Related Experiment Video
Updated: Mar 16, 2026
Continuous Manual Exchange Transfusion for Patients with Sickle Cell Disease: An Efficient Method to Avoid Iron Overload
Published on: March 14, 2017
Cardiac iron load and function in transfused patients treated with deferasirox (the MILE study)
P Joy Ho1, Lay Tay2, Juliana Teo3
1Institute of Haematology, Royal Prince Alfred Hospital, University of Sydney, Sydney, NSW, Australia.
Insights
Iron chelation therapy with deferasirox improved cardiac iron levels in patients with transfusion-dependent anemias. The treatment effectively reduced liver iron concentration, enhancing heart function over one year.
Area of Science:
- Hematology
- Cardiology
- Pharmacology
Background:
- Iron overload is a significant complication in patients with transfusion-dependent anemias like thalassemia major, sickle cell disease (SCD), and myelodysplastic syndromes (MDS).
- Accumulation of cardiac iron can lead to impaired cardiac function and increased morbidity.
Purpose of the Study:
- To evaluate the efficacy of deferasirox, an oral iron chelator, in reducing cardiac and liver iron burden.
- To assess the impact of deferasirox on cardiac function in patients with transfusion-dependent anemias.
Main Methods:
- A phase IV, open-label, single-arm study involving 46 patients with transfusion-dependent anemias.
- Patients received deferasirox (up to 40 mg/kg/d) for 53 weeks.
- Cardiac and liver iron load were assessed using magnetic resonance imaging (MRI), measuring myocardial T2* and liver iron concentration (LIC).
Main Results:
- Deferasirox significantly improved cardiac iron load (myocardial T2*, P = 0.002) overall.
- Significant improvements were observed in patients with normal and moderate baseline cardiac iron.
- Liver iron concentration (LIC) significantly decreased from a mean of 10.4 to 8.2 mg Fe/g dry tissue (P = 0.024), with notable reductions in patients with higher baseline LIC.
Conclusions:
- Once-daily deferasirox therapy over one year effectively increased myocardial T2* and reduced LIC.
- Deferasirox is effective in managing cardiac iron, particularly in patients with myocardial T2* >10 ms.
- The safety profile of deferasirox was consistent with previous studies.
Objectives:
To assess the effect of iron chelation therapy with deferasirox on cardiac iron and function in patients with transfusion-dependent thalassemia major, sickle cell disease (SCD), and myelodysplastic syndromes (MDS).
Methods:
This phase IV, single-arm, open-label study over 53 wk evaluated the change in cardiac and liver iron load with deferasirox (up to 40 mg/kg/d), measured by magnetic resonance imaging (MRI).
Results:
Cardiac iron load (myocardial T2*) significantly improved (P = 0.002) overall (n = 46; n = 36 thalassemia major, n = 4 SCD, n = 6 MDS). Results were significant for patients with normal and moderate baseline cardiac iron (P = 0.017 and P = 0.015, respectively), but not in the five patients with severe cardiac iron load. Liver iron concentration (LIC) significantly decreased overall [mean LIC 10.4 to 8.2 mg Fe/g dry tissue (dw); P = 0.024], particularly in those with baseline LIC >7 mg Fe/g dw (19.9 to 15.6 mg Fe/g dw; P = 0.002). Furthermore, myocardial T2* significantly increased in patients with LIC <7 mg Fe/g dw, but not in those with a higher LIC. Safety was consistent with previous reports.
Conclusions:
Once-daily deferasirox over 1 yr significantly increased myocardial T2* and reduced LIC. This confirms that single-agent deferasirox is effective in the management of cardiac iron, especially for patients with myocardial T2* >10 ms (Clinicaltrials.gov identifier: NCT00673608).
Related Concept Videos
01:27Disorders of Erythrocytes
Erythrocyte disorders can be broadly categorized into two main types: anemic and polycythemic conditions.
A low oxygen-carrying capacity of the blood due to the loss, lower production, or destruction of erythrocytes is termed anemia. Hemorrhagic anemia, for example, occurs when bleeding from an external wound or internal ulcer reduces erythrocyte counts.
On the other...
01:30Cardiomyopathy II: Dilated Cardiomyopathy
01:26Heart Failure Drugs: Inotropic Agents
01:11Renal Failure: Dose Adjustments
Reduced renal clearance and elimination rate are common outcomes of renal impairment. These alterations lead to a prolonged elimination half-life and an altered apparent volume of distribution for drugs. As a result, dosage adjustments are typically necessary to maintain optimal drug levels in the body.
However, dosage adjustments...
01:26Imbalances in Cardiac Output
CHF can occur due to the failure of either side of the heart. Left-side failure leads to pulmonary congestion—the right side continues to send...
01:29Cardiomyopathy IV: Restrictive Cardiomyopathy