J Clin Oncol:nab-西罗莫司有望成为恶性PEComa的治疗选择!

2021-10-14 Nebula MedSci原创

恶性血管周围上皮样细胞瘤 (PEComa) 是一种罕见的侵袭性肉瘤,nab-西罗莫司有望成为恶性PEComa的治疗选择!

恶性血管周围上皮样细胞瘤 (PEComa) 是一种罕见的侵袭性肉瘤,由组织学和免疫组织化学上独特的上皮样细胞组成。大多数PEComas在临床上是良性的并且不会转移,但恶性PEComa会表现出局部浸润和/或转移性扩散。目前尚无获批的治疗药物/方案。

nab-Sirolimus for Patients With Malignant Perivascular Epithelioid Cell Tumors

这是一项前瞻性的单臂的II期临床试验,是首个评估雷帕霉素靶点抑制剂nab-西罗莫司在PEComa患者中的疗效和安全性的研究。恶性PEComa患者接受nab-西罗莫司治疗( 100 mg/m2/周,静滴,连用两周,停一周)。主要终点是客观缓解率。次要终点包括缓解持续时间、无进展生存期和安全性。探索性终点是肿瘤标志物分析。

缓解率

共有34位患者接受了治疗(安全性评估),31位患者被纳入疗效分析。总缓解率为39%(12/31;95% CI 22-58),包括1例完全缓解和11例部分缓解;52%(16/31)的患者病情稳定,10%(3/31)的患者病情发生进展。缓解获得较快(67%的在第6周即已获得),而且持久。中位随访了2.5年后,中位缓解持续时间仍未达到。

无进展生存期

31位患者中有25位有肿瘤突变谱:9位携带TSC2突变的患者中有8位(89%)获得了缓解,相比之下,16位不携带TSC2突变的患者中只有两位(13%)获得缓解(p<0.001)。中位无进展生存期为10.6个月(95% CI 5.5-未达到),中位总生存期为40.8个月(95% CI 22.2-未达到)。

最常见的治疗相关的不良反应多少1-2级的。无4级及以上的治疗相关不良事件发生。

综上所述,nab-西罗莫司在恶性PEComa患者中具有治疗活性。缓解率、缓解持续时间、疾病控制率和安全性均支持nab-西罗莫司成为该种恶性肿瘤的重要治疗选择。

原始出处:

Andrew J. Wagner, et al. nab-Sirolimus for Patients With Malignant Perivascular Epithelioid Cell Tumors. Journal of Clinical Oncology. October 12, 2021. https://ascopubs.org/doi/full/10.1200/JCO.21.01728

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    2022-07-25 feather89
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    2022-09-28 minlingfeng
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