TY - JOUR
T1 - Eligibility and radiologic assessment in adjuvant clinical trials in bladder cancer
AU - Apolo, Andrea B.
AU - Milowsky, Matthew I.
AU - Kim, Lauren
AU - Inman, Brant A.
AU - Kamat, Ashish M.
AU - Steinberg, Gary
AU - Bagheri, Mohammadhadi
AU - Krishnasamy, Venkatesh P.
AU - Marko, Jamie
AU - Dinney, Colin P.
AU - Bangs, Rick
AU - Sweis, Randy F.
AU - Maher, Virginia Ellen
AU - Ibrahim, Amna
AU - Liu, Ke
AU - Werntz, Ryan
AU - Cross, Frank
AU - Beaver, Julia A.
AU - Singh, Harpreet
AU - Pazdur, Richard
AU - Blumenthal, Gideon M.
AU - Lerner, Seth P.
AU - Bajorin, Dean F.
AU - Rosenberg, Jonathan E.
AU - Agrawal, Sundeep
N1 - Funding Information:
Funding/Support: This work was supported by the Intramural Research Program of the Center for Cancer Research, National Cancer Institute, National Institutes of Health and the Memorial Sloan Kettering Cancer Center P30 CA008748 grant.
Funding Information:
Author Affiliations: National Institutes of Health, Bethesda, Maryland (Apolo, Kim, Bagheri, Krishnasamy, Marko, Bangs); Department of Medicine, Lineberger Comprehensive Cancer Center, University of North Carolina at Chapel Hill (Milowsky); Department of Surgery, Duke Cancer Institute, Durham, North Carolina (Inman); Department of Urology, The University of Texas MD Anderson Cancer Center, Houston (Kamat, Dinney); NYU Langone Health, New York, New York (Steinberg); University of Chicago Medicine, Chicago, Illinois (Sweis, Werntz); Center for Drug Evaluation and Research, US Food and Drug Administration, Silver Spring, Maryland (Maher, Ibrahim, Cross, Beaver, Singh, Agrawal); Center for Biologics Evaluation and Research, US Food and Drug Administration, Silver Spring, Maryland (Liu); Oncology Center of Excellence, US Food and Drug Administration, Silver Spring, Maryland (Pazdur, Blumenthal); Department of Urology, Baylor College of Medicine, Houston, Texas (Lerner); Department of Medicine, Memorial Sloan Kettering Cancer Center, New York, New York (Bajorin, Rosenberg).
Funding Information:
The US Food and Drug Administration and the National Cancer Institute, with support from the Society of Urologic Oncology, convened a public workshop on November 28, 2017, at the National Institutes of Health in Bethesda, Maryland, to discuss protocol criteria for adjuvant clinical trials of muscle-invasive bladder cancer (MIBC) and kidney cancer. This effort focused on 4 topics regarding MIBC adjuvant trials: (1) the role of patient and disease characteristics, (2) defining radiographic eligibility, (3) defining disease recurrence, and (4) management of non–muscle-invasive bladder cancer (NMIBC). Multiple virtual meetings were used to outline these topics, and major issues were discussed further at the workshop with input from investigators, patient advocates, biostatisticians, industry representatives, regulators, and the public. This report summarizes discussions from the workshop and associated meetings. The kidney cancer component of the workshop addresses many of these same issues and contains complementary material (S.A., unpublished information, September 2019). The key workshop discussion points are summarized in Table 1.1 Some recommendations in Table 1 are not included in the text.
Funding Information:
Apolo, MD, Center for Cancer Research, National Cancer Institute, National Institutes of Health, 10 Center Dr, 13N240, MSC 1906, Bethesda, MD 20892 (andrea.apolo @nih.gov).
Publisher Copyright:
© 2019 American Medical Association. All rights reserved.
PY - 2019/12
Y1 - 2019/12
N2 - Objective: To harmonize eligibility criteria and radiographic disease assessments in clinical trials of adjuvant therapy for muscle-invasive bladder cancer (MIBC). Methods: National experts in bladder cancer clinical trial research, including medical and urologic oncologists, radiologists, biostatisticians, and patient advocates, convened at a public workshop on November 28, 2017, to discuss eligibility, radiographic entry criteria, and assessment of disease recurrence in adjuvant clinical trials in patients with MIBC. Results: The key workshop conclusions for adjuvant MIBC clinical trials included the following points: (1) patients with urothelial carcinoma with divergent histologic differentiation should be allowed to enroll; (2) neoadjuvant chemotherapy is defined as at least 3 cycles of neoadjuvant cisplatin-based combination chemotherapy; (3) patients with muscle-invasive, upper-Tract urothelial carcinoma should be included in adjuvant trials of MIBC; (4) patients with severe renal insufficiency can enroll into trials using agents that are not renally excreted; (5) patients with microscopic surgical margins can be included; (6) patients should undergo a standard bilateral lymph node dissection prior to enrollment; (7) computed tomographic (CT) imaging should be performed within 4 weeks prior to enrollment. For patients with renal insufficiency who cannot undergo CT imaging with contrast, noncontrast chest CT and magnetic resonance imaging of the abdomen and pelvis with gadolinium should be done; (8) biopsy of indeterminate lesions to evaluate for malignant disease should be done when feasible; (9) a uniform approach to evaluate indeterminate radiographic lesions when biopsy is not feasible should be included in any trial design; (10) a uniform approach to determining the date of recurrence is important in interpreting adjuvant trial results; and (11) new high-grade, upper-Tract primary tumors and new MIBC tumors should be considered recurrence events. Conclusions and Relevance: A uniform approach to eligibility criteria, definitions of no evidence of disease, and definitions of disease recurrence may lead to more consistent interpretations of adjuvant trial results in MIBC.
AB - Objective: To harmonize eligibility criteria and radiographic disease assessments in clinical trials of adjuvant therapy for muscle-invasive bladder cancer (MIBC). Methods: National experts in bladder cancer clinical trial research, including medical and urologic oncologists, radiologists, biostatisticians, and patient advocates, convened at a public workshop on November 28, 2017, to discuss eligibility, radiographic entry criteria, and assessment of disease recurrence in adjuvant clinical trials in patients with MIBC. Results: The key workshop conclusions for adjuvant MIBC clinical trials included the following points: (1) patients with urothelial carcinoma with divergent histologic differentiation should be allowed to enroll; (2) neoadjuvant chemotherapy is defined as at least 3 cycles of neoadjuvant cisplatin-based combination chemotherapy; (3) patients with muscle-invasive, upper-Tract urothelial carcinoma should be included in adjuvant trials of MIBC; (4) patients with severe renal insufficiency can enroll into trials using agents that are not renally excreted; (5) patients with microscopic surgical margins can be included; (6) patients should undergo a standard bilateral lymph node dissection prior to enrollment; (7) computed tomographic (CT) imaging should be performed within 4 weeks prior to enrollment. For patients with renal insufficiency who cannot undergo CT imaging with contrast, noncontrast chest CT and magnetic resonance imaging of the abdomen and pelvis with gadolinium should be done; (8) biopsy of indeterminate lesions to evaluate for malignant disease should be done when feasible; (9) a uniform approach to evaluate indeterminate radiographic lesions when biopsy is not feasible should be included in any trial design; (10) a uniform approach to determining the date of recurrence is important in interpreting adjuvant trial results; and (11) new high-grade, upper-Tract primary tumors and new MIBC tumors should be considered recurrence events. Conclusions and Relevance: A uniform approach to eligibility criteria, definitions of no evidence of disease, and definitions of disease recurrence may lead to more consistent interpretations of adjuvant trial results in MIBC.
UR - http://www.scopus.com/inward/record.url?scp=85074486037&partnerID=8YFLogxK
UR - http://www.scopus.com/inward/citedby.url?scp=85074486037&partnerID=8YFLogxK
U2 - 10.1001/jamaoncol.2019.4114
DO - 10.1001/jamaoncol.2019.4114
M3 - Article
C2 - 31670753
AN - SCOPUS:85074486037
VL - 5
SP - 1790
EP - 1798
JO - JAMA oncology
JF - JAMA oncology
SN - 2374-2437
IS - 12
ER -