系统性硬化症

定义 英文原文（暂无中文）

Systemic sclerosis (SSc) is a generalized disorder of small arteries, microvessels and connective tissue, characterized by fibrosis and vascular obliteration in the skin and organs, particularly the lungs, heart, and digestive tract. There are two main subsets of SSc: diffuse cutaneous SSc (dcSSc) and limited cutaneous SSc (lcSSc). A third subset of SSc has also been observed, called limited Systemic Sclerosis (lSSc) or systemic sclerosis sine scleroderma.

别名

系统性硬皮病

基本事实

遗传方式

多基因/多因素、不适用

发病年龄

成年期

患病率

1-5 / 10 000（Europe）

临床表型 86

近两年的全球研究 8,101L2

2024/10 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。

• 2026-10
  Correlation Is Not Agreement: The Discordance Threshold for Patient and Physician Global Assessments in Systemic Sclerosis Requires Reliability-Based Validation
  Arthritis care & research · DOI · Europe PMC
• 2026-10综述
  Gut microbiota and systemic sclerosis: Mechanistic insights and microbial therapies
  Autoimmunity reviews · DOI · Europe PMC
• 2026-10
  Reduced serum Irisin levels in systemic sclerosis: a cross-sectional case-control study
  Clinical rheumatology · DOI · Europe PMC
• 2026-10
  Abstracts
  JPGN reports
• 2026-10综述
  Gastrointestinal ultrasound in systemic sclerosis: A scoping review of current evidence
  European journal of internal medicine · DOI · Europe PMC
• 2026-10开放获取
  Reduced humoral response to recombinant zoster vaccine in patients with SLE: a prospective cohort study across immune-mediated inflammatory diseases
  Lupus science & medicine · DOI · Europe PMC
• 2026-10综述开放获取
  Chimeric antigen receptor T (CAR-T) cell therapy in non-oncological diseases
  Signal transduction and targeted therapy · DOI · Europe PMC
• 2026-10
  Distinct Clinical Implications of Anti-Topoisomerase I Antibody Positivity and Antibody Titers in Systemic Sclerosis
  International journal of rheumatic diseases · DOI · Europe PMC
• 2026-10
  Case Report: Childhood Scleredema Following Streptococcal Infection Mimicking Systemic Sclerosis
  International journal of rheumatic diseases · DOI · Europe PMC
• 2026-10病例报告开放获取
  Drug-Induced Liver Injury During Treatment With Tocilizumab: A Case Report of an Atypical Pattern Lesion
  Pharmacology research & perspectives · DOI · Europe PMC
• 2026-10开放获取
  Association of Three HIF-1α Genotypes With Susceptibility and Severity of Chronic Kidney Disease
  Clinical and translational science · DOI · Europe PMC
• 2026-10
  Multitarget Therapy With Rituximab Plus Mycophenolate Mofetil Versus Rituximab Monotherapy in Systemic Sclerosis-Associated Interstitial Lung Disease
  The Journal of rheumatology · DOI · Europe PMC
• 2026-10
  More Than Skin Deep: Moving From a Skin-Based Phenotypic Model to Molecular Classification of Systemic Sclerosis
  The Journal of rheumatology · DOI · Europe PMC
• 2026-10开放获取
  ADAMTS14 is a novel modulator of fibroblast mechanoactivation in pulmonary fibrosis
  American journal of respiratory and critical care medicine · DOI · Europe PMC
• 2026-10
  Comparison of Warrick Scoring and Goh Staging for Detecting Progression in Systemic Sclerosis-Associated Interstitial Lung Disease
  The Journal of rheumatology · DOI · Europe PMC
• 2026-10综述开放获取
  Acute exacerbation in fibrotic interstitial lung disease: an international working group report
  American journal of respiratory and critical care medicine · DOI · Europe PMC
• 2026-10
  Mycophenolate and azathioprine in fibrotic interstitial lung disease
  American journal of respiratory and critical care medicine · DOI · Europe PMC
• 2026-09开放获取
  A Retrospective Review of 1821 Cases of Eosinophilia in a Single-Center Study in Eastern China
  EJHaem · DOI · Europe PMC
• 2026-09综述开放获取
  The Immune Checkpoint Inhibitors Journey: From Early Promise to Lasting Impact
  Journal of immunotherapy and precision oncology · DOI · Europe PMC
• 2026-09综述开放获取
  Advancing In Vivo Chimeric Antigen Receptor T-Cell Engineering to Accelerate Clinical Translation
  MedComm · DOI · Europe PMC

境外已获批用于本病的药物 2L2

欧盟 0 项、美国 2 项。同一药物在两地各批一次的，会分别列出。

• OFEV美国2019-09-06
  nintedanib
  官方记录
• Actemra美国2021-03-04
  tocilizumab
  官方记录

数据来自欧洲药品管理局（EMA）的药品与罕见病用药资格公开导出表，以及美国 FDA 的罕见病用药资格数据库（Orphan Drug Product Designation Database）。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 94L2

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 65

• 招募中NCT06417502
  Observational Study of Pediatric Rheumatic and Immunologic Diseases in China: The CAPRID Registry
  观察性 · 2022/04/29Peking Union Medical College Hospital
  中国研究中心 9 个：Beijing、Changsha、Chongqing、Hangzhou、Nanjing、Shenzhen
• 仅邀请入组NCT05859997
  Universal CAR-T Cells (BRL-301) in Relapse or Refractory Autoimmune Diseases
  不适用 · 干预性 · 2023/05/17Bioray Laboratories
  中国研究中心 1 个：Shanghai
• 招募中NCT05878717
  A Study of the Efficacy and Safety of Belimumab in Adults With Systemic Sclerosis Associated Interstitial Lung Disease
  II 期、III 期 · 干预性 · 2023/09/13GlaxoSmithKline
  中国研究中心 11 个：Beijing、Changchun、Chengdu、Luzhou、Mianyang、Nanjing 等 11 地
• 招募中NCT06058091
  RY_SW01 Cell Injection Therapy in Systemic Sclerosis
  I 期、II 期 · 干预性 · 2023/09/22Jiangsu Renocell Biotech Company
  中国研究中心 1 个：Nanjing
• 招募中NCT06189495
  A Randomized, Double-blind, Placebo-controlled Phase II Clinical Study to Evaluate the Safety, Tolerability, Efficacy, and Pharmacokinetic Profile of Genakumab Injection in Patients With Connective Tissue Disease-associated Interstitial Lung Disease
  II 期 · 干预性 · 2023/12/30Changchun GeneScience Pharmaceutical Co., Ltd.
  中国研究中心 4 个：Beijing、Chengdu、Jinan、Wuhan
• 招募中NCT06249438
  A Study of C-CAR168 in the Treatment of Autoimmune Diseases Refractory to Standard Therapy
  I 期 · 干预性 · 2024/03/20RenJi Hospital
  中国研究中心 1 个：Shanghai
• 招募中NCT06503224
  An Exploratory Clinical Study of SCAR02 Targeting BCMA and CD19 for the Treatment of Refractory Autoimmune Diseases
  不适用 · 干预性 · 2024/04/09The First Affiliated Hospital of University of Science and Technology of China
  中国研究中心 1 个：Changsha
• 尚未开始招募NCT06420154
  The Safety and Efficacy of Anti-CD19 CAR-T Cells in Patients With Relapsed/Refractory Autoimmune Diseases
  早期 I 期 · 干预性 · 2024/05/27First Affiliated Hospital of Wenzhou Medical University
  中国研究中心 1 个：Wenzhou
• 招募中NCT06414135
  Relmacabtagene Autoleucel for the Treatment of Systemic Sclerosis
  I 期 · 干预性 · 2024/06/12Liangjing Lu
  中国研究中心 1 个：Shanghai
• 招募中NCT06350110
  Fourth-gen CAR T Cells Targeting BCMA/CD19 for Refractory Systemic Lupus Erythematosus (SLE)
  I 期、II 期 · 干预性 · 2024/07/10Essen Biotech
  中国研究中心 1 个：Beijing
• 招募中NCT06548607
  Clinical Study on Targeted CD19 or CD19-BCMA CAR-T Therapy for Autoimmune Diseases
  早期 I 期 · 干预性 · 2024/09/14Nanjing Bioheng Biotech Co., Ltd.
  中国研究中心 1 个：Bengbu
• 招募中NCT06470048
  A Clinical Study to Evaluate Ianalumab in Participants With Diffuse Cutaneous Systemic Sclerosis
  II 期 · 干预性 · 2024/10/09Novartis Pharmaceuticals
  中国研究中心 7 个：Beijing、Changchun、Chengdu、Nanning、Ningbo、Tianjin 等 7 地
• 招募中NCT06888960
  Safety Study of CC312 in Autoimmune Disease Patients
  早期 I 期 · 干预性 · 2024/11/08CytoCares Inc
  中国研究中心 1 个：Tianjin
• 招募中NCT06716606
  A Study to Investigate the Long-term Safety and Efficacy of Belimumab in Adults With Interstitial Lung Disease (ILD) Associated With Systemic Sclerosis (SSc) and Other Connective Tissue Diseases (CTD) (BLISSconneCTD-OLE)
  III 期 · 干预性 · 2024/12/12GlaxoSmithKline
  中国研究中心 7 个：Beijing、Chengdu、Hangzhou、Mianyang、Shanghai、Shenyang 等 7 地
• 招募中NCT06794008
  BCMA-CD19 CAR-T Therapy for Refractory Autoimmune Diseases
  II 期 · 干预性 · 2024/12/26Peking University People's Hospital
  中国研究中心 1 个：Beijing
• 招募中NCT06775912
  Clinical Study on Targeted CD19/BCMA CAR-T Therapy for Autoimmune Diseases
  早期 I 期 · 干预性 · 2025/01/03Nanjing Bioheng Biotech Co., Ltd.
  中国研究中心 1 个：Lianyungang
• 招募中NCT06614270
  Anti-CD19 IL-10/IL15 CAR-NK Cells in Refractory/Relapsed Autoimmune Diseases
  不适用 · 干预性 · 2025/01/06Second Affiliated Hospital, Zhejiang University, School of Medicine
  中国研究中心 1 个：Hangzhou
• 招募中NCT06375005
  Efficacy and Safety of Telitacicept in the Treatment of Systemic Sclerosis
  II 期 · 干预性 · 2025/01/06Second Affiliated Hospital, Zhejiang University, School of Medicine
  中国研究中心 9 个：Hangzhou、Huzhou、Jiaxing、Ningbo、Shanghai、Wenzhou 等 7 地
• 招募中NCT06941129
  CAR T-cell Therapy Targeting CD19 and BCMA in Patients With Relapse/Refractory Autoimmune Diseases
  I 期 · 干预性 · 2025/02/11Institute of Hematology & Blood Diseases Hospital, China
  中国研究中心 1 个：Tianjin
• 招募中NCT06822881
  CT1190B in the Treatment of Patients With Moderate to Severe Refractory Systemic Lupus Erythematosus (SLE) or Refractory/Progressive Systemic Sclerosis (SSc)
  I 期 · 干预性 · 2025/02/20Beijing GoBroad Hospital
  中国研究中心 1 个：Beijing
• 招募中NCT06801119
  Efficacy and Safety of HN2301 in Autoimmune Diseases（AIDs）
  I 期 · 干预性 · 2025/03/16Shenzhen MagicRNA Biotechnology Co., Ltd
  中国研究中心 1 个：Hefei
• 尚未开始招募NCT06871644
  Clinical Study of CD7 CAR-T Cells for Relapsed/refractory Autoimmune Diseases
  不适用 · 干预性 · 2025/03/18PersonGen BioTherapeutics (Suzhou) Co., Ltd.
  中国研究中心 1 个：Hefei
• 招募中NCT06947460
  CD19-BCMA CART Cell Therapy for Refractory SLE-LN, SSc, and pSS-PAH
  I 期、II 期 · 干预性 · 2025/04/18Beijing GoBroad Hospital
  中国研究中心 1 个：Beijing
• 尚未开始招募NCT06978738
  UCAR T-cell Therapy Targeting CD19/ BCMA in Patients With Relapse/ Refractory Autoimmune Diseases
  I 期 · 干预性 · 2025/05Changzhou No.2 People's Hospital
  中国研究中心 1 个：Changzhou
• 尚未开始招募NCT06970951
  Early Clinical Study of UTAA91 Injection for the Treatment of Relapsed/Refractory Autoimmune Diseases
  早期 I 期 · 干预性 · 2025/05/18PersonGen BioTherapeutics (Suzhou) Co., Ltd.
  中国研究中心 1 个：Suzhou

另有 40 项未列出。

中国境外的在招试验 217L2

CT.gov 报告命中 217 项，此处取回并展示最近的 15 项。

• 尚未开始招募NCT07335562
  A Study to Compare the Efficacy and Safety of BMS-986353 (Zolacabtagene- Autoleucel / Zola-cel), CD19-CAR T Cells, Versus Standard of Care in Participants With Active Systemic Sclerosis
  III 期 · 干预性 · 2027/02/03Juno Therapeutics, Inc., a Bristol-Myers Squibb Company
  比利时、加拿大、法国、德国、意大利、日本、西班牙、瑞士 等 10 国
• 尚未开始招募NCT07544225
  Study Investigating the Safety of Anti-CD19 CAR-T Cells Therapy Produced at Gustave Roussy for Adults With Severe and Refractory Systemic Autoimmune Rheumatic Diseases
  I 期 · 干预性 · 2026/12/01Gustave Roussy, Cancer Campus, Grand Paris
  法国
• 尚未开始招募NCT07783269
  A Basket Study of CTA313 in Participants With Active Autoimmune Diseases
  I 期 · 干预性 · 2026/12Imviva Bio
• 尚未开始招募NCT07821398
  Hyperpolarized Xenon MRI in Adults With Pulmonary Hypertension in Interstitial Lung Disease and Systemic Sclerosis
  II 期 · 干预性 · 2026/11/01Bastiaan Driehuys
• 尚未开始招募NCT07209644
  Catestatin as a Novel Biomarker for Cardiovascular and Microvascular Involvement in Systemic Sclerosis
  观察性 · 2026/11/01Assiut University
• 尚未开始招募NCT07748000
  A Study of LIQ861 (Inhaled Treprostinil) in Adults With Systemic Sclerosis Who Have Symptomatic Raynaud's Phenomenon Attacks
  II 期 · 干预性 · 2026/10/26Liquidia Technologies, Inc.
• 尚未开始招募NCT07782450
  A Study to Learn About a Medicine Called PF-08154225 in Participants With Autoimmune Diseases
  I 期、II 期 · 干预性 · 2026/10/10Pfizer
• 尚未开始招募NCT07729995
  BAFF CAR-T Cells (LMY-922) for Treatment of Refractory Autoimmune Disease
  I 期 · 干预性 · 2026/10Luminary Therapeutics
  美国
• 尚未开始招募NCT07827742
  Rituximab vs Placebo for Maintenance in Systemic Sclerosis - Interstitial Lung Disease
  II 期 · 干预性 · 2026/10Assistance Publique - Hôpitaux de Paris
  法国
• 招募中NCT07800871
  FT839 in Autoimmune Diseases
  I 期、II 期 · 干预性 · 2026/10Fate Therapeutics
  美国
• 尚未开始招募NCT07649265
  A Trial in Healthy Adult Participants and Adults With Autoimmune Disease to Test How HBM7020 is Tolerated and Absorbed in the Body
  I 期 · 干预性 · 2026/09/15Otsuka Pharmaceutical Development & Commercialization, Inc.
• 尚未开始招募NCT07642297
  TL1A Inhibition in Systemic Sclerosis-Related Skin Fibrosis and Immunological Alterations: A Proof-of-Concept In Vitro Study (FIBROSTOP)
  观察性 · 2026/09/01Fondazione Policlinico Universitario Campus Bio-Medico
  意大利
• 尚未开始招募NCT07697274
  Hyaluronidase for Sclerodactyly in Systemic Sclerosis Trial
  早期 I 期 · 干预性 · 2026/09/01Medical University of South Carolina
  美国
• 尚未开始招募NCT07691970
  Rollover Study for Participants Who Have Completed a Previous Clinical Study With Anifrolumab (Saphnelo) and Clinically Benefited From Continued Treatment
  III 期 · 干预性 · 2026/09/01AstraZeneca
  奥地利、比利时、巴西、保加利亚、法国、希腊、印度、以色列 等 22 国
• 尚未开始招募NCT07717060
  Extracellular Vesicle Dynamics Predicting Vascular Complications and Treatment Response in Systemic Sclerosis
  不适用 · 干预性 · 2026/09/01Fondazione Policlinico Universitario Agostino Gemelli IRCCS

外部标识与链接