Exjade in MDS patients and toxicity – pro

Iron overload is a cumulative, potentially life-threatening, consequence of frequent blood transfusions. Iron starts to build up in the body after as few as 10 transfusions because the body cannot remove it on its own. Iron chelation is the only effective drug treatment for transfusion-related iron overload. In iron chelation, an agent binds to iron in the body and tissues and helps remove it through the urine and/or feces.

Exjade (deferasirox) is indicated for the treatment of chronic iron overload due to blood transfusions (transfusional hemosiderosis) in patients 2 years of age and older. In these patients, Exjade has been shown to reduce liver iron concentration and serum ferritin levels.

Exjade is administered once-daily as a drink. Exjade was developed specifically to meet the high unmet medical need for iron chelation despite the availability of deferoxamine, the standard iron chelator used around the world. While effective, deferoxamine requires nightly infusions by needle and pump, often lasting eight to 12 hours per night for five to seven nights a week as long as the patient continues to receive blood transfusions or has excess iron within the body. As a result, many patients may have stopped or avoided iron chelation therapy, thus risking the toxic effects of iron overload. Due to the burdensome administration of deferoxamine, compliance with standard chelation therapy is poor. Previous studies of patients with thalassemia have shown that good compliance with deferoxamine improves survival and quality of life.

However, patients with MDS do not tolerate Exjade as well. Exjade is contraindicated in patients with platelet counts less than 50,000. Novartis released the revised prescribing information for Exjade on February 17, stating that the kidney and liver damage or gastrointestinal bleeding could be fatal, especially for elderly patients or those with high-risk MDS.  Patients with underlying liver or kidney conditions or platelet counts less than 50×109/L were also cautioned about the side effects.  The black box warning is the strictest type of warning required by the FDA.

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