What is the diagnosis and treatment of urothelial (transitional cell) carcinoma?

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Last updated: April 18, 2025View editorial policy

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From the Guidelines

Urothelial cancer treatment should be guided by the most recent and highest quality evidence, with a focus on minimizing morbidity, mortality, and improving quality of life, as outlined in the 2020 NCCN clinical practice guidelines 1.

Overview of Urothelial Cancer

Urothelial cancer is a type of cancer that begins in the urothelial cells lining the urinary tract, most commonly affecting the bladder. The standard approach to diagnosis involves pathological examination according to the World Health Organisation (WHO) classification from a biopsy obtained during transurethral resection of the bladder tumor (TURBT) 1.

Treatment Approaches

Treatment depends on the stage and grade of the cancer. For non-muscle invasive bladder cancer, the standard approach includes TURBT followed by intravesical therapy with BCG (Bacillus Calmette-Guérin) or chemotherapy agents like mitomycin C.

  • For high-risk non-muscle invasive disease, BCG is typically administered weekly for 6 weeks as induction, followed by maintenance therapy for 1-3 years.
  • For muscle-invasive disease, radical cystectomy (surgical removal of the bladder) with pelvic lymph node dissection is often recommended, frequently combined with neoadjuvant cisplatin-based chemotherapy regimens like gemcitabine plus cisplatin for 3-4 cycles before surgery 1.
  • For metastatic disease, first-line treatment typically involves platinum-based chemotherapy combinations, while immunotherapy with PD-1/PD-L1 inhibitors like pembrolizumab or atezolizumab is used for those who are cisplatin-ineligible or as second-line therapy 1.

Surveillance and Prevention

Urothelial cancer requires regular surveillance after treatment, including cystoscopy, urine cytology, and imaging studies, as recurrence rates are high. The disease is strongly associated with tobacco use and occupational exposure to certain chemicals, making smoking cessation an important part of management and prevention 1.

Recent Guidelines and Recommendations

The NCCN guidelines insights from 2016 and 2020 emphasize the importance of continued monitoring for recurrence and the development of more refined methods to determine prognosis and guide management based on molecular staging 1. These guidelines also highlight the evolving nature of treatment options for bladder cancer, including the emergence of checkpoint inhibitors as a new therapy for the treatment of persistent disease 1.

Key Considerations

  • Pathological diagnosis should be made according to the WHO classification from a biopsy obtained during TURBT 1.
  • Treatment should be based on the pathologic findings of the biopsy, with attention to histology, grade, and depth of invasion 1.
  • Regular surveillance is essential for detecting recurrences early, as most recurrences are non–muscle-invasive and can be treated endoscopically 1.

From the FDA Drug Label

Cisplatin Injection is indicated as a single agent for patients with transitional cell bladder cancer which is no longer amenable to local treatments, such as surgery and/or radiotherapy. The FDA drug label indicates that cisplatin is used to treat urothelial cancer, specifically transitional cell bladder cancer that is no longer treatable with local therapies such as surgery and/or radiotherapy 2.

  • Key points:
    • Indication: Cisplatin is indicated as a single agent for patients with transitional cell bladder cancer.
    • Treatment: For patients with urothelial cancer that is no longer amenable to local treatments.

From the Research

Urothelial Cancer Overview

  • Urothelial cancer, also known as urothelial carcinoma, is a type of cancer that affects the urinary system, particularly the bladder, ureters, and renal pelvis.
  • The standard treatment for non-muscle invasive bladder cancer (NMIBC) includes transurethral resection followed by intravesical chemotherapy and/or bacillus Calmette-Guerin (BCG) therapy 3.
  • However, BCG therapy is not effective in all patients, and alternative treatment options are being explored, including combination intravesical therapy, immunotherapy, gene therapy, targeted therapy, photodynamic therapy, and BCG variants 3, 4.

Risk Factors and Diagnosis

  • The most important risk factor for developing urothelial cancer is tobacco smoking, which accounts for approximately 50% of cases, followed by occupational exposure to aromatic amines and ionizing radiation 5.
  • Computed tomography (CT) urography is a useful diagnostic tool for evaluating the upper urinary tract for urothelial malignancies, while magnetic resonance imaging (MRI) is being investigated for its role in local and distant staging of urothelial carcinomas 6, 7.

Treatment Options

  • The current gold standard treatment for BCG-unresponsive patients is radical cystectomy, which is associated with considerable morbidity and mortality, particularly in older and frailer patients 3.
  • Alternative treatment options, such as bladder-sparing therapies, are being explored to provide acceptable oncological outcomes for these patients 3, 4.
  • Immune checkpoint inhibitors (ICIs) have shown promise in achieving long-term durable responses in other cancers, but their effectiveness in metastatic urothelial cancer is not as well-defined 5.

Professional Medical Disclaimer

This information is intended for healthcare professionals. Any medical decision-making should rely on clinical judgment and independently verified information. The content provided herein does not replace professional discretion and should be considered supplementary to established clinical guidelines. Healthcare providers should verify all information against primary literature and current practice standards before application in patient care. Dr.Oracle assumes no liability for clinical decisions based on this content.

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