From the Guidelines
The primary drug used to lower ferritin levels is deferasirox, given orally at 14-28 mg/kg once daily, as it has been shown to produce statistically significant decreases in liver iron concentrations and is a practical option for long-term treatment. The choice of iron chelator depends on the patient's specific condition, iron overload severity, and ability to adhere to the treatment regimen 1. Deferoxamine (Desferal) is typically administered as a subcutaneous infusion at 20-40 mg/kg/day over 8-24 hours, 5-7 days per week, while deferiprone (Ferriprox) is taken orally at 75-100 mg/kg/day divided into three doses.
Key Considerations
- Treatment duration depends on the severity of iron overload and is often long-term for conditions like hemochromatosis or thalassemia.
- Regular monitoring of ferritin levels, kidney function, liver function, and hearing/vision is essential during treatment.
- Side effects vary by medication but can include gastrointestinal issues, kidney or liver problems, and hearing or vision changes.
- The goal of treatment is to decrease ferritin levels to less than 1000 ng/mL, particularly for patients with low or INT-1 risk disease who have received or are anticipated to receive greater than 20 RBC transfusions 1.
Monitoring and Assessment
- Serum ferritin is a practical measure of body iron and is readily available, but its use is limited by the influence of infection, acute and chronic inflammation, and alcohol abuse 1.
- Transferrin saturation, liver biopsy, Magnetic Resonance Imaging (MRI), and Subsequent Quantum Interference Device (SQUID) are other methods to confirm increased iron deposits, but have their own limitations and are not always readily available.
Recent Developments
- A recent NCCN task force report presents the available evidence regarding iron chelation in patients with MDS, highlighting the importance of monitoring and treatment in these patients 1.
- A black box warning has been added to deferasirox due to reports of acute renal failure, hepatic failure, and cytopenias, emphasizing the need for close monitoring of patients on deferasirox therapy 1.
From the FDA Drug Label
Deferasirox tablets for oral suspension doses of 20 to 30 mg per kg per day. Deferasirox tablets for oral suspension doses below 20 mg per kg per day failed to provide consistent lowering of LIC and serum ferritin levels
- The drug to lower Ferritin is deferasirox.
- The recommended starting dose is 20 mg per kg per day. 2
From the Research
Iron Chelation Therapy
The goal of iron chelation therapy is to reduce iron overload in the body. Several drugs are used for this purpose, including:
- Desferrioxamine: a parenteral iron chelator that is considered the gold standard of iron chelation 3
- Deferiprone: an orally active iron chelator that has the advantage of removing toxic iron from the myocardium, but can cause agranulocytosis 3
- Deferasirox: an orally active iron chelator that is well tolerated, but has limited cardiac effects 3
Comparison of Iron Chelators
Studies have compared the efficacy of different iron chelators in reducing iron overload and improving clinical outcomes. For example:
- A study found that desferrioxamine, deferiprone, and deferasirox all reduced serum ferritin and serum iron levels in patients with beta thalassemia major, but there were no significant differences between the three groups 4
- Another study found that deferiprone was more effective than desferrioxamine in removing cardiac iron and improving left ventricular ejection fraction in patients with thalassemia major 5
- A study also found that controlling iron burden through phlebotomy improved outcomes in patients with peripheral arterial disease, particularly in younger patients 6
Treatment Options
Based on the available evidence, the choice of iron chelator depends on various factors, including:
- The severity of iron overload
- The presence of cardiac iron deposition
- The patient's age and overall health
- The potential side effects of each drug
- The patient's ability to comply with the treatment regimen
Some possible treatment options include: