Greve Jens, Strassen Ulrich, Gorczyza Marina, Dominas Nina, Frahm Uta-Marie, Mühlberg Heike, Wiednig Michaela, Zampeli Vasiliki, Magerl Markus
Department of Oto-Rhino-Laryngology, Head and Neck Surgery, Ulm University Medical Center, Ulm, Germany.
Department of Otorhinolaryngology, Head and Neck Surgery, Technical University of Munich, Germany.
J Dtsch Dermatol Ges. 2016 Mar;14(3):266-75. doi: 10.1111/ddg.12856.
Hereditary angioedema (HAE) is a rare congenital disorder characterized by recurrent episodes of subcutaneous or submucosal edema. Laryngeal manifestations can be life-threatening. In the majority of cases, the disease can be adequately treated with an on-demand approach--in some cases, however, short- or long-term prophylaxis is indicated. Attenuated androgens used to be the drugs of choice, but they are associated with considerable side effects and no longer commercially available in the German-speaking countries of the EU. They are currently being replaced by more effective and more tolerable agents such C1-inhibitors, the kallikrein inhibitor ecallantide, and the B2 receptor antagonist icatibant, which have recently obtained market authorization. These new drugs have had a major impact, especially on the indications and procedures for long-term prophylaxis. According to the most recent international consensus papers and our own experience, self-administered C1-inhibitors are now the first option for long-term prophylactic therapy. The decision for prophylaxis should no longer be based on single parameters such as the frequency of attacks but on adequate overall disease control including quality of life. More drugs are currently being developed, which may lead to further changes in the treatment algorithms of HAE.
遗传性血管性水肿（HAE）是一种罕见的先天性疾病，其特征为皮下或黏膜下水肿反复发作。喉部表现可能危及生命。在大多数情况下，该病可采用按需治疗方法进行充分治疗——然而，在某些情况下，需要进行短期或长期预防。减毒雄激素曾是首选药物，但它们伴有相当多的副作用，在欧盟德语国家已不再有商业供应。目前，它们正被更有效、耐受性更好的药物所取代，如C1抑制剂、激肽释放酶抑制剂依库珠单抗和B2受体拮抗剂艾替班特，这些药物最近已获得市场授权。这些新药产生了重大影响，尤其是对长期预防的适应症和程序。根据最新的国际共识文件和我们自己的经验，自我注射C1抑制剂现在是长期预防性治疗的首选。预防决策不应再基于单一参数，如发作频率，而应基于包括生活质量在内的充分的整体疾病控制。目前正在研发更多药物，这可能会导致HAE治疗方案的进一步改变。
