Abulikemu Kailibinuer, Hu Fengxia, Liang Junqin, Kang Xiaojing
Department of Dermatology and Venereology, People's Hospital of Xinjiang Uygur Autonomous Region; Xinjiang Clinical Research Center for Dermatologic Diseases; Xinjiang Key Laboratory of Dermatology Research (XJYS1707), Urumqi 830002, China.
Heliyon. 2023 May 25;9(6):e16679. doi: 10.1016/j.heliyon.2023.e16679. eCollection 2023 Jun.
Pemphigus is a heterogeneous group of autoimmune skin disorders characterized by blistering of the skin and mucosal membranes, potentially affecting the quality of life if left unchecked. The current mainstay of treatment is systemic corticosteroids and immunosuppressive agents. Nevertheless, long-term use of these drugs can easily cause infections and other life-threatening adverse reactions. Thus, currently, researchers are trying to develop new and safer therapeutic approaches. Specifically, targeted therapies to pathogenic immune pathways have been gradually introduced and used for the treatment of pemphigus or in clinical trials, such as monoclonal anti-CD20 antibody, BAFF inhibitor, BTK inhibitor, CAAR-T therapy, FcRn antagonist, and TNF-α inhibitor. In addition, IL-4Rα antibody, IL-17 blockade, mTOR pathway inhibitor, CTLA-4Ig, and p38 MAPK inhibitors are theoretically promising treatment for pemphigus. Here, we review the research progress on the mechanism of targeted therapies for pemphigus.
天疱疮是一组异质性自身免疫性皮肤病，其特征为皮肤和黏膜出现水疱，若不加以控制，可能会影响生活质量。目前的主要治疗方法是全身使用皮质类固醇和免疫抑制剂。然而，长期使用这些药物容易引发感染及其他危及生命的不良反应。因此，目前研究人员正试图开发新的、更安全的治疗方法。具体而言，针对致病免疫途径的靶向治疗已逐渐被引入并用于治疗天疱疮或处于临床试验阶段，如单克隆抗CD20抗体、BAFF抑制剂、BTK抑制剂、嵌合抗原受体T细胞（CAR-T）疗法、新生儿Fc受体（FcRn）拮抗剂和肿瘤坏死因子-α（TNF-α）抑制剂。此外，白细胞介素-4受体α（IL-4Rα）抗体、白细胞介素-17阻断剂、雷帕霉素靶蛋白（mTOR）途径抑制剂、细胞毒性T淋巴细胞相关抗原4免疫球蛋白（CTLA-4Ig）和p38丝裂原活化蛋白激酶（p38 MAPK）抑制剂在理论上是有前景的天疱疮治疗方法。在此，我们综述天疱疮靶向治疗机制的研究进展。
