Department of Health Sciences, Section of Dermatology, University of Florence, Florence, Italy -
Department of Health Sciences, Section of Dermatology, University of Florence, Florence, Italy.
Ital J Dermatol Venerol. 2021 Apr;156(2):161-173. doi: 10.23736/S2784-8671.20.06694-8. Epub 2020 Oct 5.
Pemphigus is a rare autoimmune disease of the skin, characterized by autoantibodies targeting adhesion proteins of the epidermis, in particular desmoglein 3 and desmoglein 1, that cause the loss of cell-cell adhesion and the formation of intraepidermal blisters. Given that these autoantibodies are both necessary and sufficient for pemphigus to occur, the goal of pemphigus therapy is the elimination of autoreactive B-cells responsible for autoantibody production. Rituximab, an anti-CD20 monoclonal antibody, was the first targeted B-cell therapy approved for use in pemphigus and is now considered the frontline therapy for new onset disease. One limitation of this treatment is that it targets both autoreactive and non -autoreactive B-cells, which accounts for the increased risk of serious infections in treated patients. In addition, most rituximab-treated patients experience disease relapse, highlighting the need of new therapeutic options. This review provides a concise overview of rituximab use in pemphigus and discusses new B-cell and antibody-directed therapies undergoing investigation in clinical studies.
天疱疮是一种罕见的皮肤自身免疫性疾病，其特征是针对表皮黏附蛋白的自身抗体，特别是桥粒芯糖蛋白 3 和桥粒芯糖蛋白 1，导致细胞间黏附丧失和表皮内水疱形成。鉴于这些自身抗体对于天疱疮的发生既必要又充分，天疱疮治疗的目标是消除负责产生自身抗体的自身反应性 B 细胞。利妥昔单抗是一种抗 CD20 单克隆抗体，是第一种被批准用于天疱疮的靶向 B 细胞治疗药物，目前被认为是新发疾病的一线治疗药物。这种治疗方法的一个局限性是它针对的是自身反应性和非自身反应性 B 细胞，这导致了治疗患者严重感染的风险增加。此外，大多数接受利妥昔单抗治疗的患者会出现疾病复发，这凸显了需要新的治疗选择。本文简要概述了利妥昔单抗在天疱疮中的应用，并讨论了正在临床研究中进行的新的 B 细胞和抗体靶向治疗。
