a IRCCS Istituti Clinici Scientifici Maugeri , Italy.
b Department of Biomedical and Clinical Sciences Luigi Sacco , University of Milan , Milan , Italy.
Expert Opin Biol Ther. 2019 Jun;19(6):517-526. doi: 10.1080/14712598.2019.1595581. Epub 2019 Mar 26.
Hereditary angioedema due to C1-INH deficiency (C1-INH-HAE) is a rare disease with unpredictable, self-limiting and localized swelling episodes involving the cutaneous and subcutaneous tissues. In the last decade, the spectrum of the possibilities to control the disease has considerably changed with the development of biologic therapies making necessary a careful evaluation of the differences among current and emerging treatments to properly optimize the management of patients.
This review serves to summarize the literature regarding the use of biologics for the treatment of C1-INH-HAE. Medications already available on the market and new drugs in different phases of development are addressed.
The advent of biologic therapies dramatically improved the lives of patients with C1-INH-HAE although further improvement is still needed. Whether this is cost/effective will be answered in the next years when we will see if these major advances will benefit the majority of the patients.
由于 C1 酯酶抑制剂（C1-INH）缺乏引起的遗传性血管性水肿（C1-INH-HAE）是一种罕见疾病，具有不可预测、自限性和局限性肿胀的特点，涉及皮肤和皮下组织。在过去的十年中，随着生物疗法的发展，控制疾病的可能性范围发生了很大变化，因此需要仔细评估当前和新兴治疗方法之间的差异，以正确优化患者的管理。
本文综述了生物制剂治疗 C1-INH-HAE 的文献，涉及已上市的药物和不同开发阶段的新药。
生物疗法的出现极大地改善了 C1-INH-HAE 患者的生活，但仍需要进一步改善。当我们看到这些重大进展是否将使大多数患者受益时，未来几年将回答这是否具有成本效益。
