DEFERASIROX

Clinical safety rating: caution

Animal studies have proved adverse effects but may be safe for humans

How it works

Deferasirox is an oral iron chelator that binds trivalent iron (Fe3+) with high affinity, forming a stable complex that is excreted primarily in the feces. It reduces iron burden in transfusion-dependent patients.

What the body does with it

Metabolism	Primarily glucuronidation via UGT1A1 and UGT1A3; minor oxidation by CYP450 enzymes (CYP1A2, CYP2D6, and CYP3A4).
Excretion	Primarily fecal (84%) as unchanged drug via biliary excretion; renal excretion accounts for approximately 8% as unchanged drug and metabolites.
Half-life	Terminal elimination half-life is 8–16 hours in patients with transfusional iron overload; higher iron burden prolongs half-life due to enterohepatic recirculation.
Protein binding	99% bound to serum albumin; negligible binding to other proteins.
Volume of Distribution	Apparent Vd is 14–18 L/kg, indicating extensive extravascular distribution and tissue binding, particularly in iron-loaded organs.
Bioavailability	Oral bioavailability is approximately 70% (range 60–80%) under fed conditions; absorption is dose-proportional up to 40 mg/kg.
Onset of Action	Oral: Serum ferritin reduction observed within 2–4 weeks of continuous therapy.
Duration of Action	Duration of iron chelation persists for approximately 24 hours post-dose; urinary iron excretion peaks at 4–8 hours and declines over 24 hours.

Classification & Brands

Dosing & administration

Initial: 20 mg/kg orally once daily. Maintenance: 20-40 mg/kg orally once daily (maximum 40 mg/kg/day).

Dosage form	TABLET, FOR SUSPENSION
Renal impairment	GFR 40-60 mL/min: No adjustment. GFR 20-39 mL/min: Reduce dose by 50%. GFR <20 mL/min: Contraindicated.
Liver impairment	Child-Pugh A: No adjustment. Child-Pugh B: Reduce dose by 50%. Child-Pugh C: Contraindicated.
Pediatric use	Age ≥2 years: 20 mg/kg orally once daily. Adjust based on serum ferritin; maximum 40 mg/kg/day.
Geriatric use	Start at low end of dosing range; monitor renal function and serum ferritin closely.

Use during pregnancy

1st trimester	Consult provider
2nd trimester	Consult provider
3rd trimester	Consult provider

Clinical note

Avoid concurrent administration with aluminum-containing antacids Can cause renal impairment and hepatic failure monitor function regularly.

Breastfeeding	No data on excretion in human milk; M/P ratio unknown. Theoretical risk of infant iron deficiency. Avoid breastfeeding or discontinue drug based on importance to mother.
Teratogenic Risk	Deferasirox is contraindicated in pregnancy (FDA Category C). Animal studies show fetal malformations and mortality at clinically relevant doses. In humans, no adequate controlled studies exist; risk cannot be excluded. First trimester: potential for teratogenicity based on animal data. Second and third trimesters: risk of fetal iron deficiency and anemia due to iron chelation.

Warnings & precautions

■ FDA Black Box Warning

Risk of acute kidney injury, including renal tubular necrosis, and renal failure, which may be fatal. Monitor renal function before and during therapy. Dose reduction or interruption may be needed.

Side Effect Profile

Common Effects	Nausea
Serious Effects

Absolute Contraindications

["Severe renal impairment (CrCl < 40 mL/min) or acute kidney injury","History of hypersensitivity to deferasirox or any component","Myelodysplastic syndrome with high-risk features or advanced malignancies"]

Clinical Precautions

Precautions

["Renal toxicity: acute kidney injury, Fanconi syndrome, and renal tubular acidosis","Hepatic toxicity: elevated liver enzymes and hepatitis","Gastrointestinal hemorrhage","Cytopenias: agranulocytosis, neutropenia, and thrombocytopenia","Skin rashes and hypersensitivity reactions","Auditory and ocular disturbances"]

Clinical Tips & Counseling

Drug interactions

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