Monitoring of plasma and urine tumor-derived DNA to inform bladder-sparing approaches for patients with muscle-invasive bladder cancer

M Matthew D. Galsky (Division of Hematology and Medical Oncology, Icahn School of Medicine at Mount Sinai) S Sudeh Izadmehr (Division of Hematology and Medical Oncology, Icahn School of Medicine at Mount Sinai) M Menggang Yu (Department of Biostatistics, University of Michigan) S Samuel D. Curtis (Department of Pharmacology and Molecular Sciences, Johns Hopkins University School of Medicine) C Christopher Douville (Department of Oncology, the Sidney Kimmel Comprehensive Cancer Center, Johns Hopkins University School of Medicine) M Maria Popoli (Department of Oncology, the Sidney Kimmel Comprehensive Cancer Center, Johns Hopkins University School of Medicine) J Janine Ptak (Department of Oncology, the Sidney Kimmel Comprehensive Cancer Center, Johns Hopkins University School of Medicine) L Lisa Dobbyn (Department of Oncology, the Sidney Kimmel Comprehensive Cancer Center, Johns Hopkins University School of Medicine) N Natalie Silliman (Department of Oncology, the Sidney Kimmel Comprehensive Cancer Center, Johns Hopkins University School of Medicine) K Kevin G. Chan (Department of Urology, City of Hope Comprehensive Cancer Center) T Tanya B. Dorff (Department of Medical Oncology and Therapeutics, City of Hope Comprehensive Cancer Center) J Jeremy P. Cetnar (Division of Hematology and Medical Oncology, Oregon Health and Science University) B Brock O’Neil (Department of Urology, University of Utah) A Anishka D’Souza (Division of Hematology and Medical Oncology, Keck School of Medicine of University of Southern California, Norris Comprehensive Cancer Center) R Ronac Mamtani (Division of Hematology and Medical Oncology, University of Pennsylvania Abramson Cancer Center) C Christos E. Kyriakopoulos (Division of Hematology and Medical Oncology, University of Wisconsin Carbone Cancer Center) R Rachel Brody (Tisch Cancer Institute, Icahn School of Medicine at Mount Sinai) E Evita Sadimin (Department of Pathology, City of Hope Comprehensive Cancer Center) R Reza Mehrazin (Department of Urology, Icahn School of Medicine at Mount Sinai, Tisch Cancer Institute) D Diego Chowell (Tisch Cancer Institute, Icahn School of Medicine at Mount Sinai) J John Sfakianos (Tisch Cancer Institute, Icahn School of Medicine at Mount Sinai) S Siamak Daneshmand (Department of Urology, Keck School of Medicine of University of Southern California, Norris Comprehensive Cancer Center) S Sumanta K. Pal (Department of Medical Oncology and Therapeutics, City of Hope Comprehensive Cancer Center) C Chetan Bettegowda K Kenneth W. Kinzler (Department of Oncology, the Sidney Kimmel Comprehensive Cancer Center, Johns Hopkins University School of Medicine) N Nickolas Papadopoulos (Department of Oncology, the Sidney Kimmel Comprehensive Cancer Center, Johns Hopkins University School of Medicine) B Bert Vogelstein (Department of Oncology, the Sidney Kimmel Comprehensive Cancer Center, Johns Hopkins University School of Medicine) Y Yuxuan Wang

Abstract

We previously reported initial results from a clinical trial testing a strategy in which patients with muscle-invasive bladder cancer (MIBC) achieving a clinical complete response after cystoscopic resection of the bladder tumor plus systemic therapy could forgo removal of their entire bladder (cystectomy). While the results were highly promising, a subset of patients omitting initial cystectomy developed recurrence highlighting the need for biomarkers to refine selection of patients for this approach. We here report long-term follow-up of these patients and investigate whether tumor DNA in the plasma (ctDNA) or urine (utDNA) could inform prognosis and the need for cystectomy. Three-year bladder-intact survival among patients with a complete clinical response following four rounds of systemic therapy was 69%. Metastatic risk was significantly higher for patients with detectable versus undetectable ctDNA presystemic therapy (HR 4.68; 95% CI 1.10-43.35; log-rank P = 0.036). Only 4.5% (1 of 22) of patients with undetectable baseline ctDNA developed metastatic disease. Undetectable ctDNA before or after systemic therapy was associated with extremely low metastatic risk. Urine utDNA was more sensitive than plasma ctDNA at detecting residual disease within the bladder, and detectable urine utDNA in patients with a complete clinical response was associated with shorter bladder-intact survival (HR 6.47, 95% CI 1.34-31.31; log-rank P = 0.008). These findings establish the conceptual and experimental foundation for incorporating ctDNA and utDNA assays into the management of patients with MIBC, particularly with respect to the need for cystectomy.

Article Details

Volume / Issue Vol. 123, Issue 8
Published February 24, 2026
ISSN 0027-8424
Publisher National Academy of Sciences

Authors (28)

M

Matthew D. Galsky

Division of Hematology and Medical Oncology, Icahn School of Medicine at Mount Sinai

S

Sudeh Izadmehr

Division of Hematology and Medical Oncology, Icahn School of Medicine at Mount Sinai

M

Menggang Yu

Department of Biostatistics, University of Michigan

S

Samuel D. Curtis

Department of Pharmacology and Molecular Sciences, Johns Hopkins University School of Medicine

C

Christopher Douville

Department of Oncology, the Sidney Kimmel Comprehensive Cancer Center, Johns Hopkins University School of Medicine

M

Maria Popoli

Department of Oncology, the Sidney Kimmel Comprehensive Cancer Center, Johns Hopkins University School of Medicine

J

Janine Ptak

Department of Oncology, the Sidney Kimmel Comprehensive Cancer Center, Johns Hopkins University School of Medicine

L

Lisa Dobbyn

Department of Oncology, the Sidney Kimmel Comprehensive Cancer Center, Johns Hopkins University School of Medicine

N

Natalie Silliman

Department of Oncology, the Sidney Kimmel Comprehensive Cancer Center, Johns Hopkins University School of Medicine

K

Kevin G. Chan

Department of Urology, City of Hope Comprehensive Cancer Center

T

Tanya B. Dorff

Department of Medical Oncology and Therapeutics, City of Hope Comprehensive Cancer Center

J

Jeremy P. Cetnar

Division of Hematology and Medical Oncology, Oregon Health and Science University

B

Brock O’Neil

Department of Urology, University of Utah

A

Anishka D’Souza

Division of Hematology and Medical Oncology, Keck School of Medicine of University of Southern California, Norris Comprehensive Cancer Center

R

Ronac Mamtani

Division of Hematology and Medical Oncology, University of Pennsylvania Abramson Cancer Center

C

Christos E. Kyriakopoulos

Division of Hematology and Medical Oncology, University of Wisconsin Carbone Cancer Center

R

Rachel Brody

Tisch Cancer Institute, Icahn School of Medicine at Mount Sinai

E

Evita Sadimin

Department of Pathology, City of Hope Comprehensive Cancer Center

R

Reza Mehrazin

Department of Urology, Icahn School of Medicine at Mount Sinai, Tisch Cancer Institute

D

Diego Chowell

Tisch Cancer Institute, Icahn School of Medicine at Mount Sinai

J

John Sfakianos

Tisch Cancer Institute, Icahn School of Medicine at Mount Sinai

S

Siamak Daneshmand

Department of Urology, Keck School of Medicine of University of Southern California, Norris Comprehensive Cancer Center

S

Sumanta K. Pal

Department of Medical Oncology and Therapeutics, City of Hope Comprehensive Cancer Center

C

Chetan Bettegowda

K

Kenneth W. Kinzler

Department of Oncology, the Sidney Kimmel Comprehensive Cancer Center, Johns Hopkins University School of Medicine

N

Nickolas Papadopoulos

Department of Oncology, the Sidney Kimmel Comprehensive Cancer Center, Johns Hopkins University School of Medicine

B

Bert Vogelstein

Department of Oncology, the Sidney Kimmel Comprehensive Cancer Center, Johns Hopkins University School of Medicine

Y

Yuxuan Wang