新辅助特瑞普利单抗联合阿西替尼治疗透明细胞肾细胞癌 (ccRCC) 和下腔静脉癌栓 (IVC-TT) 病例的潜在益处尚不清楚。 NEOTAX 是一项 2 期研究，旨在调查新辅助特瑞普利单抗联合阿西替尼治疗 ccRCC 和 IVC-TT 患者的疗效和安全性 (ChiCTR2000030405)。主要终点是 IVC-TT 水平的降期率。次要终点包括 TT 长度的变化、缓解率、手术方法的百分比变化、手术发病率、无进展生存期 (PFS)、安全性和生物标志物分析。总共有 25 名患者接受了研究治疗，44.0% (11/25) 的患者血栓水平降低，没有人出现 Mayo 水平升高。肿瘤血栓长度的中位变化为-2.3厘米（范围：-7.1至1.1厘米）。总体而言，与计划的手术相比，61.9%（13/21）的患者经历了手术策略的改变，其中 3 名患者出现了严重并发症。中位 PFS 为 25.3 个月（95% CI：17.0-NE）。 1 年 PFS 为 89.1%（95% CI：62.7-97.2）。未发现任何 4 级或 5 级治疗相关不良事件。无反应者的活检样本显示 T 细胞毒性细胞浸润增加，但这些细胞主要为 PD-1 阳性。应答者的活检样本显示出较低的 T 辅助细胞，但其亚型调节性 T 细胞保持不变。在 TT 的手术样本中，无反应者表现出 CD8T_01_GZMK_CXCR4 子集 T 细胞增加。 NEOTAX 达到了预设终点，证明特瑞普利单抗与阿西替尼联合使用可以使很大一部分患者的 IVC-TT 阶段下降，从而简化手术过程。© 2024。作者。

The potential benefit of neoadjuvant toripalimab plus axitinib in cases with clear cell renal cell carcinoma (ccRCC) and inferior vena cava tumor thrombus (IVC-TT) remains unclear. NEOTAX was a phase 2 study to investigate the efficacy and safety of neoadjuvant toripalimab plus axitinib in patients with ccRCC and IVC-TT (ChiCTR2000030405). The primary endpoint was the down-staging rate of IVC-TT level. Secondary endpoints included change in TT length, response rate, percentage change in surgical approach, surgical morbidity, progression-free survival (PFS), safety, and biomarker analyses. In all, 25 patients received study treatment, 44.0% (11/25) patients had a reduction in thrombus level, and none experienced an increase in Mayo level. The median change in tumor thrombus length was -2.3 cm (range: -7.1 to 1.1 cm). Overall, 61.9% (13/21) patients experienced changes in surgical strategy compared with planned surgery, three patients experienced major complications. The median PFS was 25.3 months (95% CI: 17.0-NE). The 1-year PFS was 89.1% (95% CI: 62.7-97.2). No any of grade 4 or 5 treatment-related adverse event was identified. Biopsy samples of non-responders exhibited increased T cytotoxic cell infiltration, but these cells were predominantly PD-1 positive. Biopsy samples of responders exhibited lower T helper cells, however, their subtype, regulatory T cells remained unchanged. In surgical samples of the TT, non-responders exhibited increased CD8T_01_GZMK_CXCR4 subset T cells. NEOTAX met preset endpoints proving that toripalimab in combination with axitinib downstages IVC-TT in a significant proportion of patients leading to simplification in the procedure of surgery.© 2024. The Author(s).