Mitoxantrone for multiple sclerosis

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Mitoxantrone for multiple sclerosis


Generic 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 nerves (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 not officially approved for MS treatment in Canada, but it is used in both Canada and the United States for treatment of several forms of advancing 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

Use of mitoxantrone may cause serious, even fatal, heart damage in some people. It also can cause bone marrow problems. 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. Typically, the medicine is injected once every 3 months for 2 to 3 years.

Common but less serious side effects of mitoxantrone include:

  • Nausea.
  • Hair loss.
  • Increased risk of infections, especially urinary tract infections.
  • Changes in a woman's menstrual cycle.
  • Mouth sores.
  • Diarrhea or constipation.

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

What To Think About

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

Mitoxantrone is not officially approved in Canada for MS treatment. But some clinics and neurologists are using the medicine for relapsing-remitting MS that is rapidly getting worse or when other medicines for MS have not worked.

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 should not be used during pregnancy. Women taking mitoxantrone should use a reliable form of birth control if there is any chance they could become pregnant. If you become pregnant while you are taking this medicine or are thinking about trying to become pregnant, contact your doctor immediately.

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.


By Healthwise Staff
Primary Medical Reviewer Anne C. Poinier, MD - Internal Medicine
Primary Medical Reviewer Adam Husney, MD, MD - Family Medicine
Specialist Medical Reviewer Colin Chalk, MD, CM, FRCPC - Neurology
Last Revised April 15, 2010

This information does not replace the advice of a doctor. Healthwise, Incorporated disclaims any warranty or liability for your use of this information.