimed to identify the current status of HAE management in China. Materials and Methods An internet-based survey was sent to 129 patients with type 1 and 2 HAE diagnosed between 1983 and 2017; a total of 107 patients provided complete disease management information. Results The survey response rate was 82.9% (107 responders). Ten patients reported 18 fresh frozen plasma (FFP) transfusions for the main purpose of treating lethal laryngeal oedema. Two patients reported adverse events. Eighty-nine (83.2%) patients had taken danazol for long-term prophylaxis, and 74 (69.2%) were on long-term danazol prophylaxis. Among patients on long-term prophylaxis, 56 (75.7%) patients reported a decrease in their annual attack frequency after taking danazol. Twenty-five (33.8%) patients had an attack frequency of no more than once per year. The most common side effects were menstrual disorders, weight gain, osteoarticular pain, acne, and sebaceous hypersecretion. Patients with higher education levels, positive family histories, and laryngeal oedema attacks before medication tended to show better adherence. Patients maintaining high or medium adherence showed better control of laryngeal and gastrointestinal involvement than patients with low adherence. Four (3.7%) patients reported current or past use of tranexamic acid. Conclusion Attenuated androgen and FFP transfusions remain the mainstay in China, where specifically targeted drugs are currently lacking. Maintaining good medication adherence should be recommended to achieve improved disease control.