寻常型天疱疮
Pemphigus vulgaris
定义 英文原文（暂无中文）
A rare autoimmune bullous skin diseases characterized by painful, flaccid blisters and erosions of the oral mucosa, predominantly involving the buccal area, and with or without extension to the epidermis. Mucosa of the larynx, oesophagus, conjunctiva, nose, genitalia and anus, are less frequently affected.
基本事实
- 遗传方式
- 不适用
- 发病年龄
- 成年期、儿童期、老年期
- 患病率
- 1-9 / 1 000 000（France）
临床表型 20
极常见 99–80%10
- 皮肤棘层松解 HP:0100792
- 非典型皮肤瘢痕 HP:0000987
- 自身免疫 HP:0002960
- 口腔黏膜糜烂 HP:0031446
- 婴儿期喂养困难 HP:0008872
- 口腔粘膜水泡 HP:0200097
- 复发性皮肤脓肿 HP:0100838
- 反复感染 HP:0002719
- 荨麻疹 HP:0001025
- 体重减轻 HP:0001824
常见 79–30%7
- 皮肤的异常起疱 HP:0008066
- 抗桥粒黏蛋白-1抗体阳性 HP:4000013
- 抗桥粒黏蛋白-3抗体阳性 HP:4000014
- 焦虑 HP:0000739
- 抑郁 HP:0000716
- 喂养困难 HP:0011968
- 基底上层裂隙 HP:0034194
偶见 29–5%2
- 头皮脱发 HP:0002293
- 疼痛 HP:0012531
罕见 <4–1%1
- 荨麻疹性斑块 HP:0030351
近两年的全球研究 1,237L2
2024/08 起在 Europe PMC 检索所得，按发表时间倒序显示最近 20 篇。标题未译成中文——自动翻译需要接入 LLM 服务，尚未引入。
- 2026-08MiR-125b-5p promotes pemphigus vulgaris pathogenesis by modulating desmosome structures
- 2026-07A Case Report of a Patient With Recalcitrant Pemphigus Vulgaris Treated With Rituximab
- 2026-07综述Periodontal Involvement in Autoimmune Blistering Diseases: A Narrative Review
- 2026-07Dipeptidyl Peptidase-4 Inhibitor Use and Risk of Pemphigus Vulgaris and Foliaceus: A Retrospective Cohort Study Using Databases of the Korea National Health Insurance Claims and the National Health Screening Examination
- 2026-07Pre-Referral Provisional Diagnoses and Final Diagnostic Subtypes of Pemphigus at a Regional Tertiary Dermatology Center in Japan: A Single-Center Retrospective Study
- 2026-07Chemiluminescence Immunoassay and Enzyme-Linked Immunosorbent Assay in the Diagnosis of Pemphigoid and Pemphigus: A Comparative Study
- 2026-07Direct Immunofluorescence Intensity as a Supportive Marker of Clinical Severity in Pemphigus Vulgaris
- 2026-07Evaluating the Effect of the EAT-Lancet Diet on Pemphigus Vulgaris Severity: Insights From a Cross-Sectional Study
- 2026-07系统综述Efficacy and Safety of Rituximab in Pemphigus Foliaceus: A Systematic Review and Meta-analysis
- 2026-07Correlation of Clinical and Histopathological Features With Anti-Desmoglein Antibody Profile in Pemphigus Patients
- 2026-07临床试验CD19 CAR T-cell therapy is feasible for patients with pemphigus vulgaris treated without lymphodepletion in the RESET-PV trial
- 2026-07Pesticide exposure as a risk factor for pemphigus vulgaris: findings from a case-control study conducted in Liguria, Italy
- 2026-07系统综述Pemphigus vegetans: insights from a systematic review of the literature
- 2026-06综述The Oral Cavity as a Window to Systemic Disease: Diagnostic Clues and Pre-treatment Dental Management for Interdisciplinary Trainees
- 2026-06Mechanistic modeling of FcRn-dependent IgG drug interactions: Clinical applications and dosing implications
- 2026-06Prednisone dosage is the primary modifiable risk factor for infection in pemphigus with rituximab treatment: A retrospective analysis of 105 patients
- 2026-06综述开放获取A Systematic Review of Literature: TNF-α Blockers and JAK Inhibitors for the Treatment of Stevens-Johnson Syndrome, Toxic Epidermal Necrolysis, and Severe Forms of Erythema Multiforme
- 2026-06综述Drug therapies and immune pathways in ocular manifestations of inflammatory skin diseases
- 2026-06病例报告Pemphigus vulgaris-a blistering clinical enigma: case report
- 2026-06开放获取Protocol to quantify desmoglein 3 tension at cell-cell adhesions using FRET-based tension sensors
境外已获批用于本病的药物 2L2
欧盟 1 项、美国 1 项。同一药物在两地各批一次的，会分别列出。
「境外已获批」不等于「在中国能用」。中间隔着进口注册、临床急需境外新药通道、海南博鳌乐城国际医疗旅游先行区等几条路径，各有各的条件与费用。这一节能确定地告诉你的只有一件事：这个病在世界范围内已经有获得批准的药物，它叫什么名字。拿这个名字去问主治医生，是下一步最省力的做法。
药名一律保留英文原文，不作翻译——中国的药品通用名与英文名的音译经常不一致，译错会让人去找一个不存在的药。
已获孤儿药资格、尚未获批的在研药物（6 项）
孤儿药资格只是一种监管身份——它意味着监管机构认可这是罕见病用药并给予研发激励，不代表这个药已被证明有效，也不代表将来一定能上市。绝大多数最终不会成药。列在这里是为了看清有哪些方向正在被尝试。
- human monoclonal antibody against Fas ligand欧盟2012-02-09Treatment of pemphigus官方记录
- afamelanotide欧盟2014-07-04Treatment of familial benign chronic pemphigus (Hailey-Hailey disease)官方记录
- Desmoglein 3 synthetic peptide (PI-0824)美国2004-10-26Treatment of pemphigus vulgaris官方记录
- Autologous Desmoglein 3 (DSG3) Chimeric Autoantibody Receptor-directed美国2020-01-28Treatment of pemphigus vulgaris官方记录
- afamelanotide美国2025-10-16treatment of pemphigus官方记录
- resecabtagene autoleucel美国2026-03-14treatment of pemphigus vulgaris官方记录
数据来自欧洲药品管理局（EMA）的药品与孤儿药资格公开导出表，以及美国 FDA 孤儿药资格数据库。两边口径不同：欧盟一侧取的是当前状态仍为「已授权」的药品；美国一侧记录的是「曾获批准」这一事实，FDA 的公开表不追踪药物此后是否退市（例如 Relyvrio 于 2024 年撤市，表中仍记为已获批）。请以官方记录页为准。
在中国开展的临床试验 11L2
按病名在 ClinicalTrials.gov 检索、并校验研究中心含中国大陆而来。登记状态不等于现在真的能入组——务必按 NCT 号到原站核实，并与主治医生商量。
登记为可入组 1
- 招募中NCT07681388CD19 CAR-T Therapy for Refractory Pemphigus Vulgaris中国研究中心 1 个：Wuhan
宽泛匹配的在招试验（2 项）
这些试验只被更宽泛的病名命中（例如用「帕金森病」检索「青年型帕金森病」），很可能并不针对本病。逐条看标题再判断。
- A Clinical Study of B007 in the Treatment of Pemphigus.中国研究中心 12 个：Beijing、Changsha、Chengdu、Chongqing、Fuzhou、Guangzhou 等 10 地
- A Single-center Study of CM313 in Patients With Pemphigus中国研究中心 1 个：Chengdu
其他状态的试验（8 项）
- Efficacy and Safety of Different Doses of Rituximab Therapy in Pemphigus Patients中国研究中心 1 个：Shanghai
- A Single Cycle of Intravenous Immunoglobulin as Adjuvant to Rituximab in Patients With Pemphigus: A Retrospective Cohort Study at a Tertiary Referral Center中国研究中心 1 个：Shanghai
- 状态未知NCT04023149Efficacy of Interleukin-2 Gargle in the Treatment of Oral Mucosa Lesion in Pemphigus Vulgaris中国研究中心 1 个：Changsha
- 已完成NCT04598451A Study to Assess the Efficacy and Safety of a Subcutaneous Formulation of Efgartigimod PH20 SC in Adults With Pemphigus (Vulgaris or Foliaceus)中国研究中心 13 个：Beijing、Chendu、Chongqing、Fujian、Guangzhou、Guanzhou 等 10 地
- 状态未知NCT05303272A Study to Evaluate Efficacy and Safety of Abatacept in Participants of Pemphigus Vulgaris (PV)中国研究中心 1 个：Wuhan
- 已终止NCT04598477A Study to Assess the Long-term Safety and Efficacy of a Subcutaneous Formulation of Efgartigimod PH20 SC in Adults With Pemphigus (Vulgaris or Foliaceus)中国研究中心 13 个：Beijing、Chengdu、Chongqing、Fuzhou、Guangzhou、Guanzhou 等 10 地
- 已完成NCT06167408Identifying Factors Influencing In-Hospital Relapse in Pemphigus Patients中国研究中心 1 个：Chongqing
- 已完成NCT06654817Efficacy and Safety Evaluation of Anti-CD20 Monoclonal Antibody Combined with Azathioprine and Corticosteroids in the Treatment of Pemphigus Vulgaris中国研究中心 1 个：Fuzhou
中国境外的在招试验 10L2
这些试验在中国没有研究中心，通常无法直接报名——入组一般要求在当地居住并接受随访。列在这里是因为它另有用处：看清楚全世界正在试哪些药、做到了哪一期、由谁在做。把药名和 NCT 编号记下来去问主治医生，或据此进一步查该药是否已在境外获批、是否有拓展性用药（expanded access）通道。
美国3Syria1土耳其1
CT.gov 报告命中 10 项，此处取回并展示最近的 9 项。
- 尚未开始招募NCT07769333Rituximab With Corticosteroids Versus Corticosteroids Plus Azathioprine for Moderate-to-Severe Pemphigus Vulgaris
- 招募中NCT07641725Comparison of the Efficacy of Clobetasol Propionate 0.05% Mouthwash, Photobiomodulation, and Their Combination in Managing of Oral Lesions in Patient With Pemphigus Vulgaris.Syria
- 招募中NCT07384221Periodontal Status and Disease Severity in Pemphigus Vulgaris Patients土耳其
- 尚未开始招募NCT06663943A Randomized Study on Pemphigus Treatment With Humanized CD38 Antibody CM313.
- 尚未开始招募NCT06971172Low-level Laser Therapy on Oral Pemphigus Vulgaris Patients.
- 尚未开始招募NCT06863454Trichoscopy as a Monitoring Tool for Activity and Remission in Pemphigus Vulgaris: A Clinical Study Supported by Immunological Evaluation.
- 招募中NCT06581562Open-label Single-Center Study to Evaluate the Safety and Efficacy of Combining Rituximab and AB-101 in B-cell Associated Autoimmune Diseases.美国
- 招募中NCT04422912A Phase 1/2, Open-label, Safety and Dosing Study of Autologous CART Cells (Desmoglein 3 Chimeric Autoantibody Receptor T Cells [DSG3-CAART] or CD19-specific Chimeric Antigen Receptor T Cells [CABA-201]) in Subjects With Active, Pemphigus Vulgaris (RESET-PV)美国
- 招募中NCT02753777Autoimmune Blistering Diseases Study美国
外部标识与链接
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本页数据来源
- 疾病定义、同义词、基因、表型、流行病学：Orphanet（CC BY 4.0）
- 中文病名：Orphanet 中文包，冻结于 2020-06-01
- 表型中文标签：HPO 简体中文翻译（CHPO 上游成果）