Melphalan (Intravitreal) is a powerful medicine used as an injection into the eye to treat certain serious eye cancers like retinoblastoma and vitreoretinal lymphoma.
| Drug class | Alkylating Agent (Intravitreal) |
|---|---|
| Type | Single |
| Forms | Intravitreal Injection |
| Common strength | Varies |
| Prescription status | Prescription (hospital use) |
What Melphalan (Intravitreal) is used for
Melphalan (Intravitreal) is a specialised medicine used to treat specific eye conditions, primarily certain types of eye cancers. It is administered directly into the vitreous, which is the clear, jelly-like substance that fills the space between the lens and the retina of the eye.
- Vitreous seeds in retinoblastoma: Retinoblastoma is a rare type of eye cancer that begins in the retina, the light-sensitive tissue at the back of the eye. Sometimes, small clusters of cancer cells, called ‘vitreous seeds’, can break away from the main tumour and float in the vitreous. Melphalan (Intravitreal) is used to target and treat these seeds.
- Selected vitreoretinal lymphoma: This is a rare type of lymphoma (a cancer of the immune system cells) that affects the vitreous and retina of the eye. Melphalan (Intravitreal) may be used in selected cases to manage this condition.
How it works
Melphalan (Intravitreal) belongs to a category of medicines called immunomodulators, specifically an alkylating agent. When delivered directly into the vitreous of the eye, it works by interfering with the DNA of fast-growing cancer cells. This action prevents the cancer cells from multiplying and eventually leads to their destruction. By targeting the cancer cells within the eye, it helps to control the disease locally.
How it’s used
Melphalan (Intravitreal) is administered as an intravitreal injection, meaning it is injected directly into the vitreous of your eye. This procedure is always performed by a specialist doctor in a hospital or clinic setting. It is never for self-use.
For retinoblastoma, the typical dosing involves weekly injections during the initial treatment phase, known as the induction period. The exact strength of the medicine and the duration of treatment will vary depending on your specific condition, the severity of the disease, and your doctor’s assessment. Your doctor will determine the most appropriate treatment plan for you.
During the injection, your eye will be numbed with local anaesthetic to minimise discomfort. You may be asked to look in a certain direction, and the doctor will carefully inject the medicine into your eye. After the injection, you will be monitored for a short period to ensure there are no immediate complications.
Always follow your ophthalmologist’s exact instructions.
Brands in India
Hospital-prepared or not marketed as a retail brand in India.
Please note
Powerful medicine that suppresses the immune system. Screening for tuberculosis and hepatitis is usually required before starting, and it raises the risk of infection. Used only under specialist supervision with regular monitoring. This is given only by specialists in a hospital or clinic setting and is never for self-use.
Precautions & side effects
Melphalan (Intravitreal) is a powerful medicine and its use requires extreme caution and specialist supervision. Here are some important precautions:
- Highly Specialist Use: This medicine is used only by highly specialised ocular oncologists (eye cancer specialists) in a hospital or clinic setting. It is not a general eye medicine.
- Immune System Suppression: Melphalan is a powerful medicine that suppresses the immune system. This means your body’s ability to fight off infections may be reduced, increasing your risk of getting sick.
- Screening for Infections: Before starting treatment, you will usually need to undergo screening tests for tuberculosis and hepatitis. This is because suppressing the immune system can reactivate these infections if they are present but dormant in your body.
- Risk of Infection: Due to immune suppression, there is an increased risk of developing infections during and after treatment. It is crucial to report any signs of infection (like fever, chills, unusual tiredness, or new pain) to your doctor immediately.
- Regular Monitoring: You will require regular and close monitoring by your specialist doctor throughout the treatment period to assess the effectiveness of the medicine and to manage any potential side effects.
- Never for Self-Use: This medicine is always administered by a healthcare professional and is never to be used by patients themselves.
- Pregnancy and Breastfeeding: If you are pregnant, planning to become pregnant, or breastfeeding, it is crucial to discuss this with your doctor before starting treatment, as this medicine can have serious effects.
Understanding Retinoblastoma and Vitreoretinal Lymphoma
Melphalan (Intravitreal) is used to treat serious eye conditions like retinoblastoma and vitreoretinal lymphoma. Retinoblastoma is a rare cancer that starts in the retina, the light-sensitive tissue at the back of the eye. It primarily affects young children. When cancer cells break away from the main tumour and float in the clear jelly (vitreous) inside the eye, they are called ‘vitreous seeds’. These seeds need to be treated to prevent the cancer from spreading. Vitreoretinal lymphoma, on the other hand, is a very rare type of cancer that affects the immune cells within the eye, specifically in the vitreous and retina. Both conditions are very serious and require highly specialised care from doctors who are experts in eye cancers. Early diagnosis and treatment are crucial for the best possible outcomes. While these conditions can be frightening, modern medicine offers various treatment options, and your doctors at Pristine Eye Hospitals are dedicated to providing the best possible care.
General Eye Care and Hygiene
Maintaining good eye hygiene is always important, especially when undergoing treatment for eye conditions. Always wash your hands thoroughly with soap and water before touching your eyes or any eye medication. Avoid rubbing your eyes, as this can introduce germs and potentially irritate them. If you experience any unusual symptoms like redness, pain, discharge, or changes in vision, it’s important to inform your doctor immediately. Keep your eyes clean by gently wiping away any discharge with a clean, damp cloth, always wiping from the inner corner of your eye towards the outer corner. If you wear contact lenses, your doctor will advise you whether you should continue using them during treatment, as it’s often recommended to avoid them to prevent irritation or infection. Always follow your doctor’s specific instructions regarding eye care during your treatment.
When to Seek Medical Help
While Melphalan (Intravitreal) is administered under strict medical supervision, it’s vital for you to be aware of when to seek immediate medical attention. Because this medicine can suppress your immune system, you need to be vigilant for signs of infection. If you develop a fever (a temperature above 100.4°F or 38°C), chills, unusual tiredness, unexplained pain, or any new or worsening symptoms, contact your doctor or Pristine Eye Hospitals immediately. Also, if you experience any severe eye pain, sudden vision changes, excessive redness, swelling around the eye, or discharge from the eye after your injection, do not hesitate to seek urgent medical advice. Your medical team will provide you with specific contact numbers and instructions for emergencies. Always remember that your health and safety are our top priority, and prompt communication with your healthcare provider is key to managing your treatment effectively.
Frequently asked questions
What is Melphalan (Intravitreal) used for?
Melphalan (Intravitreal) is used to treat specific eye cancers. It is primarily used for vitreous seeds in retinoblastoma (a type of eye cancer in children) and selected cases of vitreoretinal lymphoma (a rare cancer affecting the eye's vitreous and retina).
How does Melphalan (Intravitreal) work?
Melphalan (Intravitreal) is an alkylating agent delivered directly into the vitreous of the eye. It works by interfering with the DNA of fast-growing cancer cells, preventing them from multiplying and leading to their destruction, thereby helping to control the cancer locally within the eye.
What are the common Indian brands for Melphalan (Intravitreal)?
Melphalan (Intravitreal) is typically prepared by hospitals for specific use and is not available under retail brand names in India. It is referred to as 'Hospital-prepared'.
How is Melphalan (Intravitreal) administered?
Melphalan (Intravitreal) is given as an injection directly into the vitreous (the clear jelly inside the eye). This procedure is always performed by a specialist doctor in a hospital or clinic setting and is never for self-use. For retinoblastoma, it is typically given weekly during the initial treatment phase.
Is a prescription needed for Melphalan (Intravitreal)?
Yes, Melphalan (Intravitreal) is a prescription medicine and is used only in a hospital setting under the direct supervision of specialist doctors.
What are the key precautions to take with Melphalan (Intravitreal)?
Melphalan (Intravitreal) is a powerful medicine that suppresses the immune system. Key precautions include requiring screening for tuberculosis and hepatitis before starting treatment, as it raises the risk of infection. It is used only under specialist supervision with regular monitoring and is never for self-use. You should report any signs of infection to your doctor immediately.
References
- Eye health – MedlinePlus (US National Library of Medicine)
- Eye health information – American Academy of Ophthalmology
- Learn about eye health – National Eye Institute (NIH)
- Blindness and vision impairment – World Health Organization
- StatPearls – NCBI Bookshelf
Medically reviewed by Dr. C Jagadesh Reddy, Chief Surgeon: Cornea, Cataract and Refractive Surgery — last reviewed 2026-07-28.
This page is for general education only and is not a substitute for professional medical advice, diagnosis or treatment. Never self-medicate or reuse an old prescription. Always consult a qualified ophthalmologist before starting, changing or stopping any eye medicine.