Retinoblastoma Treatment (PDQ®): Treatment - Patient Information [NCI]

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 Treatment

General Information About Retinoblastoma

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.
Eye anatomy; two-panel drawing shows the outside and inside of the eye. The top panel shows outside of the eye including the eyelid, pupil, sclera, and iris; the bottom panel shows inside of the eye including the cornea, lens, ciliary body, retina, choroid, optic nerve, and vitreous humor.
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 tumor may be in one eye or in both eyes. 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:

  • There is a family history of retinoblastoma.
  • There is more than one tumor in the eye.
  • There is a certain change in the retinoblastomagene.

Nonheritable retinoblastoma is retinoblastoma that is not the heritable form.

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.

The parents, brothers, and sisters of a child with retinoblastoma need to have their eyes checked for retinoblastoma.

The parents of a child with retinoblastoma should have an eye exam by an ophthalmologist (a doctor with special training in diseases of the eye).

The brother or sister of a child with retinoblastoma also should have regular eye exams by an ophthalmologist until age 3 to 5 years, unless it is known that the brother or sister does not have the retinoblastoma gene change. Children who have a close family member with retinoblastoma should have regular eye exams beginning early in life to check for retinoblastoma. Early diagnosis of retinoblastoma may mean the child will need less intense treatment.

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 is also at risk for a tumor in the brain. This is called trilateral retinoblastoma and usually occurs more than 20 months after retinoblastoma is diagnosed. 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 bone or soft tissue sarcoma 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:

  • Pupil of the eye appears white instead of red when light shines into it. This may be seen in flash photographs of the child.
  • Eyes appear to be looking in different directions (lazy eye).
  • Pain or redness in the eye.

Tests that examine the retina are used to detect (find) and diagnose retinoblastoma.

The following tests and procedures may be used:

  • Physical exam and history: An exam of the body to check general signs of health, including checking for signs of disease, such as lumps or anything else that seems unusual. A history of the patient's health habits and past illnesses and treatments will also be taken. The doctor will ask if there is a family history of retinoblastoma.
  • Eye exam with dilated pupil: An exam of the eye in which the pupil is dilated (opened wider) with medicated eye drops to allow the doctor to look through the lens and pupil to the retina. The inside of the eye, including the retina and the optic nerve, is examined with a light. Depending on the age of the child, this exam may be done under anesthesia.

    There are several types of eye exams that are done with the pupil dilated:

    • Ophthalmoscopy: An exam of the inside of the back of the eye to check the retina and optic nerve using a small magnifying lens and a light.
    • Slit-lamp biomicroscopy: An exam of the inside of the eye to check the retina, optic nerve, and other parts of the eye using a strong beam of light and a microscope.
    • Fluorescein angiography: A procedure to look at blood vessels and the flow of blood inside the eye. An orange fluorescent dye (fluorescein) is injected into a blood vessel in the arm and goes into the bloodstream. As the dye travels through blood vessels of the eye, a special camera takes pictures of the retina and choroid to find any areas that are blocked or leaking.
  • Ultrasound exam of the eye: A procedure in which high-energy sound waves (ultrasound) are bounced off the internal tissues of the eye to make echoes. Eye drops are used to numb the eye and a small probe that sends and receives sound waves is placed gently on the surface of the eye. The echoes make a picture of the inside of the eye and the distance from the cornea to the retina is measured. The picture, called a sonogram, shows on the screen of the ultrasound monitor.
  • MRI (magnetic resonance imaging): A procedure that uses a magnet, radio waves, and a computer to make a series of detailed pictures of areas inside the body, such as the eye. This procedure is also called nuclear magnetic resonance imaging (NMRI).
  • CT scan (CAT scan): A procedure that makes a series of detailed pictures of areas inside the body, such as the eye, taken from different angles. The pictures are made by a computer linked to an x-ray machine. A dye may be injected into a vein or swallowed to help the organs or tissues show up more clearly. This procedure is also called computed tomography, computerized tomography, or computerized axial tomography.
  • Bone scan: A procedure to check if there are rapidly dividing cells, such as cancer cells, in the bone. A very small amount of radioactive material is injected into a vein and travels through the bloodstream. The radioactive material collects in the bones and is detected by a scanner that also takes a picture of the body. Areas of bone with cancer show up brighter in the picture because they take up more radioactive material than normal bone cells do.

Retinoblastoma is usually diagnosed without a biopsy.

Certain factors affect prognosis (chance of recovery) and treatment options.

The prognosis (chance of recovery) and treatment options depend on the following:

  • Whether the tumor is in one or both eyes.
  • The size and number of tumors.
  • Whether the tumor has spread to the area around the eye, to the brain, or to other parts of the body.
  • The age of the patient.
  • How likely it is that vision can be saved in one or both eyes.
  • Whether a second type of cancer has formed.

Stages of Retinoblastoma

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:

  • Eye exam with dilatedpupil: An exam of the eye in which the pupil is dilated (opened wider) with medicated eye drops to allow the doctor to look through the lens and pupil to the retina. The inside of the eye, including the retina and the optic nerve, is examined using a light. Depending on the age of the child, this exam may be done under anesthesia.
  • Ultrasound exam: A procedure in which high-energy sound waves (ultrasound) are bounced off internal tissues or organs and make echoes. The echoes form a picture of body tissues called a sonogram.
  • CT scan (CAT scan): A procedure that makes a series of detailed pictures of areas inside the body taken from different angles. The pictures are made by a computer linked to an x-ray machine. A dye may be injected into a vein or swallowed to help the organs or tissues show up more clearly. This procedure is also called computed tomography, computerized tomography, or computerized axial tomography.
  • MRI (magnetic resonance imaging): A procedure that uses a magnet, radio waves, and a computer to make a series of detailed pictures of areas inside the body. This procedure is also called nuclear magnetic resonance imaging (NMRI).
  • Bone scan: A procedure to check if there are rapidly dividing cells, such as cancer cells, in the bone. A very small amount of radioactive material is injected into a vein and travels through the bloodstream. The radioactive material collects in the bones and is detected by a scanner that also takes a picture of the body. Areas of bone with cancer show up brighter in the picture because they take up more radioactive material than normal bone cells do.
  • Bone marrow aspiration and biopsy: The removal of bone marrow and a small piece of bone by inserting a hollow needle into the hipbone or breastbone. A pathologist views the bone marrow under a microscope to look for signs of cancer. A bone marrow aspiration and biopsy is done if it is thought the cancer has spread outside of the eye.

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.

Stage 0

The eye has not been removed and the tumor was treated without surgery.

Stage I

The eye has been removed and no cancer cells are seen.

Stage II

The eye has been removed and there are cancer cells left that can be seen only with a microscope.

Stage III

Stage III is divided into stages IIIa and IIIb:

  • In stage IIIa, cancer has spread to tissues around the eye socket.
  • In stage IIIb, cancer has spread to lymph nodes near the ear or in the neck.

Stage IV

Stage IV is divided into stages IVa and IVb:

  • In stage IVa, cancer has spread to the blood but not to the brain or spinal cord. There are one or more tumors.
  • In stage IVb, cancer has spread to the brain or spinal cord. It may have spread to other parts of the body as well.

Treatment for retinoblastoma depends on whether it is intraocular (within the eye) or extraocular (outside the eye).

Intraocular retinoblastoma

In intraocular retinoblastoma, cancer is found in the eye and may be only in the retina 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 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:

  • Tissue. The cancer spreads from where it began by growing into nearby areas.
  • Lymph system. The cancer spreads from where it began by getting into the lymph system. The cancer travels through the lymph vessels to other parts of the body.
  • Blood. The cancer spreads from where it began by getting into the blood. The cancer travels through the blood vessels to other parts of the body.

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.

  • Lymph system. The cancer gets into the lymph system, travels through the lymph vessels, and forms a tumor (metastatic tumor) in another part of the body.
  • Blood. The cancer gets into the blood, travels through the blood vessels, and forms a tumor (metastatic tumor) in another part of the body.

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.

Recurrent Retinoblastoma

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.

Treatment Option Overview

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:

  • Pediatric surgeon.
  • Radiation oncologist.
  • Pediatrician.
  • Pediatric nurse specialist.
  • Rehabilitation specialist.
  • Psychologist.
  • Social workers.
  • Geneticist or genetic counselor.

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:

  • Physical problems such as trouble hearing clearly or, if the eye is removed, a change in the shape and size of the bone around the eye.
  • Changes in mood, feelings, thinking, learning, or memory.
  • Second cancers (new types of cancer), such as osteosarcoma, soft tissue sarcoma, or melanoma.

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 on Late Effects of Treatment for Childhood Cancer for more information.)

Children with the heritable form of retinoblastoma are at an even greater risk of forming second cancers. Children, especially those younger than 12 months, who have been treated for retinoblastoma with radiation therapy have an increased risk of second cancers. Regular follow-up by health professionals who are experts in diagnosing and treating late effects is important.

Five types of standard treatment are used:

Surgery (enucleation)

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 the tumor is large and there is little or no chance that vision can be saved. 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 eye and to check the other eye.

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. 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. The way the radiation therapy is given depends on the type and stage of the cancer being treated. Methods of radiation therapy used to treat retinoblastoma include the following:

  • Intensity-modulated radiation therapy (IMRT): A type of 3-dimensional (3-D) radiation therapy that uses a computer to make pictures of the size and shape of the tumor. Thin beams of radiation of different intensities (strengths) are aimed at the tumor from many angles. This type of radiation therapy causes less damage to healthy tissue near the tumor.
  • Stereotactic radiation therapy: Radiation therapy that uses a rigid head frame attached to the skull to aim high-dose radiation beams directly at the tumors, causing less damage to nearby healthy tissue. It is also called stereotactic external-beam radiation and stereotaxic radiation therapy.
  • Proton beam radiation therapy: Radiation therapy that uses protons made by a special machine. A proton is a type of high-energy radiation that is different from an x-ray.
  • Plaque radiotherapy: Radioactive seeds are attached to one side of a disk, called a plaque, and placed directly on the outside wall of the eye near the tumor. The side of the plaque with the seeds on it faces the eyeball, aiming radiation at the tumor. The plaque helps protect other nearby tissue from the radiation.
    Plaque radiotherapy of the eye; drawing shows a cross-section of the eye. An inset shows a plaque with radioactive seeds; it is placed on the outside of the eye with the seeds aimed at the cancer. Also shown are the sclera, choroid, retina, and optic nerve.
    Plaque radiotherapy of the eye. A type of radiation therapy used to treat eye tumors. Radioactive seeds are placed on one side of a thin piece of metal (usually gold) called a plaque. The plaque is sewn onto the outside wall of the eye. The seeds give off radiation which kills the cancer. The plaque is removed at the end of treatment, which usually lasts for several days.

Cryotherapy

Cryotherapy is a treatment that uses an instrument to freeze and destroy abnormal tissue, such as carcinoma in situ. This type of treatment is also called cryosurgery.

Thermotherapy

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 type of treatment is also called laser therapy.

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. 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:

  • Systemic chemotherapy: 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 is given to shrink the tumor (chemoreduction) and avoid surgery to remove the eye. After chemoreduction, other treatments may include radiation therapy, cryotherapy, laser therapy, or regional chemotherapy. Systemic chemotherapy may also be given to kill any cancer cells that are left after the initial treatment. Treatment given after the initial treatment, to lower the risk that the cancer will come back, is called adjuvant therapy.
  • Regional chemotherapy: When chemotherapy is placed directly into the cerebrospinal fluid (intrathecal chemotherapy), an organ (such as the eye), or a body cavity, the drugs mainly affect cancer cells in those areas. Several types of regional chemotherapy:are used to treat retinoblastoma.
    • Ophthalmic artery infusion chemotherapy: Ophthalmic artery infusion chemotherapy carries anticancer drugs directly to the eye. A catheter is put into an artery that leads to the eye and the anticancer drug is given through the catheter. During this treatment, a small balloon may be inserted into the artery to block it and keep most of the anticancer drug trapped near the tumor. This type of chemotherapy may be given as the initial treatment when the tumor is only in one eye or when the tumor has not responded to other types of treatment. Ophthalmic artery infusion chemotherapy is given at specialized retinoblastoma treatment centers.
    • Subtenon chemotherapy: Subtenon chemotherapy is the use of drugs injected through the membrane covering the muscles and nerves at the back of the eyeball. It is usually combined with systemic chemotherapy and local treatment (such as radiation therapy, cryotherapy, or thermotherapy) in order to avoid surgery to remove the eye.

See Drugs Approved for Retinoblastoma for more information.

New types of treatment are being tested in clinical trials.

This summary section describes treatments that are being studied in clinical trials. It may not mention every new treatment being studied. Information about clinical trials is available from the NCI Web site.

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.

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. This is sometimes called re-staging.

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 Options for Retinoblastoma

Intraocular Retinoblastoma

If the tumor is large and it is expected that the eye cannot be saved, treatment may include the following:

  • Enucleation. After surgery, systemic chemotherapy may be given to lower the risk that the cancer will spread to other parts of the body.

If it is expected that the eye can be saved, treatment may include the following:

  • Systemic chemotherapy, ophthalmic artery infusion chemotherapy, or subtenon chemotherapy to shrink the tumor, followed by one or more of the following:
    • Cryotherapy.
    • Thermotherapy.
    • Plaque radiotherapy.
  • External-beam radiation therapy.

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.

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.

Extraocular Retinoblastoma

Treatment for extraocular retinoblastoma that has spread to the area around the eye may include the following:

  • Systemic chemotherapy and external-beam radiation therapy.
  • Systemic chemotherapy followed by surgery (enucleation). Radiation therapy and more chemotherapy may be given after surgery.

Treatment for extraocular retinoblastoma that has spread to the brain may include the following:

  • Systemic or intrathecal chemotherapy.
  • Radiation therapy to the brain and spinal cord.
  • Chemotherapy followed by high-dose chemotherapy with stem cell rescue.

It is not clear whether extraocular retinoblastoma that has spread to the brain responds to chemotherapy or radiation therapy.

For trilateral retinoblastoma, treatment may include the following:

  • Chemotherapy followed by high-dose chemotherapy with stem cell rescue.

For retinoblastoma that has spread to other parts of the body, treatment may include the following:

  • Chemotherapy followed by high-dose chemotherapy with stem cell rescue.
  • A clinical trial of chemotherapy followed by high-dose chemotherapy with stem cell rescue and radiation therapy.

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.

Recurrent Retinoblastoma

Treatment of recurrent retinoblastoma may include the following:

  • Enucleation.
  • External-beam radiation therapy or plaque radiotherapy.
  • Cryotherapy.
  • Thermotherapy.
  • Systemic chemotherapy or ophthalmic artery infusion chemotherapy.

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.

To Learn More About Childhood Cancer

For more information from the National Cancer Institute about the treatment of retinoblastoma, see the following:

  • Retinoblastoma Home Page
  • Computed Tomography (CT) Scans and Cancer
  • Drugs Approved for Retinoblastoma
  • Biological Therapies for Cancer
  • Genetic Testing for Hereditary Cancer Syndromes

For more childhood cancer information and other general cancer resources, see the following:

  • Childhood Cancers
  • CureSearch for Children's Cancer
  • Late Effects of Treatment for Childhood Cancer
  • Adolescents and Young Adults with Cancer
  • Young People with Cancer: A Handbook for Parents
  • Care for Children and Adolescents with Cancer
  • Cancer Staging
  • Coping with Cancer: Supportive and Palliative Care
  • Questions to Ask Your Doctor About Cancer
  • Cancer Library
  • Information for Survivors/Caregivers/Advocates

Changes to This Summary (01 / 28 / 2014)

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.

Changes were made to this summary to match those made to the health professional version.

About This PDQ Summary

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This PDQ cancer information summary has current information about the treatment of retinoblastoma. It is meant to inform and help patients, families, and caregivers. It does not give formal guidelines or recommendations for making decisions about health care.

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Editorial Boards write the PDQ cancer information summaries and keep them up to date. These Boards are made up of experts in cancer treatment and other specialties related to cancer. The summaries are reviewed regularly and changes are made when there is new information. The date on each summary ("Date Last Modified") is the date of the most recent change.

The information in this patient summary was taken from the health professional version, which is reviewed regularly and updated as needed, by the PDQ Pediatric Treatment Editorial Board.

Clinical Trial Information

A clinical trial is a study to answer a scientific question, such as whether one treatment is better than another. Trials are based on past studies and what has been learned in the laboratory. Each trial answers certain scientific questions in order to find new and better ways to help cancer patients. During treatment clinical trials, information is collected about the effects of a new treatment and how well it works. If a clinical trial shows that a new treatment is better than one currently being used, the new treatment may become "standard." Patients may want to think about taking part in a clinical trial. Some clinical trials are open only to patients who have not started treatment.

Clinical trials are listed in PDQ and can be found online at NCI's Web site. Many cancer doctors who take part in clinical trials are also listed in PDQ. For more information, call the Cancer Information Service 1-800-4-CANCER (1-800-422-6237).

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Last Revised: 2014-01-28


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