Treatment Outcomes of High-Risk Non-Muscle Invasive Bladder Cancer (HR-NMIBC) in Real-World Evidence (RWE) Studies: Systematic Literature Review (SLR)

Abstract

Mihaela Georgiana Musat,1 Christina Soeun Kwon,1 Elizabeth Masters,2 Slaven Sikirica,3 Debduth B Pijush,3 Anna Forsythe4 1Evidence Generation, Purple Squirrel Economics, a Wholly Owned Subsidiary of Cytel, Inc., Waltham, MA, USA; 2Real World Evidence, Pfizer, New York, NY, USA; 3Global Health Economics and Outcomes Research, Pfizer, New York, NY, USA; 4Value and Access, Purple Squirrel Economics, a Wholly Owned Subsidiary of Cytel, Inc., Waltham, MA, USACorrespondence: Mihaela Georgiana MusatPurple Squirrel Economics, a Wholly Owned Subsidiary of Cytel, Inc, 1050 Winter Street, Waltham, MA, 02451, USAEmail [email protected]: To date, there has been limited synthesis of RWE studies in high-risk non-muscle invasive bladder cancer (HR-NMIBC). The objective of this research was to conduct a systematic review of published real-world evidence to better understand the real-world burden and treatment patterns in HR-NMIBC.Methods: An SLR was conducted following the Preferred Reporting Items for Systematic Reviews and Meta-Analyses (PRISMA) guidelines with the scope defined by the Population, Intervention Comparators, Outcomes, and Study design (PICOS) criteria. EMBASE, MEDLINE, and Cochrane databases (Jan 2015–Jul 2020) were searched, and relevant congress abstracts (Jan 2018–Jul 2020) identified. The final analysis only included studies that enrolled ≥ 100 patients with HR-NMIBC from the US, Europe, Canada, and Australia.Results: The SLR identified 634 RWE publications in NMIBC, of which 160 studies reported data in HR-NMIBC. The average age of patients in the studies was 71 years, and 79% were males. The rates of BCG intravesical instillations ranged from 3% to 86% (29– 95% for induction and 8– 83% for maintenance treatment). Five-year outcomes were 17– 89% recurrence-free survival (longest survival in patients completing BCG maintenance), 58– 89% progression-free survival, 71– 96% cancer-specific survival (lowest survival in BCG-unresponsive patients), and 28– 90% overall survival (lowest survival in patients who did not receive BCG or instillation therapy).Conclusion: BCG treatment rates and survival outcomes in patients with HR-NMIBC vary in the real world, with better survival seen in patients completing maintenance BCG, responding to treatment, and not progressing to muscle-invasive disease. There is a need to better understand the factors associated with BCG use and discontinuation and for an effective treatment that improves outcomes in HR-NMIBC. Generalization of these results is limited by variations in data collection, reporting, and methodologies used across RWE studies.Keywords: real-world outcomes, high-risk NMIBC

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