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Trade/other name(s): L-Phenylalanine Mustard, L-PAM, Alkeran, L-Sarcolysin

Why would this drug be used?

Melphalan is used to treat multiple myeloma, as well as ovarian, breast, and prostate cancer. It is also used for other cancers and sometimes for non-cancerous conditions.

How does this drug work?

Melphalan is a member of the general group of chemotherapy drugs known as alkylating agents. It works by interfering with and stopping the growth of cancer cells, which causes them to die.

Before taking this medicine

Tell your doctor…

  • If you are allergic to anything, including medicines, dyes, additives, or foods.
  • If you have any medical conditions such as kidney disease, liver disease (including hepatitis), heart disease, congestive heart failure, diabetes, gout, or infections. These conditions may require that your medicine dose, regimen, or timing be changed.
  • If you are pregnant, trying to get pregnant, or if there is any chance of pregnancy. This drug might cause birth defects if either the man or woman is taking it at the time of conception or during pregnancy. Check with your doctor about what kinds of birth control can be used with this medicine.
  • If you think you might want to have children in the future. This drug has been reported to cause temporary or permanent sterility in some people. Talk with your doctor about the possible risk with this drug and the options that may preserve your ability to have children.
  • If you are breast-feeding. The drug may pass into breast milk and affect the baby.
  • About any other prescription or over-the-counter medicines you are taking, including vitamins and herbs. In fact, keeping a written list of each of these medicines (including the doses of each and when you take them) with you in case of emergency may help prevent complications if you get sick.

Interactions with other drugs

People who took cyclosporine (a drug to help prevent rejection of organ transplants) during or after taking melphalan had a higher risk of kidney failure. Cisplatin (a cancer treatment drug) may affect the body's processing of melphalan. The cancer drug BCNU is more likely to cause damage to lungs if IV melphalan is used at the same time. When nalidixic acid and IV melphalan are given to children at the same time, severe bleeding and intestinal damage may occur.

Any drugs or supplements that interfere with blood clotting can raise the risk of bleeding during treatment with melphalan. These include:

  • vitamin E
  • non-steroidal anti-inflammatory drugs (see above examples)
  • warfarin (Coumadin)
  • ticlopidine (Ticlid)
  • clopidogrel (Plavix)

Note that many cold, flu, fever, and headache remedies contain aspirin or ibuprofen. Ask your pharmacist if you aren't sure what's in the medicines you take.

Check with your doctor, nurse, or pharmacist about other medicines, vitamins, herbs, and supplements, and whether alcohol can cause problems with this medicine.

Interactions with foods

The pill form of this medicine must be taken on an empty stomach, at least 1 hour before or 2 hours after eating. Check with your pharmacist, doctor, or nurse for more information.

Tell all the doctors, dentists, nurses, and pharmacists you visit that you are taking this drug.

How is this drug taken or given?

Melphalan comes in pill form and is taken by mouth or it can be given in high doses as an injection in a vein. You should take the pill on an empty stomach, usually once a day for 5 days, every 6 weeks. Melphalan is known to cause nausea and vomiting, so you will likely need anti-nausea medicine before taking it. The dose depends on your size, why you are taking it, and what regimen is used.

Store the medicine in a tightly closed container away from heat and moisture and out of the reach of children and pets.


Melphalan can cause allergic reactions in some people, most often while it is being given into the vein. Symptoms can include feeling lightheaded or dizzy (due to low blood pressure), fever or chills, hives, nausea, itching, headache, coughing, shortness of breath, or swelling of the face, tongue, or throat. Tell your doctor or nurse right away if you notice any of these symptoms as you are being given the drug.

You may have nausea and vomiting on the day you receive this drug, especially if you get it in the vein, or in the first few days afterward. Your doctor may give you medicine before your treatment to help prevent nausea and vomiting. You will likely also get a prescription for an anti-nausea medicine that you can take at home. It is important to have these medicines on hand and to take them as prescribed by your doctor.

Your doctor will likely test your blood throughout your treatment, looking for possible effects of the drug on blood counts (described below) or on other body organs. Based on the test results, you may be given medicines to help treat any effects. Your doctor may also need to reduce or delay your next dose of this drug, or even stop it altogether.

This drug can lower your white blood cell count, especially in the weeks after the drug is given. Having a low white blood cell count increases your chance of getting an infection. Be sure to let your doctor or nurse know right away if you have any signs of infection, such as fever (100.5° or higher), chills, pain when passing urine, a new cough, or bringing up sputum.

Melphalan may lower your platelet count in the weeks after it is given, which can increase your risk of bleeding. Speak with your doctor before taking any drugs or supplements that might affect your body's ability to stop bleeding, such as aspirin or aspirin-containing medicines, warfarin (Coumadin), or vitamin E. Tell your doctor right away if you have unusual bruising, or bleeding such as nosebleeds, bleeding gums when you brush your teeth, or black, tarry stools.

Melphalan may lower your red blood cell count in the weeks after it is given. A low red blood cell count (known as anemia) can cause trouble getting your breath, or make you to feel weak or tired all the time. Your doctor may give you medicines to help prevent or treat this condition, or you may need to get blood transfusions.

This drug is often given into the vein (IV). If the drug leaks out of the vein and under the skin, it may damage the tissue, causing pain, ulceration, and scarring. Tell the doctor or nurse right away if you notice redness, pain, swelling or other symptoms near the IV.

Do not get any immunizations (vaccinations), either during or after treatment with this drug, without your doctor's OK. Melphalan may affect your immune system. This could make vaccinations ineffective, or even lead to serious infections if you get a live virus vaccine during or soon after treatment. Try to avoid contact with people who have recently received a live virus vaccine, such as the oral polio vaccine or smallpox vaccine.

This drug can rarely cause scarring of the lungs (fibrosis), a serious problem. Tell your doctor right away if you have cough, trouble breathing, shortness of breath, or chest pain.

Women of childbearing age should avoid pregnancy while on this drug, due to its risk of causing birth defects.

Call your doctor if you have skin rash, bleeding, fever, weight loss, fatigue (tiredness), aches and pains, unusual lumps on your body, or other new problems.

Because of the way this drug acts on cells in the body, it may increase your long-term risk of getting a second type of cancer, such as leukemia. This is generally rare, but risk goes up with higher doses of melphalan. If cancer does occur it would likely be years after the drug is used. If you are getting this drug, your doctor feels this risk is outweighed by the risk of what might happen if you do not get this drug. You may want to discuss these risks with your doctor.

Possible side effects

You will probably not have most of the following side effects, but if you have any talk to your doctor or nurse. They can help you understand the side effects and cope with them.


  • nausea (at higher doses)*
  • vomiting (at higher doses)*
  • low white blood cell count with increased risk of infection*
  • low platelet count with increased risk of bleeding*
  • anemia (low red blood cell count) with symptoms like tiredness, paleness, or trouble catching your breath*

Less common

  • short-term or long-term infertility (inability to have children)
  • weakness


  • severe allergic reaction*
  • loss of appetite
  • scarring (fibrosis) or inflammation of lungs*
  • hair loss, including face and body hair
  • rash
  • itching
  • second type of cancer (may happen years after treatment)*
  • death from lung damage or other causes

*See "Precautions" section for more detailed information.

There are some other side effects not listed above that can also occur in some patients. Tell your doctor or nurse if you develop these or any other problems.

FDA approval

Yes – pill form first approved before 1984 (FDA cannot verify dates of drugs approved before 1984; IV form first approved 1992.)

Disclaimer: This information does not cover all possible uses, actions, precautions, side effects, or interactions. It is not intended as medical advice, and should not be relied upon as a substitute for talking with your doctor, who is familiar with your medical needs.

Last Medical Review: 09/18/2009
Last Revised: 09/18/2009