You’ve just been diagnosed with iron overload (also called hemosiderosis) secondary to the numerous red blood cell transfusions you’ve had to receive. You may have known about this risk early in your treatment, but maybe you didn’t. Iron overload can cause numerous complications, so it’s important to have a good treatment plan.
Iron chelators (medications that remove iron from the body) are typically used in people with thalassemia or other forms of anemia that require multiple red blood cell transfusions. People with hereditary hemochromatosis are treated with serial phlebotomy (physical removal of blood).
Iron Chelation Options
Currently, in the United States, there are three different iron chelators available. Each has its own pros and cons. Multiple factors are weighed when deciding which medication to use.
Deferoxamine (brand name Desferal) has been used for more than 40 years in the United States. Deferoxamine can be given as an intravenous (IV) infusion at the hospital, but more commonly people with iron overload take deferoxamine subcutaneously (under the skin) at home. The infusion is given over 8 to 12 hours at night using a small battery-powered pump. Local skin reactions may occur, but they can be treated by reducing the dose or rate of infusion or with hydrocortisone cream.
Most people tolerate deferoxamine well, but because the infusions must be given over long periods of time, many people grow weary of this treatment. Hearing or vision loss can occur at high doses, so while being treated with deferoxamine, you should have regular hearing and vision screening.
The most common iron chelator used in the United States is deferasirox. It is effective at removing iron from the liver and heart, the two organs most likely to have iron overload. Deferasirox comes in two formulations: a dispersible tablet (dissolved in liquid), called Exjade, and a tablet that can be swallowed, called Jadenu.
One of the biggest benefits of deferasirox is that it is an oral medication taken once per day. Unfortunately, there is no liquid form of deferasirox. Although Jadenu comes as an easy-to-swallow tablet, most children with transfusion-dependent anemias will develop iron overload before they are able to swallow pills. In these children, Exjade may be used.
The downside to this formulation is that it simply tastes icky. The tablet is dissolved in liquid, but often it does not fully dissolve and the liquid takes on a chalky texture. The taste can be somewhat improved by putting the tablet in apple or orange juice. Deferasirox granules can also be sprinkled over food.
Deferasirox is also FDA approved for use in people with non-transfusion dependent thalassemia age 10 or older.
Deferiprone (Ferriprox) is the latest iron chelator approved in the United States. Although deferiprone is an oral medication, it is currently taken three times per day. It comes as a pill or a liquid. It appears deferiprone may be the best medication for removing iron from the heart. Although most people tolerate deferiprone without issues, the most concerning potential side effect is agranulocytosis—severely low neutrophil counts that can increase the risk of infections. While on this medication, your neutrophil count should be followed closely with weekly complete blood counts.
Some people with iron overload may require treatment with more than one chelator at once. This may mean taking deferasirox or deferiprone every day with deferoxamine infusions three to four times a week.
If you have questions about whether a particular iron chelator is right for you, discuss it with your healthcare provider. Make sure you take the medication as directed and inform your physician of any concerns you have.