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

被引:0
作者
Fasshauer, Maria [1 ]
Wedi, Bettina [2 ]
机构
[1] ImmunDefektCentrum Leipzig IDCL, Clin Pediat & Adolescent Med, Klinikum St Georg Leipzig, Delitzscher Str 141, D-04129 Leipzig, Germany
[2] Treatment Ctr Hereditary Angioedema MHH, Interdisciplinary Allergy Ctr MHH, Hannover Med Sch MHH, Dept Dermatol Allergol & Venereol, Hannover, Germany
关键词
hereditary angioedema (HAE); children and adolescents; on-demand treatment; long-term prophylaxis; plasma kallikrein and prekallikrein inhibition; factor XIIa inhi- bition; bradykinin B2 receptor (BK2R) inhibition; gene editing; quality of life; EFFICACY; ATTACKS;
D O I
10.5414/ALX02532Ecitation
中图分类号
R392 [医学免疫学];
学科分类号
100102 ;
摘要
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 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.
引用
收藏
页码:336 / 345
页数:10
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