General Updates | The importance of chelation therapy in thalassemia and iron chelators available for patients

We discussed a lot during ASCAT the asked the future, the alternative treatments or approaches, alternative to transfusion. But we have to keep in mind, patients should keep in mind that the standard of care with transfusion and iron chelation remain the main cure for thalassemia. We will have other possibilities, but to reach that point, we need to have patients well treated with the standard of care. So if we are treating them with a good transfusion regimen, we must have a proper chelation therapy, because with transfusion, iron accumulates. Each unit of blood contains 200mg of iron. That iron goes in the liver, in the heart, in the endocrine glands. So absolutely we need to remove it.

And at present we have three main iron chelators. One is the historical one, let’s say, which is deferoxamine. That has been the only iron chelator available for almost 40 years. A good chelator, it’s a big molecule. To be efficacious, it should be given subcutaneously with a pump for at least six-eight hours a day every day. So it’s a very, very demanding treatment. That’s the reason why back in the 90s, several companies, but especially research groups, were looking for alternative chelators, particularly and possibly oral instead of subcutaneous. It was very difficult because, you know, a chelator usually has a huge structure. It’s a big molecule and it is difficult to have a molecule which can be taken orally. But eventually we have now two oral iron chelators, one is deferiprone and one is deferasirox.

To make the long story short, deferasirox is the compound that underwent extensive clinical trials, and in 2005 was approved by the FDA and in 2006 by EMA for treating iron overload in transfused patients. Deferiprone had a more, let’s say, complicated registration pathway, because it was actually around since the 80s, but unfortunately didn’t undergo proper clinical trials, so it wasn’t approved by the FDA, and the FDA approved it only recently, and EMA approved with some restrictions mainly related to side effect of neutropenia and agranulocytosis.

So just to conclude, to my knowledge, all over the world now, I should say 80% of the patients are taking deferasirox, another 10%, maybe a bit more, may take deferiprone alone. And there is a percentage of patients, who are characterized by very, very severe iron overload due to poor compliance in the past or whatever, and they can be treated actually with a combination. A combination which can be deferoxamine plus deferiprone or deferoxamine plus deferasirox or, there isn’t a real trial on that, but there are also reported cases using deferiprone and deferasirox.

So we have quite a good availability of iron chelators. It is extremely important that iron chelation should be maintained properly. I’ll just mention there is a molecule, which is in development under the clinical trial is still under evaluation. It is a molecule which can be given twice a week, but I cannot present any data on that. So this is the scenario for iron chelation.

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