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Review
. 2022 Jan 10:14:35-48.
doi: 10.2147/CEOR.S341896. eCollection 2022.

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

Mihaela Georgiana Musat  1 , Christina Soeun Kwon  1 , Elizabeth Masters  2 , Slaven Sikirica  3 , Debduth B Pijush  3 , Anna Forsythe  4
Affiliations
Review

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

Mihaela Georgiana Musat et al. Clinicoecon Outcomes Res. .

Abstract

Background: 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: high-risk NMIBC; real-world outcomes.

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Conflict of interest statement

The following authors were Pfizer employees at the time the study was conducted: Elizabeth Masters and Slaven Sikirica. Debduth B Pijush was an employee of Atrium Staffing, which was a paid contractor to Pfizer in connection with the development of this manuscript. Mihaela Georgiana Musat, Christina Soeun Kwon, and Anna Forsythe are employees of Purple Squirrel Economics, which was a paid consultant to Pfizer in connection with the development of this manuscript. The authors report no other conflicts of interest in this work.

Figures

Figure 1
Figure 1
PRISMA flow diagram of the real-world SLR in NMIBC.
Figure 2
Figure 2
Tumor stages/grades reported in HR-NMIBC real-world studies (weighted-average %).
Figure 3
Figure 3
Five-year survival outcomes. Five-year survival outcomes for (A) recurrence-free survival (RFS), (B) progression-free survival (PFS), (C) cancer-specific survival (CSS), and (D) overall survival (OS).
Figure 4
Figure 4
Median OS (months) in HR-NMIBC.
Figure 5
Figure 5
CSS and OS rates by follow-up time (months) in HR-NMIBC. Follow-up time in HR-NMIBC in (A) CSS and (B) OS rates. Each blue dot corresponds to a value from an individual study.
See this image and copyright information in PMC

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