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- Retinoblastoma
- Retinoblastoma Treatment
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Experts at MD Anderson have partnered with physicians at three other leading institutions to form the Retinoblastoma Center of Houston. This group is one of the world's foremost teams in caring for retinoblastoma. They will work together to develop a customized treatment plan just for your child.
Our retinoblastoma treatments
The team that diagnoses and treats your child for retinoblastoma will include several highly trained doctors, including medical oncologists, surgical oncologists, radiation oncologists, ocular pathologists and geneticists. Together, they offer every possible treatment for retinoblastoma, including:
- Intra-arterial chemotherapy
- Intravitreal chemotherapy
- High-dose chemotherapy with stem cell rescue
- Proton therapy
- Brachytherapy
- Laser therapy
- Surgery
- Specialized ocular pathology and tissue harvesting
Doctors who treat retinoblastoma will tell you they have three goals, in order: Save the life. Save the eye. Save the vision. To achieve these goals, they may use the following treatments.
Chemotherapy
Chemotherapy drugs kill cancer cells, control their growth or relieve disease-related symptoms. Chemotherapy may involve a single drug or a combination of two or more drugs, depending on the type of cancer and how fast it is growing.
Learn more about chemotherapy.
Retinoblastoma chemotherapy is delivered in one of the following ways:
Intra-arterial: A catheter is inserted into a blood vessel near the groin and then steered through the body up to the eye, where chemo is directly delivered. This avoids exposing the patient to systemic chemotherapy. Under certain circumstances, this is the preferred technique for delivering chemotherapy medications.
Intravenous: The drug is injected into a blood vessel and is pumped throughout the body.
Intravitreal: Chemotherapy is injected directly into the eye.
Periocular: The drug is placed around the eye.
High dose chemotherapy with stem cell rescue: This is a form of intravenous chemotherapy. It is an option for patients whose cancer has spread beyond the eye into areas like the liver or central nervous system. These patients are given a very powerful dose of chemotherapy to kill cancer cells.
As a side effect, the medicine also kills the patient’s bone marrow, which makes new blood cells.
The patient then undergoes a stem cell transplant. This procedure is not designed to kill the cancer, but to restart the body’s ability to create blood cells.
A stem cell transplant (also known as a bone marrow transplant) replaces bone marrow with new, healthy bone marrow stem cells. For retinoblastoma, the stem cells are usually taken from the patient before chemotherapy. Patients usually stay in the hospital for three to four weeks after the transplant. Learn more about stem cell transplants.
Laser treatment
Doctors can use lasers to destroy a retinoblastoma tumor. Laser treatment is often used after chemotherapy to help kill any remaining cancer cells. This is effective for small tumors that are confined to the retina and do not involve the optic disc or the area near it, called the macula, which is the most important part of the retina for vision.
Surgery
Enucleation surgery, or removal of the eye, may be performed when the tumor fills more than half the eyeball, when other structures in the eye are involved or when the retina is detached. Children who undergo this procedure are fitted with an ocular implant and later an artificial eye. Thanks to advances in detection and treatment, enucleation is not used as often as it once was.
Cryoablation
Cryoablation, also known as cryotherapy or cryosurgery, uses cold to kill tumor cells. During the procedure, a special probe is inserted into the tumor and then cooled to temperatures well below freezing. A ball of ice forms at the tip of the probe, freezing and destroying cancerous tissue. Cryotherapy is not as invasive as surgery. It is generally performed as an outpatient procedure.
Learn more about cryoablation.
Radiation therapy
Radiation therapy uses powerful, focused beams of energy to kill cancer cells. There are several different radiation therapy techniques. Doctors can use these to accurately target a tumor while minimizing damage to healthy tissue.
Learn more about radiation therapy.
Radiation treatments for retinoblastoma may include the following:
Brachytherapy: Brachytherapy delivers radiation therapy with small pieces of radioactive material that are placed on or inside the patient’s body as close to the tumor as possible. This allows doctors to deliver very high doses of radiation directly to the patient’s tumor while limiting radiation exposure to healthy tissue. Also called plaque therapy, this treatment is used for patients with very small tumors. Learn more about brachytherapy.
Proton therapy: Proton therapy is a sophisticated way of delivering radiation therapy. It uses a different type of energy that may allow doctors to target tumors with much more accuracy. This limits damage to nearby healthy tissue and allows for the delivery of a more powerful dose of radiation.
Proton therapy may be used to treat retinoblastoma that has spread outside the eyeball into nearby structures. Learn more about proton therapy.
Life after cancer
After treatment, retinoblastoma patients should undergo regular cancer screenings and follow-up care to look for cancer recurrence. This is especially important for patients with hereditary disease. These patients are at an increased risk of developing a second cancer over the course of their life.
MD Anderson’s Children’s Cancer Hospital offers expert survivorship care to retinoblastoma patients, including regular screening exams, genetic counseling and testing, and ongoing monitoring for long-term side effects.
Learn more about retinoblastoma:
Treatment at MD Anderson
Retinoblastoma is treated in our Children's Cancer Hospital and Proton Therapy Center.
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Retinoblastoma: 7 insights on this rare eye cancer
Retinoblastoma is a cancer that forms in the retina, a layer of tissue in the eye that’s sensitive to light. It’s a rare eye cancer, but it’s the most common cancer that starts in the eye in children. It’s typically diagnosed in children under age 2.
We spoke with ophthalmologist and retinoblastoma specialist Dan Gombos, M.D., to learn more about retinoblastoma, including symptoms, how it’s diagnosed and how it’s treated.
What causes retinoblastoma?
Each retina cell normally has two retinoblastoma genes, called RB1 genes, involved in cell division. If a cell loses both copies of the RB1 gene, you can develop retinoblastoma.
About 40% of cases are germline. This means either a child inherits this gene mutation from one of their parents, or a child develops a new gene mutation spontaneously. In these cases, retinoblastoma can be passed down to the next generation. In most germline cases, children have retinoblastoma in both eyes. This is called bilateral retinoblastoma.
About 60% of cases are caused by a sporadic gene mutation. In these cases, retinoblastoma is not inherited and cannot be passed down to the next generation. This will almost always be unilateral retinoblastoma, affecting only one eye.
Is genetic testing recommended for retinoblastoma?
Yes, it’s our standard to refer patients with retinoblastoma and, at times, their family members, for genetic counseling and genetic testing.
When you have a potential genetic disorder, you’re really referring a family. Families need genetic counseling, especially young parents who have a child with retinoblastoma. They often want to know what the odds are that their other children or potential children will develop retinoblastoma. It could be less than 1% in some cases and as high as 45% in other cases. So, whenever you have a genetic disorder that makes you more likely to have cancer, it’s vital to see a geneticist who provides both genetic testing and genetic counseling.
What are the symptoms of retinoblastoma?
The most common symptom of retinoblastoma in the United States is a white reflex in the eye, known as leukocoria. If a photo is taken with the flash on, it will look like a white spot in the center of the pupil. This is an abnormal reflection of light from the retina.
Other symptoms that could indicate retinoblastoma are strabismus (crossed eyes) or a lazy eye.
Several conditions can cause these symptoms in the eye, many of which are not cancer. But they could negatively affect a child’s sight, so it’s important to have your child evaluated by an ophthalmologist, a medical doctor trained in eye surgery, as soon as possible if you notice these symptoms.
How is retinoblastoma diagnosed?
The early signs of retinoblastoma, such as a white reflex in the eye, are usually found when a child visits a pediatrician. If the assessment is concerning, they will refer the child to an ophthalmologist or an ocular oncologist for a diagnostic eye exam, which may include:
- Dilated eye exam: The child will be placed under anesthesia and their pupils will be dilated so the doctor can examine all parts of the eye.
- Ultrasound: The doctor looks for signs of calcium inside the eye, which is often associated with retinoblastoma.
- Fluorescein angiogram: The doctor dilates the child’s eyes and then injects a colored dye into a vein in the arm. Once the dye reaches the blood vessels in the eye, the doctor uses a special camera to examine the retina.
- MRI: The doctor scans the brain and eye socket.
A biopsy is not used to diagnose retinoblastoma because it can potentially harm the eye, and removing tissue from the tumor can cause the cancer cells to spread.
“After the exams and testing are done, the cancer specialist will determine whether it’s retinoblastoma,” says Gombos, stressing the importance of seeing someone with extensive experience diagnosing eye cancers.
How is retinoblastoma treated?
Treatment for retinoblastoma depends on several factors, including how advanced the tumor is.
“Our treatment approach to retinoblastoma is: first, save the child’s life; second, save the child’s eye; and third, save the child’s sight,” says Gombos.
Chemotherapy kills cancer cells, and it can be delivered in different ways to treat retinoblastoma. One way is intra-arterially, in which chemo is delivered through a catheter inserted into a blood vessel in the groin. It then travels to the blood vessels that feed the eye, delivering the chemo directly into the eye. Intravenous means the chemo is injected into a blood vessel and pumped throughout the body. Chemo can also be injected directly into the eye with a needle while a patient is under anesthesia. This method is called intravitreal.
“We use these techniques to salvage the eye and often the sight,” says Gombos. “But if the cancer is not responding, the disease progresses or if there’s concern of the tumor growing near the optic nerve, we may surgically remove the eye. We don’t want to risk it spreading elsewhere.”
If the tumor in the eye is advanced, then there is a potential risk of the tumor spreading. If that’s the case, sometimes we will remove the eye at the beginning and avoid chemotherapy altogether. If the eye is so advanced that the vision will likely not be salvaged, this is often the best option.
“We always discuss the risks and benefits with every type of treatment with the family,” says Gombos. “We factor in everything and determine what’s in the best interest of the child.”
Can you recover from retinoblastoma?
Retinoblastoma is a highly curative disease when caught early. That’s why it’s important to get treatment at a specialized cancer center with specialists who treat this type of cancer.
What research is being done to advance retinoblastoma treatment?
MD Anderson has contributed to several retinoblastoma clinical trials. A new clinical trial is exploring whether intravenous chemotherapy combined with intravitreal chemotherapy will improve the success rates of salvaging the eyes during retinoblastoma treatment.
Experts are also looking at using a liquid biopsy to diagnose retinoblastoma. While this test has been successful in detecting blood cancers and, more recently, solid tumors, experts are trying to figure out if this would apply to eye cancers.
“We’re researching to see if tapping into the aqueous, which is the fluid in the front of the eye, will be helpful in assessing cancer,” says Gombos.
Gombos began researching retinoblastoma during medical school 30 years ago, and he sees a lot of promise in the treatment advances.
“Retinoblastoma is a disease with a lot of hope,” he says. “When I see these advances, I continue to see opportunities for better treatments across the globe.”
Request an appointment at MD Anderson online or call 1-877-632-6789.
How I supported my mom through eye cancer treatment
For as long as I can remember, my mom has been a source of strength. She’s my comforter, advisor, mentor and role model.
But in 2017, the tables turned when a doctor discovered my mom had ocular melanoma, a type of eye cancer. It was in her right eye. My mom would need many months of treatment, with moral support along the way.
Now it was my turn to support her, just as she had always supported me.
Early eye cancer warning sign
The first hint of cancer happened in 2009 in Guatemala, where we lived at the time. While cleaning dishes, my mom accidentally got soap in her eye. When she looked in the mirror while rinsing her eye with water, she noticed a curious dark spot. She visited a doctor in Guatemala, who said it was nothing to worry about.
But the spot began interfering with my mom’s vision, so she visited two more doctors. Each told her it was the equivalent of a freckle, and not to worry. We put the spot out of our minds, and life continued as normal.
Finally, an eye cancer diagnosis: ocular melanoma
In 2011, my family’s dream came true when our green card application was approved and we were granted U.S. permanent residency. My mom, dad, sister and I moved to Texas and settled in a Dallas suburb. My dad got a factory job, my sister and I enrolled in school, and my mom cared for our home and cooked amazing Guatemalan dishes like pollo en pipian – chicken in a creamy pumpkin seed sauce – and Guatemalan tamales wrapped in banana leaves.
All the while, the spot in my mom’s eye had been growing larger and darker. Her vision was becoming more obscured, and she felt as though something were floating in her eye. Unable to tolerate it any longer, she visited an ophthalmologist in Dallas. He biopsied the spot, then delivered the news: my mom had ocular melanoma in her right eye. The doctor explained that most people think of melanoma as an aggressive form of skin cancer, but it can also affect the eyes.
He recommended treatment at MD Anderson.
Overcoming fear about my mom’s ocular melanoma diagnosis
My family was scared and upset. In our culture, we equate cancer with death. I wanted to be strong for my mom, but I could only think about the worst-case scenario.
That changed the minute we walked in the doors of MD Anderson. Everyone was so warm and welcoming. They truly cared about my mom – not just her physical health, but also her mental well-being. They called her by her name and gave her one-on-one personal attention which made her feel special.
I could see my mom’s anxieties begin to disappear. She knew she was in the best of hands, and she would beat this disease.
Combination therapy allowed my mom to keep her eye
From December 2017 to January 2019, my mom went to MD Anderson every three weeks. Her eye cancer specialist prescribed intravenous injections of the immunotherapy drug Keytruda to help her immune system recognize and attack the cancer more effectively. Her doctor also prescribed mitomycin eye drops to deter the growth of cancer cells and reduce scarring. My mom’s melanoma rapidly improved with this combination therapy.
The only other option would have been to remove the entire eye, so we’re very grateful that MD Anderson recommended immunotherapy.
By June 2019, my mom’s tumor had shrunk enough to be surgically removed. Today, she shows no signs of cancer, and her vision is excellent. We return to MD Anderson every six months to check for signs of recurrence. So far, so good.
Looking to the future
I’ve always been fascinated by science, but accompanying my mom during her cancer experience has inspired me to become an oncologist. I’ll graduate from Texas A&M University in two years with a bachelor’s degree in biomedical science. After that, my sights are set on medical school.
My mom’s experience also motivated me to volunteer with myCancerConnection, MD Anderson’s one-on-one cancer support community. The program trains survivor and caregiver volunteers to share their cancer experiences with others who have the same diagnoses, treatments and other similarities. Talking with someone who has gone through the same thing can be so uplifting.
I often think back to where we would be today had my mom not come to MD Anderson. We feel blessed that God led us here, and we’re grateful beyond measure to the MD Anderson health care professionals who have given my mom a future.
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