Mitoxantrone is a drug that a healthcare provider can give in a clinical setting as an intravenous infusion. It is a treatment option for multiple sclerosis.

It is a type of disease-modifying therapy (DMT). DMTs are an emerging class of drug that can help treat multiple sclerosis (MS), some types of cancer, including prostate cancer, and acute nonlymphocytic leukemia.

The Food and Drug Administration (FDA) require mitoxantrone to carry a black box warning about side effects.

These include:

  • a risk of heart damage that can lead to heart failure
  • reduced immunity and a higher risk of infection

The FDA also warn of a higher risk of developing secondary leukemia.

Mitoxantrone is an early form of DMT. For people with advanced-stage MS and severe symptoms, a doctor may prescribe mitoxantrone. However, newer medications are appearing that can replace its use in many cases.

Current guidelines recommend prescribing mitoxantrone for MS only if the benefits outweigh any possible risks.

A person who is using mitoxantrone for MS may wish to speak to their doctor about new options for treatment.

nurse preparing infusion of mitoxantroneShare on Pinterest
nurse preparing infusion of mitoxantrone

Doctors prescribe mitoxantrone to treat some types of cancer.

They may prescribe it:

  • as part of a combination therapy to treat acute, nonlymphocytic leukemia in people aged over 18 years
  • with corticosteroids for the treatment of patients with pain related to advanced hormone-refractory prostate cancer

Mitoxantrone can also reduce problems associated with MS when the MS is:

  • secondary (chronic) progressive
  • progressive relapsing or worsening relapsing-remitting (RRMS)

Mitoxantrone is considered an antineoplastic agent and a multiple sclerosis agent.

It works in different ways to treat MS and acute nonlymphocytic leukemia and prostate cancer.

  • In MS: Immune cells damage the protective covering of nerves. Mitoxantrone limits the ability of these cells to damage the protective nerve covering.
  • In cancer: For acute nonlymphocytic leukemia and prostate cancer, mitoxantrone targets cells that divide quickly. These include cancer cells. Mitoxantrone causes these cells to die.

This drug can have a number of side effects, and some of them can be serious.

Anyone who is going to start using this drug should have a special visit with the clinician, who will discuss with them the risks and benefits of the drug, what they can expect, and what their preferences are.

According to guidelines published in 2018, there is evidence that mitoxantrone can lead to:

  • cardiomyopathy, or diseases of the heart muscle
  • ovarian failure
  • male infertility
  • chromosomal changes
  • promyelocytic leukemia

The guidelines note that clinicians should only prescribe mitoxantrone if the possible benefits greatly outweigh the possible risks. The guidelines add that other drugs are now available now with a lower risk to the individual.

Heart failure

Mitoxantrone may cause heart failure, which can be life-threatening. This can happen during therapy or months to years after therapy.

Lowered immunity

This drug can cause the white blood cell count to go down, indicating reduced immunity. This increases the chance of having an infection.

A doctor will not normally give this drug if a person's neutrophil count is less than 1,500, unless they have acute nonlymphocytic leukemia.

Bleeding problems

The platelet count may also go down, which increases the chance of bleeding.

Discoloration of urine or eyes

Mitoxantrone may turn the urine blue-green for a few days after each dose.

It may also make the whites of the eyes a little blue, but this, too, will go away.

Other common side effects

The most common side effects of mitoxantrone include:

  • nausea
  • hair loss
  • menstrual problems, including a loss of menstrual periods
  • upper respiratory tract infections
  • urinary infections

These are not usually serious, and a doctor can treat them.

If any of these symptoms occur, call a doctor right away.

If the symptoms may be life-threatening or if there may be a medical emergency, call 9-1-1.

Decreased ability to make blood cells

Symptoms may include:

Congestive heart failure

Symptoms may include:

  • trouble breathing
  • swelling of your legs or ankles
  • sudden weight gain
  • uneven or fast heartbeat

Mitoxantrone does not cause drowsiness.

Cancer and secondary cancer

Some research has suggested that there is a higher risk of some types of leukemia when using this drug.

Patients using this medication for MS have developed acute myelogenous leukemia (AML), and those with cancer may have a higher risk of a secondary cancer if they take this drug.

Some people should not use this drug.

These include:

  • those with low immunity, as mitoxantrone can reduce immunity further
  • people with a heart condition, as it can decrease the heart's ability to pump blood
  • anyone who is pregnant or breastfeeding
  • people with MS who have liver problems
  • those with an allergy to the drug

Pregnancy

Mitoxantrone can harm a developing baby, so the doctor may give a pregnancy test before each dose. Studies have linked DMTs with growth problems and a higher risk of preterm birth.

It is important not to become pregnant while taking mitoxantrone. Women should talk to their doctor about birth control before starting mitoxantrone.

Anyone who is pregnant or planning on becoming pregnant should inform their doctor as soon as possible.

Breastfeeding

Mitoxantrone is not suitable during breastfeeding, as it may be passed to the baby through the breast milk. This can be harmful to the baby.

The chemicals can remain in breast milk for up to a month after discontinuing the medication.

Allergy

Allergic reactions have also been reported in some people. Symptoms include low blood pressure, hives and rashes, and difficulty breathing. In rare cases, anaphylaxis may occur.

Who can administer it?

Mitoxantrone can cause serious health problems if a healthcare provider does not administer it correctly. Only a doctor with experience of giving this kind of medication should administer mitoxantrone.

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Some patients will need to receive their drugs as part of their hospital treatment. Mitoxantrone is always given in a healthcare setting.

The doctor will determine a suitable dose, based on the individual's needs. It is important to take this medication exactly as prescribed by the doctor.

How long the treatment takes will depend on the condition.

  • For treating MS and prostate cancer, it usually takes 5 to 15 minutes to infuse.
  • People with acute nonlymphocytic leukemia may need to stay in the hospital for treatment, as the drug is generally given as part of a combination therapy.

A person's general health may affect their dose. It is essential to tell the doctor about any existing health conditions before receiving this drug.

If you miss an appointment for a dose, it is important to call the doctor's office to make another appointment as soon as possible.

How do I know it is working?

This is a long-term treatment, and it may take time for the benefits to appear.

If the drug is working for MS, symptoms will not get worse, and there will be fewer flare-ups.

The doctor will do tests to see how well the drug is working for your cancer treatment.

How do I stop using it?

A person should not stop using this drug without medical advice.

Anyone who is using mitoxantrone as a treatment for stable RRMS should receive counseling from a health professional about the need for ongoing follow-up.

A person with secondary progressive MS should undergo an assessment to see how likely they are to have a relapse in the future.

If mitoxantrone is not working, or if the adverse effects are significant, a doctor may prescribe a different DMT for a person with MS. There are now 17 different drugs available.

The doctor may perform these tests to monitor the individual's progress and health as they take mitoxantrone:

  • complete blood count
  • chest X-ray
  • heart function tests: electrocardiography, echocardiograph, and ejection fraction
  • cholesterol level tests
  • liver function and bile tests
  • pregnancy tests

Travel: Mitoxantrone is only given by a healthcare provider, so anyone who is planning to travel should discuss their travel plans with their provider. In this way, they can arrange for a place to receive mitoxantrone while travelling.

Insurance: Many insurance companies will need prior authorization before they approve the payment for mitoxantrone.