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.This page explains what melphalan is, how it is given and some of the possible side effects. Each person reacts differently to medicines, so your child will not necessarily suffer from every side effect mentioned.

Melphalan is given intravenously into a vein through a central venous catheter or implantable port.

What are the side effects?

Nausea and vomiting

Anti-sickness medicines can be given to reduce or prevent these symptoms. Please tell your doctor or nurse if your child’s sickness is very bad or continues for more than a few days.

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 (reduced red blood cells), 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 our leaflet. If your child complains of having a sore mouth, please tell your doctor or nurse.


If your child has a sore mouth, they 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 very bad or continues for more than a few days. It is important that your child drinks lots of fluids.

Hair loss

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


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

Temporary effect on 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 (LFTs) will be taken to monitor your child’s liver function will be recorded. Please contact your doctor immediately if your child complains of pain in their right side or the whites of their eyes or their skin develops a yellow tinge.

Warm or tingling feeling

Your child may experience a warm or tingling feeling while they are 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.

Altered 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.

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.


If melphalan leaks into the tissues underneath your child’s skin, they can damage the tissue in this area. This is called extravasation.

  • If given through a cannula and your child complains of stinging and burning around the cannula, please tell your doctor or nurse immediately.
  • If given through a central venous catheter or implantable port and your child complains of pain around their chest or neck, please tell your doctor or nurse immediately.
Compiled by:
The Pharmacy department in collaboration with the Child and Family Information Group.
Last review date:
May 2020