Childhood MEN2 Syndrome – NCI

There are different types of treatment for children and adolescents with tumors related to MEN2 syndrome. You and your child’s cancer team will work together to decide treatment. Many factors will be considered, such as your child’s overall health and whether the cancer is newly diagnosed or has come back. 

Your child’s treatment plan will include information about the cancer, the goals of treatment, treatment options, and the possible side effects. It will be helpful to talk with your child’s cancer care team before treatment begins about what to expect. For help every step of the way, see our booklet, Children with Cancer: A Guide for Parents. 

Types of treatment your child might have include: 

Surgery

Surgery may be done to remove the thyroid gland when a child has medullary thyroid cancer caused by MEN2 syndromes. The thyroid may also be removed to prevent medullary thyroid cancer from developing.

Targeted therapy 

Targeted therapy uses drugs or other substances to block the action of specific enzymes, proteins, or other molecules involved in the growth of tumor cells. 

Selpercatinib and vandetanib are used to treat medullary thyroid cancer caused by MEN2 syndrome.

Learn more about Targeted Therapy to Treat Cancer.

Clinical trials

For some children, joining a clinical trial may be an option. There are different types of clinical trials for childhood cancer. For example, a treatment trial tests new treatments or new ways of using current treatments. Supportive care and palliative care trials look at ways to improve quality of life, especially for those who have side effects from cancer and its treatment.

You can use the clinical trial search to find NCI-supported cancer clinical trials accepting participants. The search allows you to filter trials based on the type of cancer, your child’s age, and where the trials are being done. Clinical trials supported by other organizations can be found on the ClinicalTrials.gov website.

Learn more about clinical trials, including how to find and join one, at Cancer Clinical Trial Information for Patients and Caregivers.

Treatment of tumors caused by childhood MEN2A syndrome

There is an increased risk of medullary thyroid cancer, an aggressive form of thyroid cancer, in people with MEN2A syndrome. Children with MEN2A syndrome and certain changes in the RET gene usually have surgery to remove the thyroid before age 5 years to lessen the chance cancer will form or spread. Treatment is also given for pheochromocytoma and hyperparathyroidism as needed. 

Treatment of medullary thyroid cancer in children who have MEN2A syndrome may include: 

Treatment of tumors caused by childhood MEN2B syndrome

There is an increased risk of medullary thyroid cancer, an aggressive form of thyroid cancer, in people with MEN2B syndrome. Infants with MEN2B syndrome and certain changes in the RET gene usually have surgery to remove the thyroid to lessen the chance cancer will form. Treatment is also given for pheochromocytoma and other conditions as needed. 

Children with MEN2B syndrome who have medullary thyroid cancer may be treated with: 

Treatment of recurrent tumors caused by childhood MEN2 syndrome

If the cancer linked to MEN2 syndrome comes back after treatment, your child’s doctor will talk with you about what to expect and possible next steps. There might be treatment options that may shrink the cancer or control its growth. If there are no treatments, your child can receive care to control symptoms from cancer so they can be as comfortable as possible.