Generic name: melphalan (injection) [ MEL-fa-lan ]
Other brand names of melphalan (injection) include: Evomela, Hepzato
Drug class: Alkylating agents

Medically reviewed by Drugs.com on Jun 30, 2025. Written by Cerner Multum.

What is melphalan?

Melphalan is used as a "palliative" treatment to relieve symptoms of multiple myeloma (a type of blood cancer).

Melphalan injection is also used as a "conditioning" treatment in people with multiple myeloma before receiving a stem cell transplant.

Melphalan injection is also used in the treatment of uveal melanoma (a type of eye cancer) in adults.

Melphalan for intra-arterial is available only from a certified pharmacy under a special program.

Melphalan may also be used for purposes not listed in this medication guide.

Melphalan side effects

Get emergency medical help if you have signs of an allergic reaction: hives, difficult breathing, swelling of your face, lips, tongue, or throat.

Hepzato may cause serious side effects. Call your doctor at once if you have:

• low white blood cell counts--fever, mouth sores, skin sores, sore throat, cough;

• low red blood cells (anemia)--pale skin, tiredness, feeling light-headed or short of breath, cold hands and feet;

• easy bruising, unusual bleeding, purple or red spots under your skin;

• sores or white patches in or around your mouth, trouble swallowing or talking, dry mouth, bad breath, altered sense of taste;

• fast heart rate, low blood pressure;

• severe ongoing nausea, vomiting, or diarrhea;

• new or worsening cough, fever, trouble breathing;

• unusual lumps;

• missed menstrual periods;

• inflammation of your blood vessels--numbness or tingling, skin rash, fever, headache, body aches, night sweats, weight loss, feeling or weak or tired;

• liver problems--loss of appetite, nausea, vomiting, stomach pain (upper right side), tiredness, itching, dark urine, clay-colored stools, jaundice (yellowing of the skin or eyes); or

• low blood potassium--leg cramps, constipation, irregular heartbeats, fluttering in your chest, increased thirst or urination, numbness or tingling, muscle weakness or limp feeling.

Common side effects of Hepzato may include:

• diarrhea, nausea, vomiting;

• muscle pain, fatigue;

• mouth sores;

• tiredness;

• abnormal lab results;

• low potassium levels; or

• anemia.

This is not a complete list of side effects and others may occur. Call your doctor for medical advice about side effects. You may report side effects to FDA at 1-800-FDA-1088.

Related/similar drugs

Warnings

While using melphalan, you may get serious or fatal infections or bleed more easily. Call your doctor if you have unusual bleeding, signs of infection (fever, cough, skin sores, painful urination), have flu symptoms, cough, night sweats, neck stiffness, confusion, or vision problems.

Using melphalan may increase your risk of developing other cancers. Ask your doctor about this risk.

Before taking this medicine

You should not use melphalan if you are allergic to it, or if you received this medicine in the past and it did not work.

Tell your doctor if you have or have ever had:

• nausea, vomiting, or diarrhea;

• an infection or weak immune system (caused by disease or by using certain medicine);

• chemotherapy or radiation; or

• liver or kidney disease.

Using melphalan may increase your risk of developing other cancers. Ask your doctor about this risk.

You may need to have a negative pregnancy test before starting this treatment.

Melphalan can harm an unborn baby if the mother or the father is using this medicine.

• If you are a woman, you may need a pregnancy test to make sure you are not pregnant. Use birth control while using this medicine and for at least 6 months after your last dose.

• If you are a man, use birth control if your sex partner is able to get pregnant. Keep using birth control for at least 3 months after your last dose.

• Tell your doctor right away if a pregnancy occurs.

Melphalan may affect fertility in men or women. Pregnancy could be harder to achieve while either parent is using this medicine.

Do not breastfeed while using this medicine, and for at least 1 week after your last dose.

How is melphalan given?

Follow all directions on your prescription label and read all medication guides or instruction sheets.

Melphalan injection is given as an infusion into a vein. A healthcare provider will give you this injection.

Some side effects may occur during the injection. Tell your medical caregiver if you feel dizzy, nauseated, light-headed, itchy, sweaty, or have a headache, chest tightness, back pain, trouble breathing, or swelling in your face.

You may be given other medications to help prevent serious side effects or allergic reaction. Keep taking these medicines for as long as your doctor has prescribed.

You may need frequent medical tests and your cancer treatments may be delayed based on the results.

You may get serious or fatal infections or bleed more easily. Your doctor will need to examine you on a regular basis.

It may take several months before your body responds to melphalan. Your doctor will determine how long to treat you with this medicine. For best results, keep using melphalan as directed. You may not get the full benefit of this medicine if you stop using it too soon.

Store melphalan in the refrigerator, do not freeze. Protect from light.

What happens if I miss a dose?

In a medical setting you are not likely to miss a dose.

What happens if I overdose?

In a medical setting an overdose would be treated quickly.

Overdose symptoms may include extreme weakness, vision problems, severe mouth sores, increased salivation or sweating, cough with mucus, shortness of breath, severe nausea and vomiting, diarrhea, bloody or tarry stools, severe stomach pain, loss of muscle movement, or seizure.

What should I avoid while using melphalan?

Avoid receiving a "live" vaccine. The vaccine may not work as well while you are using melphalan. Live vaccines include measles, mumps, rubella (MMR), rotavirus, typhoid, yellow fever, varicella (chickenpox), zoster (shingles), and nasal flu (influenza) vaccine.

Avoid being near people who are sick or have infections. Tell your doctor at once if you develop signs of infection.

Avoid activities that may increase your risk of bleeding or injury. Use extra care while shaving or brushing your teeth.

What other drugs will affect melphalan?

Other drugs may affect melphalan, including prescription and over-the-counter medicines, vitamins, and herbal products. Tell your doctor about all other medicines you use.

Further information

Remember, keep this and all other medicines out of the reach of children, never share your medicines with others, and use this medication only for the indication prescribed.

Always consult your healthcare provider to ensure the information displayed on this page applies to your personal circumstances.

Medical Disclaimer

Copyright 1996-2025 Cerner Multum, Inc. Version: 9.01.