Wilms Tumor and Other Childhood Kidney Tumors Treatment Option Overview

There are different types of treatment for patients with Wilms tumor and other childhood kidney tumors.

Different types of treatment are available for children with Wilms and other childhood kidney tumors. Some treatments are standard (the currently used treatment), and some are being tested in clinical trials. A treatment clinical trial is a research study meant to help improve current treatments or obtain information on new treatments for patients with cancer. When clinical trials show that a new treatment is better than the standard treatment, the new treatment may become the standard treatment.

Because cancer in children is rare, taking part in a clinical trial should be considered. Some clinical trials are open only to patients who have not started treatment.

Children with Wilms tumor or other childhood kidney tumors should have their treatment planned by a team of health care providers who are experts in treating cancer in children.

Your child's treatment will be overseen by a pediatric oncologist, a doctor who specializes in treating children with cancer. The pediatric oncologist works with other pediatric health care providers who are experts in treating children with Wilms tumor or other childhood kidney tumors and who specialize in certain areas of medicine. These may include the following specialists:

• Pediatrician.
• Pediatric surgeon or urologist.
• Radiation oncologist.
• Rehabilitation specialist.
• Pediatric nurse specialist.
• Social worker.

Six types of treatment are used:

Surgery

Two types of surgery are used to treat kidney tumors:

• Nephrectomy: Wilms tumor and other childhood kidney tumors are usually treated with nephrectomy (surgery to remove the whole kidney). Nearby lymph nodes may also be removed and checked for signs of cancer. Sometimes a kidney transplant (surgery to remove the kidney and replace it with a kidney from a donor) is done when the cancer is in both kidneys and the kidneys are not working well.
• Partial nephrectomy: If cancer is found in both kidneys or is likely to spread to both kidneys, surgery may include a partial nephrectomy (removal of the cancer in the kidney and a small amount of normal tissue around it). Partial nephrectomy is done to keep as much of the kidney working as possible. A partial nephrectomy is also called renal-sparing surgery.

After the doctor removes all the cancer that can be seen at the time of the surgery, some patients may be given chemotherapy or radiation therapy after surgery to kill any cancer cells that are left. Treatment given after the surgery, to lower the risk that the cancer will come back, is called adjuvant therapy. Sometimes, a second-look surgery is done to see if cancer remains after chemotherapy or radiation therapy.

Sometimes the tumor cannot be removed by surgery for one of the following reasons:

• The tumor is too close to important organs or blood vessels.
• The tumor is too large to remove.
• The cancer is in both kidneys, unless the tumors are very small.
• There is a blood clot in the vessels near the liver.
• The patient has trouble breathing because cancer has spread to the lungs.

In this case, a biopsy is done first. Then chemotherapy is given to reduce the size of the tumor before surgery, in order to save as much healthy tissue as possible and lessen problems after surgery. This is called neoadjuvant chemotherapy. Radiation therapy is given after surgery.

Radiation therapy

Radiation therapy is a cancer treatment that uses high-energy x-rays or other types of radiation to kill cancer cells or keep them from growing. External radiation therapy uses a machine outside the body to send radiation toward the area of the body with cancer.

External radiation therapy is used to treat Wilms tumor and other childhood kidney tumors.

Chemotherapy

Chemotherapy is a cancer treatment that uses drugs to stop the growth of cancer cells, either by killing the cells or by stopping them from dividing. When chemotherapy is taken by mouth or injected into a vein or muscle, the drugs enter the bloodstream and can reach cancer cells throughout the body (systemic chemotherapy). Combination chemotherapy is treatment using two or more anticancer drugs.

Systemic chemotherapy is used to treat Wilms tumor and other childhood kidney tumors.

Sometimes chemotherapy is given to reduce the size of the tumor before surgery, in order to save as much healthy tissue as possible and lessen problems after surgery. This is called neoadjuvant chemotherapy.

See Drugs Approved for Wilms Tumor and Other Childhood Kidney Cancers for more information.

Immunotherapy

Immunotherapy is a treatment that uses the patient's immune system to fight cancer. Substances made by the body or made in a laboratory are used to boost, direct, or restore the body's natural defenses against cancer. This cancer treatment is a type of biologic therapy.

Interferon and interleukin-2 (IL-2) are types of immunotherapy used to treat childhood renal cell cancer. Interferon may slow tumor growth and may help kill the cancer cells. IL-2 boosts the growth and activity of many immune cells, especially lymphocytes (a type of white blood cell). Lymphocytes can attack and kill cancer cells.

High-dose chemotherapy with stem cell rescue

High doses of chemotherapy are given to kill cancer cells. Healthy cells, including blood-forming cells, are also destroyed by the cancer treatment. Stem cell rescue is a treatment to replace the blood-forming cells. Stem cells (immature blood cells) are removed from the blood or bone marrow of the patient and are frozen and stored. After the patient completes chemotherapy, the stored stem cells are thawed and given back to the patient through an infusion. These stem cells grow into (and restore) the body's blood cells.

High-dose chemotherapy with stem cell rescue may be used to treat rhabdoid tumor of the kidney or recurrent Wilms tumor.

Targeted therapy

Targeted therapy is a type of treatment that uses drugs or other substances to identify and attack specific cancer cells. Targeted therapies usually cause less harm to normal cells than chemotherapy or radiation therapy do. Targeted therapy used to treat childhood kidney tumors may include the following:

• Tyrosine kinase inhibitors: This targeted therapy blocks signals that cancer cells need to grow and divide.
  • Larotrectinib and entrectinib may be used to treat congenital mesoblastic nephroma with a certain gene change that cannot be removed by surgery, has spread to other parts of the body, or has continued to grow during treatment. This combination is also being studied to treat congenital mesoblastic nephroma that has come back after treatment.
  • Sunitinib or cabozantinib may be used to treat renal cell carcinoma.
  • Axitinib is being studied in combination with a monoclonal antibody (nivolumab) to treat renal cell carcinoma that cannot be removed by surgery or has spread to other parts of the body.
• Histone methyltransferase inhibitors: This targeted therapy may keep the cancer cells from growing and dividing. Tazemetostat is being studied to treat rhabdoid tumor of the kidney that does not respond or has come back after treatment.
• Monoclonal antibody therapy: Monoclonal antibodies are immune system proteins made in the laboratory to treat many diseases, including cancer. As a cancer treatment, these antibodies can attach to a specific target on cancer cells or other cells that may help cancer cells grow. The antibodies are able to then kill the cancer cells, block their growth, or keep them from spreading. Monoclonal antibodies are given by infusion. They may be used alone or to carry drugs, toxins, or radioactive material directly to cancer cells. Nivolumab or a combination of nivolumab and a tyrosine kinase inhibitor (axitinib) are being studied to treat renal cell carcinoma that cannot be removed by surgery or has spread to other parts of the body.

Other targeted therapies are being studied for the treatment of childhood kidney tumors that have recurred (come back).

New types of treatment are being tested in clinical trials.

Information about clinical trials is available from the NCI website.

Treatment for Wilms tumor and other childhood kidney tumors may cause side effects.

To learn more about side effects that begin during treatment for cancer, visit Side Effects.

Side effects from cancer treatment that begin after treatment and continue for months or years are called late effects. Late effects of cancer treatment may include the following:

• Physical problems, such as problems with the heart, the kidney, or during pregnancy.
• Infertility.
• Changes in mood, feelings, thinking, learning, or memory.
• Second cancers (new types of cancer), such as leukemias, cancer of the gastrointestinal tract, or breast cancer.

Some late effects may be treated or controlled. It is important to talk with your child's doctors about the effects cancer treatment can have on your child. (See the PDQ summary about Late Effects of Treatment for Childhood Cancer for more information).

Clinical trials are being done to find out if lower doses of chemotherapy and radiation can be used to lessen the late effects of treatment without changing how well the treatment works.

Monitoring for late effects involving the kidneys in patients with Wilms tumor and related conditions includes the following:

• Children with WAGR syndrome are monitored throughout their lives because they are at increased risk of developing hypertension and kidney disease.
• Children with Wilms tumor and abnormal genitourinary system are monitored because they are at increased risk of late kidney failure.
• Patients with Wilms tumor and aniridia without abnormal genitourinary system are at lower risk but are monitored for kidney disease or kidney failure.

Patients may want to think about taking part in a clinical trial.

For some patients, taking part in a clinical trial may be the best treatment choice. Clinical trials are part of the cancer research process. Clinical trials are done to find out if new cancer treatments are safe and effective or better than the standard treatment.

Many of today's standard treatments for cancer are based on earlier clinical trials. Patients who take part in a clinical trial may receive the standard treatment or be among the first to receive a new treatment.

Patients who take part in clinical trials also help improve the way cancer will be treated in the future. Even when clinical trials do not lead to effective new treatments, they often answer important questions and help move research forward.

Patients can enter clinical trials before, during, or after starting their cancer treatment.

Some clinical trials only include patients who have not yet received treatment. Other trials test treatments for patients whose cancer has not gotten better. There are also clinical trials that test new ways to stop cancer from recurring (coming back) or reduce the side effects of cancer treatment.

Clinical trials are taking place in many parts of the country. Information about clinical trials supported by NCI can be found on NCI’s clinical trials search webpage. Clinical trials supported by other organizations can be found on the ClinicalTrials.gov website.

Follow-up tests may be needed.

As your child goes through treatment, they will have follow-up tests or check-ups. Some tests that were done to diagnose or stage the cancer may be repeated to see how well the treatment is working. Decisions about whether to continue, change, or stop treatment may be based on the results of these tests.

Some of the tests will continue to be done from time to time after treatment has ended. The results of these tests can show if your child's condition has changed or if the cancer has recurred (come back).