title: Safety profile of a liquid formulation of deferiprone in young children with transfusion-induced iron overload: a 1-year experience.
abstract: Data on the use of deferiprone in young children with iron overload are limited.
  
  
  
  To study the safety profile of a liquid formulation of deferiprone in chelating young children with transfusion-induced iron overload.
  
  
  
  A daily dose of 50-100 mg/kg BW in three divided doses of oral deferiprone was given to young patients who had received at least ten packed red cell transfusions and achieved a serum ferritin level >1000 μg/L during a 12-month period from 2011 to 2012.
  
  
  
  Nine children (four males) diagnosed with various types of thalassaemia (n = 8) and hereditary spherocytosis (n = 1) were enrolled. Their mean (SD) age was 4.5 (1.9) years. The patients received 15-20 ml/kg BW of packed red cell transfusions every 4-8 weeks from a mean (SD) age of 2.1 (1.7) years to maintain a pre-transfusion haematocrit at 27%. A mean (SD) total of packed red cells of 5132 (2725) ml were given within a mean (SD) duration of 2.4 (1.1) years before the study. During the 1-year study period, they received a mean (SD) total of packed red cells of 2194 (680) ml or 138 (50) ml/kg BW with a mean (SD) daily iron load of 0.29 (0.12) mg/kg BW. The pre-treatment geometric mean of serum ferritin of 1863.8 μg/L decreased to 1279.7 μg/L after 1 year of treatment (P = 0.05). All patients tolerated the liquid formulation well and did not experience any gastro-intestinal discomfort, nausea or vomiting.
  
  
  
  The liquid formulation of deferiprone is safe in young children with transfusion-induced iron overload.
authors.affiliation: a Department of Pediatrics, Faculty of Medicine, Ramathibodi Hospital , Mahidol University , Bangkok , Thailand.
authors.forename: Ampaiwan
authors.initials: A
authors.lastname: Chuansumrit
authors.affiliation: a Department of Pediatrics, Faculty of Medicine, Ramathibodi Hospital , Mahidol University , Bangkok , Thailand.
authors.forename: Duantida
authors.initials: D
authors.lastname: Songdej
authors.affiliation: a Department of Pediatrics, Faculty of Medicine, Ramathibodi Hospital , Mahidol University , Bangkok , Thailand.
authors.forename: Nongnuch
authors.initials: N
authors.lastname: Sirachainan
authors.affiliation: b Department of Nursing, Faculty of Medicine, Ramathibodi Hospital , Mahidol University , Bangkok , Thailand.
authors.forename: Pakawan
authors.initials: P
authors.lastname: Wongwerawattanakoon
authors.affiliation: a Department of Pediatrics, Faculty of Medicine, Ramathibodi Hospital , Mahidol University , Bangkok , Thailand.
authors.forename: Praguywan
authors.initials: P
authors.lastname: Kadegasem
authors.affiliation: a Department of Pediatrics, Faculty of Medicine, Ramathibodi Hospital , Mahidol University , Bangkok , Thailand.
authors.forename: Werasak
authors.initials: W
authors.lastname: Sasanakul
mesh_terms: D000743:Anemia, Hemolytic / Q000628:therapy*; D002648:Child; D002675:Child, Preschool; D000077543:Deferiprone; D005260:Female; D005293:Ferritins / Q000097:blood; D006801:Humans; D007502:Iron Chelating Agents / Q000008:administration & dosage* / Q000009:adverse effects; D019190:Iron Overload / Q000188:drug therapy* / Q000209:etiology; D008297:Male; D011728:Pyridones / Q000008:administration & dosage* / Q000009:adverse effects; D065227:Transfusion Reaction
chemical_list: D007502:Iron Chelating Agents; D011728:Pyridones; D000077543:Deferiprone; D005293:Ferritins
keywords: Iron chelation; Iron overload; Liquid deferiprone