Hereditary angioedema (HAE) in children and adolescents: New treatment options

儿童和青少年遗传性血管性水肿 (HAE):新的治疗选择

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Abstract

Modern management of hereditary angioedema (HAE) due to reduced C1 inhibitor (C1-INH) function or concentration (HAE-C1-INH) focuses on individualized therapeutic strategies to address the specific needs of children and adolescents as well as the severity of the disease. Psychosocial factors such as the burden of disease and therapy on quality of life and participation play an important role. New medications have already significantly improved the prognosis and health related quality of life in HAE patients, but not all of these therapies have yet been approved for children. Further treatment options that inhibit bradykinin effects are currently being investigated. They target factor XIIa, prekallikrein, plasma kallikrein, or the bradykinin B2 receptor. Modern research focuses on oral options or long-acting parenteral therapy approaches to further optimize care and, in particular, the needs of children. There are also initial developments in the field of gene therapy, which could represent a causal treatment option for HAE in the future. This article focuses on the presentation and treatment of HAE type I (reduced C1-INH concentration) and HAE type II (impaired C1-INH function) in children and adolescents. Acquired AE and HAE with normal C1-INH are rare in the pediatric age group and are not discussed in detail here.

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