Salvage Therapy for Non–muscle-invasive Bladder Cancer

Khaled, Dunia; Taylor, John A.; Holzbeierlein, Jeffrey M.

doi:10.1016/j.ucl.2019.09.014

Cited by 5 publications

(2 citation statements)

References 35 publications

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“…This important decision naturally involves a high degree of subjectivity and patient preference. In addition, any benefits of delaying RC must be weighed against the risk of progression from NMIBC to MIBC which would result in a worsening of prognosis from 80% survival to 30% survival at 5 years [43][44][45]. Given these challenges, delay of cystectomy would need to be done judiciously and appropriately, with predetermined and clinically sound criteria for when a cystectomy should be offered in order to mitigate the subjectivity of the endpoint and, more importantly, reduce the likelihood of progression to more advanced MIBC or metastatic disease.…”

Section: Regulatory Considerations For Incorporating a Cystectomy-fre...mentioning

confidence: 99%

Advancing Clinical Trial Design for Non-Muscle Invasive Bladder Cancer

Chang,

Hahn,

Lerner

et al. 2023

Bladder Cancer

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BACKGROUND: Despite recent drug development for non-muscle invasive bladder cancer (NMIBC), few therapies have been approved by the US Food and Drug Administration (FDA), and there remains an unmet clinical need. Bacillus Calmette-Guerin (BCG) supply issues underscore the importance of developing safe and effective drugs for NMIBC. OBJECTIVE: On November 18–19, 2021, the FDA held a public virtual workshop to discuss NMIBC research needs and potential trial designs for future development of effective therapies. METHODS: Representatives from various disciplines including urologists, oncologists, pathologists, statisticians, basic and translational scientists, and the patient advocacy community participated. The workshop format included invited lectures, panel discussions, and opportunity for audience discussion and comment. RESULTS: In a pre-workshop survey, 92% of urologists surveyed considered the development of alternatives to BCG as a high drug development priority for BCG-naïve high-risk patients. Key topics discussed included definitions of disease states; trial design for BCG-naïve NMIBC, BCG-unresponsive carcinoma in situ, and BCG-unresponsive papillary carcinoma; strengths and limitations of single-arm trial designs; assessing patient-reported outcomes; and considerations for assessing avoidance of cystectomy as an efficacy measure. CONCLUSIONS: The workshop discussed several important opportunities for trial design refinement in NMIBC. FDA encourages sponsors to meet with the appropriate review division to discuss trial design proposals for NMIBC early in drug development.

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Section: Regulatory Considerations For Incorporating a Cystectomy-fre...mentioning

confidence: 99%

Advancing Clinical Trial Design for Non-Muscle Invasive Bladder Cancer

Chang,

Hahn,

Lerner

et al. 2023

Bladder Cancer

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“…The past decade has witnessed the rapid development of combination therapy for improved therapeutic outcomes in bladder cancer. Combination therapy has been a successful strategy to enhance efficacy, increase response, reverse resistance and reduce toxicity as well as address tumor heterogeneity upon using different drugs of different dynamics and molecular targets ( 5 , 6 ). Previously, the common combination regimens are drugs composed of different chemotherapeutic anticancer drugs or combining chemotherapy with radiotherapy.…”

Section: Introductionmentioning

confidence: 99%

Novel Combination Therapies for the Treatment of Bladder Cancer

Peng

Xiao

et al. 2021

Front. Oncol.

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Bladder cancer is the ninth most frequently diagnosed cancer world-wide and ranks 13th in cancer-related deaths. Two tremendous breakthroughs in bladder cancer therapy over the last decades are the approval of immune checkpoint inhibitors(ICIs)and the fibroblast growth factor receptor tyrosine kinase inhibitor (FGFR-TKI) erdafitinib for treating this deadly disease. Despite the beneficial effects of these approaches, the low response rate and the potential resistance of the cancer are major concerns. Hence, novel combination therapies to overcome these limitations have been investigated. In this context, combining immunotherapy with targeted drugs is an appealing therapeutic option to improve response and reduce the emergence of resistance in the management of bladder cancer. In this review, the rationale of using different therapeutic combinations is discussed according to the mechanistic differences, emphasizing the efficacy and safety based on evidence collected from preclinical and clinical studies. Finally, we highlight the limitations of these combinations and provide suggestions for further efforts in this challenging field.

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