Background: Hereditary angioedema with C1 inhibitor deficiency (HAE-C1INH) is a rare disease characterized by swelling episodes. It affects quality of life (QoL) and can be lethal, when involving upper airways. Treatment is individualized, with therapeutic options including on-demand treatment (ODT), and short- and long-term prophylaxis (STP, LTP). However, available guidelines are not always clear about treatment selection, its goals or assessment of goal achievement. Objective: To review the available evidence for the management of HAE-C1INH and build a Spanish expert consensus to steer HAE-C1INH management towards a treat-to-target (T2T) approach, while addressing some of the unclear aspects of the Spanish guidelines. Methods: We reviewed the literature on the management of HAE-C1INH with a T2T approach, focusing on: 1) treatment selection and goals; and 2) available tools to assess goal achievement. We discussed the literature based on clinical experience and drew up 45 statements on undefined management aspects. A panel of 53 HAE experts validated the statements through a two-round Delphi process. Results: The goals for ODT and STP are minimizing the morbidity and mortality of attacks, and preventing attacks caused by known triggers, respectively, while the main goal of LTP is to decrease the rate, severity and duration of attacks. Furthermore, when prescribing, clinicians should consider the reduction in adverse events, while increasing patient QoL and satisfaction. Appropriate instruments for assessing goal achievement have also been indicated. Conclusions: We provide recommendations on previously unclear aspects of HAE-C1INH management with ODT, STP and LTP, focusing particularly on clinical and patient-oriented goals.
scite is a Brooklyn-based organization that helps researchers better discover and understand research articles through Smart Citations–citations that display the context of the citation and describe whether the article provides supporting or contrasting evidence. scite is used by students and researchers from around the world and is funded in part by the National Science Foundation and the National Institute on Drug Abuse of the National Institutes of Health.
customersupport@researchsolutions.com
10624 S. Eastern Ave., Ste. A-614
Henderson, NV 89052, USA
This site is protected by reCAPTCHA and the Google Privacy Policy and Terms of Service apply.
Copyright © 2024 scite LLC. All rights reserved.
Made with 💙 for researchers
Part of the Research Solutions Family.