Systematic Review of the Definition of Cure and of Curative-intent Treatments in Early Bladder Cancer.

BACKGROUND AND

OBJECTIVE

Current guidelines on bladder cancer lack consensus on which outcomes should be used to define cure, the optimal time point for measurement, and which treatments can be given with curative intent in early bladder cancer. Our aim was to determine the optimal definition of cure by assessing cure definitions used in non-muscle invasive bladder cancer (NMIBC) and muscle invasive bladder cancer (MIBC) studies.

METHODS

We conducted a systematic literature review via Embase and Medline on July 10, 2024, to identify real-world evidence (RWE) studies published within the previous 5 yr reporting key effectiveness outcomes (time to recurrence [TTR], overall survival [OS], and recurrence-free survival [RFS]) of NMIBC and MIBC treatments.

KEY FINDINGS AND LIMITATIONS: Among 83 publications included in the review, median TTR ranged from 10 to 89 mo across seven NMIBC studies, and from 6 to 19 mo across five MIBC studies. RFS was the first and second most commonly reported outcome among the NMIBC and MIBC publications, respectively. OS and RFS were commonly reported at 1, 2, and 5 yr among NMIBC studies, and at 3 and 5 yr among MIBC studies. Limitations include the use of RWE only, the 5-yr cut-off for the publications included, and the scarcity of relevant data.

CONCLUSIONS AND CLINICAL IMPLICATIONS: According to the evidence reviewed, 5-yr RFS appears to be a suitable definition of cure in early bladder cancer. Consensus on this or another surrogate outcome for measurement of cure could support the development of clinical trial designs and inform decision-making in health care from both clinician-patient and reimbursement perspectives.