Increasing compliance in thalassemia treatment

The leading cause of death in patients with β-thalassaemia — a hereditary form of anaemia also known as Mediterranean anaemia — is heart failure resulting from iron overload. Despite effective iron-chelation treatment with deferoxamine, which has reduced the overall mortality rate, almost 50% of patients still suffer from cardiac iron overload. One of the main reasons for this effect is proposed to be low patient compliance with the demanding injection regimen. The oral iron chelator deferasirox might be preferred by patients and therefore improve compliance.

Researchers conducted a 1-year trial of deferasirox to determine its efficacy in controlling iron overload. The patients were divided into two groups: a reduction arm, in which patients had a high starting level of iron; and a prevention arm, in which patients were at risk but had normal iron levels.

Patients in the reduction arm experienced a 16% decrease in myocardial iron concentrations and maintained the left ventricular-ejection fraction (LVEF). In the prevention arm, medication lead to an improvement in the LVEF while maintaining myocardial iron at normal levels.

The study suggests that a high patient compliance to the medication regimen offers significantly better results. The oral pathway might offer better compliance than older iron chelators that are given as injections. Other oral drugs, such as deferiprone, show better cardiac efficacy than deferoxamine, and current evidence suggests that they might be superior. However, there are no direct studies to confirm this yet. A trial is now underway comparing deferasirox with deferoxamine directly.

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