尼沃卢马布治疗高风险口腔白血病患者:非随机对照试验
Glenn J Hanna1, Alessandro Villa2, Shuvro P Nandi3,4
1Department of Medical Oncology, Dana-Farber Cancer Institute, Boston, Massachusetts.
JAMA oncology
|November 16, 2023
概括
抗编程细胞死亡1蛋白 (PD-1) 疗法在治疗多发性白血病 (PVL) 方面具有潜力,这是一种具有侵略性的口腔癌前期癌症. 该研究满足了其响应终点,确定了免疫基因组标志物,以便在这种高风险疾病中进行未来研究.
科学领域:
- 在瘤学瘤学.
- 免疫学 免疫学 免疫学
- 口腔医学是指口腔医学.
背景情况:
- 增殖性白血病 (PVL) 是一种具有高风险的侵袭性口腔癌前期癌症,具有转化为口腔状细胞癌 (OSCC) 的高风险.
- 该PVL微环境富含细胞毒性T细胞,表明免疫检查点疗法作为一种潜在的治疗策略.
- 目前没有一种疗法能够有效地改变PVL的自然史.
研究的目的:
- 评估抗编程细胞死亡1蛋白 (PD-1) 治疗对高风险PVL患者的安全性和临床活性.
- 根据病变大小和发育不良的复合得分来评估应答率.
- 探索与免疫相关的不良事件,无癌症生存率和与治疗反应相关的免疫基因组因素.
主要方法:
- 一个非随机的,开放的,第二阶段临床试验,涉及33名PVL患者.
- 患者每28天内静脉注射4剂尼沃卢马布 (抗PD-1疗法) 治疗.
- 进行了治疗前和后的活检,口内摄影和全面的免疫基因组分析.
主要成果:
- 十二名患者 (36%) 通过复合得分显示出反应,其中3人实现了主要反应 (≥80%的减少).
- 在试验期间,9名患者 (27%) 患上OSCC;无癌症2年生存率为73%.
- 9p21.3 在治疗前活检中体质拷贝数损失与尼沃卢马布治疗后的OSCC进展有关.
结论:
- 尼沃卢马布在高风险PVL中显示出潜在的临床活性,满足了该研究的主要反应终点.
- 这些发现支持免疫检查点治疗作为PVL的可行策略.
- 已识别的免疫基因组关联,特别是9p21.3损失,可以为这种未满足的医疗需求提供未来临床试验的设计信息.
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