A prognostic model for survival of patients with metastatic upper tract urothelial carcinoma with first-line systemic therapy.

Purpose: Upper tract urothelial carcinoma (UTUC) presents distinct clinicopathological, molecular features, and biological behaviors compared to urothelial bladder carcinoma (UBC). Currently, no prognostic model exists for metastatic UTUC (mUTUC). This study aimed to develop a prognostic model for patients with mUTUC receiving first-line systemic therapy.

Patients and methods: A total of 476 patients with mUTUC who received first-line systemic therapy were included and retrospectively analyzed. Patients were randomly assigned to development and validation cohorts in a 3:1 ratio, with potential prognostic factors recorded prospectively. Univariate analyses identified clinical and laboratory factors significantly associated with median overall survival (mOS) in the development cohort, followed by multivariate analyses to determine independent prognostic factors. These factors were utilized to develop a prognostic model. Internal validation was conducted using the validation cohort.

Results: The number of metastatic organs, Eastern Cooperative Oncology Group Performance Status (ECOG PS), time to distant metastasis (TTDM), white blood cell (WBC) count and alkaline phosphatase (ALP) were identified as independent prognostic factors for mUTUC. Patients were stratified into three risk categories: favorable (0 risk factors, mOS 65.0 months, 95% confidence interval [CI] 35.5-94.6]), intermediate (1 risk factor, mOS 32.0 months, 95% CI 25.8-38.2), and poor (2 + risk factors, mOS 16.0 months, 95% CI 12.1-19.9) (P < 0.001). The model's concordance statistic (c-statistic) was 0.71.

Conclusion: We developed and validated a prognostic model to estimate survival of patients with mUTUC receiving first-line systemic therapy. This model is applicable to real-world clinical practice and may inform the design of future clinical trials.