LAUSR

BACKGROUND Stage-specific guideline recommendations are lacking for chemotherapy in micropapillary carcinoma of the urinary bladder (MCUB). OBJECTIVE To test the efficacy of stage-specific chemotherapy for MCUB. DESIGN, SETTING, AND PARTICIPANTS Within the Surveillance, Epidemiology and End Results (SEER) registry (2001-2016), we identified patients with MCUB and pure urothelial carcinoma of the urinary bladder (UCUB) of all stages. OUTCOME MEASUREMENTS AND STATISTICAL ANALYSIS Kaplan-Meier survival analyses and multivariate Cox regression models were used to determine cancer-specific mortality (CSM) in addition to power analyses. RESULTS AND LIMITATIONS Of 210 491 patients of all stages, 518 (0.2%) harboured MCUB versus 209 973 (99.8%) UCUB. Stage at presentation was invariably higher in MCUB than in UCUB patients. Of the MCUB patients, 223 (43.1%) received chemotherapy versus 42 921 (20.4%) of the UCUB patients. In MCUB patients, chemotherapy improved CSM-free survival significantly in metastatic stage (hazard ratio [HR] 0.36, p = 0.04). Longer median CSM-free survival was also associated with chemotherapy use in addition to radical cystectomy (RC) versus RC alone in non-organ-confined MCUB (HR 0.69, p = 0.2). Additional power analyses revealed an underpowered comparison. Finally, no CSM difference was recorded in organ-confined MCUB according to the use of chemotherapy in addition to RC versus RC alone (HR 0.98, p = 1). CONCLUSIONS Stage at presentation was invariably higher in MCUB than in UCUB patients. Very important CSM reduction was associated with chemotherapy use in metastatic MCUB. A promising protective effect of perioperative chemotherapy might also be applicable to non-organ-confined MCUB, but without sufficient statistical power. Conversely, no association was recorded in organ-confined MCUB. PATIENT SUMMARY Patients with micropapillary carcinoma of the urinary bladder (MCUB) present in higher tumour stages than those with urothelial carcinoma of the urinary bladder. Chemotherapy for MCUB is effective in metastatic stages, but of no beneficial effect in organ-confined stage. In not-yet-metastatic but already non-organ-confined stages, we did not have enough observations to show a statistically significant protective effect of chemotherapy.