Editor’s Note: Radical nephrectomy combined with venous tumor thrombus resection is the preferred treatment for renal cell carcinoma (RCC) with inferior vena cava tumor thrombus (IVC-TT) and can significantly improve patient prognosis. Preoperative neoadjuvant therapy aimed at reducing tumor thrombus level and grade is of great importance in reducing perioperative risks. Recently, the NEOTAX study, led by Academician Xu Zhang, Professor Xin Ma, and Professor Liangyou Gu from the Chinese People's Liberation Army General Hospital, was presented at the ESMO Congress (1701P). The study demonstrated that the neoadjuvant regimen of toripalimab combined with axitinib reduced tumor thrombus levels in 96.0% of RCC patients with IVC-TT and achieved thrombus downstaging in 44% of patients, thereby simplifying the surgical process. The study results were published in the Nature sub-journal Signal Transduction and Targeted Therapy (IF: 40.8). The combined treatment showed significant efficacy in reducing IVC-TT and improving surgical feasibility, offering a new direction for improving the prognosis of such patients. Below are the study details.

In a Phase II study, the combination of toripalimab and axitinib as a neoadjuvant therapy for patients with renal cell carcinoma (RCC) and inferior vena cava tumor thrombus (IVC-TT) demonstrated significant efficacy in thrombus downstaging. Between March 2020 and October 2023, 25 patients underwent the study treatment, and after 12 weeks, 44.0% experienced a reduction in Mayo staging level, with a median thrombus height reduction of 2.3 cm. The overall response rate was 44.0%, with 61.9% of patients who were candidates for surgery seeing changes in their surgical strategy due to the downstaging. The treatment was well-tolerated, with the majority of treatment-related adverse events being of grade 1 or 2, and no clinically significant thrombus progression was observed. This study confirms the potential of neoadjuvant targeted and immunotherapy to simplify surgery and improve outcomes for patients with RCC and IVC-TT. The high rate of thrombus downstaging and the reduction in thrombus height are particularly encouraging, suggesting that this approach could become a new standard of care for these patients.