Retinoblastoma grows from the retina, the light-sensing layer at the back of the eye, in babies and children under five. Parents spot it first in most cases, as a white glow in the pupil on a flash photograph or a new squint.
One child in three has it in both eyes.
Those children, and some with a tumor in one eye only, carry a change in the RB1 gene in every cell of the body, which explains the young age, the tumors in both eyes and the need to examine brothers, sisters and future children. Outcomes in countries with full treatment access put survival above 95 percent, and the aim of eye cancer surgery in a child has moved on from saving the life alone to keeping the eye and, wherever the tumor allows, useful sight.
Which treatments keep the eye, and when must it come out?
Treatment moves from the gentlest option to the strongest, and many eyes need two or three of these together.
- Laser (transpupillary thermotherapy) and freezing (cryotherapy) for small tumors.
- Plaque radiotherapy, a small radioactive disc stitched to the outside of the eye for a few days.
- Chemotherapy through a vein, six cycles.
- Intra-arterial chemotherapy, delivered straight into the artery that feeds the eye.
- Chemotherapy injected into the gel of the eye (intravitreal injection) for floating tumor seeds.
- Enucleation, removal of the eye, when the tumor cannot be controlled safely or sight cannot be kept.
Chemoreduction alone or combined with cryotherapy or thermotherapy is effective for treatment of retinoblastoma, but tumor recurrence rate is highest for those located in the macula and those with greater thickness.
Single-center series of 457 tumors, American Journal of Ophthalmology, 2004
Chemotherapy through a vein
Six cycles of vincristine, etoposide and carboplatin, every three to four weeks, shrink tumors in both eyes at once, and laser or freezing then treats what remains. Seven years after treatment in the Philadelphia series of 457 tumors, 45 percent of tumors treated with chemotherapy alone had come back, against 18 percent when local treatment followed, and tumors in the macula, the center of vision, and thick tumors recurred most.
Side effects of intravenous chemotherapy
Low blood counts in the second week of each cycle, a risk of infection that needs antibiotics within the hour if fever appears, hair loss that grows back, and constipation from vincristine. Carboplatin can affect hearing, so children have hearing tests during and after treatment.
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Eye-saving treatments compared
| Treatment |
How it is given |
What the studies showed |
| Intravenous chemotherapy |
Six cycles through a vein, every three to four weeks |
Recurrence at seven years in 18 percent of tumors when laser or freezing followed |
| Intra-arterial chemotherapy |
A catheter from the groin to the eye's artery, three sessions on average, four weeks apart |
Group D eyes kept without radiotherapy in 85.1 percent when used first |
| Intravitreal injection |
A fine needle into the gel of the eye, weekly to monthly |
Eyes kept at two years in 94.2 percent, with a small loss of retinal function per injection |
Intra-arterial chemotherapy
Intra-arterial chemotherapy, also called ophthalmic artery chemosurgery, sends melphalan, with topotecan or carboplatin in many cases, straight into the eye's own artery, so the eye receives a high dose and the rest of the body very little. Memorial Sloan Kettering in New York treated 112 group D eyes this way between 2006 and 2012. Eyes treated first with this method stayed without removal or external radiotherapy in 85.1 percent, and eyes that had failed other treatment elsewhere in 72.4 percent. No child died of spread.
OAC is effective for curing group D retinoblastoma, achieving rates of globe salvage many times higher than systemic chemotherapy (10-47%), even in eyes that previously failed other treatments.
Memorial Sloan Kettering series of 112 eyes, PLoS One, 2016
- General anesthesia, with the child asleep throughout.
- A thin catheter passes from the artery in the groin up to the artery of the eye under X-ray guidance.
- The drug runs in over thirty minutes.
- The catheter comes out, the groin is pressed and the child lies flat for a few hours, going home the same evening or the next morning.
Tumor seeds floating in the gel of the eye escape both forms of chemotherapy, because the gel has no blood supply. A fine needle places melphalan directly among them. In 130 eyes treated with these injections, 94.2 percent were kept at two years.
Intravitreous melphalan is an effective treatment for vitreous seeding in retinoblastoma, resulting in high rates of ocular survival and disease-free survival.
Series of 130 eyes, Ophthalmology, 2017
Effects of intra-arterial treatment and injections
Swelling and redness of the eyelid, loss of eyelashes and a patch of pale skin on the forehead settle within weeks. Low blood counts follow in some children, more when both eyes are treated. Each melphalan injection into the eye lowers retinal function slightly, measured as a fall of 5.3 microvolts on the retinal electrical test, and the team weighs the number of injections against the sight it is trying to keep.
Enucleation, removal of the eye
When removal is the safer choice
The operation takes close to an hour.
Removal of the eye cures most group E eyes and many eyes in one-sided disease that would need months of treatment for little sight. A child with one healthy eye sees, reads, plays and later drives with one eye, and the operation removes the risk of the cancer leaving the eye. Children adapt within weeks. Judging distance for catching a ball takes a little longer, and school needs no special arrangements. Glasses with impact-resistant lenses protect the remaining eye during sport.
How the operation is done
Under general anesthesia, in close to an hour, the surgeon removes the eye whole, without opening it, together with a long section of the optic nerve, and places a round implant in the socket attached to the eye muscles so that the future artificial eye moves. A clear shell sits behind the lids while the socket heals.
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What the pathologist looks for after enucleation
| Finding |
What it means |
What follows |
| Tumor confined to the retina and gel |
Low risk of spread |
Observation with regular checks |
| Tumor in the optic nerve beyond the lamina cribrosa, or 3 millimeters or more into the choroid |
High-risk features |
Chemotherapy for several months to prevent spread |
| Tumor at the cut end of the optic nerve or outside the eye |
Very high risk |
Chemotherapy and radiotherapy to the eye socket |
The artificial eye
An ocularist fits a custom prosthesis four to six weeks after surgery, painted to match the other eye. Children take it out for cleaning a few times a year and get a new one as the face grows.
Removing one eye protects the child
Parents find this the hardest decision in retinoblastoma. A group E eye kept at all costs gives little sight and exposes the child to months of treatment and a real chance of the tumor spreading, while removal ends that risk in one operation and leaves a child who adapts to one-eyed vision quickly.
What the pathologist decides
Pathology decides the rest.
The Children's Oncology Group enrolled 321 children whose eye had been removed for one-sided retinoblastoma, sent every eye for central review, gave chemotherapy to the children whose eyes showed high-risk features and watched the others with examinations and scans, and two years later 96 percent of the treated group and 99 percent of the watched group were free of cancer. Central review disagreed with the local pathologist in 23 percent of children with high-risk features, so the slides travel with the child for a second reading.
Adequate handling and interpretation of histopathology of eyes with retinoblastoma is necessary to assign metastatic risk.
Children's Oncology Group, 321 children, Journal of Clinical Oncology, 2019
Recovery and the flight home
After enucleation children stay one night.
They go home with antibiotic ointment and fly within a week, once the swelling settles. Intra-arterial chemotherapy brings the family back every four weeks for three sessions or more, and each visit needs three to four days for the treatment and an examination under anesthesia. Published packages for pediatric eye cancer treatment in Turkey cover the anesthesia, the procedure, the hospital night and the interpreter, and leave out flights, insurance and further cycles beyond the plan, so the number of sessions, the drugs used in each, extra examinations under anesthesia, chemotherapy after removal and the custom prosthesis decide the final figure. A written estimate, free after the team reads your child's photographs and scans, names the treatment and the sessions it assumes.
Your companion, your language, your food
A parent stays in the child's room every night on the companion bed. The international patients office books the hotel nights around each admission, arranges the airport pickup and daily transport, and sends the visa invitation letter ten days before the flight.
Your coordinator speaks with you in English, Arabic, French, Russian, Serbian, Romanian or Spanish and brings an interpreter for other languages. Meals come halal, vegetarian or diabetic from the hospital kitchen, the hospital has a prayer room, and a request for a female doctor is met wherever the rota allows.
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Eye checks after treatment
| Period |
What happens |
Where |
| First year after eye-saving treatment |
Examination under anesthesia every four to six weeks |
Here or with a pediatric eye oncologist at home |
| Until five years of age |
Examinations spaced to every three to six months |
Local eye oncologist, photos sent to the team here |
| Children with the RB1 change |
Lifelong check for second cancers, and eye checks for brothers and sisters |
Local pediatric oncologist |
