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Deferasirox.

Janice L Stumpf¹

¹University of Michigan Health System and College of Pharmacy, Ann Arbor, Michigan, USA.

American Journal of Health-System Pharmacy : AJHP : Official Journal of the American Society of Health-System Pharmacists

|March 14, 2007

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Deferasirox offers an effective oral alternative to deferoxamine for managing chronic iron overload in patients with hematologic disorders. This oral chelator demonstrates non-inferiority to deferoxamine and is preferred by most patients.

Area of Science:

Pharmacology
Hematology
Toxicology

Background:

Iron overload is a common complication of chronic blood transfusions for hematologic disorders.
Current management involves parenteral deferoxamine, but compliance is often poor due to treatment burden.
Deferasirox, an oral iron chelator, was approved in 2005 as an alternative therapy.

Purpose of the Study:

To review the pharmacology, clinical efficacy, adverse effects, toxicities, and economic considerations of deferasirox.
To evaluate deferasirox as an alternative to deferoxamine for managing transfusion-related iron overload.

Main Methods:

Review of studies involving over 700 adult and pediatric patients with transfusion-related iron overload.
Comparison of deferasirox (20 or 30 mg/kg/day) with subcutaneous deferoxamine (>/=35 mg/kg/day, 5 days/week) in patients with high hepatic iron burdens.

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Main Results:

Deferasirox demonstrated non-inferiority to deferoxamine in a key clinical study.
Deferasirox was generally well-tolerated in clinical trials.
Approximately 97% of patients preferred deferasirox over their prior deferoxamine treatment.

Conclusions:

Deferasirox is an effective oral tridentate chelator for treating chronic iron overload from blood transfusions.
It provides a well-tolerated and preferred alternative for patients with transfusion-dependent anemias.
Deferasirox offers a significant advantage for patients unable or unwilling to comply with deferoxamine therapy.