Mitoxantrone for Multiple Sclerosis

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Mitoxantrone for Multiple Sclerosis


Generic NameBrand Name

How It Works

Mitoxantrone is an immunosuppressant, a type of medicine that reduces the activity of the immune system. It has been used to treat leukemia and some advanced prostate cancer. Multiple sclerosis (MS) appears to be a disease in which the immune system attacks the covering of the nerve fibers (myelin sheath) within the brain and spinal cord, so immunosuppressants may slow the progression of the disease. Immunosuppressants other than mitoxantrone, though, have not been shown to significantly reduce the progression of MS or the frequency of relapses.

Mitoxantrone may be injected once every 3 months for up to 3 years. A higher total dose increases the risk of serious heart damage.

Why It Is Used

Mitoxantrone is used to treat several forms of worsening MS, including secondary progressive MS, progressive relapsing MS, and advanced relapsing-remitting MS.

Mitoxantrone is one of only two medicines that have been shown to benefit people who have secondary progressive MS that is relapsing. The interferon beta medicine Betaseron is the other. Interferon beta will likely remain the first choice for treating secondary progressive MS, because it does not carry the risk of heart damage associated with mitoxantrone. Mitoxantrone may be tried if you develop severe side effects from interferon beta, cannot take the medicine for some other reason, or continue to have active disease.

How Well It Works

Mitoxantrone reduces the frequency of relapses and helps slow the development of disability in people who have secondary progressive MS that is relapsing or advanced relapsing-remitting MS. Some studies have shown that when mitoxantrone is combined with other drugs such as intravenous methylprednisolone (IVMP) or interferon beta, the rate of new MS lesions in the brain has been reduced.1

Side Effects

All medicines have side effects. But many people don't feel the side effects, or they are able to deal with them. Ask your pharmacist about the side effects of each medicine you take. Side effects are also listed in the information that comes with your medicine.

Here are some important things to think about:

  • Usually the benefits of the medicine are more important than any minor side effects.
  • Side effects may go away after you take the medicine for a while.
  • If side effects still bother you and you wonder if you should keep taking the medicine, call your doctor. He or she may be able to lower your dose or change your medicine. Do not suddenly quit taking your medicine unless your doctor tells you to.

Because mitoxantrone can lower your number of platelets (cells that help blood clot), you may have problems with bleeding. Make sure you know the signs of abnormal bleeding.

Call 911 or other emergency services right away if you have:

  • Trouble breathing.
  • Swelling of your face, lips, tongue, or throat.

Call your doctor right away if you have:

  • Hives.
  • Shortness of breath.
  • Signs of an infection, such as a sore throat, fever, sneezing, or coughing.
  • Sores in your mouth or on your lips.
  • Stomach pain.

Common side effects of this medicine include:

  • Body aches and pains.
  • Constipation.
  • Hair loss.
  • Headache.
  • Nausea and vomiting.
  • Change in the color of urine or the whites of the eyes. Mitoxantrone may make them turn a blue-green color.

See Drug Reference for a full list of side effects. (Drug Reference is not available in all systems.)

What To Think About

Use of mitoxantrone may cause serious, even fatal, heart damage in some people, including heart failure. It also can cause leukemia. Frequent tests, including a heart scan, are recommended before each injection of this medicine. To reduce the risk of heart damage associated with the medicine, you should receive only a limited number of doses. The U.S. Food and Drug Administration (FDA) recommends up to 12 total doses, or no more than 140 mg/m³ total cumulative dose. Typically, the medicine is injected once every 3 months for 2 to 3 years.

Due to these safety concerns and the limitations on its use, mitoxantrone is for the most part being used only in specialized MS clinics or centers staffed by neurologists and other health professionals who specialize in treating people with MS.

As with interferon beta, people who are taking mitoxantrone need to have regular blood tests to monitor white and red blood cell counts and liver function. These are in addition to the regular testing needed to monitor heart function. Mitoxantrone is not recommended for people who already have heart disease, liver problems, or certain blood disorders, because it may make these problems worse.

Mitoxantrone can keep your immune system from fighting infection. When you are taking this medicine (and even when you have finished taking it), try not to be around people who are sick. And make sure you talk to your doctor before you get any vaccinations.

Taking medicine

Medicine is one of the many tools your doctor has to treat a health problem. If you don't take your medicines properly, you may be putting your health (and perhaps your life) at risk.

There are many reasons why people have trouble taking their medicine. But in most cases, there is something you can do. For suggestions on how to work around common problems, see the topic Taking Medicines as Prescribed.

Advice for women

Women who use this medicine during pregnancy have a higher chance of having a baby with birth defects. If you are pregnant or planning to get pregnant, you and your doctor must weigh the risks of using this medicine against the risks of not treating your condition. If you need to use this medicine, talk to your doctor about how you can prevent pregnancy.


Follow-up care is a key part of your treatment and safety. Be sure to make and go to all appointments, and call your doctor if you are having problems. It's also a good idea to know your test results and keep a list of the medicines you take.

Complete the new medication information form (PDF)(What is a PDF document?) to help you understand this medication.



  1. Stuart WH, Vermersch P (2004). Concomitant therapy for multiple sclerosis. Neurology, 63(Suppl 5): S28–S34.


ByHealthwise Staff
Primary Medical ReviewerAdam Husney, MD - Family Medicine
Primary Medical ReviewerAnne C. Poinier, MD - Internal Medicine
Specialist Medical ReviewerBarrie J. Hurwitz, MD - Neurology
Last RevisedMay 14, 2012

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