The outcome of patients with the MEN 1 syndrome is generally good provided adequate treatment can be obtained for parathyroid, pancreatic, and pituitary tumors. The outcome for patients with the MEN 2A syndrome is also generally good, yet the possibility exists for recurrence of medullary thyroid carcinoma and pheochromocytoma.[31,32,33] Patients who have the MEN 2B syndrome have a worse outcome primarily due to more aggressive medullary thyroid carcinoma. Prophylactic thyroidectomy has the potential to improve the outcome in MEN 2B, but there are no long-term outcome reports published to date. For patients with the Carney complex, prognosis depends on the frequency of recurrences of cardiac and skin myxomas and other tumors.
Refer to the PDQ summary on Genetics of Medullary Thyroid Cancer for more information about MEN 2A and MEN 2B.
Treatment options under clinical evaluation
- NCI-07-C-0189: This phase I/II NCI trial is investigating vandetanib, an orally available tyrosine kinase receptor inhibitor, for patients aged 5 years to 18 years, with hereditary thyroid medullary carcinoma.[34,35]
Skin Cancer (Melanoma, Basal Cell Carcinoma, and Squamous Cell Carcinoma)
Melanoma is the most common skin cancer in children, followed by basal cell and squamous cell carcinomas (SCCs).[36,37,38,39,40,41,42,43,44] There are approximately 425 cases of melanoma diagnosed each year in the United States in patients younger than 20 years, representing about 1% of all new cases of melanoma that are diagnosed annually in this country. Melanoma annual incidence in the United States (2002-2006) increased with age, from 1 to 2 per million in children younger than 10 years to 4.1 per million in children aged 10 to 14 years and 16.9 per million in children aged 15 to 19 years. Melanoma accounts for about 8% of all cancers seen in children aged 15 to 19 years. The incidence of pediatric melanoma (in children younger than 20 years) increased by 1.7% per year between 1975 and 2006; increased ambient ultraviolet radiation increases the risk of the disease.
Conditions associated with an increased risk of melanoma in children and adolescents include giant melanocytic nevi, xeroderma pigmentosum (a rare recessive disorder characterized by extreme sensitivity to sunlight, keratosis, and various neurologic manifestations), immunodeficiency, immunosuppression, and Werner syndrome. Other phenotypic traits that are associated with an increased risk of melanoma in adults have been documented in children and adolescents with melanoma and include exposure to ultraviolet sunlight, red hair, blue eyes,[49,50,51,52] poor tanning ability, freckling, dysplastic nevi, increased number of melanocytic nevi, and family history of melanoma.[53,54,55] Neurocutaneous melanosis is an unusual condition associated with large or multiple congenital nevi of the skin in association with meningeal melanosis or melanoma; approximately 2.5% of patients with large congenital nevi develop this condition, and those with increased numbers of satellite nevi are at greatest risk.[56,57]