Deferasirox is a once-daily, oral iron chelator used for the treatment of chronic iron overload due to blood transfusions (transfusional hemosiderosis) in patients 2 years and older, and for the treatment of non-transfusion-dependent thalassemia (NTDT) syndromes with a liver iron concentration (LIC) of at least 5 mg Fe/g dw and a serum ferritin >300 mcg/L. It selectively binds iron with high affinity in a 2:1 ratio, forming a stable complex excreted primarily in feces.
Adult: Initial dose: 20 mg/kg body weight orally once daily. Dose is titrated every 3-6 months based on serum ferritin trends, therapeutic goals, and safety. Maintenance: 20-40 mg/kg/day. Max recommended: 40 mg/kg/day.
Note: Take on an empty stomach, at least 30 minutes before food, preferably at the same time each day. Tablet must be dispersed in a glass of water, orange juice, or apple juice (100-200 mL). Stir to form a fine suspension and drink immediately. Do not chew, swallow whole, or take with antacids containing aluminum. The suspension can also be administered via nasogastric tube.
Deferasirox is a tridentate oral chelator with high selectivity and affinity for ferric iron (Fe3+). It binds iron in a 2:1 ratio (drug:iron). The formed complex is neutral, lipophilic, and stable, preventing iron from participating in harmful redox reactions that generate reactive oxygen species (ROS). This complex is then excreted via the bile into the feces.
Pregnancy: Category D (US FDA). There is positive evidence of human fetal risk. Use only if the potential benefit justifies the potential risk to the fetus. Iron chelation is generally avoided during pregnancy unless severe maternal iron overload is life-threatening.
Driving: May cause dizziness and visual disturbances. Patients should be cautioned about operating machinery or driving until they know how the medication affects them.
| Aluminum-containing Antacids | Decreased absorption of deferasirox. Separate administration by at least 4 hours. | Moderate |
| Cholestyramine | May decrease deferasirox absorption. | Moderate |
| Potent UGT inducers (e.g., Rifampicin, Phenytoin, Phenobarbital, Ritonavir) | May decrease deferasirox plasma concentration, reducing efficacy. | Major |
| Drugs metabolized by CYP2C8 (e.g., Repaglinide, Paclitaxel) | Deferasirox may increase their plasma concentration, risk of toxicity. | Moderate |
| Drugs metabolized by CYP3A4 (e.g., Simvastatin, Midazolam) | Deferasirox may decrease their plasma concentration. | Moderate |
| CYP1A2 substrates (e.g., Theophylline, Tizanidine) | Deferasirox may increase their plasma concentration. | Moderate |
| NSAIDs, Corticosteroids, Bisphosphonates | Increased risk of GI irritation/ulceration. | Moderate |
| Other Nephrotoxic drugs (e.g., Aminoglycosides, Amphotericin B, Cyclosporine) | Increased risk of renal impairment. | Major |