冯·希佩尔-林道病的全身治疗进展：罕见病的巨大贡献
Systemic Therapy Development in Von Hippel-Lindau Disease: An Outsized Contribution from an Orphan Disease.
作者信息
Narayan Vivek, Jonasch Eric
机构信息
Division of Hematology/Medical Oncology, University of Pennsylvania, Abramson Cancer Center, Philadelphia, PA 19104, USA.
Genitourinary Medical Oncology, MD Anderson Cancer Center, Houston, TX 77030, USA.
出版信息
Cancers (Basel). 2022 Oct 28;14(21):5313. doi: 10.3390/cancers14215313.
Abstract
Over the last several decades, an improved understanding of von Hippel-Lindau disease and its underlying biology has informed the successful development of numerous anti-cancer agents, particularly for the treatment of advanced renal cell carcinoma. Most recently, this has culminated in the first regulatory approval for a systemic therapy for VHL disease-associated neoplasms. This review will trace the clinical development of systemic therapies for VHL disease and additionally highlight anticipated challenges and opportunities for future VHL systemic therapy.
摘要
在过去几十年里，对希佩尔-林道病及其潜在生物学机制的深入了解推动了众多抗癌药物的成功研发，尤其是用于治疗晚期肾细胞癌。最近，这一进展促成了首个用于治疗与VHL病相关肿瘤的全身性疗法获得监管批准。本综述将追溯VHL病全身性疗法的临床发展历程，并着重介绍未来VHL病全身性疗法预期面临的挑战与机遇。
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