Hereditary angioedema (HAE) in children and adolescents—a consensus on therapeutic strategies

TLDR

Hereditary angioedema caused by C1‑inhibitor deficiency is a rare, potentially life‑threatening disease that typically presents in childhood or adolescence, and early diagnosis is essential to avoid ineffective treatments and to manage swelling attacks, though existing recommendations cannot be directly applied in German‑speaking countries because of drug approval constraints. The article aims to review available HAE therapies, their approval status, and study outcomes in adults and children. At a 2011 consensus meeting, pediatricians and dermatologists from Germany, Austria, and Switzerland reviewed the literature and international consensus recommendations for HAE therapy across all age groups. The authors recommend plasma‑derived C1‑inhibitor concentrate as the preferred treatment for acute attacks and for short‑term and long‑term prophylaxis of pediatric HAE in German‑speaking countries.

Abstract

Hereditary angioedema due to C1 inhibitor (C1 esterase inhibitor) deficiency (types I and II HAE-C1-INH) is a rare disease that usually presents during childhood or adolescence with intermittent episodes of potentially life-threatening angioedema. Diagnosis as early as possible is important to avoid ineffective therapies and to properly treat swelling attacks. At a consensus meeting in June 2011, pediatricians and dermatologists from Germany, Austria, and Switzerland reviewed the currently available literature, including published international consensus recommendations for HAE therapy across all age groups. Published recommendations cannot be unconditionally adopted for pediatric patients in German-speaking countries given the current approval status of HAE drugs. This article provides an overview and discusses drugs available for HAE therapy, their approval status, and study results obtained in adult and pediatric patients. Recommendations for developing appropriate treatment strategies in the management of HAE in pediatric patients in German-speaking countries are provided.Conclusion Currently, plasma-derived C1 inhibitor concentrate is considered the best available option for the treatment of acute HAE-C1-INH attacks in pediatric patients in German-speaking countries, as well as for short-term and long-term prophylaxis.