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Home Knowledge Center Wellness Library Childhood Bladder Cancer Treatment (PDQ®): Treatment - Health Professional Information [NCI]

Childhood Bladder Cancer Treatment (PDQ®): Treatment - Health Professional Information [NCI]

This information is produced and provided by the National Cancer Institute (NCI). The information in this topic may have changed since it was written. For the most current information, contact the National Cancer Institute via the Internet web site at http://cancer.gov or call 1-800-4-CANCER.

Clinical Presentation

Clinical Presentation

Urothelial bladder neoplasms are extremely rare in children. The most common presenting symptom of bladder cancer in children is hematuria.[1,2]

References:

  1. Saltsman JA, Malek MM, Reuter VE, et al.: Urothelial neoplasms in pediatric and young adult patients: A large single-center series. J Pediatr Surg 53 (2): 306-309, 2018.
  2. Rezaee ME, Dunaway CM, Baker ML, et al.: Urothelial cell carcinoma of the bladder in pediatric patients: a systematic review and data analysis of the world literature. J Pediatr Urol 15 (4): 309-314, 2019.
Risk Factors

Risk Factors

Bladder cancer in adolescents may develop as a result of exposure to alkylating-agent chemotherapy that was given to treat other childhood tumors or leukemia.[1,2,3,4] The association between cyclophosphamide exposure and bladder cancer is the only established relationship between a specific anticancer drug and a solid tumor.[1]

References:

  1. Johansson SL, Cohen SM: Epidemiology and etiology of bladder cancer. Semin Surg Oncol 13 (5): 291-8, 1997 Sep-Oct.
  2. IARC Working Group on the Evaluation of Carcinogenic Risks to Humans. International Agency for Research on Cancer: Overall evaluations of carcinogenicity: an updating of IARC monographs, volumes 1 to 42. IARC Monographs on the Evaluation of Carcinogenic Risks to Humans, Supplement 7. International Agency for Research on Cancer, 1987.
  3. Di Carlo D, Ferrari A, Perruccio K, et al.: Management and follow-up of urothelial neoplasms of the bladder in children: a report from the TREP project. Pediatr Blood Cancer 62 (6): 1000-3, 2015.
  4. Ritchey M, Ferrer F, Shearer P, et al.: Late effects on the urinary bladder in patients treated for cancer in childhood: a report from the Children's Oncology Group. Pediatr Blood Cancer 52 (4): 439-46, 2009.
Histology

Histology

Histological classification of these neoplasms includes the following:

  • Urothelial papillomas.
  • Papillary neoplasms of low malignant potential.
  • Low-grade urothelial carcinoma.
  • High-grade urothelial carcinoma.

An alternative designation is transitional cell carcinoma of the bladder. The most common histology is papillary urothelial neoplasm of low malignant potential, while high-grade, invasive urothelial carcinomas are extremely rare in young patients.[1,2,3,4,5]

References:

  1. Alanee S, Shukla AR: Bladder malignancies in children aged <18 years: results from the Surveillance, Epidemiology and End Results database. BJU Int 106 (4): 557-60, 2010.
  2. Paner GP, Zehnder P, Amin AM, et al.: Urothelial neoplasms of the urinary bladder occurring in young adult and pediatric patients: a comprehensive review of literature with implications for patient management. Adv Anat Pathol 18 (1): 79-89, 2011.
  3. Stanton ML, Xiao L, Czerniak BA, et al.: Urothelial tumors of the urinary bladder in young patients: a clinicopathologic study of 59 cases. Arch Pathol Lab Med 137 (10): 1337-41, 2013.
  4. Di Carlo D, Ferrari A, Perruccio K, et al.: Management and follow-up of urothelial neoplasms of the bladder in children: a report from the TREP project. Pediatr Blood Cancer 62 (6): 1000-3, 2015.
  5. Berrettini A, Castagnetti M, Salerno A, et al.: Bladder urothelial neoplasms in pediatric age: experience at three tertiary centers. J Pediatr Urol 11 (1): 26.e1-5, 2015.
Special Considerations for the Treatment of Children With Cancer

Special Considerations for the Treatment of Children With Cancer

Cancer in children and adolescents is rare, although the overall incidence has been slowly increasing since 1975.[1] Referral to medical centers with multidisciplinary teams of cancer specialists experienced in treating cancers that occur in childhood and adolescence should be considered. This multidisciplinary team approach incorporates the skills of the following health care professionals and others to ensure that children receive treatment, supportive care, and rehabilitation that will achieve optimal survival and quality of life:

  • Primary care physicians.
  • Pediatric surgeons.
  • Radiation oncologists.
  • Pediatric medical oncologists/hematologists.
  • Rehabilitation specialists.
  • Pediatric nurse specialists.
  • Social workers.
  • Child-life professionals.
  • Psychologists.

(Refer to the PDQ Supportive and Palliative Care summaries for specific information about supportive care for children and adolescents with cancer.)

The American Academy of Pediatrics has outlined guidelines for pediatric cancer centers and their role in the treatment of pediatric patients with cancer.[2] At these pediatric cancer centers, clinical trials are available for most types of cancer that occur in children and adolescents, and the opportunity to participate is offered to most patients and their families. Clinical trials for children and adolescents diagnosed with cancer are generally designed to compare potentially better therapy with current standard therapy. Most of the progress made in identifying curative therapy for childhood cancers has been achieved through clinical trials. Information about ongoing clinical trials is available from the NCI website.

Dramatic improvements in survival have been achieved for children and adolescents with cancer. Between 1975 and 2010, childhood cancer mortality decreased by more than 50%.[3] Childhood and adolescent cancer survivors require close monitoring because side effects of cancer therapy may persist or develop months or years after treatment. (Refer to the PDQ summary on Late Effects of Treatment for Childhood Cancer for specific information about the incidence, type, and monitoring of late effects in childhood and adolescent cancer survivors.)

Childhood cancer is a rare disease, with about 15,000 cases diagnosed annually in the United States in individuals younger than 20 years.[4] The U.S. Rare Diseases Act of 2002 defines a rare disease as one that affects populations smaller than 200,000 people. Therefore, all pediatric cancers are considered rare.

The designation of a rare tumor is not uniform among pediatric and adult groups. In adults, rare cancers are defined as those with an annual incidence of fewer than six cases per 100,000 people. They account for up to 24% of all cancers diagnosed in the European Union and about 20% of all cancers diagnosed in the United States.[5,6] Also, the designation of a pediatric rare tumor is not uniform among international groups, as follows:

  • A consensus effort between the European Union Joint Action on Rare Cancers and the European Cooperative Study Group for Rare Pediatric Cancers estimated that 11% of all cancers in patients younger than 20 years could be categorized as very rare. This consensus group defined very rare cancers as those with annual incidences of fewer than 2 cases per 1 million people. However, three additional histologies (thyroid carcinoma, melanoma, and testicular cancer) with incidences of more than 2 cases per 1 million people were also included in the very rare group because there is a lack of knowledge and expertise in the management of these tumors.[7]
  • The Children's Oncology Group defines rare pediatric cancers as those listed in the International Classification of Childhood Cancer subgroup XI, which includes thyroid cancer, melanoma and nonmelanoma skin cancers, and multiple types of carcinomas (e.g., adrenocortical carcinoma, nasopharyngeal carcinoma, and most adult-type carcinomas such as breast cancer, colorectal cancer, etc.).[8] These diagnoses account for about 4% of cancers diagnosed in children aged 0 to 14 years, compared with about 20% of cancers diagnosed in adolescents aged 15 to 19 years.[9]

    Most cancers in subgroup XI are either melanomas or thyroid cancer, with other types accounting for only 1.3% of cancers in children aged 0 to 14 years and 5.3% of cancers in adolescents aged 15 to 19 years.

These rare cancers are extremely challenging to study because of the low number of patients with any individual diagnosis, the predominance of rare cancers in the adolescent population, and the lack of clinical trials for adolescents with rare cancers.

References:

  1. Smith MA, Seibel NL, Altekruse SF, et al.: Outcomes for children and adolescents with cancer: challenges for the twenty-first century. J Clin Oncol 28 (15): 2625-34, 2010.
  2. American Academy of Pediatrics: Standards for pediatric cancer centers. Pediatrics 134 (2): 410-4, 2014. Also available online. Last accessed February 1, 2022.
  3. Smith MA, Altekruse SF, Adamson PC, et al.: Declining childhood and adolescent cancer mortality. Cancer 120 (16): 2497-506, 2014.
  4. Ward E, DeSantis C, Robbins A, et al.: Childhood and adolescent cancer statistics, 2014. CA Cancer J Clin 64 (2): 83-103, 2014 Mar-Apr.
  5. Gatta G, Capocaccia R, Botta L, et al.: Burden and centralised treatment in Europe of rare tumours: results of RARECAREnet-a population-based study. Lancet Oncol 18 (8): 1022-1039, 2017.
  6. DeSantis CE, Kramer JL, Jemal A: The burden of rare cancers in the United States. CA Cancer J Clin 67 (4): 261-272, 2017.
  7. Ferrari A, Brecht IB, Gatta G, et al.: Defining and listing very rare cancers of paediatric age: consensus of the Joint Action on Rare Cancers in cooperation with the European Cooperative Study Group for Pediatric Rare Tumors. Eur J Cancer 110: 120-126, 2019.
  8. Pappo AS, Krailo M, Chen Z, et al.: Infrequent tumor initiative of the Children's Oncology Group: initial lessons learned and their impact on future plans. J Clin Oncol 28 (33): 5011-6, 2010.
  9. Howlader N, Noone AM, Krapcho M, et al., eds.: SEER Cancer Statistics Review, 1975-2012. National Cancer Institute, 2015. Also available online. Last accessed June 22, 2021.
Treatment and Outcome of Childhood Bladder Cancer

Treatment and Outcome of Childhood Bladder Cancer

Treatment options for childhood bladder cancer include the following:

  1. Surgery.

In contrast to adults, most pediatric bladder carcinomas are low grade and superficial, and patients have an excellent prognosis after transurethral resection.[1,2,3,4] In one review of the literature, recurrence risk and mortality rates were low in pediatric patients (8.6% and 3.7%, respectively).[5] Most recurrences were observed within 9 months, and all recurrences occurred within 32 months. This finding suggests that patients should be monitored for at least 3 years.

Squamous cell carcinomas and more aggressive carcinomas have been reported and may require a more aggressive surgical approach.[3,6,7,8]

References:

  1. Fine SW, Humphrey PA, Dehner LP, et al.: Urothelial neoplasms in patients 20 years or younger: a clinicopathological analysis using the world health organization 2004 bladder consensus classification. J Urol 174 (5): 1976-80, 2005.
  2. Paner GP, Zehnder P, Amin AM, et al.: Urothelial neoplasms of the urinary bladder occurring in young adult and pediatric patients: a comprehensive review of literature with implications for patient management. Adv Anat Pathol 18 (1): 79-89, 2011.
  3. Stanton ML, Xiao L, Czerniak BA, et al.: Urothelial tumors of the urinary bladder in young patients: a clinicopathologic study of 59 cases. Arch Pathol Lab Med 137 (10): 1337-41, 2013.
  4. Berrettini A, Castagnetti M, Salerno A, et al.: Bladder urothelial neoplasms in pediatric age: experience at three tertiary centers. J Pediatr Urol 11 (1): 26.e1-5, 2015.
  5. Rezaee ME, Dunaway CM, Baker ML, et al.: Urothelial cell carcinoma of the bladder in pediatric patients: a systematic review and data analysis of the world literature. J Pediatr Urol 15 (4): 309-314, 2019.
  6. Sung JD, Koyle MA: Squamous cell carcinoma of the bladder in a pediatric patient. J Pediatr Surg 35 (12): 1838-9, 2000.
  7. Lezama-del Valle P, Jerkins GR, Rao BN, et al.: Aggressive bladder carcinoma in a child. Pediatr Blood Cancer 43 (3): 285-8, 2004.
  8. Korrect GS, Minevich EA, Sivan B: High-grade transitional cell carcinoma of the pediatric bladder. J Pediatr Urol 8 (3): e36-8, 2012.
Treatment Options Under Clinical Evaluation for Childhood Bladder Cancer

Treatment Options Under Clinical Evaluation for Childhood Bladder Cancer

Information about National Cancer Institute (NCI)–supported clinical trials can be found on the NCI website. For information about clinical trials sponsored by other organizations, refer to the ClinicalTrials.gov website.

The following is an example of a national and/or institutional clinical trial that is currently being conducted:

  • APEC1621 (NCT03155620) (Pediatric MATCH: Targeted Therapy Directed by Genetic Testing in Treating Pediatric Patients with Relapsed or Refractory Advanced Solid Tumors, Non-Hodgkin Lymphomas, or Histiocytic Disorders): NCI-COG Pediatric Molecular Analysis for Therapeutic Choice (MATCH), referred to as Pediatric MATCH, will match targeted agents with specific molecular changes identified in a patient's tumor (refractory or recurrent). Children and adolescents aged 1 to 21 years are eligible for the trial.

    Patients with tumors that have molecular variants addressed by treatment arms included in the trial will be offered treatment on Pediatric MATCH. Additional information can be obtained on the NCI website and ClinicalTrials.gov website.

Changes to This Summary (02 / 23 / 2022)

Changes to This Summary (02 / 23 / 2022)

The PDQ cancer information summaries are reviewed regularly and updated as new information becomes available. This section describes the latest changes made to this summary as of the date above.

Editorial changes were made to this summary.

This summary is written and maintained by the PDQ Pediatric Treatment Editorial Board, which is editorially independent of NCI. The summary reflects an independent review of the literature and does not represent a policy statement of NCI or NIH. More information about summary policies and the role of the PDQ Editorial Boards in maintaining the PDQ summaries can be found on the About This PDQ Summary and PDQ® - NCI's Comprehensive Cancer Database pages.

About This PDQ Summary

About This PDQ Summary

Purpose of This Summary

This PDQ cancer information summary for health professionals provides comprehensive, peer-reviewed, evidence-based information about the treatment of pediatric bladder cancer. It is intended as a resource to inform and assist clinicians in the care of their patients. It does not provide formal guidelines or recommendations for making health care decisions.

Reviewers and Updates

This summary is reviewed regularly and updated as necessary by the PDQ Pediatric Treatment Editorial Board, which is editorially independent of the National Cancer Institute (NCI). The summary reflects an independent review of the literature and does not represent a policy statement of NCI or the National Institutes of Health (NIH).

Board members review recently published articles each month to determine whether an article should:

  • be discussed at a meeting,
  • be cited with text, or
  • replace or update an existing article that is already cited.

Changes to the summaries are made through a consensus process in which Board members evaluate the strength of the evidence in the published articles and determine how the article should be included in the summary.

The lead reviewers for Childhood Bladder Cancer Treatment are:

  • Denise Adams, MD (Children's Hospital Boston)
  • Karen J. Marcus, MD, FACR (Dana-Farber Cancer Institute/Boston Children's Hospital)
  • Paul A. Meyers, MD (Memorial Sloan-Kettering Cancer Center)
  • Thomas A. Olson, MD (Aflac Cancer and Blood Disorders Center of Children's Healthcare of Atlanta - Egleston Campus)
  • Alberto S. Pappo, MD (St. Jude Children's Research Hospital)
  • Arthur Kim Ritchey, MD (Children's Hospital of Pittsburgh of UPMC)
  • Carlos Rodriguez-Galindo, MD (St. Jude Children's Research Hospital)
  • Stephen J. Shochat, MD (St. Jude Children's Research Hospital)

Any comments or questions about the summary content should be submitted to Cancer.gov through the NCI website's Email Us. Do not contact the individual Board Members with questions or comments about the summaries. Board members will not respond to individual inquiries.

Levels of Evidence

Some of the reference citations in this summary are accompanied by a level-of-evidence designation. These designations are intended to help readers assess the strength of the evidence supporting the use of specific interventions or approaches. The PDQ Pediatric Treatment Editorial Board uses a formal evidence ranking system in developing its level-of-evidence designations.

Permission to Use This Summary

PDQ is a registered trademark. Although the content of PDQ documents can be used freely as text, it cannot be identified as an NCI PDQ cancer information summary unless it is presented in its entirety and is regularly updated. However, an author would be permitted to write a sentence such as "NCI's PDQ cancer information summary about breast cancer prevention states the risks succinctly: [include excerpt from the summary]."

The preferred citation for this PDQ summary is:

PDQ® Pediatric Treatment Editorial Board. PDQ Childhood Bladder Cancer Treatment. Bethesda, MD: National Cancer Institute. Updated <MM/DD/YYYY>. Available at: https://www.cancer.gov/types/bladder/hp/child-bladder-treatment-pdq. Accessed <MM/DD/YYYY>.

Images in this summary are used with permission of the author(s), artist, and/or publisher for use within the PDQ summaries only. Permission to use images outside the context of PDQ information must be obtained from the owner(s) and cannot be granted by the National Cancer Institute. Information about using the illustrations in this summary, along with many other cancer-related images, is available in Visuals Online, a collection of over 2,000 scientific images.

Disclaimer

Based on the strength of the available evidence, treatment options may be described as either "standard" or "under clinical evaluation." These classifications should not be used as a basis for insurance reimbursement determinations. More information on insurance coverage is available on Cancer.gov on the Managing Cancer Care page.

Contact Us

More information about contacting us or receiving help with the Cancer.gov website can be found on our Contact Us for Help page. Questions can also be submitted to Cancer.gov through the website's Email Us.

Last Revised: 2022-02-23

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