MP16-08: Challenging the Paradigm of "BCG Unresponsive" Bladder Cancer: does additional BCG have an effect?
Friday, May 3, 2024 1:00 PM to 3:00 PM · 2 hr. (US/Central)
221B
Abstract
Information
Full Abstract and Figures
Author Block
Amanda A Myers*, Wei Shen Tan, Valentina Grajales, Hyunsoo Hwang, Kelly K Bree, Neema Navai, Byron H Lee, Colin P N Dinney, Ashish M Kamat, Houston, TX
Introduction
The category "BCG unresponsive disease", was formulated by experts and adopted by the United States Food and Drug Administration in 2018, mainly to facilitate single arm registration studies for novel agents. This paradigm has had tremendous success with 2 agents approved and additional trials ongoing. Over time, this term has become incorporated into clinical use with the implication that additional BCG should not be offered to these patients. We aim to report response rates to additional (rescue) BCG in patients meeting BCG unresponsive criteria.
Methods
We performed an IRB approved review of patients diagnosed with NMIBC between January 2000 and September 2021 to identify patients who met BCG unresponsive criteria. We analyzed the outcomes of patients who received rescue BCG as primary therapy. The primary outcome evaluated was event free survival (EFS): defined as any HG recurrence, progression, or death. The Kaplan-Meier (KM) method was used to estimate EFS, cystectomy free survival (CFS), progression to muscle-invasive or metastatic free survival (PFS) and overall survival (OS).
Results
We identified 163 patients with BCG unresponsive disease. Of these, 35 patients (T1: 19, Ta: 7, CIS only: 9 and concomitant CIS: 8) received rescue BCG as primary therapy. 26 (74%) patients showed no disease after rescue BCG; of these 25 (96%) received maintenance. Median follow-up was 5.9 years (IQR 2.8-8.5). KM estimates of EFS after rescue BCG were 68% at 12 months, 62% at 24 months and 48% at 36 months. Individual patient trajectories are displayed in Figure 1. In the 2 years after rescue BCG, 21 patients remained NED, 4 progressed to MIBC and 9 recurred without progression – of these, 2 had cystectomy and 7 had further bladder sparing therapies. This resulted in CFS at 12, 24 and 36 months (95% CI) of 88% (72-95), 79% (61-90) and 79% (61-90), respectively. PFS at 12, 24 and 36 months (95% CI) was 91% (82-100), 88% (77-100) and 76% (61-94), respectively. OS at 12, 24 and 36 months (95% CI) was 94% (86-100), 91% (82-100) and 85% (73-98), respectively.
Conclusions
In selected patients who meet the “BCG unresponsive” definition, additional BCG therapy can demonstrate appreciable efficacy. This challenges current paradigms which recommend against using further BCG in these patients and emphasizes the need for randomized controlled trials.
Source Of Funding
n/a
Author Block
Amanda A Myers*, Wei Shen Tan, Valentina Grajales, Hyunsoo Hwang, Kelly K Bree, Neema Navai, Byron H Lee, Colin P N Dinney, Ashish M Kamat, Houston, TX
Introduction
The category "BCG unresponsive disease", was formulated by experts and adopted by the United States Food and Drug Administration in 2018, mainly to facilitate single arm registration studies for novel agents. This paradigm has had tremendous success with 2 agents approved and additional trials ongoing. Over time, this term has become incorporated into clinical use with the implication that additional BCG should not be offered to these patients. We aim to report response rates to additional (rescue) BCG in patients meeting BCG unresponsive criteria.
Methods
We performed an IRB approved review of patients diagnosed with NMIBC between January 2000 and September 2021 to identify patients who met BCG unresponsive criteria. We analyzed the outcomes of patients who received rescue BCG as primary therapy. The primary outcome evaluated was event free survival (EFS): defined as any HG recurrence, progression, or death. The Kaplan-Meier (KM) method was used to estimate EFS, cystectomy free survival (CFS), progression to muscle-invasive or metastatic free survival (PFS) and overall survival (OS).
Results
We identified 163 patients with BCG unresponsive disease. Of these, 35 patients (T1: 19, Ta: 7, CIS only: 9 and concomitant CIS: 8) received rescue BCG as primary therapy. 26 (74%) patients showed no disease after rescue BCG; of these 25 (96%) received maintenance. Median follow-up was 5.9 years (IQR 2.8-8.5). KM estimates of EFS after rescue BCG were 68% at 12 months, 62% at 24 months and 48% at 36 months. Individual patient trajectories are displayed in Figure 1. In the 2 years after rescue BCG, 21 patients remained NED, 4 progressed to MIBC and 9 recurred without progression – of these, 2 had cystectomy and 7 had further bladder sparing therapies. This resulted in CFS at 12, 24 and 36 months (95% CI) of 88% (72-95), 79% (61-90) and 79% (61-90), respectively. PFS at 12, 24 and 36 months (95% CI) was 91% (82-100), 88% (77-100) and 76% (61-94), respectively. OS at 12, 24 and 36 months (95% CI) was 94% (86-100), 91% (82-100) and 85% (73-98), respectively.
Conclusions
In selected patients who meet the “BCG unresponsive” definition, additional BCG therapy can demonstrate appreciable efficacy. This challenges current paradigms which recommend against using further BCG in these patients and emphasizes the need for randomized controlled trials.
Source Of Funding
n/a