Melphalan is a chemotherapy medicine used before bone marrow transplant or high dose therapy with stem cell rescue to help treat certain types of cancer, immunology and metabolic conditions.

How is melphalan given?

It is given intravenously into a vein through a central venous catheteror implantable port.

What are the side effects of melphalan?

Nausea and vomiting

Anti-sickness drugs will be given to reduce or prevent these symptoms. Please tell your doctor or nurse if your child’s sickness is not controlled or persists.

Bone marrow suppression

There will be a temporary reduction in how well your child’s bone marrow works. This means your child may become anaemic, bruise or bleed more easily than usual, and have a higher risk of infection. Your child’s blood counts will be checked regularly to see how the bone marrow is working. Please tell your doctor if your child seems unusually tired, has bruising or bleeding, or any signs of infection, especially a high temperature.

Mouth sores and ulcers

Your child may get painful or bleeding gums, ulcers or a sore mouth. You will be given advice about appropriate mouth care including a copy of the mouth care leaflet. If your child complains of having a sore mouth, please tell your doctor or nurse.


If your child has a sore mouth, he or she will often have a sore tummy too. This can cause pain and bloating as well as diarrhoea. Please tell the doctor or nurse if your child has diarrhoea that is not controlled or persists. It is important that your child drinks lots of fluids.

Hair loss

Your child may lose some or all of his or her hair, including eyebrows and eyelashes. This is temporary and the hair will grow back once the treatment has finished.


Depending on the combination of medicines and the dose that your child is given, his or her fertility may be affected. If you feel you would like more information, please discuss this with your doctor.

Liver function

Melphalan can sometimes cause some changes to your child’s liver function. This should return to normal when the treatment is finished. Blood tests will be taken to monitor your child’s liver function and his or her weight and tummy measurement will be recorded. If you notice the whites of your child’s eyes or his or her skin become yellow, please let your doctor or nurse know immediately.

Warm or tingling feeling

Your child may experience a warm or tingling feeling while he or she is being given melphalan. This disappears once the melphalan has been given.

Allergic reaction

Some children receiving melphalan may have an allergic reaction to the drug. This reaction may be mild to severe. Signs of a mild allergic reaction include skin rashes and itching, high temperature, shivering, redness of the face, a feeling of dizziness or a headache. If you see any of these signs, please report them to a doctor or nurse.

Signs of a severe allergic reaction include any of the above, as well as shortness of breath or chest pain. If your child shows signs of a severe allergic reaction, call a doctor or nurse immediately.

Kidney function

Melphalan may change how well your child’s kidneys work. Kidney function will be monitored with regular blood tests.

Lung problems

Melphalan may cause changes to the lung function. If your child develops a cough, has difficulty breathing or chest pain, please tell your doctor or nurse immediately.

Secondary cancers

There is a very small risk of your child developing a second cancer after many years. If you would like more information, please discuss this with your doctor.

Melphalan and interactions with other medicines

Some medicines can react with melphalan, altering how well it works. Always check with your doctor or pharmacist before giving your child any other medicine, including medicines on prescription from your family doctor (GP), medicines bought from a pharmacy (chemist) or any herbal or complementary medicines.

Compiled by: 
The Pharmacy department in collaboration with the Child and Family Information Group.
Last review date: 
December 2017


Please read this information sheet from GOSH alongside the patient information leaflet (PIL) provided by the manufacturer. If you do not have a copy of the manufacturer’s patient information leaflet please talk to your pharmacist. A few products do not have a marketing authorisation (licence) as a medicine and therefore there is no PIL.

For children in particular, there may be conflicts of information between the manufacturer’s patient information leaflet (PIL) and guidance provided by GOSH and other healthcare providers. For example, some manufacturers may recommend, in the patient information leaflet, that a medicine is not given to children aged under 12 years. In most cases, this is because the manufacturer will recruit adults to clinical trials in the first instance and therefore the initial marketing authorisation (licence) only covers adults and older children.  

For new medicines, the manufacturer then has to recruit children and newborns into trials (unless the medicine is not going to be used in children and newborns) and subsequently amend the PIL with the approved information. Older medicines may have been used effectively for many years in children without problems but the manufacturer has not been required to collect data and amend the licence. This does not mean that it is unsafe for children and young people to be prescribed such a medicine ‘off-licence/off-label’. However, if you are concerned about any conflicts of information, please discuss with your doctor, nurse or pharmacist.