The prognostic significance of histologic variant on survival outcomes in patients with metastatic urothelial carcinoma receiving immune checkpoint inhibitor therapy

• Published in: BMC cancer Vol. 23; no. 1; pp. 1 - 10
• Main Authors: Tsai, Tsung-Han, Su, Po-Jung, Huang, Shih-Yu, Kuo, Ming-Chun, Lin, Chang-Ting, Wu, Chia-Che, Luo, Hao-Lun, Chen, Chien-Hsu, Chou, Chih-Chi, Liu, Ting-Ting, Huang, Chun-Chieh, Tsai, Kai-Lung, Su, Yu-Li
• Format: Journal Article
• Language: English
• Published: London BioMed Central 15.09.2023; BioMed Central Ltd; BMC
• Subjects: Analysis; Biomedical and Life Sciences; Biomedicine; Bladder cancer; Cancer; Cancer Research; Carcinoma; Care and treatment; Chemotherapy; Development and progression; FDA approval; Genetic aspects; Genetic variation; Health Promotion and Disease Prevention; Hemoglobin; Immune checkpoint inhibitors; Medical prognosis; Medicine/Public Health; Metastases; Metastasis; Metastatic urothelial carcinoma; Monoclonal antibodies; Multivariate analysis; Oncology; Patient outcomes; Patients; Performance evaluation; Prognosis; Real-world data; Response rates; Statistical significance; Surgical Oncology; Survival; Survival analysis; Urothelial carcinoma; Variant histology; United States; Taiwan; Immune checkpoint inhibitors; Variant histology; Metastatic urothelial carcinoma; Real-world data
• ISSN: 1471-2407; 1471-2407
• DOI: 10.1186/s12885-023-11398-w

Background While the treatment guidelines have been established for pure urothelial carcinoma (pUC), patients with variant type urothelial carcinoma (vUC) face limited effective treatment options. The effectiveness of immune checkpoint inhibitors (ICI) in patients with vUC remains uncertain and necessitates additional research. Method We conducted a retrospective, multicenter study to explore the effectiveness of ICI in patients with pUC or vUC in Taiwan. We evaluated the overall response rate (ORR) through univariate logistic regression analysis and examined the overall survival (OS) and progression-free survival (PFS) using Kaplan-Meier analysis. Additionally, we employed univariate and multivariate Cox proportional hazards models to analyze the data. Result A total of 142 patients (116 pUC, 26 vUC) were included in our final analysis. The ORR was marginally higher in patients with pUC compared to those with vUC (34.5% vs. 23.1%, p = 0.26). Among all patients, 12.9% with pUC achieved a complete response (CR) after ICI treatment, while no vUC cases achieved CR (p = 0.05). There were no significant differences in PFS (median 3.6 months vs. 4.1 months, p = 0.34) or OS (median 16.3 months vs. 11.0 months, p = 0.24) when comparing patients with pUC or vUC. In the subgroup analysis, patients with pUC who underwent first-line ICI treatment exhibited significantly improved OS compared to those with vUC (24.6 months vs. 9.1 months, p = 0.004). Conclusion The use of ICI as monotherapy is a feasible and effective treatment approach for patients with metastatic vUC.