TY - JOUR
T1 - Prognostic Implication of the United States Food and Drug Administration-defined BCG-unresponsive Disease [Figure presented]
AU - Li, Roger
AU - Tabayoyong, William B.
AU - Guo, Charles C.
AU - González, Graciela M.Nogueras
AU - Navai, Neema
AU - Grossman, H. Barton
AU - Dinney, Colin P.
AU - Kamat, Ashish M.
N1 - Funding Information:
Funding/Support and role of the sponsor: This work was supported in part by the Wayne B. Duddlesten Professorship and Floyd Family Research Support for AMK; Database was supported by Cancer Center Support Grant (NCI Grant P30 CA016672) and the GU Bladder SPORE.
Publisher Copyright:
© 2018 European Association of Urology
PY - 2019/1
Y1 - 2019/1
N2 - The category “BCG-unresponsive disease” formulated by experts at the request of the United States Food and Drug Administration, denotes a group of patients with recurrent non-muscle-invasive bladder cancer for whom continued BCG treatment is unlikely to provide benefit. Although quickly adopted for trial design, many of the nuances within the definition lack validation. In this study, we evaluated the prognostic value of BCG unresponsive designation (i.e. recurrence after induction plus at least 1 maintenance course of BCG) by comparing the oncologic outcomes of these patients with those recurring after induction BCG alone. We confirm that appropriately defined, BCG-unresponsive patients are more likely to require salvage radical cystectomy (54.5% vs 17.9%, p = 0.002). Moreover, those opting for second-line bladder-sparing therapies are less likely to remain free of tumor recurrence (23% vs 69.2%, p = 0.003). On multivariate analysis, BCG-unresponsive disease independently predicts inferior high-grade recurrence-free survival (hazard ratio [HR]: 6.25, 95% confidence interval [CI]: 2.27–16.67; p < 0.001) and cystectomy-free survival (HR: 3.85, 95% CI: 1.49–10.0; p = 0.006). Our data confirm the prognostic implication of the BCG unresponsive definition i.e. recurrence of high grade disease after induction and one course of maintenance BCG, and support its use in counseling and risk stratification of patients with tumor recurrence after BCG. Patient summary: Patients who have BCG-unresponsive disease, that is, high-grade non-muscle-invasive bladder cancer recurring after BCG induction and maintenance, have a low likelihood to respond to further BCG treatment and should consider radical cystectomy or clinical trial enrollment. Bacillus Calmette-Guerin (BCG)-unresponsive disease, which is high-grade non-muscle-invasive bladder cancer recurring after BCG induction and at least one course maintenance, is a distinct risk category and must be recognized as such. These patients have a very low likelihood to respond to further BCG treatment and should consider radical cystectomy or clinical trial enrollment.
AB - The category “BCG-unresponsive disease” formulated by experts at the request of the United States Food and Drug Administration, denotes a group of patients with recurrent non-muscle-invasive bladder cancer for whom continued BCG treatment is unlikely to provide benefit. Although quickly adopted for trial design, many of the nuances within the definition lack validation. In this study, we evaluated the prognostic value of BCG unresponsive designation (i.e. recurrence after induction plus at least 1 maintenance course of BCG) by comparing the oncologic outcomes of these patients with those recurring after induction BCG alone. We confirm that appropriately defined, BCG-unresponsive patients are more likely to require salvage radical cystectomy (54.5% vs 17.9%, p = 0.002). Moreover, those opting for second-line bladder-sparing therapies are less likely to remain free of tumor recurrence (23% vs 69.2%, p = 0.003). On multivariate analysis, BCG-unresponsive disease independently predicts inferior high-grade recurrence-free survival (hazard ratio [HR]: 6.25, 95% confidence interval [CI]: 2.27–16.67; p < 0.001) and cystectomy-free survival (HR: 3.85, 95% CI: 1.49–10.0; p = 0.006). Our data confirm the prognostic implication of the BCG unresponsive definition i.e. recurrence of high grade disease after induction and one course of maintenance BCG, and support its use in counseling and risk stratification of patients with tumor recurrence after BCG. Patient summary: Patients who have BCG-unresponsive disease, that is, high-grade non-muscle-invasive bladder cancer recurring after BCG induction and maintenance, have a low likelihood to respond to further BCG treatment and should consider radical cystectomy or clinical trial enrollment. Bacillus Calmette-Guerin (BCG)-unresponsive disease, which is high-grade non-muscle-invasive bladder cancer recurring after BCG induction and at least one course maintenance, is a distinct risk category and must be recognized as such. These patients have a very low likelihood to respond to further BCG treatment and should consider radical cystectomy or clinical trial enrollment.
KW - BCG
KW - BCG-unresponsive disease
KW - Bladder cancer
KW - Intravesical therapy
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U2 - 10.1016/j.eururo.2018.09.028
DO - 10.1016/j.eururo.2018.09.028
M3 - Article
C2 - 30301695
AN - SCOPUS:85054434577
SN - 0302-2838
VL - 75
SP - 8
EP - 10
JO - European urology
JF - European urology
IS - 1
ER -