Molecular Profiling of Metastatic Bladder Cancer Early-Phase Clinical Trial Participants Predicts Patient Outcomes

• Published in: Molecular cancer research Vol. 19; no. 3; pp. 395 - 402
• Main Authors: Alhalabi, Omar, Hahn, Andrew W., Msaouel, Pavlos, Andreev-Drakhlin, Alexander Y., Meric-Bernstam, Funda, Naing, Aung, Piha-Paul, Sarina, Filip, Janku, Pant, Shubham, Yap, Timothy A., Hong, David S., Fu, Siqing, Karp, Daniel, Campbell, Erick, Le, Hung, Campbell, Matthew T., Shah, Amishi Y., Tannir, Nizar M., Siefker-Radtke, Arlene O., Gao, Jianjun, Roszik, Jason, Subbiah, Vivek
• Format: Journal Article
• Language: English
• Published: United States 01.03.2021
• ISSN: 1541-7786; 1557-3125; 1557-3125
• DOI: 10.1158/1541-7786.MCR-20-0751

Prognosis for patients with metastatic bladder carcinoma (mBC) remains limited and in need of novel therapies. We retrospectively analyzed medical records of 43 patients with platinum-refractory metastatic bladder cancer (mBC) who participated in one or more phase I trials of various investigational therapies. Patients' tumors or circulating tumor DNA were analyzed by next-generation sequencing. The median progression-free survival was 4.2 months, the median overall survival was 9.6 months, and the overall response rate was 17.5%. , and alterations were detected in 66%, 29%, 27%, 24%, and 22% of all patients, respectively. Alterations in were almost mutually exclusive of . More than half (64%) of patients with an alt received an FGFR inhibitor, 67% of which achieved disease control. Among patients with urothelial carcinoma histology, those harboring a alteration had a shorter median progression-free survival (PFS) compared with those whose tumors carry wild-type . The reverse relationship was observed in patients harboring an alteration. IMPLICATIONS: Patients with platinum-refractory mBC derive clinical benefit from participating in early-phase clinical trials and their survival outcomes correlate with the genetic profile of the tumor. VISUAL OVERVIEW: http://mcr.aacrjournals.org/content/molcanres/19/3/395/F1.large.jpg.