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周周血管上皮细胞瘤的系统治疗和分子生物标志物:单一机构的回顾性分析

Stefano Testa^1,2, Nam Q Bui², Kristen N Ganjoo²

¹Department of Medicine, Stanford University, Stanford, California.

Cancer research communications

|July 14, 2023

概括

系统性治疗,如mTOR抑制剂,对于晚期周血管上皮质细胞瘤 (PEComa) 的化学疗法具有类似的疗效. 过度表达TFE3表明PEComa患者的预后较差.

科学领域:

在瘤学瘤学.
肉瘤研究研究肉瘤研究
分子病理学分子病理学

背景情况:

周周血管上皮质细胞瘤 (PEComa) 是一个多样化的瘤组,具有可变的临床行为.
对于治疗晚期PEComa的证据有限,只有一种FDA批准的治疗方法.
确定预后生物标志物对于优化患者管理至关重要.

研究的目的:

评估晚期PEComas患者的全身治疗结果.
为了确定PEComa的预后生物标志物,包括TFE3表达.
为了比较不同治疗方式的疗效,包括mTOR抑制剂和化疗.

主要方法:

对29名患有晚期PEComa需要全身治疗的患者进行了回顾性分析.
评估的结果包括总生存率 (OS) 和无进展生存率 (PFS).
使用卡普兰-梅尔分析和考克斯比例危险模型来分析数据,包括通过IHC或FISH过度表达TFE3.

主要成果:

平均生存期为204.9个月;中位数第一线PFS为92.4个月,综合PFS为15.8个月.
过度表达TFE3与明显更高的死亡风险和更短的生存期相关.
在OS和一线PFS方面,mTOR抑制剂的疗效与化疗相当.

结论:

mTOR抑制剂在晚期PEComa中具有与化疗相似的疗效,这表明由于相关发病率较低,它们可能更可取.
过度表达TFE3识别出一个具有更具侵略性的疾病过程和更糟糕的预后的PEComa子组.
成本和可用性应指导不同mTOR抑制剂之间的选择,因为它们的疗效相似.

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