Mitoxantrone hydrochloride was the first FDA-approved medication for both secondary-progressive multiple sclerosis (MS) and progressive-relapsing MS. It’s also used to treat relapsing-remitting MS that’s getting worse. It was initially (and still is) a chemotherapy drug prescribed for certain types of cancer.
This treatment is a disease-modifying drug (DMD) that can help increase time between symptom relapses. But mitoxantrone has many side effects and raises your risk for certain complications. So doctors don't typically choose it as treatment unless your MS is severe.
How Does Mitoxantrone Work?
When you have MS, your immune system attacks normal brain and spinal nerves and their coverings (myelin sheaths.) This damage causes the symptoms of MS.
Mitoxantrone curbs your immune system. Specifically, it lowers the number of T cells, B cells, and macrophages in your blood. This helps ease inflammation in your brain and spinal cord.
The drug helps control your MS but it can’t cure it. You should continue to take it even if you aren't having any symptoms.
How Do You Take Mitoxantrone?
Mitoxantrone is a liquid you get through a vein with an IV. A doctor or nurse gives it to you.
You typically get one dose every 3 months. The infusion takes about 5-15 minutes to go into your bloodstream. You continue treatment for about 2 to 3 years for a total of 8 to 12 doses. Because it is such a toxic drug, you can only take a certain amount of it in your lifetime.
Before you get an infusion, your doctor will want to know what other medications you take. They’ll also want to know about any vitamins or herbal supplements you take since they can also interact with the medication.
You might take mitoxantrone at the same time as other MS medications. Your doctor will tell you when to take each medication so they work best.
Who Is Mitoxantrone Best For?
Your doctor will likely only choose this treatment if your MS is getting worse and other drugs haven’t helped. The FDA hasn’t approved it for people with the primary progressive form of MS. You may take it if you have:
- Secondary progressive MS
- Progressive-relapsing MS
- Relapsing-remitting MS
You should not take mitoxantrone if you’re pregnant or breastfeeding. If you get pregnant while on the drug, your doctor will tell you to stop the treatment.
What Are the Side Effects of Mitoxantrone?
Mitoxantrone is a powerful drug. Possible side effects include:
- Changes in your period
- Nausea, vomiting, and diarrhea
- Hair loss
The temporary decrease of white blood cells in your blood can lead to infections because it’s harder for your body to fight off germs. Because of this risk, you shouldn’t get live vaccines while taking the drug.
Mitoxantrone is dark blue, so it can sometimes turn the whites of your eyes blue. Your pee might also have a blue-green color for about 24 hours after the infusion.
Call 911 or go to the nearest emergency room if you have these symptoms:
- Black stools
- Bladder pain
- Bloody or cloudy urine
- Cough or shortness of breath
- Difficult, burning, or painful urination
- Irregular heartbeat
- Frequent urge to urinate
- Lower back or side pain
- Pale skin
- Rapid heart rate (or slower heart rate)
- Stomach pain
- Swollen mouth
- Tiredness, weakness
- Trouble breathing when you exert yourself
- Ulcers, sores, or white spots in the mouth
- Unexplained bleeding or bruising
Mitoxantrone can have long-lasting effects in some people, even months and years after you take it. These complications can include:
- Heart damage that can lead to congestive heart failure
- Therapy-related leukemia
- Liver damage
Your doctor will likely test your liver regularly to see how well it’s tolerating the drug. They’ll also check how well your heart is working, specifically how well it pumps blood, with an echocardiogram. This helps monitor your heart function over time on the drug.