Hereditary angioedema (HAE) in chil-dren and adolescents: New treatment op-tions

被引:0
作者
Fasshauer, M. [1 ]
Wedi, B.
机构
[1] Klinikum St Georg Leipzig, ImmunDefektCtr Leipzig IDCL, Klin Kinder & Jugendmed, Gemass 116 B,SGB 5,Delitzscher Str 141, D-04129 Leipzig, Germany
关键词
hereditary angioedema (HAE); children and adolescents; on-demand treatment; longterm pro- phylaxis; plasma kallikrein and prekallikrein inhibition; factor XIIa inhibition; bradykinin B2 receptor (BK2R) inhibition; gene editing; quality of life; EFFICACY; ATTACKS;
D O I
10.5414/ALX02532E
中图分类号
R392 [医学免疫学];
学科分类号
100102 ;
摘要
Modern management of hereditary an-gioedema (HAE) due to reduced C1 inhibitor (C1-INH) function or concentration (HAE-C1- NH) focuses on individualized therapeutic strategies to address the specific needs of children and adolescents as well as the se-verity 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 al-ready significantly improved the prognosis and health related quality of life in HAE pati-ents, but not all of these therapies have yet been approved for children. Further treat-ment options that inhibit bradykinin effects are currently being investigated. They target factor XIIa, prekallikrein, plasma kallikrein, or the bradykinin B2 receptor. Modern re-search 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 re-present a causal treatment option for HAE in the future.
引用
收藏
页码:681 / 690
页数:10
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