Our Health Library information does not replace the advice of a doctor. Please be advised that this information is made available to assist our patients to learn more about their health. Our providers may not see and/or treat all topics found herein. 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. Uveal melanoma (iris, ciliary body, choroid) is the most common primary intraocular malignancy. About 2,000 cases of uveal melanoma are diagnosed each year in the United States. It accounts for 5% of all cases of melanoma.[1] This tumor is most commonly diagnosed in older patients, and the incidence peaks at age 70 years.[2] Pediatric uveal melanoma is extremely rare and accounts for 0.8% to 1.1% of all cases of uveal melanoma.[3] A retrospective, 24-center, observational study conducted by the European Ophthalmic Oncology Group from 1968 to 2014 identified 114 children (aged 1–17 years) and 185 young adults (aged 18–25 years) with ocular melanoma.[3] The median age at the time of diagnosis for children was 15.1 years. The incidence of disease increased by 0.8% per year between the ages of 5 and 10 years and 8.8% per year between the ages of 17 and 24 years. Other series have also documented the higher incidence of the disease in adolescents.[4,5] References: Risk factors for developing uveal melanoma include the following:[1,2,3] In a European Oncology Group study, 57% of the pediatric patients were female. Four patients had a preexisting condition that included oculodermal melanocytosis (n = 2) and neurofibromatosis (n = 2).[4] In a review of 13 cases of uveal melanoma in the first 2 years of life, four patients had familial atypical melanoma mole syndrome, one patient had dysplastic nevus syndrome, and one patient had café au lait spots.[5] References: Uveal melanoma is characterized by activating variants of GNAQ and GNA11, which lead to activation of the mitogen-activated protein kinases (MAPK) pathway. In addition, variants in BAP1 are seen in 84% of metastasizing tumors. Variants in SF3B1 and EIF1AX are associated with a good prognosis.[1,2,3,4,5,6] References: Prognostic factors for uveal melanoma include the following:[1,2] The survival of children appears to be more favorable than that of young adults and adults, suggesting that the biology of ocular melanoma might be different in children.[1,2] A retrospective, multicenter, cohort study identified 133 young children aged 1 to 12 years with choroidal or ciliary body (n = 66; 50%), iris (n = 33; 25%), conjunctival (n = 26; 19%), and eyelid (n = 8; 6%) melanomas.[3] References: 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: For information about supportive care for children and adolescents with cancer, see the summaries on Supportive and Palliative Care. 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 2020, childhood cancer mortality decreased by more than 50%.[3,4,5] Childhood and adolescent cancer survivors require close monitoring because side effects of cancer therapy may persist or develop months or years after treatment. For information about the incidence, type, and monitoring of late effects in childhood and adolescent cancer survivors, see Late Effects of Treatment for Childhood Cancer. Childhood cancer is a rare disease, with about 15,000 cases diagnosed annually in the United States in individuals younger than 20 years.[6] 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.[7,8] Also, the designation of a pediatric rare tumor is not uniform among international groups, as follows: Most cancers in subgroup XI are either melanomas or thyroid cancers, with other cancer types accounting for only 2% of the cancers in children aged 0 to 14 years and 9.3% of the 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. Information about these tumors may also be found in sources relevant to adults with cancer, such as Intraocular (Uveal) Melanoma Treatment. References: Treatment options for childhood intraocular (uveal) melanoma include the following: A retrospective single-institution review identified 18 patients younger than 21 years with uveal melanoma.[3][Level of evidence C1] Patients were treated with enucleation (n = 9), transscleral en bloc resection (n = 3), plaque radiation therapy (n = 5), or proton-beam radiation therapy (n = 1). Laser surgery has been used to treat childhood intraocular melanoma.[1,2] An open-label, randomized, phase III trial of adult patients with previously untreated HLA-A*02:01–positive metastatic uveal melanoma investigated treatment with tebentafusp. Tebentafusp is a bispecific restricted T-cell receptor for the glycoprotein 100 peptide; it is fused to a CD3 single-chain variable fragment. Patients were randomly assigned to receive either tebentafusp or treatment according to the investigator's choice.[4] For information about the treatment of uveal melanoma in adults, see Intraocular (Uveal) Melanoma Treatment. References: 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, see the ClinicalTrials.gov website. 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. Prognostic Factors Added text about the results of a retrospective, multicenter, cohort study that identified 133 young children aged 1 to 12 years with choroidal or ciliary body, iris, conjunctival, and eyelid melanomas (cited Masoomian et al. as reference 3). 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® Cancer Information for Health Professionals pages. Purpose of This Summary This PDQ cancer information summary for health professionals provides comprehensive, peer-reviewed, evidence-based information about the treatment of pediatric intraocular (uveal) melanoma. 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: 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 Intraocular (Uveal) Melanoma Treatment are: 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 Intraocular (Uveal) Melanoma Treatment. Bethesda, MD: National Cancer Institute. Updated <MM/DD/YYYY>. Available at: https://www.cancer.gov/types/eye/hp/child-intraocular-melanoma-treatment-pdq. Accessed <MM/DD/YYYY>. [PMID: 31909938] 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: 2024-04-11 This information does not replace the advice of a doctor. Ignite Healthwise, LLC disclaims any warranty or liability for your use of this information. Your use of this information means that you agree to the Terms of Use and Privacy Policy. Learn how we develop our content. Healthwise, Healthwise for every health decision, and the Healthwise logo are trademarks of Ignite Healthwise, LLC.Topic Contents
Childhood Intraocular (Uveal) Melanoma Treatment (PDQ®): Treatment - Health Professional Information [NCI]
Incidence
Risk Factors
Molecular Features
Prognostic Factors
Special Considerations for the Treatment of Children With Cancer
Treatment and Outcome of Childhood Intraocular (Uveal) Melanoma
Treatment Options Under Clinical Evaluation for Childhood Intraocular (Uveal) Melanoma
Latest Updates to This Summary (04 / 11 / 2024)
About This PDQ Summary
Our Health Library information does not replace the advice of a doctor. Please be advised that this information is made available to assist our patients to learn more about their health. Our providers may not see and/or treat all topics found herein.Childhood Intraocular (Uveal) Melanoma Treatment (PDQ®): Treatment - Health Professional Information [NCI]