11 August 2020
Promising combined therapy for iron-overloaded transfusion patients
Published online 20 May 2015
A combination drug may be helpful to prevent cardiac failure in patients dependent on regular blood transfusions.
Chronic anaemia sufferers require frequent blood transfusions, which can result in dangerously high levels of iron in the body, sometimes causing cardiac failure.
An international team of scientists, including researchers from Egypt and Turkey, conducted a two-year study to see how effective a combination of two iron-binding drugs is in treating patients with very high iron levels in heart muscle and liver tissue1.
Deferasirox (DFX) and deferoxamine (DFO) are two drugs often used separately to treat transfusion-dependent thalassemia patients with myocardial iron overload. However, neither has been effective in treating people who also have severe liver iron levels.
The researchers gave a combination of DFX and DFO to chronic anaemia patients with high iron tissue overload until myocardial iron levels fell as indicated by MRI scan data. This was followed by continuing medication with DFX alone. Approximately one-third of the patients who remained on treatment at the end of the two-year study showed clinically meaningful improvements in myocardial iron levels, along with rapid decreases in liver iron concentration, with no increase in adverse effects compared to monotherapies.
But Yesim Aydinok, the lead author and a paediatrician at Ege University in Izmir, Turkey, warns that combination DFX-DFO therapy may be cumbersome, since DFO is often given by prolonged subcutaneous infusion, which takes time. “Combination therapy should therefore be limited to as short a duration as possible followed by monotherapy with deferasirox, which may be sufficient to control iron overload once patients have reached a lower-risk status for heart failure.”
Aydinok, Y. et al. Effects of deferasirox-deferoxamine on myocardial and liver iron in patients with severe transfusional iron overload. Blood http://dx.doi.org/10.1182/blood-2014-07-586677 (2015).