Currently Enrolling Trials
Ferriprox (deferiprone) is a chelating agent with an affinity for ferric ion (iron III). Deferiprone binds with ferric ions to form neutral complexes that are stable over a wide range of pH values.
Ferriprox is specifically indicated for patients with transfusional iron overload in adult and pediatric patients 8 years of age and older with thalassemia syndromes or with sickle cell disease or other anemias.
Ferriprox is supplied as tablets for oral administration. Ferriprox Tablets are available in two different 1,000 mg formulations, which have different dosing regimens to achieve the same total daily dosage.
To prevent medication errors, before prescribing and dispensing, ensure that the tablet formulation is appropriate for the dosing regimen. Each tablet has distinct identifying characteristics.
Ferriprox Tablets (three times a day):
- Starting oral dosage: 75 mg/kg/day (actual body weight) in three divided doses
- Maximum oral dosage: 99 mg/kg/day (actual body weight) in three divided doses
Ferriprox Tablets (twice a day):
- Starting oral dosage: 75 mg/kg/day (actual body weight) in two divided doses.
- Maximum oral dosage: 99 mg/kg/day (actual body weight) in two divided doses.
Mechanism of Action
Ferriprox is a chelating agent with an affinity for ferric ion (iron III). Deferiprone binds with ferric ions to form neutral complexes that are stable over a wide range of pH values.
Adverse effects associated with the use of Ferriprox in patients with thalassemia may include, but are not limited to, the following:
- abdominal pain
- ALT increased
Adverse effects associated with the use of Ferriprox in patients with sickle cell disease or other anemias may include, but are not limited to, the following:
- abdominal pain
- bone pain
- vomiting, pain in extremity
- sickle cell anemia with crisis
- back pain
- ALT increased
- AST increased
- oropharyngeal pain
- neutrophil count decreased
The Ferriprox drug label comes with the following Black Box Warning: Ferriprox can cause agranulocytosis that can lead to serious infections and death. Neutropenia may precede the development of agranulocytosis. Measure the absolute neutrophil count (ANC) before starting Ferriprox and monitor weekly while on therapy. Interrupt Ferriprox if infection develops and monitor the ANC more frequently. Advise patients taking Ferriprox to report immediately any symptoms indicative of infection.
Clinical Trial Results
Transfusional Iron Overload in Patients with Thalassemia Syndromes:
The FDA approval of Ferriprox was based on 12 clinical studies in 236 subjects who did not respond to prior iron chelation therapy. Ferriprox therapy (35-99 mg/kg/day) was considered successful in individuals who experienced a greater than or equal to 20% decline in serum ferritin within one year of starting therapy. Of the 236 enrolled subjects, 50% reached the endpoint of at least a 20% reduction in serum ferritin.
Transfusional Iron Overload in Patients with Sickle Cell Disease and other Anemia:
A controlled study that compared the efficacy of Ferriprox to that of deferoxamine in patients with SCD and other transfusion-dependent anemias met the non-inferiority criterion for change in liver iron concentration from baseline after 12 months. Data from an extension study confirmed that liver iron concentration continued to decrease progressively over time with the mean value dropping from 14.93 mg/g dw at baseline to 12.30 mg/g dw after one year, to 11.19 mg/g dw after two years, and to 10.45 mg/g dw after three years of Ferriprox treatment.