WHAT IS THE DEEP PROJECT FOR?

Continuous check-ups and periodic transfusion of healthy blood make it possible even for patients suffering from serious diseases, such as thalassaemia and other inherited anaemias, to live a normal life. The main effect of continuous transfusion, however, is iron overload, i.e. the accumulation of iron in the body, which is extremely damaging over the course of time.

To reduce this load, the patient has to undergo treatment known as iron chelation. Many years ago, researchers developed deferoxamine (DFO), a drug that has proven very effective in reducing iron overload. DFO has significantly improved patient life expectancy, but also has a serious drawback: its active form does not last long in the body. To administer an appropriate dose, deferoxamine needs to be injected 5 to 7 days a week for 8-12 hours at a time. Even though many thalassaemia patients have become accustomed to the procedure, at such a frequency the treatment is still long, uncomfortable and difficult to carry out, particularly for younger patients. Some patients also experience allergic reactions and intolerance to the drug.

A true treatment revolution occurred with the development of deferiprone and deferasirox, that can both be taken orally, i.e. in the form of syrup and tablets (either swallowed whole or dissolved in water). Both drugs significantly reduce the amount of iron in the body. As well as its main beneficial effect, deferiprone is also particularly effective at reducing the accumulation of iron in the heart.

Despite all its advantages, deferiprone has not yet been extensively studied in paediatric patients, so it has not yet been approved specifically for use in children. The DEEP project (“DEferiprone Evaluation in Paediatrics”) aims to study deferiprone in the paediatric population and to develop a formulation that is particularly suited to children, to be made available in the coming years.