Multi-country clinical practice patterns, including use of biomarkers, among physicians' treatment of BCG-unresponsive non-muscle invasive bladder cancer (NMIBC)

Abstract

Background: Intravesical bacillus Calmette-Guérin (BCG) fails in a considerable proportion of non-muscle invasive bladder cancer (NMIBC) patients despite treatment per recommended protocol. This real-world study aimed to understand the current patterns of treatment and disease management for the broad BCG-unresponsive NMIBC patient population, alongside collecting sufficient data on patients who do not undergo cystectomy.

Methods: This was a multicenter, retrospective survey of physicians treating BCG-unresponsive NMIBC patients. Data were collected in eight countries - France, Germany, Spain, Italy, United Kingdom, United States, China, and Japan - between January and May 2019. The study consisted of a short online physician survey and a retrospective chart review of eligible BCG-unresponsive NMIBC patients. Physicians abstracted chart data for the last 10 (five patients in Japan) eligible BCG-unresponsive NMIBC patients meeting the inclusion criteria, and the data were analysed for all countries combined using descriptive statistics. Country-specific analyses were also carried out, as appropriate.

Results: Overall, 508 physicians participated in the study. Almost one-quarter (22.9%) of physicians' current NMIBC patient caseload was BCG-unresponsive, whereby BCG therapy was no longer considered an option. Half of physicians (49.4%) did not regularly use biomarker tests in their practice, with particularly few physicians undertaking biomarker testing in Spain and Japan. Biomarker testing varied considerably, with the proportions of physicians selecting 'none' ranging from 11.4% in China to 70.3% in Japan. Physicians reported transurethral resection of the bladder tumor (TURBT) and BCG as the most common current treatments received by their patients. Chemotherapy and anti-PD-L1 treatment options were considered impactful new therapies by 94.7% and 90.0% of physicians surveyed in this study, respectively.

Conclusions: The most common treatments received by patients in this study were TURBT and BCG. Emerging new treatments are driven by exploring biomarkers, but in real-world clinical practice only half of physicians or fewer regularly tested their NMIBC patients for biomarkers; PD-1/PD-L1 was the most common biomarker test used. Most physicians reported that, in addition to chemotherapy, anti-PD-L1 was an impactful new therapy.

Keywords: Biomarkers; Clinical practice patterns; Disease management; Immunotherapy; Non-muscle invasive bladder cancer; Real-world evidence; Urinary bladder neoplasms.

Fig. 1

Physicians’ (n = 508) techniques for diagnosing NMIBC BCG-unresponsiveness (a) and biomarker tests in NMIBC patients (b)

Fig. 2

Physicians’ (n = 508) considerations when recommending radical cystectomy over BCG treatment (a), and vice versa (b)

Fig. 3

a, b Physician proportions (n = 508) reporting current management approaches for NMIBC patients, split by ‘intermediate-‘ and ‘high-risk’

Fig. 4

Top 3 ‘most impactful new therapies for BCG-unresponsive NMIBC patients’, as ranked by physicians (n = 508)

Fig. 5

Country-specific top 3 ‘most impactful new therapies for BCG-unresponsive NMIBC patients’, as ranked by physicians