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A to Z of MS Mitoxantrone

Product names

Novantrone (USA)

Mitoxantrone is a chemotherapy drug that is sometimes used in MS as a disease modifying therapy (DMT) to reduce the number of relapses a person is experiencing. Although it is not licensed for MS in the UK, it is sometimes used in some specialist centres. However, in recent years its use has decreased due to the introduction of newer therapies such as natalizumab (Tysabri), fingolimod (Gilenya) and alemtuzumab (Lemtrada). The risk of severe adverse events when using mitoxantrone, especially therapy-related acute leukaemia (TRAL), are also greater than previously thought.

How is mitoxantrone given?

Mitoxantrone is given by intravenous infusion in hospital. The actual dose given varies depending on the patient's weight. As mitoxantrone is not licensed for use in MS in the UK, there are no hard-and-fast prescribing guidelines, use of the drug is entirely at the discretion of the neurologist.

How mitoxantrone works

Mitoxantrone appears to work by suppressing the body's immune system for the period of treatment, depleting the number of cells that are attacking the myelin around nerves. This effectively gives the body a chance to 'restart' and sort out what has gone wrong with the immune system.

As mitoxantrone suppresses the immune system, the white blood cell count is likely to fall, making the recipient more prone to infections. Therefore, regular blood tests are carried out for the duration of treatment.

Side effects and contraindications

Many people experience some, or all, of these common temporary side effects:

  • changes in menstruation pattern and/or temporary cessation of periods, in some women this can be permanent
  • nausea
  • temporary hair thinning (in common with other anti-cancer drugs)
  • blue-green urine for 24 hours after infusion.

Mitoxantrone can also have some long-term permanent side effects:

  • cardiotoxicity - mitoxantrone may damage the heart, and can cause congestive heart disease if taken over a long period of time. To reduce this risk, the total amount any one person may take in their lifetime is limited to between 8-12 doses taken over two to three years
  • therapy-related acute leukaemia (TRAL) - treatment with mitoxantrone can cause some people to develop leukaemia and cases are increasingly being reported. The overall risk of developing TRAL is currently estimated at 1 in 137 people, or 0.73%. This compares with a risk of 1 in 33,333, or 0.003%, of developing acute myeloblastic leukaemia in the general population
  • liver damage – mitoxantrone can cause changes to liver enzyme levels and harm the liver, so blood tests are carried out to monitor liver function during treatment
  • pregnancy and breastfeeding - mitoxantrone may cause birth defects if either parent is receiving it when a baby is conceived, so effective contraception should be used whilst on treatment and for up to six months after treatment stops. Mitoxantrone may be passed on through breast milk, so should not be taken whilst a woman is breastfeeding.


Ellis R, et al.
Therapy-related acute leukaemia with mitoxantrone: four years on, what is the risk and can it be limited?
Multiple Sclerosis Journal 2014; Jul 10 [Epub ahead of print].

Cocco E, Marrosu MG.
The current role of mitoxantrone in the treatment of multiple sclerosis.
Expert Reviews in Neurotherapeutics 2014; 14(6): 607-16.

Morrissey SP, et al.
Mitoxantrone in the treatment of multiple sclerosis.
The International MS Journal 2005; 12: 74-87.

Edan G, et al.
Rationale for the use of mitoxantrone in multiple sclerosis.
Journal of the Neurological Sciences 2004; 223: 35-9.

Read more about mitoxantrone
Patient Information Leaflets (EMC website)
Find out more


Disease modifying therapies

Drugs used in the treatment of MS

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