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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.
Retinoblastoma is a disease in which malignant (cancer) cells form in the tissues of the retina.
The retina is the nerve tissue that lines the inside of the back of the eye. The retina senses light and sends images to the brain by way of the optic nerve.Anatomy of the eye, showing the outside and inside of the eye including the sclera, cornea, iris, ciliary body, choroid, retina, vitreous humor, and optic nerve. The vitreous humor is a gel that fills the center of the eye.
Although retinoblastoma may occur at any age, it usually occurs in children younger than 5 years, most often younger than 2 years. The cancer may be in one eye (unilateral) or in both eyes (bilateral). Retinoblastoma rarely spreads from the eye to nearby tissue or other parts of the body.
Retinoblastoma occurs in heritable and nonheritable forms.
A child is thought to have the heritable form of retinoblastoma when one of the following is true:
After diagnosis and treatment in a child with heritable retinoblastoma, new tumors may continue to form for a few years. Regular eye exams to check for new tumors are usually done every 2 to 4 months for at least 28 months.
Nonheritable retinoblastoma is retinoblastoma that is not the heritable form. Most cases of retinoblastoma are the nonheritable form.
Treatment for both forms of retinoblastoma should include genetic counseling.
Parents should receive genetic counseling (a discussion with a trained professional about genetic diseases) to discuss whether genetic testing is needed and the risk of retinoblastoma for the child's brothers or sisters.
A child who has heritable retinoblastoma has an increased risk of trilateral retinoblastoma and other cancers.
A child with heritable retinoblastoma has an increased risk of a pineal tumor in the brain. When retinoblastoma and a brain tumor occur at the same time it is called trilateral retinoblastoma. The brain tumor is usually diagnosed between 20 and 36 months of age. Regular screening using MRI (magnetic resonance imaging) every 6 months for 5 years may be done for a child thought to have heritable retinoblastoma or for a child with retinoblastoma in one eye and a family history of the disease. CT scans (computerized tomography) should not be used for routine screening to avoid exposing the child to ionizing radiation.
Heritable retinoblastoma also increases the child's risk of other types of cancer such as lung cancer, bladder cancer, or melanoma in later years. Regular follow-up exams are important.
Signs and symptoms of retinoblastoma include "white pupil" and eye pain or redness.
These and other signs and symptoms may be caused by retinoblastoma or by other conditions. Check with a doctor if your child has any of the following:
Tests that examine the retina are used to detect (find) and diagnose retinoblastoma.
The following tests and procedures may be used:
There are several types of eye exams that are done with the pupil dilated:
Retinoblastoma can usually be diagnosed without a biopsy.
When retinoblastoma is in one eye, it sometimes forms in the other eye. Exams of the unaffected eye are done until it is known if the retinoblastoma is the heritable form.
Certain factors affect prognosis (chance of recovery) and treatment options.
The prognosis (chance of recovery) and treatment options depend on the following:
After retinoblastoma has been diagnosed, tests are done to find out if cancer cells have spread within the eye or to other parts of the body.
The process used to find out if cancer has spread within the eye or to other parts of the body is called staging. The information gathered from the staging process determines the stage of the disease. It is important to know the stage in order to plan treatment.
The following tests and procedures may be used in the staging process:
The International Retinoblastoma Staging System (IRSS) may be used for staging retinoblastoma.
There are several staging systems for retinoblastoma. The IRSS stages are based on how much cancer remains after surgery to remove the tumor and whether the cancer has spread.
The tumor is in the eye only. The eye has not been removed and the tumor was treated without surgery.
The tumor is in the eye only. The eye has been removed and no cancer cells remain.
The tumor is in the eye only. The eye has been removed and there are cancer cells left that can be seen only with a microscope.
Stage III is divided into stages IIIa and IIIb:
Stage IV is divided into stages IVa and IVb:
Treatment for retinoblastoma depends on whether it is intraocular (within the eye) or extraocular (outside the eye).
In intraocular retinoblastoma, cancer is found in one or both eyes and may be in the retina only or may also be in other parts of the eye such as the choroid, ciliary body, or part of the optic nerve. Cancer has not spread to tissues around the outside of the eye or to other parts of the body.
Extraocular retinoblastoma (metastatic)
In extraocular retinoblastoma, cancer has spread beyond the eye. It may be found in tissues around the eye (orbital retinoblastoma) or it may have spread to the central nervous system (brain and spinal cord) or to other parts of the body such as the liver, bones, bone marrow, or lymph nodes.
There are three ways that cancer spreads in the body.
Cancer can spread through tissue, the lymph system, and the blood:
Cancer may spread from where it began to other parts of the body.
When cancer spreads to another part of the body, it is called metastasis. Cancer cells break away from where they began (the primary tumor) and travel through the lymph system or blood.
The metastatic tumor is the same type of cancer as the primary tumor. For example, if retinoblastoma spreads to the bone, the cancer cells in the bone are actually retinoblastoma cells. The disease is metastatic retinoblastoma, not bone cancer.
Progressive retinoblastoma is retinoblastoma that does not respond to treatment. Instead, the cancer grows, spreads, or gets worse.
Recurrent retinoblastoma is cancer that has recurred (come back) after it has been treated. The cancer may recur in the eye, in tissues around the eye, or in other places in the body.
There are different types of treatment for patients with retinoblastoma.
Different types of treatment are available for patients with retinoblastoma. 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 retinoblastoma should have their treatment planned by a team of health care providers who are experts in treating cancer in children.
The goals of treatment are to save the child's life, to save vision and the eye, and to prevent serious side effects. Treatment will be overseen by a pediatric oncologist, a doctor who specializes in treating children with cancer. The pediatric oncologist works with other health care providers who are experts in treating children with eye cancer and who specialize in certain areas of medicine. These may include a pediatric ophthalmologist (children's eye doctor) who has a lot of experience in treating retinoblastoma and the following specialists:
Some cancer treatments cause side effects months or years after treatment has ended.
Side effects from cancer treatment that begin during or after treatment and continue for months or years are called late effects. Late effects of treatment for retinoblastoma may include the following:
The following risk factors may increase the risk of having another cancer:
It is important to talk with your child's doctors about the effects cancer treatment can have on your child. Regular follow-up by health professionals who are experts in diagnosing and treating late effects is important. See the PDQ summary on Late Effects of Treatment for Childhood Cancer for more information.
Six types of standard treatment are used:
Enucleation is surgery to remove the eye and part of the optic nerve. The eye will be checked with a microscope to see if there are any signs that the cancer is likely to spread to other parts of the body. Enucleation is done if there is little or no chance that vision can be saved and when the tumor is large, did not respond to treatment, or comes back after treatment. The patient will be fitted for an artificial eye.
Close follow-up is needed for 2 years or more to check for signs of recurrence in the area around the affected eye and to check the other eye.
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. There are two types of radiation therapy. External radiation therapy uses a machine outside the body to send radiation toward the cancer. Internal radiation therapy uses a radioactive substance sealed in needles, seeds, wires, plaques, or catheters that are placed directly into or near the cancer.
Methods of radiation therapy used to treat retinoblastoma include the following:
The way the radiation therapy is given depends on how the cancer responded to other treatments and whether cancer has spread to other parts of the body.
Cryotherapy is a treatment that uses an instrument to freeze and destroy abnormal tissue. This type of treatment is also called cryosurgery.
Thermotherapy is the use of heat to destroy cancer cells. Thermotherapy may be given using a laser beam aimed through the dilated pupil or onto the outside of the eyeball. Thermotherapy may be used alone for small tumors or combined with chemotherapy for larger tumors. This treatment is a type of laser therapy.
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. The way the chemotherapy is given depends on the stage of the cancer and where the cancer is in the body.
There are different types of chemotherapy:
See Drugs Approved for Retinoblastoma for more information.
High-dose chemotherapy with stem cell rescue
High-dose chemotherapy with stem cell rescue is a way of giving high doses of chemotherapy and replacing blood -forming cells destroyed by the cancer treatment. Stem cells (immature blood cells) are removed from the blood or bone marrow of the patient and are frozen and stored. After the chemotherapy is completed, the stored stem cells are thawed and given back to the patient through an infusion. These reinfused stem cells grow into (and restore) the body's blood cells.
New types of treatment are being tested in clinical trials.
Information about clinical trials is available from the NCI Web site.
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. See the Treatment Options section that follows for links to current treatment clinical trials. These have been retrieved from NCI's listing of clinical trials.
Follow-up tests may be needed.
Some of the tests that were done to diagnose the cancer or to find out the stage of the cancer may be repeated. Some tests will be repeated in order 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). These tests are sometimes called follow-up tests or check-ups.
Treatment of Unilateral and Bilateral Retinoblastoma
If it is expected that the eye can be saved, treatment may include the following:
If the tumor is large and it is expected that the eye cannot be saved, treatment may include the following:
When retinoblastoma is in both eyes, the treatment for each eye may be different, depending on the size of the tumor and whether it is expected that the eye can be saved. The dose of systemic chemotherapy is usually based on the eye that has more cancer.
Check for U.S. clinical trials from NCI's list of cancer clinical trials that are now accepting patients with intraocular retinoblastoma. For more specific results, refine the search by using other search features, such as the location of the trial, the type of treatment, or the name of the drug. Talk with your child's doctor about clinical trials that may be right for your child. General information about clinical trials is available from the NCI Web site.
Treatment of Extraocular Retinoblastoma
Treatment for extraocular retinoblastoma that has spread to the area around the eye may include the following:
Treatment for extraocular retinoblastoma that has spread to the brain may include the following:
It is not clear whether treatment with chemotherapy, radiation therapy, or high-dose chemotherapy with stem cell rescue improves survival for patients with extraocular retinoblastoma.
For trilateral retinoblastoma, treatment may include the following:
For retinoblastoma that has spread to other parts of the body, treatment may include the following:
Check for U.S. clinical trials from NCI's list of cancer clinical trials that are now accepting patients with extraocular retinoblastoma. For more specific results, refine the search by using other search features, such as the location of the trial, the type of treatment, or the name of the drug. Talk with your child's doctor about clinical trials that may be right for your child. General information about clinical trials is available from the NCI Web site.
Treatment of Progressive or Recurrent Retinoblastoma
Treatment of progressive or recurrent intraocular retinoblastoma may include the following:
Treatment of progressive or recurrent extraocular retinoblastoma may include the following:
Check for U.S. clinical trials from NCI's list of cancer clinical trials that are now accepting patients with recurrent retinoblastoma. For more specific results, refine the search by using other search features, such as the location of the trial, the type of treatment, or the name of the drug. Talk with your child's doctor about clinical trials that may be right for your child. General information about clinical trials is available from the NCI Web site.
For more information from the National Cancer Institute about the treatment of retinoblastoma, see the following:
For more childhood cancer information and other general cancer resources, see the following:
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.
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National Cancer Institute: PDQ® Retinoblastoma Treatment. Bethesda, MD: National Cancer Institute. Date last modified <MM/DD/YYYY>. Available at: http://www.cancer.gov/types/retinoblastoma/patient/retinoblastoma-treatment-pdq. Accessed <MM/DD/YYYY>.
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