Bradykinin-mediated angioedema The path towards complete disease control

This thesis examines various aspects of bradykinin-mediated angioedema, focusing on hereditary angioedema (HAE) and angioedema due to acquired C1-inhibitor deficiency. It is divided into three parts: Part I explores innovative treatments targeting the kallikrein-kinin pathway, showing promising results in reducing angioedema attacks with therapies like donidalorsen, NTLA-2002 and deucrictibant. Part II addresses the inconsistency in efficacy outcomes used in clinical trials assessing HAE treatment, establishing consensus on a core outcome set for future studies. Part III investigates residual risks associated with C1-inhibitor deficiency, such as potential thromboembolic complications, and the impact of HAE on reproductive decisions. The overall goal is to enhance treatment strategies, standardize clinical trial outcomes, and address broader implications of the disease, ultimately improving the quality of life for patients with bradykinin-mediated angioedema.