OBJECTIVES: Muscle invasive bladder cancer (MIBC) has a poor prognosis, with half of patients developing metastases. NCCN and ESMO guidelines recommend patients with MIBC receive neoadjuvant cisplatin-based chemotherapy (NAC) followed by radical cystectomy (RC). However, many patients may be ineligible for cisplatin. This systematic literature review (SLR) evaluated real-world treatment effectiveness and economic and humanistic burden in patients with MIBC who received NAC+RC or RC alone to characterize unmet need in these patients.

METHODS: Literature searches conducted in accordance with PRISMA guidelines identified real-world studies (January 2018–June 2023) of adult patients with MIBC who received RC in the US, UK, Germany, France, Italy, and Spain. Publications were screened for those reporting real-world effectiveness (including median overall survival [mOS] and progression-free survival [PFS]), economic burden and humanistic burden.

RESULTS: Of 4,192 references identified, 76 were included following screening. Of these, 61 reported real-word effectiveness, 12 economic burden and 5 humanistic burden. The range of reported mOS was 1.9–6.9 years with NAC+RC (n=9) and 1.5–6 years with RC alone (n=7). Risk of progression was lower with NAC+RC than RC alone (3 studies reported real-world PFS outcomes). Eight studies reported direct costs, with inpatient costs post-RC a large component of total costs. In the US, patients receiving NAC+RC had a shorter median length of hospital stay than those with RC alone. Health-related quality of life decreased following either NAC+RC or RC alone.

CONCLUSIONS: Patients with MIBC who receive either NAC+RC or RC alone experience a high economic and humanistic burden. While NAC may improve outcomes for some patients, not all patients can tolerate NAC and more effective treatments are needed. New data are required to better characterize the impact of new treatments on patient outcomes.