Olbrich Henning, Sadik Christian D, Schmidt Enno
Department of Dermatology, University of Lübeck, Lübeck, Germany.
Lübeck Institute of Experimental Dermatology, University of Lübeck, Lübeck, Germany.
Expert Opin Investig Drugs. 2023 Jul-Dec;32(7):615-623. doi: 10.1080/13543784.2023.2242778. Epub 2023 Aug 7.
Treatment options for autoimmune bullous diseases (AIBD) are currently limited to corticosteroids and traditional immunomodulants and immunosuppressants that are associated with unfavorable adverse effect profiles. The most frequent AIBDs, i.e. bullous pemphigoid, pemphigus vulgaris, and mucous membrane pemphigoid, impose a high disease burden onto affected patients and can be detrimental due to infections, exsiccosis, and impaired food intake. Significant progress has been made in elucidating disease mechanisms and key mediators by in vivo and in vitro models, thus identifying a multifaceted range of possible drug targets. However, except for rituximab for pemphigus vulgaris, no new drugs have been approved for the treatment of AIBDs in the last decades.
This review covers new drug developments and includes ongoing or completed phase 2 and 3 clinical trials. Studies were identified by querying the registries of ClinicalTrials.gov and Cochrane Library.
Promising results were shown for a variety of new agents including nomacopan, efgartigimod, omalizumab, dupilumab, as well as chimeric autoantibody receptor T cells. Clinical translation in the field of AIBDs is highly active, and we anticipate significant advances in the treatment landscape.
自身免疫性大疱性疾病（AIBD）的治疗选择目前仅限于皮质类固醇以及与不良副作用相关的传统免疫调节剂和免疫抑制剂。最常见的AIBD，即大疱性类天疱疮、寻常型天疱疮和黏膜类天疱疮，给受影响的患者带来了很高的疾病负担，并且可能因感染、脱水和食物摄入受损而产生不利影响。通过体内和体外模型在阐明疾病机制和关键介质方面取得了重大进展，从而确定了多方面的可能药物靶点。然而，除了用于寻常型天疱疮的利妥昔单抗外，在过去几十年中没有新的药物被批准用于治疗AIBD。
本综述涵盖新药研发，包括正在进行或已完成的2期和3期临床试验。通过查询ClinicalTrials.gov和Cochrane图书馆的注册信息来确定研究。
包括诺马卡潘、艾加莫德、奥马珠单抗、度普利尤单抗以及嵌合自身抗体受体T细胞在内的多种新药都显示出了有前景的结果。AIBD领域的临床转化非常活跃，我们预计治疗前景将取得重大进展。
