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Cell therapy for scleroderma: progress in mesenchymal stem cells and CAR-T treatment.
Front Med (Lausanne)
January 2025
Department of Rheumatology and Immunology, Qinzhou First People's Hospital, Qinzhou, Guangxi, China.
Cell therapy is an emerging strategy for precision treatment of scleroderma. This review systematically summarizes the research progress of mesenchymal stem cell (MSC) and chimeric antigen receptor T cell (CAR-T) therapies in scleroderma and discusses the challenges and future directions for development. MSCs possess multiple functions, including immunomodulation, anti-fibrosis, and promotion of vascular regeneration, all of which can improve multiple pathological processes associated with scleroderma.
View Article and Find Full Text PDFDevelopment and testing the psychometric properties of 20 bolt-on items for the EQ-5D-5L across 31 rare diseases.
Value Health
January 2025
Department of Cardiology and State Key Laboratory of Complex Severe and Rare Diseases, Peking Union Medical College Hospital, Chinese Academy of Medical Sciences, Beijing, China.
Objective: Our objective was to develop and assess the psychometric properties of relevant bolt-on items for the EQ-5D-5L in patients with rare diseases (RDs).
Methods: Nineteen new EQ-5D-5L bolt-ons were developed based on literature review, expert input and qualitative interviews and focus groups with patients, caregivers and representatives of patient associations. A nationwide, cross-sectional, web-based survey in China included patients or caregivers of patients with 31 RDs in China (n=9,190).
A comparative analysis of risk stratification tools in systemic sclerosis-associated pulmonary arterial hypertension: a EUSTAR analysis.
Rheumatology (Oxford)
January 2025
Department of Rheumatology, University Hospital Zurich, University of Zurich, Zurich, Switzerland.
Objectives: The 2022 European Society of Cardiology and European Respiratory Society (ESC/ERS) Guidelines for pulmonary arterial hypertension (PAH) recommend risk stratification to optimize management. However, the performance of generic PAH risk stratification tools in patients with systemic sclerosis (SSc)-associated PAH remains unclear. Our objective was to identify the most accurate approach for risk stratification at SSc-PAH diagnosis.
View Article and Find Full Text PDFFoot peripheral sensory neuropathy: a frequent disabling manifestation in systemic sclerosis.
Rheumatology (Oxford)
January 2025
Leeds Institute of Rheumatic and Musculoskeletal Medicine, School of Medicine, University of Leeds, Leeds, UK.
Objectives: Peripheral Sensory Neuropathy (PSN) is an under-recognized feature in systemic sclerosis (SSc). Moreover, SSc foot involvement is frequent but poorly investigated. We aimed to provide a detailed characterization of foot PSN in a large cohort of SSc patients, describing its associations with disease-specific features, physical disability, and Quality of Life (QoL).
View Article and Find Full Text PDFRegulation of involucrin and signalling pathway in scleroderma epidermal keratinocytes.
Postepy Dermatol Alergol
December 2024
Department of Dermatology, Second Affiliated Hospital of Zhejiang University School of Medicine, Hangzhou, China.
Introduction: Systemic sclerosis is a complex disease characterized by the fibrosis and vasculopathy.
Aim: We aimed to assess scleroderma by examining involucrin, an early terminal differentiation marker of epidermal keratinocytes.
Material And Methods: Immunolocalization of involucrin was performed in healthy controls and patients with scleroderma lesions by using an immunofluorescence (IF) assay.
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