Improved overall survival with immune checkpoint inhibitors in the treatment of metastatic urothelial carcinoma: A national retrospective comparative study.

person Chih-Chin Yu Taipei Tzu Chi Hospital, Buddhist Tzu Chi Medical Foundation, New Taipei City, Taiwan info_outline Chih-Chin Yu, Po-Jung SU, Yao-Chou Tsai

Authors person Chih-Chin Yu Taipei Tzu Chi Hospital, Buddhist Tzu Chi Medical Foundation, New Taipei City, Taiwan info_outline Chih-Chin Yu, Po-Jung SU, Yao-Chou Tsai Organizations Taipei Tzu Chi Hospital, Buddhist Tzu Chi Medical Foundation, New Taipei City, Taiwan, Division of Medical Oncology, Department of Internal Medicine, Chang Gung Memorial Hospital, Linkou, Taoyuan, Taiwan Abstract Disclosures Research Funding Taiwan Urological Association Merck Background: Numerous randomized trials have illustrated the survival advantages of immune checkpoint inhibitors (ICI) in metastatic urothelial carcinoma. However, the application of ICI varies in different trials, encompassing first-line, second-line, monotherapy, combination with chemotherapy, or maintenance therapy. Real-world clinical practices often diverge from trial criteria. This study aims to evaluate the effectiveness of any ICI regimen in treating metastatic urothelial carcinoma using real-world data. Methods: This retrospective comparative study included patients diagnosed with locally advanced or metastatic urothelial carcinoma between January 2015 and July 2023, sourced from the Taiwan Upper Tract Urothelial Cancer and Bladder Cancer Collaboration Database. Patients with prior neo-adjuvant or adjuvant systemic therapy were excluded. Results: Among 427 patients, 152 (35.6%) who received any ICI as the first-line or second-line regimen constituted the ICI group, while the remaining 275 (64.4%) patients receiving only chemotherapy formed the chemotherapy group. Within the ICI group, 55 (36.2%) patients received monotherapy, 49 (32.2%) received combination therapy with chemotherapy, and 4 (2.6%) received maintenance therapy as the first-line regimen. The remaining 44 (28.9%) patients received ICI as the second-line regimen. No significant differences were observed in age, sex, performance status, renal function, tumor site, ratio of locally advanced disease, ratio of visceral metastasis, and ratio of previous radical surgery between the two groups. Median overall survival (OS) from the start of the first-line therapy was 15.8 months in the ICI group and 10.2 months in the chemotherapy group. Kaplan-Meier curves demonstrated that the ICI group exhibited superior OS and cancer-specific survival compared to the chemotherapy group (log-rank p = 0.009 and 0.004, respectively). In the multivariable analysis, the ICI group also demonstrated favorable outcomes in OS [hazard ratio (HR) 0.726, p =0.011] and cancer-specific survival (HR 0.67, p =0.004). Conclusions: Our study confirms the clinical efficacy of ICI in metastatic urothelial carcinoma. The use of ICI monotherapy or combination in the first-line or second-line regimen for metastatic urothelial carcinoma improves patient survival. Patient characteristics. Characteristic –no. (%) Immune Checkpoint Inhibitors (N=152) Chemotherapy (N=275) p -value Age≧70 yr 83 (54.6) 147 (53.5) 0.819 Male sex 84 (55.3) 142 (51.6) 0.472 Upper tract 107 (70.4) 197 (71.6) 0.786 ECOG 0-1 121 (79.6) 216 (78.5) 0.527 eGFR≧60 mL/min/1.73m 2 49 (32.2) 93 (33.8) 0.727 Locally advanced 29 (19.1) 73 (26.5) 0.083 Visceral metastasis 96 (63.2) 161 (58.5) 0.351 Prior radical surgery 58 (38.2) 116 (42.2) 0.418