Chelation Therapy with Oral Solution of Deferiprone in Transfusional Iron-Overloaded Children with Hemoglobinopathies

Iron overload in hemoglobinopathies is secondary to blood transfusions, chronic hemolysis, and increased iron absorption and leads to tissue injury requiring the early use of chelating agents. The available agents are parenteral deferoxamine and oral deferiprone and deferasirox. There are limited data on the safety and efficacy of deferiprone at a very young age. The aim of our study was the presentation of data regarding the use of oral solution of deferiprone in 9 children (mean age 6.5, range 2–10) with transfusion dependent hemoglobinopathies (6 beta thalassemia major, 1 thalassemia intermedia, and 2 sickle cell beta thalassemia). The mean duration of treatment was 21.5 months (range 15–31). All children received the oral solution without any problems of compliance. Adverse reactions were temporary abdominal discomfort and diarrhea (1 child), mild neutropenia (1 child) that resolved with no need of discontinuation of treatment, and transient arthralgia (1 child) that resolved spontaneously. The mean ferritin levels were significantly reduced at the end of 12 months (initial 2440 versus final 1420？μg/L, ). This small study shows that oral solution of deferiprone was well tolerated by young children and its use was not associated with major safety concerns. Furthermore, it was effective in decreasing serum ferritin. 1. Introduction Children with transfusion dependent hemoglobinopathies rapidly develop potentially damaging levels of toxic iron overload in many vital organs. Iron removal with chelating agents is required early in life and in some cases after the age of two [1]. Therefore, the safety, efficacy, and compliance regarding the use of iron chelators are of high significance because of the long-term nature of the treatment. Currently available chelators are deferoxamine, which is administered intravenously or subcutaneously, and the deferasirox and deferiprone that are given orally. The oral administration of iron chelation is most welcome by children with hemoglobinopathies who have problems with the discomfort of deferoxamine injections. Furthermore, early and aggressive chronic administration with deferoxamine can affect many organs and especially skeletal maturation and result in growth retardation [2, 3]. But even more important is deferiprone’s superior ability to protect the heart, which has been shown to improve survival in many studies [4, 5]. Limited clinical data on the safety and efficacy of deferiprone on very young age allow us to add our experience [6–8]. The aim of our study was the presentation of data regarding the clinical