How it is administered

Deferasirox is taken by mouth and is available in several forms, including tablets for oral suspension, film-coated tablets, and oral granules.

• Tablets for oral suspension should be dispersed in water, orange juice, or apple juice and taken on an empty stomach at least 30 minutes before food.
• Film-coated tablets and oral granules can be taken on an empty stomach or with a light meal. Tablets can be swallowed whole or, if needed, crushed and mixed with soft food (like applesauce or yogurt) and taken immediately.
• Oral granules are sprinkled on soft food and taken immediately.

Dosing is usually once daily, and the exact dose is based on your body weight and iron levels. Your healthcare provider will determine the best form and dose for you.

How it works

Deferasirox is an iron chelator, which means it binds to excess iron in the body. People with myelodysplastic syndromes (MDS) or other blood disorders often receive frequent blood transfusions, which can lead to iron overload. Too much iron can damage organs like the liver, heart, and endocrine glands.

Deferasirox works by binding to iron (specifically Fe3+) in the bloodstream. Once bound, the iron-deferasirox complex is excreted mainly through the feces. This helps reduce the total amount of iron in the body and prevents complications from iron overload. The medication is selective for iron, but it can also affect levels of other metals like zinc and copper, though the clinical significance of this is not fully known.

By lowering iron levels, deferasirox helps protect organs from iron-induced damage, which is especially important for people with chronic transfusion needs, such as those with MDS.

Who Should take it

Deferasirox is used to treat chronic iron overload caused by frequent blood transfusions in patients 2 years of age and older. It is also approved for treating chronic iron overload in patients 10 years of age and older with non-transfusion-dependent thalassemia (NTDT) syndromes, provided their liver iron concentration is at least 5 mg Fe/g dry weight and their serum ferritin is greater than 300 mcg/L.

For people with myelodysplastic syndromes (MDS), deferasirox can be helpful in managing iron overload due to repeated transfusions. It is especially considered in patients with low or intermediate-1 risk MDS, as studies have shown it can help reduce iron levels and may improve event-free survival. Your doctor will monitor your iron levels and overall health to determine if deferasirox is appropriate for you.

Who should not take it

Deferasirox should not be taken by:

• Patients with an estimated glomerular filtration rate (eGFR) less than 40 mL/min/1.73 m² (significant kidney impairment)
• Patients with poor performance status
• Patients with high-risk myelodysplastic syndromes (MDS), as this group was not studied and is not expected to benefit from chelation therapy
• Patients with advanced malignancies
• Patients with platelet counts less than 50 x 10⁹/L
• Anyone with known hypersensitivity to deferasirox or any of its components

If you have severe liver impairment or are pregnant, discuss the risks and benefits with your doctor. Deferasirox is not recommended for patients under 2 years old for transfusional iron overload or under 10 years old for NTDT syndromes.

Commonly used with

Deferasirox is often used alone to manage iron overload. It is not typically combined with other iron chelation therapies, as the safety and efficacy of such combinations have not been established.

However, people taking deferasirox may also be on other supportive treatments for MDS or their underlying blood disorder, such as growth factors, antibiotics, or other medications as needed for their condition.

Commonly tested with

Deferasirox has been studied in combination with other supportive care measures for patients with MDS and other transfusion-dependent anemias. It has also been evaluated alongside other iron chelators in research settings, but combination therapy is not standard.

In clinical trials, deferasirox is often compared to deferoxamine (another iron chelator) or placebo to assess its effectiveness and safety in reducing iron overload.