SYSTEMATIC LITERATURE REVIEW OF PROGNOSTIC FACTORS AND TREATMENT EFFECT MODIFIERS IN NON-MUSCLE INVASIVE BLADDER CANCER
Author(s)
Ashish Kamat, MD1, Ruhee Jain, MBA, MPH2, Siobhán Mulhern-Haughey, PhD3, Jiayue Yu, MS4, Ben Mayer, MPH5, Sarah Cote, MS6, Jianming He, PhD2;
1MD Anderson, Houston, TX, USA, 2Johnson & Johnson, Raritan, NJ, USA, 3Johnson & Johnson, Dublin, Ireland, 4Cytel, Toronto, ON, Canada, 5Cytel, London, United Kingdom, 6Johnson & Johnson, Toronto, ON, Canada
1MD Anderson, Houston, TX, USA, 2Johnson & Johnson, Raritan, NJ, USA, 3Johnson & Johnson, Dublin, Ireland, 4Cytel, Toronto, ON, Canada, 5Cytel, London, United Kingdom, 6Johnson & Johnson, Toronto, ON, Canada
OBJECTIVES: To identify prognostic factors (PFs) and treatment-effect modifiers (TEMs) in intermediate or high risk non-muscle-invasive bladder cancer (NMIBC) to inform indirect treatment comparisons.
METHODS: Systematic literature review of real-world evidence and clinical trials published between 2014 and 2025 in intermediate or high risk NMIBC. Data extraction included study design, patient characteristics, associations between baseline variables and outcomes, as well as subgroup analysis.
RESULTS: We screened 5,237 records and included 79 unique analyses (64 retrospective/database analyses, 5 prospective observational studies, 9 randomized controlled trials, 1 non-randomized trial) in intermediate or high risk NMIBC. Included publications evaluated 13 outcome measures. Seven baseline factors were consistently associated with outcomes: age, tumor stage, tumor burden (size/number), tumor grade, carcinoma in situ (CIS), prior BCG therapy, and histological subtype. Age was most consistently associated with worse overall survival (OS), cancer-specific survival (CSS), and disease-free survival (DFS). The presence of CIS was strongly linked to progression and mortality. Prior BCG exposure correlated with recurrence risk. Tumor burden, tumor stage, tumor grade, and histological subtype were associated with recurrence-free survival (RFS), progression-free survival (PFS), and CSS. Only three publications (all RCTs) reported TEM analyses; tumor stage and CIS status emerged as potential TEMs, but evidence was limited by the small number of subgroup analyses and inconsistent reporting.
CONCLUSIONS: Seven PFs are consistently associated with outcomes in intermediate or high-risk NMIBC and should be considered when matching baseline characteristics for indirect comparisons. Robust TEM evidence is scarce. These findings can inform future trial design and analytical approaches to reduce bias in comparative effectiveness research in localized bladder cancer.
METHODS: Systematic literature review of real-world evidence and clinical trials published between 2014 and 2025 in intermediate or high risk NMIBC. Data extraction included study design, patient characteristics, associations between baseline variables and outcomes, as well as subgroup analysis.
RESULTS: We screened 5,237 records and included 79 unique analyses (64 retrospective/database analyses, 5 prospective observational studies, 9 randomized controlled trials, 1 non-randomized trial) in intermediate or high risk NMIBC. Included publications evaluated 13 outcome measures. Seven baseline factors were consistently associated with outcomes: age, tumor stage, tumor burden (size/number), tumor grade, carcinoma in situ (CIS), prior BCG therapy, and histological subtype. Age was most consistently associated with worse overall survival (OS), cancer-specific survival (CSS), and disease-free survival (DFS). The presence of CIS was strongly linked to progression and mortality. Prior BCG exposure correlated with recurrence risk. Tumor burden, tumor stage, tumor grade, and histological subtype were associated with recurrence-free survival (RFS), progression-free survival (PFS), and CSS. Only three publications (all RCTs) reported TEM analyses; tumor stage and CIS status emerged as potential TEMs, but evidence was limited by the small number of subgroup analyses and inconsistent reporting.
CONCLUSIONS: Seven PFs are consistently associated with outcomes in intermediate or high-risk NMIBC and should be considered when matching baseline characteristics for indirect comparisons. Robust TEM evidence is scarce. These findings can inform future trial design and analytical approaches to reduce bias in comparative effectiveness research in localized bladder cancer.
Conference/Value in Health Info
2026-05, ISPOR 2026, Philadelphia, PA, USA
Value in Health, Volume 29, Issue S6
Code
CO181
Topic
Clinical Outcomes
Topic Subcategory
Comparative Effectiveness or Efficacy
Disease
SDC: Oncology, SDC: Urinary/Kidney Disorders