From the Northwell Health Cancer Institute, New Hyde Park.
Cancer J. 2024;30(2):92-101. doi: 10.1097/PPO.0000000000000708.
Uveal melanoma (UM), arising from intraocular melanocytes, poses a complex clinical challenge with a substantial risk of distant metastasis, often to the liver. Molecular profiling, encompassing genetic, cytogenetic, gene expression, and immunological subsets, plays a pivotal role in determining prognoses. The evolving landscape includes promising systemic treatments, such as tebentafusp, a novel immune-modulating bispecific fusion protein, and targeted therapies. Combined regional and systemic approaches, including immune checkpoint inhibitors and innovative liver-directed therapy, are also under investigation. Although recent progress has improved outcomes, ongoing research aims to address the unique challenges of UM and develop effective therapies, particularly for HLA-A*02:01-negative patients who represent a significant unmet medical need. This review comprehensively discusses the molecular characteristics of UM, risk stratification methods, and the current and future spectrum of regional and systemic therapeutic modalities.
葡萄膜黑色素瘤 (UM) 源于眼内黑素细胞，具有很高的远处转移风险，常转移至肝脏，这给临床带来了极大的挑战。分子分析包括遗传、细胞遗传学、基因表达和免疫学亚群，对确定预后起着关键作用。目前正在研究新的系统治疗方法，如新型免疫调节双特异性融合蛋白替本福司，以及靶向治疗。联合区域和全身治疗方法，包括免疫检查点抑制剂和创新的肝脏定向治疗，也在研究中。尽管最近的进展改善了预后，但仍需要进行研究以解决 UM 的独特挑战并开发有效的治疗方法，特别是对于 HLA-A*02:01 阴性患者，他们存在着巨大的未满足的医疗需求。本文全面讨论了 UM 的分子特征、风险分层方法以及目前和未来的区域和全身治疗模式。
