Genna Vincenzo Giuseppe, Maurizi Eleonora, Rama Paolo, Pellegrini Graziella
Holostem Terapie Avanzate S.r.l., Modena, Italy.
Centre for Regenerative Medicine ''S. Ferrari'', University of Modena and Reggio Emilia, Modena, Italy.
Ocul Surf. 2025 Jan;35:57-67. doi: 10.1016/j.jtos.2024.11.005. Epub 2024 Nov 22.
Ocular vision can be hampered by corneal damages, sensibly reducing patients' quality of life and having important social and economic consequences. Ocular surface diseases, which often lead to corneal opacities with visual impairment are the most severe forms of the Limbal Stem Cell Deficiency (LSCD). The present review provides an updated perspective on the available treatments for LSCD, focusing on clinical and biological features, as well as critical points to monitor during clinical translation. Recently developed surgical treatments for LSCD are described, along with their benefits and limitations, with the aim of addressing the issue of correct patient selection. Autologous surgical approaches have been attempted, such as conjunctival limbal autograft (CLAU), simple limbal epithelial transplantation (SLET), and others. Allogeneic limbal stem cell transplantation represents an alternative but carries risk of rejection and requires immunosuppression. Other potential treatments are based on induced pluripotent stem cells (iPSCs), but they require further investigation. The development of advanced therapy medicinal products (ATMPs) such as cultivated limbal epithelial transplantation (CLET), or the use of other epithelia as cultivated oral mucosal epithelial cell transplantation (COMET), has opened additional therapeutic possibilities. Some common critical issues in clinical translation are described, such as patient selection, biopsy procurement, or the use of human/animal derived components, which require rigorous validation to ensure safety and efficacy. Personalized medicine is a promising field for ocular surface restoration, where long-term follow-up studies and standardized criteria are crucial to evaluate the efficacy of these treatments and their cost-effectiveness in providing high-value healthcare.
角膜损伤会阻碍眼部视力，显著降低患者的生活质量，并产生重要的社会和经济后果。眼表疾病常导致角膜混浊并伴有视力障碍，是角膜缘干细胞缺乏症（LSCD）最严重的形式。本综述提供了关于LSCD现有治疗方法的最新观点，重点关注临床和生物学特征，以及临床转化过程中需要监测的关键点。文中描述了最近开发的LSCD手术治疗方法及其优缺点，目的是解决正确选择患者的问题。已经尝试了自体手术方法，如结膜角膜缘自体移植（CLAU）、单纯角膜缘上皮移植（SLET）等。同种异体角膜缘干细胞移植是一种替代方法，但存在排斥风险且需要免疫抑制。其他潜在的治疗方法基于诱导多能干细胞（iPSC），但它们需要进一步研究。先进治疗药品（ATMP）的发展，如培养角膜缘上皮移植（CLET），或使用其他上皮组织如培养口腔黏膜上皮细胞移植（COMET），开辟了更多的治疗可能性。文中描述了临床转化中的一些常见关键问题，如患者选择、活检取材或使用人源/动物源成分，这些都需要严格验证以确保安全性和有效性。个性化医疗是眼表修复的一个有前景的领域，长期随访研究和标准化标准对于评估这些治疗方法的疗效及其在提供高价值医疗保健方面的成本效益至关重要。
