预约演示

更新于：2025-05-07

Pemphigus Vulgaris, Familial

家族性寻常型天疱疮

更新于：2025-05-07

基本信息

别名

Familial pemphigus vulgaris、PEMPHIGUS VULGARIS, FAMILIAL、Pemphigus vulgaris, familial

+ [1]

简介-

关联

项与家族性寻常型天疱疮相关的药物

Rituximab

利妥昔单抗

靶点

CD20

作用机制

CD20抑制剂 [+3]

在研机构

原研机构

在研适应症

非在研适应症

最高研发阶段批准上市

首次获批国家/地区

美国

首次获批日期1997-11-26

DSG3-CAART

靶点

DSG3

作用机制

DSG3调节剂

在研机构

Cabaletta Bio, Inc.

原研机构

Cabaletta Bio, Inc.

在研适应症

家族性寻常型天疱疮

非在研适应症-

最高研发阶段临床1/2期

首次获批国家/地区-

首次获批日期1800-01-20

100 项与家族性寻常型天疱疮相关的临床结果

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100 项与家族性寻常型天疱疮相关的转化医学

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0 项与家族性寻常型天疱疮相关的专利（医药）

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项与家族性寻常型天疱疮相关的文献（医药）

2025-03-26·Clinical and Experimental Dermatology

Refractory benign familial pemphigus successfully treated with dupilumab: a case series

Article

作者: Simon Gozalbo, Ana ; Martin Diaz, Maria A ; Carrascosa de Lome, Raquel ; de la Cueva Dobao, Pablo ; Mauleon Fernandez, Cristina

AbstractBenign familial pemphigus (BFP) is a chronic autosomal dominant genodermatosis characterized by the appearance of flaccid blisters that progress to painful erythematous macerated plaques and erosions in intertriginous areas. Although various treatments for BFP exist, current treatments are not consistently effective, and BFP symptoms often cause a decreased quality of life. We report a case series of three patients with refractory moderate-to-severe BFP who were successfully treated with dupilumab. We also describe their clinical evolution over a follow-up period of 18 months. All patients reported a decrease in pruritus and improved skin findings, with no adverse side-effects. However, increased dosing was required in one patient to achieve optimal control of the disease. We also discuss previously reported cases of BFP treated with biologics. Dupilumab appears to be a useful and safe treatment option for patients with refractory BFP.

2025-03-01·Pediatric Dermatology

Pemphigus Vulgaris With Esophageal Involvement in an Atopic Child Successfully Treated With Dupilumab

Article

作者: Leiferman, Kristin M. ; Milner, Joshua D. ; Yun, Sonora ; Lauren, Christine T. ; Scollan, Margaret E. ; Jenkins, Blair A. ; Defelice, Amy R.

ABSTRACTWe report the case of a 3‐year‐old boy who was diagnosed with childhood pemphigus vulgaris having developed oral lesions, gastrointestinal symptoms with esophageal involvement, and failure to thrive. He had a markedly increased total serum IgE level and peripheral blood eosinophilia. The pemphigus was recalcitrant to conventional therapies and, based on the coexisting characteristics of Th2 immune deviation, he was treated with dupilumab and has had sustained clinical improvement since starting treatment. The case illustrates the importance of recognizing pemphigus vulgaris in childhood, considerations for dupilumab therapy, and a potential pathophysiological links between pemphigus autoantibodies in early life, Th2 inflammation, and atopic disorder.

2022-11-01·Pediatric Dermatology

Management of pediatric pemphigus vulgaris with rituximab: A case report and review of the literature

Review

作者: Mistry, Bhavik Dipak ; Levy, Rebecca ; Lee, David Michael ; Leis, Maria

AbstractPemphigus vulgaris (PV), an acquired autoimmune bullous disease, is caused by autoantibodies targeting desmosomal proteins in the skin and mucous membranes. Recent data from the adult PV population supports the use of rituximab, a chimeric anti‐CD20 IgG1 antibody, as a primary treatment strategy, but limited data exist regarding treatment in the pediatric population. We report the case of a 13‐year‐old male with PV treated successfully with systemic corticosteroids and rituximab, and review the literature supporting the treatment of pediatric PV with rituximab.

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