Retinoblastoma - Childhood: Types of Treatment

Approved by the Cancer.Net Editorial Board, 09/2023

ON THIS PAGE: You will learn about the different types of treatments doctors use for children with retinoblastoma. Use the menu to see other pages.

In general, cancer in children is uncommon. This means it can be hard for doctors to plan treatments unless they know what has been most effective in other children. That is why more than 60% of children with cancer are treated as part of a clinical trial. A clinical trial is a research study that tests a new approach to treatment. The “standard of care” is the best treatments known based on previous clinical trials. Clinical trials may test such approaches as a new drug, a new combination of existing treatments, or new doses of current therapies. The health and safety of all children participating in clinical trials are closely monitored.

To take advantage of these newer treatments, children with cancer should be treated at a specialized cancer center. Doctors at these centers have extensive experience in treating children with cancer and have access to the latest research. A doctor who specializes in treating children with cancer is called a pediatric oncologist. An ocular oncologist is a doctor who specializes in treating eye cancer. If a pediatric cancer center is not nearby, general cancer centers sometimes have pediatric specialists who are able to be part of your child’s care.

How retinoblastoma is treated

In many cases, a team of doctors works with a child and their family to provide care. This is called a multidisciplinary team. Pediatric cancer centers often have extra support services for children and their families, such as child life specialists, dietitians, physical therapists, occupational therapists, social workers, and counselors. Special activities and programs to help the child and family cope may also be available. Learn more about the clinicians who provide cancer care.

Several types of therapies are used for retinoblastoma, and more than 90% of children can be cured. In addition to curing the retinoblastoma, an important goal of treatment is the preservation of vision. Many of the treatment approaches described below have become available as a result of clinical trials. The Children’s Oncology Group has recently developed clinical trials about treatment for retinoblastoma. (Please note this link takes you to another website.)

Treatment options and recommendations depend on several factors, including the type and stage of cancer, possible side effects, the family’s preferences, and the patient’s overall health. Take time to learn about all of your child’s treatment options and be sure to ask questions about things that are unclear. Talk with your child’s doctor about the goals of each treatment and what your child can expect while receiving the treatment. These types of conversations are called "shared decision-making." Shared decision-making is when your family and your child's doctors work together to choose treatments that fit the goals of your child's care. Shared decision-making is important for retinoblastoma because there are different treatment options. Learn more about making treatment decisions.

The common types of treatments used for retinoblastoma are described below. Your child's care plan also includes treatment for symptoms and side effects, an important part of cancer care. Learn more about preparing your child for treatment.



Surgery is the removal of the tumor and some surrounding healthy tissue during an operation. Surgery to remove the eye is called enucleation. Your child's ocular oncologist will determine if surgery is the best option to treat your child's retinoblastoma.

Children with a tumor in 1 eye only can often be cured with this treatment. For children with a tumor in both eyes, enucleation is only used if other types of treatment to preserve vision are not possible. Surgical treatment for retinoblastoma should be performed by a specially trained pediatric ophthalmologist. Talk with your child's pediatric ophthalmologist or ocular oncologist about the recovery and possible side effects for the surgery your child is having. Learn more about the basics of cancer surgery.

If enucleation is the best treatment to cure the cancer, there will be some loss of eyesight. This may result in trouble with depth perception, but most children adapt well to these differences over time. Talk with your child's doctor about what to expect if enucleation is recommended and what support services are available to help your child adapt to any visual changes.

Many people worry about what their child will look like when they have an eye removed. Your child can have cosmetic surgery to fill the area left by the missing eye. They will be fitted with an artificial eye called a prosthesis. The prosthesis will look and behave almost the same as a natural eye. The artificial eye will move along with the person's remaining eye, though not as much as a natural eye moves. Most people will be unable to tell the difference. If enucleation is required, talk with your child's doctor about a prosthesis.

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Radiation therapy

Radiation therapy is the use of high-energy x-rays or other particles to destroy cancer cells. A doctor who specializes in giving radiation therapy to treat cancer is called a radiation oncologist. A radiation therapy regimen, or schedule, usually consists of a specific number of treatments given over a set period of time.

The most common type of radiation treatment is called external-beam radiation therapy, which is radiation therapy given from a machine outside the body. Proton beam radiation therapy is a type of external-beam radiation therapy that uses protons rather than x-rays. At high energy, protons can destroy cancer cells. Proton beam radiation therapy is a targeted way of delivering radiation therapy, which could decrease both the severity and frequency of short-term and long-term side effects. However, this treatment is not widely available in all areas.

Additionally, there is a type of internal radiation therapy, also called brachytherapy, for retinoblastoma called radioactive plaque therapy. Radioactive plaque therapy is the delivery of radiation therapy directly to the eye with a disc containing radiation.

Fatigue, drowsiness, nausea, vomiting, and headaches are common side effects of radiation therapy, and these usually go away after treatment. Radiation therapy in young children can interfere with the body’s normal development, including growth of the bones around the eye, depending on the dose the child received. The increased risk of additional tumors later in life for children with the hereditary form of retinoblastoma (see Risk Factors) is further increased after external-beam radiation therapy. These effects are not seen after radioactive plaque therapy.

Talk with your child's health care team about the potential side effects of your child's radiation therapy plan. Learn more about the basics of radiation therapy.

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Cryosurgery, also called cryotherapy or cryoablation, uses liquid nitrogen to freeze and destroy cancer cells. More than 1 freezing may be needed.

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Laser therapy

Laser therapy uses heat in the form of a laser to shrink a smaller tumor. It may be called thermotherapy or "TTT" for transpupillary thermotherapy. It may be used alone or in addition to cryotherapy or radiation therapy. Photocoagulation is a different type of laser therapy that uses light to shrink tumors.

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Chemotherapy is the use of drugs to destroy cancer cells, usually by keeping the cancer cells from growing, dividing, and making more cells. It may be used to shrink an eye tumor. Chemotherapy is given by a pediatric oncologist or a medical oncologist, a doctor who specializes in treating cancer with medication. A chemotherapy regimen, or schedule, usually consists of a specific number of cycles given over a set period of time.

Chemotherapy often makes it possible to completely get rid of any remaining smaller tumors with the following focal (localized) measures, described above:

  • Thermotherapy or photocoagulation (laser therapy)

  • Cryotherapy

  • Radioactive plaque therapy

Chemoreduction uses chemotherapy to reduce the size of the tumor. This is a treatment approach that is often used for children with retinoblastoma in both eyes in the hope of avoiding enucleation and preserving vision in at least 1 eye. The ophthalmologist, working closely with the pediatric oncologist, will determine if this treatment is appropriate.

Children with low-risk disease and some children with intermediate-risk disease may be able to avoid chemotherapy after surgery or reduce the intensity of chemotherapy. Talk with your child's doctor about whether this approach is recommended for your child. Both doctors will regularly monitor how well the treatment is working and may recommend additional treatment to prevent the cancer from returning.

The drugs used most often for retinoblastoma are vincristine (Oncovin, Vincasar PFS), carboplatin (Paraplatin), and etoposide (Toposar, VePesid). Depending on the extent of the tumor, a combination of 2 or more drugs may be recommended. All chemotherapy has side effects that occur during treatment. Some drugs may also cause specific long-term side effects. Talk with your child's doctor about the specific drugs used and the possible short-term and long-term side effects before treatment begins.

There are different ways to give chemotherapy for retinoblastoma.

  • Systemic chemotherapy is when medication is given through the bloodstream to reach cancer cells throughout the body. Common ways to give chemotherapy include an intravenous (IV) tube placed into a vein using a needle or in a pill or capsule that is swallowed (orally).

  • Intra-arterial chemotherapy (IA therapy) is chemotherapy that is delivered directly into the artery or the blood vessel that supplies the affected eye. IA therapy is done by inserting a catheter in the child’s groin area. The doctor moves the catheter with the help of imaging technology to reach the blood vessel in the head that directly supplies the eye. This procedure may be used in an effort to save the eye in children with advanced disease if the tumor is still confined to the eye. This method of giving chemotherapy with 1 or several drugs is being used both in children with 1 affected eye or 2 affected eyes. This method is performed by an interventional radiologist.

  • Intravitreal chemotherapy (IVIT therapy) is chemotherapy that is delivered by directly injecting it into the cavity of the eye. IVIT therapy is effective in treating vitreous seeds, which are found in the fluid inside the eye. Vitreous seeds are difficult to treat with systemic chemotherapy alone.

Learn more about the basics of chemotherapy.

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Bone marrow transplant/stem cell transplant

For some children with stage IV extraocular retinoblastoma, a bone marrow transplant may be recommended. The child is first treated with high doses of chemotherapy (and/or radiation therapy) to destroy as many cancer cells as possible, then the transplant takes place.

A bone marrow transplant is a medical procedure in which bone marrow that contains cancer is replaced by highly specialized cells. These cells, called hematopoietic stem cells, develop into healthy bone marrow. Hematopoietic stem cells are blood-forming cells found both in the bloodstream and in the bone marrow. This procedure is also called a stem cell transplant or hematopoietic stem cell transplant.

Before recommending a transplant, doctors will talk with the patient about the risks of this treatment. They will also consider several other factors, such as results of any previous treatment and the patient’s age and general health.

There are 2 types of hematopoietic stem cell transplantation depending on the source of the replacement blood stem cells: allogeneic (ALLO) and autologous (AUTO). ALLO uses donated stem cells, while AUTO uses the patient’s own stem cells. In both types, the goal is to destroy all of the cancer cells in the marrow, blood, and other parts of the body using high doses of chemotherapy and/or radiation therapy, and then allow replacement blood stem cells to create healthy bone marrow.

Side effects depend on the type of transplant, your child’s general health, and other factors. Learn more about the basics of stem cell and bone marrow transplant.

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Physical, emotional, social, and financial effects of cancer

Cancer and its treatment cause physical symptoms and side effects, as well as emotional, social, and financial effects. Managing all of these effects is called palliative and supportive care. It is an important part of your child's care that is included along with treatments intended to slow, stop, or eliminate the cancer.

Palliative and supportive care focuses on improving how your child feels during treatment by managing symptoms and supporting patients and their families with other, non-medical needs. Any person, regardless of age or type and stage of cancer, may receive this type of care. And it often works best when it is started right after a cancer diagnosis. People who receive palliative and supportive care along with treatment for the cancer often have less severe symptoms, better quality of life, and report that they are more satisfied with treatment.

Palliative treatments vary widely and often include medication, nutritional changes, relaxation techniques, emotional and spiritual support, and other therapies. Your child may also receive palliative treatments, such as chemotherapy, surgery, or radiation therapy, to improve symptoms.

Before treatment begins, talk with your child's doctor about the goals of each treatment in the recommended treatment plan. You should also talk about the possible side effects of the specific treatment plan and palliative and supportive care options. Many patients also benefit from talking with a social worker and participating in support groups. Ask your doctor about these resources, too.

Cancer care is often expensive, and navigating health insurance can be difficult. Ask your doctor or another member of your health care team about talking with a financial navigator or counselor who may be able to help with your financial concerns.

During treatment, your child's health care team may ask you to answer questions about your child's symptoms and side effects and to describe each problem. Be sure to tell the health care team if your child experiences a problem. This helps the health care team treat any symptoms and side effects as quickly as possible. It can also help prevent more serious problems in the future.

Learn more about the importance of tracking side effects in another part of this guide. Learn more about palliative and supportive care in a separate section of this website.

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Remission and the chance of recurrence

A remission is when cancer cannot be detected in the body and there are no symptoms. This may also be called having “no evidence of disease” or NED.

A remission may be temporary or permanent. This uncertainty causes many people to worry that the cancer will come back. Although many remissions are permanent, it is important to talk with your child’s doctor about the possibility of the cancer returning. Understanding your child’s risk of recurrence and the treatment options may help you and your child feel more prepared if the cancer does return. Learn more about coping with the fear of recurrence.

If the cancer returns after the original treatment, it is called recurrent cancer. It may come back in the same place (called a local recurrence), nearby (regional recurrence), or in another place (distant recurrence).

If a recurrence happens, a new cycle of testing will begin to learn as much as possible about it. After this testing is done, you and your child’s doctor will talk about the treatment options. If retinoblastoma returns after treatment, the treatment plan depends on where the cancer recurred and how aggressive the new tumor is. The doctor may recommend surgery, radiation therapy, chemotherapy, and/or focal measures, such as photocoagulation, thermotherapy, or cryotherapy (see above). The doctor may suggest clinical trials that are studying new ways to treat recurrent retinoblastoma. Whichever treatment plan you choose, palliative and supportive care will be important for relieving symptoms and side effects.

People with recurrent cancer and their families sometimes experience emotions such as disbelief or fear. Parents are encouraged to talk with their child's health care team about these feelings and ask about support services to help them cope. Learn more about dealing with cancer recurrence.

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If treatment does not work

Although treatment is successful for most children with retinoblastoma, sometimes it is not. If a child’s cancer cannot be cured or controlled, this is called advanced or terminal cancer. This diagnosis is stressful, and advanced cancer may be difficult to discuss. However, it is important to have open and honest conversations with your child’s health care team to express your family’s feelings, preferences, and concerns. The health care team has special skills, experience, and knowledge to support patients and their families and is there to help.

Hospice care is designed to provide the best possible quality of life for people who are expected to live less than 6 months. Parents or guardians are encouraged to talk with the health care team about hospice options, which include hospice care at home, a special hospice center, or other health care locations. Nursing care and special equipment can make staying at home a workable option for many families. Some children may be happier and more comfortable if they can attend school part-time or keep up other activities and social connections. The child’s health care team can help parents or guardians decide on an appropriate level of activity. Making sure a child is physically comfortable and free from pain is extremely important as part of end-of-life care. Learn more about caring for a terminally ill child and advanced cancer care planning.

The death of a child is an enormous tragedy, and families may need support to help them cope with the loss. Pediatric cancer centers often have professional staff and support groups to help with the process of grieving. Learn more on grieving the loss of a child.

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The next section in this guide is About Clinical Trials. It offers more information about research studies that are focused on finding better ways to care for children with cancer. Use the menu to choose a different section to read in this guide.