Safety, Tolerability, and Preliminary Efficacy of TAR-200 in Patients with Muscle-invasive Bladder Cancer Who Refused or Were Unfit for Curative-intent Therapy: A Phase 1 Study


Por: Tyson M.D., Morris D., Palou J., Rodriguez O., Mir M.C., Dickstein R.J., Guerrero-Ramos F., Scarpato K.R., Hafron J.M., Messing E.M., Cutie C.J., Maffeo J.C., Raybold B., Chau A., Stromberg K.A., Keegan K.A.

Publicada: 1 ene 2023 Ahead of Print: 7 abr 2023
Resumen:
PURPOSE: Half of patients with muscle-invasive bladder cancer worldwide may not receive curative-intent therapy. Elderly or frail patients are most affected by this unmet need. TAR-200 is a novel, intravesical drug delivery system that provides sustained, local release of gemcitabine into the bladder over a 21-day dosing cycle. The phase 1 TAR-200-103 study evaluated the safety, tolerability, and preliminary efficacy of TAR-200 in patients with muscle-invasive bladder cancer who either refused or were unfit for curative-intent therapy. MATERIALS AND METHODS: Eligible patients had cT2-cT3bN0M0 urothelial carcinoma of the bladder. TAR-200 was inserted for 4 consecutive 21-day cycles over 84 days. The primary end points were safety and tolerability at 84 days. Secondary end points included rates of clinical complete response and partial response as determined by cystoscopy, biopsy, and imaging; duration of response; and overall survival. RESULTS: Median age of the 35 enrolled patients was 84 years, and most were male (24/35, 68.6%). Treatment-emergent adverse events related to TAR-200 occurred in 15 patients. Two patients experienced treatment-emergent adverse events leading to removal of TAR-200. At 3 months, complete response and partial response rates were 31.4% (11/35) and 8.6% (3/35), respectively, yielding an overall response rate of 40.0% (14/35; 95% CI 23.9-57.9). Median overall survival and duration of response were 27.3 months (95% CI 10.1-not estimable) and 14 months (95% CI 10.6-22.7), respectively. Progression-free rate at 12 months was 70.5%. CONCLUSIONS: TAR-200 was generally safe, well tolerated, and had beneficial preliminary efficacy in this elderly and frail cohort with limited treatment options.

Filiaciones:
Tyson M.D.:
 Mayo Clinic Arizona, Phoenix, AZ, United States

Morris D.:
 Urology Associates, Nashville, TN, United States

Palou J.:
 Fundació Puigvert, Universitat Autónoma de Barcelona, Barcelona, Spain

Rodriguez O.:
 Fundació Puigvert, Universitat Autónoma de Barcelona, Barcelona, Spain

Mir M.C.:
 Fundacion Instituto Valenciano de Oncologia, Valencia, Spain

Dickstein R.J.:
 Chesapeake Urology, Baltimore, MD, United States

Guerrero-Ramos F.:
 University Hospital 12 de Octubre, Madrid, Spain

Scarpato K.R.:
 Vanderbilt University Medical Center, Nashville, TN, United States

Hafron J.M.:
 Michigan Institute of Urology, Troy, MI, United States

Messing E.M.:
 University of Rochester, Rochester, NY, United States

Cutie C.J.:
 Janssen Research and Development, Lexington, MA, United States

Maffeo J.C.:
 Janssen Research and Development, Lexington, MA, United States

Raybold B.:
 Janssen Research and Development, Spring House, PA, United States

Chau A.:
 Datacision Limited, London, United Kingdom

Stromberg K.A.:
 Janssen Research and Development, Raritan, NJ, United States

Keegan K.A.:
 Vanderbilt University Medical Center, Nashville, TN, United States

 Janssen Research and Development, Lexington, MA, United States
ISSN: 00225347
Editorial
LIPPINCOTT WILLIAMS & WILKINS, TWO COMMERCE SQ, 2001 MARKET ST, PHILADELPHIA, PA 19103 USA, Estados Unidos America
Tipo de documento: Article
Volumen: 209 Número: 5
Páginas: 890-900
WOS Id: 001144475200027
ID de PubMed: 37026631
imagen hybrid, All Open Access; Hybrid Gold

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