Small Cell Bladder Cancer: Treatment Patterns for Local Disease and Associated Outcomes. A Retrospective Cohort Study.

Background: Small cell bladder cancer (SCBC) is a rare histologic subtype with relative paucity of data regarding treatment response and outcomes. We reviewed 2 databases to compare outcomes in patients with localized SCBC treated with cystectomy versus concurrent chemoradiotherapy (CCRT). We hypothesized that survival would be similar with these therapy approaches.
Methods: We retrospectively reviewed our institutional and SEER-Medicare databases to identify patients with SCBC. Overall survival (OS) was determined from the date of diagnosis to last follow-up/death. For those with nonmetastatic disease, a multivariate Cox analysis was used to compare locoregional therapy with neoadjuvant chemotherapy (NAC) + cystectomy versus CCRT.
Results: We identified 53 patients in our institutional database and 1166 patients in SEER-Medicare with localized SCBC. Median OS (mOS) with NAC + cystectomy was 46 months (95% CI, 21-72) and 45 months (95% CI, 0-104) in the institutional and SEER-Medicare databases, respectively, whereas mOS with CCRT was 26 months (95% CI, 5-47) and 23 months (95% CI, 18-28) in the 2 series, respectively. In multivariate analysis, NAC followed by cystectomy was associated with an approximately 30% reduction in mortality compared to CCRT in both institutional and national databases but did not reach statistical significance (Institution HR 0.71, 95% CI, 0.22-2.4, P = .58; SEER HR 0.73, 95% CI, 0.49-1.08; P = .11).
Conclusions: SCBC is very aggressive with limited survival observed in our institutional and SEER-Medicare datasets regardless of locoregional therapy used. There is an unmet need to define the optimal locoregional therapy for nonmetastatic stage and identify novel therapeutic targets.
Competing Interests: Disclosure Dimitra Rafailia Bakaloudi: no conflicts to disclose. Elizabeth L. Koehne: no conflicts to disclose. Leonidas N. Diamantopoulos: no conflicts to disclose. Sarah K. Holt: no conflicts to disclose. Rishi R. Sekar: no conflicts to disclose. Fady Ghali: Consulting: Immunitybio Funda Vakar-Lopez: no conflicts to disclose. Yaw A. Nyame: no conflicts to disclose. Sarah P. Psutka: Research Funding: National Institute on Aging; Bladder Cancer Advocacy Network; PRIME Education, Inc; Guidelines Committee: American Urological Association: Upper Tract Urothelial Carcinoma Guidelines 2023 Advisory/Consultancy: Janssen (SunRise-4 Global Co-PI), Immunity Bio, Merck, CG Oncology John L. Gore: Research Grant funding from Ferring Pharmaceuticals. Claire de la Calle: no conflicts to disclose. Daniel W. Lin: DSMB for the POTOMAC study with AstraZeneca; Consulting or Advisory Role—Astellas Pharma, Clovis Oncology, Janssen Oncology, and Lantheus, Research Funding –Veracyte; MDxHealth. George R. Schade: Advisor ImmunityBio, Consultant EDAP. Intellectual property licensed to Petal Surgical. Jay Liao: no conflicts to disclose. Andrew C. Hsieh: Honoraria—Hotspot Therapeutics; Research Funding—eFFECTOR Inc; Patents, Royalties, Other Intellectual Property—MTOR modulators and uses thereof Patent number: 9629843; Use Of Translational Profiling To Identify Target Molecules For Therapeutic Treatment, Publication number: 20140288097. Todd Yezefski: Institutional research funding from AstraZeneca, Astellas, participation in educational programs for Dendreon Jessica E. Hawley: consulting for Seagen, Daiichi-Sankyo, Immunity Bio; institutional research support from AstraZeneca, Bristol-Meyers Squibb, Crescendo Biologics, Macrogenics, Johnson & Johnson Innovative Medicine, PromiCell, Amgen, and Vaccitech. Evan Y. Yu: consulting for Aadi Bioscience, Advanced Accelerator Applications, Bayer, Bristol-Myers Squibb, Janssen, Lantheus, Loxo, Merck, Oncternal; institutional research support from Bayer, Blue Earth, Daiichi-Sankyo, Dendreon, Lantheus, Merck, Oncternal, Seagen, Surface, Taiho, Tyra. Robert B. Montgomery: Research Funding—AstraZeneca, Bayer, Clovis, Janssen Oncology. Petros Grivas (unrelated to this manuscript in the last 2 years): consulting for MSD, Bristol Myers Squibb, AstraZeneca, EMD Serono, Seagen, Pfizer, Janssen, Roche, Astellas Pharma, Gilead Sciences, Silverback Therapeutics, BostonGene, Fresenius Kabi, Lucence Health, PureTech, G1 Therapeutics, Aadi Biosciences, CG Oncology, Strata Oncology, ImmunityBio, Asieris Pharmaceuticals, AbbVie; Institutional research funding: Pfizer, Bristol Myers Squibb, MSD, QED Therapeutics, GlaxoSmithKline, Mirati Therapeutics, EMD Serono, G1 Therapeutics, Gilead Sciences, Acrivon Therapeutics, ALX Oncology, Genentech. Jonathan L. Wright: Royalties—UpToDate; Clinical Trials/Research—Merck, Nucleix, Altor Biosciences, Pacific Edge, Seagen, Janssen, Veracyte; Consulting—ImmunityBio; Pacific Edge.
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