Safety and efficacy of early start of iron chelation therapy with deferiprone in young children newly diagnosed with transfusion-dependent thalassemia: a randomized controlled trial

Hematology Center, Children Hospital, Ain Shams University, Egypt. Hematology Center, Children Hospital, Ain Shams University, Egypt. Department of Tanning Materials and Leather Technology National Research Center, Egypt. Hematology Center, Children Hospital, Ain Shams University, Egypt. Oasis International Hospital, Beijing, China. ApoPharma Inc., Toronto, Canada.

American Journal of Hematology. 2017;93((2):):262-268
Iron overload is inevitable in patients who are transfusion dependent. In young children with transfusion-dependent thalassemia (TDT), current practice is to delay the start of iron chelation therapy due to concerns over toxicities, which have been observed when deferoxamine was started too early. However, doing so may increase the risk of iron accumulation that will be manifested as toxicities later in life. This study investigated whether deferiprone, a chelator with a lower affinity for iron than deferoxamine, could postpone transfusional iron overload while maintaining a good safety profile. Recently diagnosed TDT infants (N=64 their age ranged from10-18 (median 12) months, 54.7% males; receiving < or = 6 transfusions; serum ferittin (SF) >400-< 1000 ng/ml were randomized to "early start deferiprone" (.ES-DFP) at a low dose (50 mg/kg/day) or to "delay chelation" (DC), and remained in the study until their serum ferritin (SF) level reached ≥1000 mug/L. 61 patients continued the study Levels of transferrin saturation (TSAT) and labile plasma iron (LPI) were measured as well. By approximately 6 months post-randomization, 100% of the subjects in DC group had achieved SF > 1000 microg/L and TSAT > 70% compared with none in the ES-DFP group. LPI level >0.6 microM was observed in 97% vs. 40% of the DS and ES groups, respectively, (p<0.001). The time to reach SF>1000 microg/L was delayed by 6 months in the ES-DFP group (P<0.001) without escalating DFP dose. No unexpected, serious, or severe adverse events were seen in the ES-DFP group. This article is protected by copyright. All rights reserved.
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Language : English
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