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免疫检查点抑制剂疗法用于侵袭性垂体神经内分泌瘤

Andrew L Lin^1,2,3, Vasilisa Rudneva⁴, Adam Newton²

¹Department of Neurosurgery, Memorial Sloan Kettering Cancer Center, New York, NY.

The Journal of clinical endocrinology and metabolism

|March 20, 2025

概括

免疫检查点抑制剂 (ICI) 显示出对侵袭性垂体神经内分泌瘤 (PitNETs) 的安全性和可行性. 不匹配修复缺陷 (MMRd) 和temozolomide高突变可能预测PitNET患者对ICI治疗的反应.

科学领域:

在瘤学瘤学.
免疫治疗是一种免疫疗法.
遗传学是一个遗传学.

背景情况:

下垂体神经内分泌瘤 (PitNETs) 在手术和放射治疗后进展,需要新的治疗策略.
在PitNET中,免疫检查点抑制剂 (ICI) 的疗效和预测生物标志物在很大程度上仍未被描述.

研究的目的:

评估PitNET患者联合伊皮利穆马布和尼沃卢马布的可行性和活性.
确定潜在的基因生物标志物,预测PitNETs中对ICI治疗的反应.

主要方法:

针对PitNET患者进行了一项前性,单中心的2期临床试验,使用伊皮利马布和尼沃马布.
使用iRANO标准评估了客观反应,并对瘤样本进行了基因生物标志物分析.

主要成果:

在9名可评估患者中没有观察到客观反应,尽管2/9的患者经历了瘤缩.
不匹配修复缺陷 (MMRd) 和temozolomide高突变与生物标志物队列中的免疫反应有关.
在ICI治疗前后对瘤的分析显示了免疫编辑的证据,包括MMRd的丧失和瘤突变负担的降低.

结论:

治疗ICI,包括ipilimumab和nivolumab,对于侵略性的PitNETs是安全的和可行的.
鉴定出MMRd和temozolomide高突变是预测PitNET中ICI反应的潜在生物标志物.
ICI代表了PitNETs的潜在额外治疗选择,需要在更大规模的研究中进一步调查.

关键词:

生物标志物生物标志物临床试验是指临床试验中的临床试验.免疫疗法免疫疗法下垂体神经内分泌瘤

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