Retinoblastoma Treatment

Introduction

Learning that your child has retinoblastoma — a cancer of the retina, the light-sensitive layer at the back of the eye — is one of the hardest moments a family can face. The diagnosis often arrives suddenly, sometimes after a paediatrician notices a white pupil in a photograph or a wandering eye on examination. From that point forward, decisions come quickly: which therapies, in what order, in which eye, and how to protect both your child’s life and their future vision.

This guide is written for parents and families whose child has been diagnosed with retinoblastoma and who are now planning the treatment phase. It explains how modern retinoblastoma treatment works, the different therapies that ocular oncology teams use, what to expect during and after treatment, and the long-term follow-up that is part of caring for a child who has had this cancer. The aim is to help you walk into conversations with your child’s specialist team with a clearer sense of the landscape, so you can ask the questions that matter most to your family.

Retinoblastoma is rare, but the field has changed substantially over the past two decades. With early diagnosis and care in a specialised centre, the great majority of children survive, and many keep one or both eyes — sometimes with useful vision. The treatment journey is demanding, but it is also one in which families are not alone: paediatric oncologists, ocular oncologists, ophthalmic surgeons, anaesthetists, geneticists, ocularists, and child-life and counselling support all play a role.

What Is Retinoblastoma Treatment?

Retinoblastoma treatment is a coordinated set of cancer therapies used to treat a tumour that grows in the retina of a child’s eye. The retina is the layer at the back of the eye that captures light and sends visual signals to the brain. Retinoblastoma develops when immature retinal cells multiply abnormally, forming one or more tumours inside the eye.

Most children diagnosed are under the age of five. The cancer may affect only one eye (unilateral retinoblastoma) or both eyes (bilateral retinoblastoma). Bilateral disease — and a portion of unilateral disease — is caused by a change in a gene called RB1, which can be inherited or arise newly in the child.

Treatment has four broad goals, which doctors balance in this order:

1. Save the child’s life by controlling the cancer and preventing it from spreading beyond the eye.
2. Preserve the eye whenever this can be done safely.
3. Preserve as much useful vision as possible.
4. Prevent recurrence, monitor for late effects, and protect long-term health.

The specific plan depends on whether one or both eyes are affected, the size and location of each tumour, whether there are “seeds” (small clusters of tumour cells floating in the vitreous gel or under the retina), whether the cancer has spread outside the eye, and the child’s overall health. Plans are made by a multidisciplinary team, usually after a detailed examination under anaesthesia and imaging.

How Retinoblastoma Treatment Works

Modern retinoblastoma care uses several types of therapy, often in combination. Some treatments aim to shrink large tumours so that more focused treatments can finish the job. Others destroy small tumours directly. Surgery to remove the eye is reserved for situations where the eye cannot be saved or where keeping it would put the child’s life at risk.

Broadly, treatments fall into four groups:

• Chemotherapy — drugs that kill cancer cells, delivered into a vein, into the artery that supplies the eye, or directly into the eye itself.
• Focal therapies — treatments such as laser and freezing that are applied directly to small tumours during an examination under anaesthesia.
• Radiation — either as a small radioactive plaque placed temporarily on the outside of the eye (brachytherapy), or, much less commonly today, external beam radiotherapy.
• Surgery — removal of the affected eye (enucleation), when the disease is advanced or when other treatments will not be enough.

The order in which these are used is guided by international staging systems. The International Classification of Retinoblastoma (ICRB) divides intraocular tumours into Groups A through E based on size, location, and the presence of seeding. The American Joint Committee on Cancer (AJCC) TNM system is also used. In general terms:

• Groups A and B — smaller tumours, often well controlled with focal therapy alone or with a short course of chemotherapy.
• Groups C and D — larger or seeded tumours, where eye-sparing treatment is generally favoured by ocular oncology teams when feasible, often using systemic or intra-arterial chemotherapy combined with focal therapy and sometimes intravitreal chemotherapy.
• Group E — the most advanced intraocular disease, where enucleation is often the safest option, particularly when there is no useful vision potential or there are features that raise the risk of spread.

If imaging or pathology suggests the cancer has spread outside the eye — into the optic nerve, the orbit (the bony socket), or further — treatment becomes more intensive and includes higher-dose chemotherapy and sometimes radiation.

Who Receives Retinoblastoma Treatment?

Retinoblastoma almost always affects young children. Most diagnoses are made before the age of three, and the great majority before the age of five. Older children and adults are very rarely affected.

Children are referred for treatment after a diagnosis is made, usually through a combination of:

• A detailed eye examination under anaesthesia by an ocular oncologist or paediatric ophthalmologist.
• Imaging of the eye, such as ocular ultrasound and high-resolution fundus photography.
• Magnetic resonance imaging (MRI) of the brain and orbits, to check the optic nerve and look for any extension of the tumour and for a rare associated brain tumour.
• Genetic testing of blood and, where the eye is removed, of the tumour, to identify changes in the RB1 gene.
• Pre-treatment blood tests and a paediatric assessment of overall health.

Children with bilateral retinoblastoma almost always carry an RB1 change in every cell of their body (a “germline” change). About one in seven children with unilateral disease also carries such a change. Genetic counselling helps families understand what this means for the child’s future health, for siblings, and for any future children in the family.

Decisions about which therapies to use are made by a multidisciplinary team in a specialised centre. Treating retinoblastoma well requires experience: the eye is small, the cancer behaves in particular ways, and many of the techniques — especially intra-arterial and intravitreal chemotherapy — need specific expertise and equipment.

Types of Retinoblastoma Treatment

The following sections describe each of the main treatments your child’s team may discuss. In most children, more than one of these is used over the course of care.

Systemic Chemotherapy

Systemic chemotherapy is given through a vein and travels throughout the body. In retinoblastoma, it is often used to shrink tumours so that focal treatments can finish the job — an approach sometimes called chemoreduction. The most common drug combination uses vincristine, etoposide, and carboplatin, usually given in cycles every three to four weeks for several months.

Systemic chemotherapy is also used when there is a risk that the cancer has spread beyond the eye, or after enucleation when pathology shows high-risk features such as significant optic nerve involvement.

Intra-Arterial Chemotherapy

Intra-arterial chemotherapy delivers a chemotherapy drug, most often melphalan, directly into the ophthalmic artery — the small artery that supplies the eye. A specialist passes a very fine catheter from a vessel in the groin up to the base of the brain, under image guidance and general anaesthesia. The drug then reaches the eye in high concentration while the rest of the body is exposed to much less.

This technique has changed retinoblastoma care over the past two decades. For many children with Group C, D, or selected Group E tumours, intra-arterial chemotherapy has made it possible to save eyes that previously would have been removed. It is usually given in two to four sessions, spaced about a month apart, and is often combined with focal therapy.

Intravitreal Chemotherapy

Some retinoblastomas produce small floating clusters of tumour cells in the vitreous gel inside the eye, called vitreous seeds. These seeds are difficult to reach with systemic or intra-arterial chemotherapy because the eye’s blood supply does not deliver drugs efficiently to the vitreous.

Intravitreal chemotherapy — a tiny injection of melphalan or topotecan directly into the eye, given under anaesthesia — can clear vitreous seeds in many children. A careful technique is used to avoid spreading cancer cells outside the eye through the needle track. This treatment is usually given as several injections over a few months.

Laser Therapy (Transpupillary Thermotherapy)

Laser therapy uses heat from a laser beam applied through the pupil to destroy small tumours. It is most effective for small tumours away from the optic nerve and the macula (the central part of the retina responsible for sharp vision). Laser is often used in combination with chemotherapy — the chemotherapy shrinks the tumour first, and the laser then finishes treating what remains.

Cryotherapy

Cryotherapy uses extreme cold to destroy tumours, applied with a small probe placed on the outside of the eye over the tumour’s location. It is well suited to small tumours in the front part of the retina that are harder to reach with a laser. Like laser, cryotherapy is often combined with chemotherapy and is delivered during examinations under anaesthesia.

Plaque Brachytherapy

Plaque brachytherapy is a focused form of radiation. A small disc containing radioactive material (commonly ruthenium-106 or iodine-125) is stitched onto the outside of the eye, directly over a tumour. It stays in place for a few days, delivering radiation to the tumour while sparing most of the rest of the eye and the body. The plaque is then removed in a second short operation.

Brachytherapy is useful for medium-sized tumours that have not responded fully to chemotherapy and for tumours in locations that are difficult to treat with laser or cryotherapy.

External Beam Radiotherapy

External beam radiotherapy — radiation delivered from a machine outside the body — was once a mainstay of retinoblastoma treatment. It is used much less often today because it can affect the growth of the bones around the eye and, in children with a germline RB1 change, increases the risk of second cancers later in life. It is now generally reserved for situations where other treatments are not sufficient, such as orbital disease or recurrent disease where eye-sparing therapy has failed.

Enucleation (Surgical Removal of the Eye)

Enucleation is the surgical removal of the eye. For families, this is often the most difficult treatment to consider, but for advanced disease — particularly Group E tumours with no realistic chance of useful vision, or when the cancer threatens to spread beyond the eye — it remains the safest option and is sometimes the treatment that saves a child’s life.

The operation is done under general anaesthesia. The surgeon removes the eye along with as long a segment of the optic nerve as possible — an important step because retinoblastoma can spread along the optic nerve toward the brain. An orbital implant, usually a small sphere, is placed in the socket to maintain shape and to allow movement of a future prosthesis. The eye muscles are attached to the implant so that, later, the artificial eye will move naturally with the other eye.

After healing, a custom-made ocular prosthesis (a thin, painted shell that sits in front of the implant) is fitted by a specialist called an ocularist. Modern prostheses are highly realistic, and many people who meet the child will not notice that one eye is artificial.

The Treatment Plan and What to Expect

Before Treatment Begins

Once retinoblastoma is suspected, your child will usually undergo an examination under anaesthesia (EUA) by an ocular oncologist. The EUA allows the doctor to see the whole retina in detail, take wide-field photographs, perform ocular ultrasound, and map every tumour. MRI of the brain and orbits is performed to check the optic nerve and to rule out a rare related tumour in the brain called trilateral retinoblastoma.

Blood tests, a paediatric assessment, and genetic counselling are arranged. If chemotherapy is planned, a small central venous catheter (often a port placed under the skin) may be inserted so that drugs and blood samples can be taken without repeated needle pricks.

Parents typically meet several members of the team during this phase — the ocular oncologist, the paediatric oncologist, an anaesthetist, a genetic counsellor, and often a nurse coordinator who will be your main point of contact. This is the right time to ask questions about each proposed treatment, the order in which they will be used, and what the team expects in terms of vision and side effects.

During Treatment

Most retinoblastoma treatments are given in cycles or rounds:

• Systemic chemotherapy is usually given as a short hospital stay every three to four weeks, often for six cycles, with blood counts checked between cycles.
• Intra-arterial chemotherapy is delivered as a day or short-stay procedure under general anaesthesia, repeated every three to four weeks for two to four sessions.
• Intravitreal chemotherapy, laser, and cryotherapy are typically performed during EUAs, every three to four weeks, until tumours are controlled.
• Plaque brachytherapy involves a short admission for placement, a few days of inpatient stay while the plaque is in place, and a second short procedure to remove it.
• Enucleation usually involves a one- to two-night stay in hospital, with prosthesis fitting beginning several weeks later once the socket has healed.

Throughout treatment, the team will continue to perform regular EUAs to monitor how tumours are responding and to deliver focal therapy as needed. These examinations are essential because young children cannot reliably describe what they see, and tumour changes are best assessed under anaesthesia using specialised equipment.

After Active Treatment

Once active treatment ends, children move into a surveillance phase. EUAs continue at regular intervals — initially every few weeks, then gradually spaced further apart over months and years — until the child is old enough to cooperate with awake eye examinations. Hearing tests, kidney function tests, and other checks may be done because some chemotherapy drugs can affect these systems.

For children with a germline RB1 change, lifelong follow-up is important because of the increased risk of other cancers later in life, including bone and soft-tissue sarcomas and melanoma.

Side Effects and How They Are Managed

Side effects depend on which treatments your child receives. Modern care places a strong focus on minimising them.

From Chemotherapy

• Low blood counts. Chemotherapy can lower white blood cells, red blood cells, and platelets, increasing the risk of infection, anaemia, and bleeding. Blood tests between cycles guide treatment timing, and supportive medications and transfusions are used when needed.
• Nausea and reduced appetite. Anti-sickness medicines are routinely given. Small frequent meals, favourite foods, and high-calorie supplements help during cycles.
• Hair loss. Hair usually thins and falls during systemic chemotherapy and grows back after treatment ends.
• Tiredness. Most children have low energy on the days around chemotherapy and rebound between cycles.
• Hearing changes. Carboplatin can occasionally affect hearing, so hearing is monitored.
• Effects on kidneys. Kidney function is checked before and during treatment.
• Local effects from intra-arterial chemotherapy. The eyelids may become swollen or red, eyelashes may thin, and rare effects on the retina’s blood supply can occur. Most local changes resolve over time.
• Local effects from intravitreal chemotherapy. The retina may show some pigment changes around the injection site.

From Focal Therapy

Laser and cryotherapy can cause temporary swelling around the eye and small changes in the retina near the treated area. These are usually mild and improve over days to weeks.

From Radiation

Plaque brachytherapy is well tolerated by most children. Possible later effects include changes in the retina, cataract (clouding of the lens), and dry eye. External beam radiotherapy carries a wider range of late effects, including changes to facial bone growth and an increased risk of second cancers in children with a germline RB1 change, which is why it is used selectively.

From Enucleation

After enucleation, the socket heals over several weeks. Some swelling, bruising, and discharge are normal. Once a prosthesis is fitted, families learn how to clean and care for it. Children adapt remarkably to vision in one eye, and depth perception — while different — generally develops well enough for school, sport, and daily life. Protective glasses are usually recommended to safeguard the remaining eye.

Response and Monitoring

Response to treatment is judged primarily by the appearance of tumours during EUAs. Doctors look for signs that tumours have become smaller, flatter, calcified, or scarred — all features of successful response. They also look for new tumours, because in children with the germline form of the disease, new tumours can develop in untreated areas of the retina until around the age of three to four.

Imaging, blood tests, and clinical assessments complete the picture. If a treatment is not controlling a tumour well enough, the team may change approach — for example, adding intravitreal chemotherapy for persistent seeds, switching from systemic to intra-arterial chemotherapy, or, where eye-sparing treatment is no longer safe, recommending enucleation.

Across all of this, success means different things for different children. For some, success is a clear retina and good vision in both eyes. For others, it is the safe removal of one eye and a healthy child playing as before. The team will share their honest expectations with you at each stage.

Combining With Other Treatments

Most retinoblastoma plans combine therapies. A child with bilateral Group C disease, for example, may receive systemic chemotherapy to shrink the tumours, then focal laser or cryotherapy to treat what remains, then intravitreal chemotherapy if vitreous seeds appear, with regular EUAs to monitor.

A child with advanced Group E disease in one eye may undergo enucleation early, with chemotherapy added afterward if pathology shows high-risk features such as cancer cells in the cut end of the optic nerve, in the choroid (the layer beneath the retina), or in the front of the eye.

When cancer has spread beyond the eye — into the orbit, the lymph nodes, the bone marrow, or the central nervous system — treatment becomes more intensive. This may include high-dose chemotherapy with stem cell rescue (a procedure where the child’s own blood-forming cells are collected, stored, and given back after very strong chemotherapy) and, in selected cases, radiation. These approaches are delivered in specialised paediatric oncology centres.

Living During and After Treatment

Day-to-Day Life During Treatment

Treatment for retinoblastoma is intensive but rarely confines children to hospital. Most chemotherapy is given as short admissions or day visits, and most children spend the majority of their time at home. Between cycles, children often play, eat, and behave much as before, particularly as side effects fade.

Practical points many families find helpful:

• Keep a simple notebook or app with treatment dates, blood counts, medications, and questions to ask the team.
• Watch for fever, unusual bleeding or bruising, or rapid changes in behaviour during chemotherapy cycles — these should be reported to the team promptly.
• Maintain routine vaccinations as advised by the oncology team; some vaccines are timed around treatment.
• Protect the remaining or treated eye from injury — ask the team about protective eyewear for play and sport.
• Use sunglasses and a hat outdoors, as some treatments can make eyes more sensitive to light.

School and Development

Many children continue at nursery or school during treatment, with adjustments for hospital visits and on days when blood counts are low. As they grow, vision needs become more apparent. Some children have full vision in both eyes; some have full vision in one eye after the other has been treated or removed; some have reduced vision in one or both eyes because tumours involved the macula or optic nerve.

Specialised input from low-vision services, paediatric ophthalmology, and educators can help children make the most of their vision. Early intervention — with high-contrast materials, larger print, magnifiers, or other adaptations — often makes a meaningful difference in learning.

Emotional Wellbeing

A cancer diagnosis is hard on the whole family. Young children often cope better than parents expect, but they sense parental stress and benefit from honest, age-appropriate explanations. Older siblings may feel anxious or left out and benefit from being included in conversations and given their own time.

Many specialised centres offer child-life and counselling services. Parent support groups — including dedicated retinoblastoma communities — can be a source of practical advice and shared experience. There is no “right” way to feel during this time, and asking for emotional support is part of looking after your child.

Long-Term Follow-Up

Follow-up for retinoblastoma is long-term and structured. It typically includes:

• Eye examinations under anaesthesia, gradually replaced by awake examinations as the child grows.
• MRI scans at intervals for children with the germline form, particularly in the first few years, to monitor the brain.
• Hearing and kidney monitoring during and after chemotherapy.
• Prosthesis adjustments and replacements as the child grows, where an eye has been removed.
• Vision support and updates as the child develops.
• Counselling around the genetic findings, future family planning, and screening of siblings and any future children.

For children who carry a germline RB1 change, lifelong awareness of the risk of second cancers is important. This is balanced with allowing the child to live as normally as possible. Adult oncology and dermatology follow-up are usually arranged as the child grows.

Genetic Counselling and the Family

Genetic counselling helps the family understand whether the child’s retinoblastoma is sporadic (limited to that child) or germline (present in every cell of the body). Blood testing for RB1 is offered to the affected child, and, where appropriate, to parents and siblings. Where a germline change is found, paediatric eye examinations of siblings — and screening of any future children — are part of the family’s care plan. These decisions are personal and unfold over time, and the genetic counselling team supports families through them.

Frequently Asked Questions

What is the long-term outlook for a child with retinoblastoma?

When retinoblastoma is diagnosed early and treated in a specialised centre, survival is very high in high-income settings. Outcomes depend on whether the disease is limited to the eye or has spread, on the size and number of tumours, and on response to treatment. Your child’s team can give you a personalised estimate based on their specific stage, group, and imaging findings.

Can my child keep the affected eye?

Eye preservation has improved substantially with intra-arterial and intravitreal chemotherapy alongside focal therapy. Whether the eye can be saved depends mainly on the stage at diagnosis, the location of tumours, and the response to treatment. For some children — particularly with Group E disease — enucleation remains the safest option, but for many children with earlier-stage disease, the eye can be saved.

Will my child be able to see?

Vision depends on where the tumours are and how the retina responds to treatment. A tumour involving or near the macula or optic nerve will affect central vision more than one in the periphery. Some children retain excellent vision; others have reduced vision in the treated eye but full vision in the other. Children with bilateral disease may have differing levels of vision in each eye. The ocular oncology team will explain what to expect for your child specifically.

Is retinoblastoma hereditary?

Around 40% of children with retinoblastoma carry a germline change in the RB1 gene that is present in every cell of their body. This can be inherited from a parent or, more commonly, occur newly in the child. Children with bilateral disease almost always carry such a change. Children with unilateral disease carry it less often, but genetic testing is generally offered to all children with retinoblastoma to clarify the risk and guide family screening.

If my child has a germline RB1 change, what does that mean for siblings and future children?

It means that siblings should have specialist eye examinations to make sure they are not affected, and that any future children may need similar screening from birth. Genetic counsellors guide families through these decisions and the practical steps involved.

How will my young child cope with so many examinations under anaesthesia?

Repeated anaesthesia is a real concern for families. Paediatric anaesthesia for short EUAs is generally very safe in experienced centres, and children typically tolerate it well. The team minimises the number of anaesthetics by planning examinations and treatments together where possible. Discuss specific concerns — including the cumulative effect of anaesthesia on a young child — with the anaesthetist and oncologist.

What can my child do during treatment? Are there activities to avoid?

Most children continue with play, family life, and often nursery or school, with breaks around chemotherapy or anaesthesia. Activities to be careful about include those with a risk of eye injury, swimming during periods of low immunity, and contact with people who have infectious illnesses. Your team will give specific advice based on your child’s treatment phase.

What happens if a tumour comes back?

Recurrence is more common in the first year or two after treatment and is one of the reasons EUAs continue at intervals. If a recurrence is found, the team reassesses the eye and either intensifies focal therapy, adds intravitreal or intra-arterial chemotherapy, or, where the eye cannot be safely preserved, recommends enucleation. Recurrence does not necessarily mean a worse overall outlook, particularly when it is caught early.

What about second cancers later in life?

Children with a germline RB1 change have an increased risk of certain cancers later in life, including bone and soft-tissue sarcomas and melanoma. This risk is higher in those who received external beam radiation, which is part of the reason this treatment is now used so selectively. Lifelong follow-up, sun protection, and prompt attention to new lumps or pain are part of long-term care.

Conclusion

Retinoblastoma treatment has changed enormously over the past two decades. Where this cancer was once a leading cause of childhood death from eye disease, it is now one of the most successfully treated paediatric cancers when diagnosed early and managed in a specialised centre. Eye-sparing approaches — intra-arterial chemotherapy, intravitreal chemotherapy, laser, cryotherapy, and plaque brachytherapy — have made it possible to save eyes that previously would have been removed. When enucleation is needed, modern surgical and prosthetic techniques mean children grow up confident and active.

For families, the path through treatment is demanding. There will be examinations under anaesthesia, chemotherapy cycles, difficult decisions, and long-term follow-up. There will also be milestones — the first scan showing tumour shrinkage, the day chemotherapy ends, the prosthesis fitting that goes smoothly, the first day back at nursery, the year-on-year clear examinations. A specialised ocular oncology team, with paediatric oncology, genetics, ophthalmic surgery, and family support working together, walks with you through each of these.

The most important thing a family can take from this guide is that retinoblastoma is highly treatable, that the choices ahead are real but rarely final, and that you do not need to make them alone. Bring your questions to your child’s team, ask for time when you need it, and let the people caring for your child help you decide what is right for your family.