Long-Term Outcomes and Costs Among BCG-treated High-Risk Non-Muscle Invasive Bladder Cancer Patients in an Equal Access Setting

Abstract

Management of high-risk non-muscle invasive bladder cancer (NMIBC) represents a clinical challenge due to high failure rates despite prior bacillus Calmette-Guérin (BCG) therapy. We describe real-world patient characteristics, long-term outcomes, as well as the economic burden in a high-risk NMIBC population. We retrospectively identified a random sample of 412 high-risk NMIBC patients who received ≥ 1 dose of BCG within Veterans Affairs (VA) centers from Jan 1, 2000, to Dec 31, 2016. HR NMIBC was defined as high-grade Ta (TaHG), T1, and/or carcinoma-in-situ (CIS). Adequate BCG induction included at least 5 of 6 instillations, and adequate BCG therapy was at least 7 instillations. We used the Kaplan-Meier method to estimate outcomes including event-free survival. All-cause expenditures were summarized as medians with corresponding interquartile ranges (IQR) and adjusted to 2019 USD. The total follow-up was 2,694 person-years. At high-risk NMIBC diagnosis, 69 (17%) patients had CIS +/- T1 or TaHG, and 341 (83%) had TaHG or T1, no CIS. A total of 392 (95%) patients received adequate BCG induction and 152 (37%) patients received adequate BCG therapy. Recurrence and progression were observed in 61 (32%) and 71 (17%) patients, respectively. There were 166 deaths during follow-up, of which 27 (7%) patients died from bladder cancer. Total median costs at 1, 2 and 5-year were $29,459 ($14,991-$52,060), $55,267 ($28,667-$99,846), and $117,361 ($59,680-$211,298), respectively. Patients which progressed had significantly higher costs (5-yr, $232,729 vs. $94,879, p<0.001) with outpatient care, pharmacy, and surgery related costs contributed largely to the higher costs associated with disease progression. From initial BCG dose to end of follow-up among patients that underwent radical cystectomy, the median all-cause expenditure per patient was $366,857 (278,462 – 668,378). In an equal access setting, the vast majority of BCG-treated patients with high-risk NMIBC received adequate BCG induction; however, less than 50% of patients received adequate maintenance therapy. Patients with CIS had increased risk of progression which was associated with significantly increased costs up to 5-yrs after diagnosis. These findings associated with $366,857 per patient that underwent subsequent radical cystectomy further highlight the considerable economic burden of managing BCG treated high-risk NMIBC.