Deferiprone is an oral iron chelating agent used to treat transfusion related, chronic iron overload. Deferiprone has been linked to a low rate of transient serum aminotransferase elevations during therapy and to rare instances of clinically apparent liver injury.


Deferiprone (de fer’ i prone) is an orally available iron chelating agent that binds iron with a high affinity and zinc and copper to a lesser extent. In clinical trials in patients with transfusion related iron overload, deferiprone therapy lowered both circulating and tissue (cardiac, liver) iron levels and maintained reduced iron concentrations in patients previously treated long term with subcutaneous infusions of deferoxamine. Deferiprone was approved for use in Europe in 1994, but was not approved in the United States until 2011. Current indications are restricted to patients with transfusion related iron overload due to thalassemic syndromes. Deferiprone is available in tablets of 500 mg under the brand name Ferriprox. The recommended dose is 25 to 33 mg/kg three times daily. Side effects can include nausea, abdominal pain, arthralgias and neutropenia (6%). Severe adverse events include agranulocytosis (1% to 2%) and infections. Weekly monitoring of white blood cell counts is recommended.


In large clinical trials, elevations in serum aminotransferase levels occurred in 7.5% of patients treated with deferiprone and led to drug discontinuation in ~1%. In many situations, it was unclear whether the ALT elevations were due to deferiprone therapy as opposed to spontaneous worsening of an underlying chronic hepatitis B or C, which is common in patients with transfusion related iron overload. Furthermore, there have been very few reports of clinically apparent liver injury attributed to deferiprone therapy and the clinical features of hepatic injury from deferiprone (latency to onset, pattern of serum enzyme elevations, clinical symptoms and laboratory findings, subsequent course) have not been defined.

Iron overload itself can cause liver injury and result in significant fibrosis and even cirrhosis. By decreasing hepatic iron stores, deferiprone and other iron chelators should improve liver disease and prevent progression of fibrosis. In a controversial open label study of deferiprone therapy for up to 4 years in 19 patients with thalassemia and iron overload, progression of fibrosis was found in 5 of 12 subjects who underwent repeat liver biopsy after an average of 4 years, compared to none of 12 subjects who were separately followed while being treated with deferoxamine. Several subsequent studies, however, failed to show fibrosis progression in subjects with thalassemia and iron overload treated with deferiprone, particularly among those without concurrent hepatitis C.

Likelihood score: E* (unproven but suspected cause of clinically apparent liver injury).

Mechanism of Injury

The mechanism of injury accounting for serum enzyme elevations during deferiprone therapy is not known. Deferiprone is metabolized in the liver largely by glucuronidation to a more water soluble complex with subsequent urinary excretion.

Outcome and Management

Serum aminotransferase elevations above 5 times the upper limit of normal (if confirmed) should lead to dose reduction or temporary cessation. Deferiprone has not been implicated in cases of severe hepatitis, acute liver failure, chronic hepatitis or vanishing bile duct syndrome. Despite this, the product label recommends monitoring of serum ALT levels during treatment and interuption if levels are persistently elevated. There does not appear to be cross reactivity in risk for hepatic injury between deferiprone and other iron chelators including deferoxamine and deferasirox.

Drug Class: Hematological Agents; Chelating Agents, Iron Chelators

Other Drugs in the Subclass, Iron Chelators: Deferasirox, Deferoxamine



Deferiprone – Ferriprox®


Hematological Agents


Product labeling at DailyMed, National Library of Medicine, NIH



References updated: 27 December 2017

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    (Among 73 children with severe thalassemia major treated with deferiprone for at least one year, serum ferritin levels did not change overall, but did decrease in those with initially very high levels; elevations in ALT occurred in 16% which led to stopping therapy in 6 patients).
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    (Systematic review and metaanalysis of 16 publications comparing iron chelating agents found differences in myocardial content, but not "live iron" concentrations with different therapies, and that the combination of deferoxamine and deferiprone had "higher risk" than deferoxamine).
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    (Among 33 children with beta-thalassemia and iron overload being treated with deferiprone or deferasirox who were then placed on both agents, 27% developed elevations of ALT or AST above twice ULN, which were usually transient and self-limited or responded to dose reduction of deferasirox).
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    (Among 297 children with beta-thalassemia observed following initiation of deferiprone therapy for an average of 2.5 years, 32 [10%] developed serum aminotransferase elevations which led to discontinuation in 21 [6.7%]; no mention of clinically apparent liver injury or acute liver failure).
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