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Treatment of Retinoblastoma, Based on Extent of the Disease

If your child has retinoblastoma, a number of factors can affect the treatment options your child’s doctor recommends. Some of these include:

  • The size and location of the tumor(s)
  • Whether the cancer is just in one eye or both
  • The chance for saving vision in the eye(s)
  • Whether the cancer has spread outside the eye

Many of these factors are taken into account as part of the stage of the cancer.

If the retinoblastoma is only in one eye, treatment depends on whether vision in the eye can be saved.

If the retinoblastoma is in both eyes, doctors will try to save at least one eye if at all possible so that the child maintains some vision.

Many children will get several types of treatment. Treatment might be needed for months or even years.

No matter which types of treatment are used, it’s very important that they are given by experts at centers experienced in treating these tumors.

If the eye can see and probably can be saved

For some smaller tumors, focal treatments such as laser therapy or cryotherapy might be the only treatment needed.

But more often, tumors are larger or in hard-to-treat areas. Treatment usually includes a combination of chemotherapy and focal treatments.

If systemic chemotherapy (chemotherapy given by vein) is used, it is typically given for about 6 months to shrink the tumor as much as possible. More recently, many centers have begun to give chemotherapy directly into the artery that feeds the eye (known as intra-arterial chemotherapy) instead of systemic chemotherapy. If the cancer has spread widely inside the eye, chemo might also be injected directly into the eye (known as intravitreal chemotherapy). Usually a few treatments are needed, each given a few weeks apart.

Depending on how much the tumor shrinks and where it is in the eye, different focal treatments can then be applied, usually starting after the first or second cycle of chemotherapy. Treatment options might include brachytherapy (plaque radiotherapy), cryotherapy, or laser therapy (photocoagulation or thermotherapy). External radiation therapy might also be given, but if so, it's usually delayed until the end of chemotherapy.

If the combination of these treatments doesn’t control the disease, the eye might need to be removed.

If the eye cannot see or cannot be saved

If there is no vision in the eye, if the tumor is so advanced within the eye that there is no hope of cure by other means, or if there is painful glaucoma (increased pressure inside the eye), then the eye is removed and an orbital implant is placed in the socket.

If the cancer affects only one eye, no other treatments may be needed. But sometimes, after looking at the removed eye under the microscope, the doctors find that some retinoblastoma cells might have escaped the eye, which means the cancer might come back later in other parts of the body. These children may be given chemotherapy, possibly along with radiation therapy to the area around the eye, to try to lower this risk.

In some instances where there are large tumors in both eyes, chemo may be used first to try to shrink the tumors and avoid the need for surgery in both eyes. If chemo shrinks the tumors enough, focal therapies such as brachytherapy (plaque radiotherapy), cryotherapy, or laser therapy (photocoagulation or thermotherapy) might allow at least one eye (and some vision) to be saved.

Some children with retinoblastoma in only one eye may actually have the hereditary form of retinoblastoma, which means they will probably develop cancer in the other eye as well. Therefore, it's very important to consider genetic counseling and testing to determine if the child has hereditary retinoblastoma. If so, they need to have the other eye examined regularly after treatment.

If the cancer has spread outside the eye

In this uncommon situation, treatment is usually a combination of chemotherapy, radiation, and in some cases surgery.

If the cancer has spread only to the orbit (the area around the eye), treatment with chemotherapy, surgical removal (enucleation) of the eye, and radiation therapy to the orbit is often successful.

If the cancer has spread outside the orbit to distant parts of the body such as the liver or the bones and bone marrow, the chances of a cure using standard chemotherapy and other treatments are not as good. In these cases, using higher doses of chemotherapy followed by a stem cell transplant is usually recommended.

Cancers that have spread to the brain (including trilateral retinoblastomas, which are retinoblastomas that usually start in the pineal gland) are very hard to treat. High-dose chemotherapy and stem cell transplant has shown some promise for these cancers. Clinical trials using other newer treatments may be an option in this situation as well.

If the cancer comes back in the eye after initial treatment

Treatment of cancer that recurs in the eye depends on the size and location of the tumor and on what treatments were used the first time.

If the tumor is small, the child’s sight can often be saved and the cancer destroyed with local treatments such as cryotherapy, laser therapy, radiation therapy (if not already used), or other treatments. Chemotherapy might be given first to shrink the tumor, which might help the other treatments work better.

If the child’s sight cannot be saved, the eye might need to be removed.

Either way, the chance of a cure is very good as long as the cancer is still confined to the eye.

If the cancer comes back outside the eye after initial treatment

Cancers that recur outside the eye are harder to treat. Options might include chemotherapy and radiation, or high-dose chemotherapy with a stem cell transplant in some cases. In this situation, the treatment and the chances of cure are similar to what is described above (when the cancer has spread outside the eye before initial treatment).

Summary of treatment

If the cancer is in only one eye and the potential for saving sight is good, chemotherapy (either by vein or directly into the artery of the eye) and focal treatments such as cryotherapy, laser therapy (photocoagulation or thermotherapy), or plaque radiation are used (although very small tumors, which are very rare, can be treated with focal treatments alone). If there is little or no chance to save the eye (or useful vision), the eye will likely need to be removed.

If the cancer is in both eyes, then the doctors will try to save as much vision as possible. The treatment usually starts with chemotherapy, followed by focal treatments. Surgery might still be needed if these treatments aren’t effective.

If the cancer has spread outside of the eye, treatment is usually a combination of chemotherapy, radiation, and in some cases surgery.

In all cases, children who have had retinoblastoma need to be followed closely for some time after treatment.

The American Cancer Society medical and editorial content team

Our team is made up of doctors and oncology certified nurses with deep knowledge of cancer care as well as journalists, editors, and translators with extensive experience in medical writing.

Hurwitz RL, Shields CL, Shields JA, et al. Chapter 27: Retinoblastoma. In: Pizzo PA, Poplack DG, eds. Principles and Practice of Pediatric Oncology. 7th ed. Philadelphia, Pa: Lippincott Williams & Wilkins; 2016.

Kaufman PL, Kim J, Berry JL. Retinoblastoma: Treatment and outcome. UpToDate. Accessed
www.uptodate.com/contents/retinoblastoma-treatment-and-outcome on September 25, 2018.

National Cancer Institute. Retinoblastoma Treatment (PDQ®). 2018. Accessed at www.cancer.gov/types/retinoblastoma/hp/retinoblastoma-treatment-pdq on September 25, 2018.

Rodriguez-Galindo C, Orbach DB, VanderVeen D. Retinoblastoma. Pediatr Clin North Am. 2015;62(1):201-223.

Last Revised: December 3, 2018

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