Tislelizumab Combined With Axitinib in Neoadjuvant Treatment of Locally Advanced Clear Cell Renal Cell Carcinoma: A Single‐Center, Phase II Clinical Study

Clear cell renal cell carcinoma (ccRCC) accounts for 70%‒80% of renal cell carcinoma cases and often shows no symptoms in early stages. Thus, approximately 30% of patients are diagnosed with advanced ccRCC. This single-center prospective single-arm study evaluated the efficacy and safety of tislelizumab combined with axitinib in patients with locally advanced ccRCC. A total of 20 eligible patients were enrolled at Nanjing Drum Tower Hospital from September 2021 to June 2024. The primary endpoint was objective response rate (ORR) before surgery, and secondary endpoints included disease-free survival (DFS), overall survival, safety, and tissue biomarker analysis. All patients completed neoadjuvant treatment, and 19 underwent planned surgery; 70% (14/20) had cT3 stage disease with a median tumor diameter of 8.3 cm. The ORR was 55% (11 partial responses), 73.6% (14/19) achieved pathological downstaging, one patient attained pathological complete response, and no grade ≥3 perioperative complications occurred. The 2-year DFS rate was approximately 90%, and biomarker analysis showed significantly higher tumor shrinkage rates in patients with RTK/RAS pathway alterations. In conclusion, tislelizumab combined with axitinib exhibits substantial efficacy and acceptable safety in neoadjuvant treatment of locally advanced ccRCC, providing preliminary clinical evidence for its application.