Therapeutic Efficacy and Mechanisms of Mesenchymal Stem Cells and Their Exosomes in the Treatment of Ocular Graft-Versus-Host Disease

Several human studies show positive signals, while research methods and sample sizes continue to develop.

• Level A · Stronger Clinical Evidence
• Level B · Emerging clinical evidence with positive signals
• Level C · Early human research exploring benefits
• Level D · Scientific groundwork from lab and animal studies
• Emerging · Emerging topic under active research

Abstract (original English)

Purpose To provide a comprehensive overview of the clinical manifestations, pathophysiology, and current treatments of ocular graft-versus-host disease (oGVHD), and to explore the therapeutic potential of mesenchymal stem cells (MSCs) and their exosomes (MSC-Exos) as novel treatments. Methods A literature review was conducted using major databases (PubMed, Web of Science, Scopus) to identify studies published between 2000 and 2024. The focus was on clinical trials, case series, and experimental studies related to oGVHD. Data were synthesized to summarize current knowledge. Results oGVHD affects 40% to 60% of Hematopoietic Stem Cell Transplantation recipients, causing symptoms such as dryness, irritation, and vision impairment. Current treatments often fail to provide long-term relief and may cause significant side effects. MSCs and MSC-Exos offer a promising alternative because of their immunosuppressive and regenerative properties. Preclinical and clinical studies have demonstrated their safety and efficacy in improving oGVHD symptoms and promoting corneal health. Conclusions MSCs and MSC-Exos show promise in treating oGVHD, with potential to improve symptoms and promote tissue repair. Further research is needed to establish their long-term safety and efficacy.

Evidence level

Several human studies show positive signals, while research methods and sample sizes continue to develop.

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