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

Abstract Prognosis for patients with metastatic bladder carcinoma (mBC) remains limited and in need of novel therapies. We retrospectively analyzed medical records 43 platinum-refractory cancer who participated one or more phase I trials various investigational Patients' tumors circulating tumor DNA were by next-generation sequencing. The median progression-free survival was 4.2 months, the overall 9.6 response rate 17.5%. TP53, ERBB2, PI3KCA, FGFR3, ARID1A alterations detected 66%, 29%, 27%, 24%, 22% all patients, respectively. Alterations FGFR3 almost mutually exclusive TP53. More than half (64%) an FGFR alt received inhibitor, 67% which achieved disease control. Among urothelial histology, those harboring a TP53 alteration had shorter (PFS) compared whose carry wild-type reverse relationship observed alteration. Implications: Patients mBC derive clinical benefit from participating early-phase their outcomes correlate genetic profile tumor. Visual Overview: http://mcr.aacrjournals.org/content/molcanres/19/3/395/F1.large.jpg.