I spent some time at the Singapore Conference talking with Gerry from Apo Pharma, makers of the oral iron chelator, Ferriprox (deferiprone, L1). I approached Gerry about an issue that confronts many users of deferiprone, neutropenia (a drop in neutrophils, a type of white blood cell) and how it should be managed. Most doctors will take a patient off deferiprone as soon as neutropenia is present. I asked if this is the correct strategy and was told that it is not. What has been found is that when a patient develops mild neutropenia, ferriprox does not need to be stopped, but instead patients should be closely monitored to see if there is any further drop. Most patients will see a return to normal white cell counts without further intervention in less than two weeks. Defriprone is almost always blamed when a patient experiences a drop in white cells and deferiprone can lead to this, but it is not the only cause and ceasing deferiprone may not be the answer. Even if patients do stop deferiprone while experiencing neutropenia, in most cases they can resume the drug after white cells return to normal. In the more serious Agranulocytosis, which affects about 1% of Ferriprox users, the drug should be stopped and it is risky to resume, as there is a 50% recurrence rate of Agranulocytosis. This is extremely important information concerning neutropenia and patients need to be made aware that a bout of neutropenia does NOT disqualify them from further use of this excellent chelation drug.
Gerry also talked to me about the new oral solution that Apo Pharma has developed and that is now available in some countries and coming to more in the future. Ferriprox oral solution is a liquid form of deferiprone and should be taken 3 times daily. It is suitable for children and has fewer adverse reactions than with use of the tablets, in terms of nausea, stomach pain and vomiting. The reduction in nausea was significant, dropping from 16% with the tablets to 1% with the liquid solution. This is exciting news for patients who have been unable to use Ferriprox because of stomach issues and may be of special significance in a country like Maldives where stomach problems have been a major reason for stopping Ferriprox. Incidence of neutropenia were similar to that with the tabs (6%). Agranulocytosis was found in 2 % compared to 1 % with tabs but this could be due to the relatively low number of patients in the study. The 1% difference may be more of a statistical difference than what will eventually be seen in real use of the solution.
Ferriprox oral solution brings one more chelation choice into the world of thalassemia and is a welcome option. I think this will be of special benefit to children whose stomachs could not tolerate the tablet form of Ferriprox and thus will provide cardiac protection that has not been equaled by the other chelators to patients. This is indeed good news, as patients need as many chelation choices as possible as they search for the drug that works best for themselves.