title: Experience with the oral iron chelator deferiprone in transfusion-dependent children.
abstract: OBJECTIVE
  To establish efficacy and safety of deferiprone.
  
  
  METHODS
  Prospective study.
  
  
  METHODS
  The Lady Ridgeway Hospital for Children, Colombo.
  
  
  METHODS
  Transfusion-dependent children in the age group 1 to 15 years.
  
  
  METHODS
  Patients were given 75 mg/kg/day of deferiprone orally in divided doses.
  
  
  METHODS
  Efficacy of deferiprone therapy was assessed by 4 to 6 monthly serum ferritin (SF) assays. Safety of therapy was assessed by 4-weekly white cell counts and serum alanine aminotransferase (ALT) levels. The Z-score was used to assess the significance of the difference between the mean initial and final SF level.
  
  
  RESULTS
  82 patients received deferiprone therapy for a mean duration of 30 +/- 14 months. Initial SF levels ranged from 1115 to 12,165 micrograms/l with a mean of 5156 +/- 2631 micrograms/l. Final SF levels ranged from 312 to 15,285 micrograms/l with a mean of 2809 +/- 2380 micrograms/l (Z score 5.99; p < 0.001). Two (2.4%) children developed agranulocytosis which reverted to normal on discontinuation of treatment. 41 (50%) developed arthropathy and in 17 this was severe enough to require discontinuation of therapy. Serum ALT levels were raised in 35 (43%) patients but reverted to pretreatment values or lower despite continuation of deferiprone therapy. There was one death in a 9-year old child who developed diabetes mellitus and heart failure despite deferiprone therapy for 3 years.
  
  
  CONCLUSIONS
  A final SF level < 2500 micrograms/l was achieved in 52% children. Severe arthropathy and agranulocytosis may necessitate permanent discontinuation of therapy.
authors.affiliation: Lady Ridgeway Hospital for Children, Colombo 8.
authors.forename: G N
authors.initials: GN
authors.lastname: Lucas
authors.forename: B J C
authors.initials: BJ
authors.lastname: Perera
authors.forename: E A N
authors.initials: EA
authors.lastname: Fonseka
authors.forename: D D S
authors.initials: DD
authors.lastname: De Silva
authors.forename: M
authors.initials: M
authors.lastname: Fernandopulle
authors.forename: D H
authors.initials: DH
authors.lastname: Karunatilaka
authors.forename: I
authors.initials: I
authors.lastname: Weerasinghe
mesh_terms: D000284:Administration, Oral; D000293:Adolescent; D000740:Anemia / Q000097:blood / Q000628:therapy*; D001803:Blood Transfusion; D002648:Child; D002675:Child, Preschool; D000077543:Deferiprone; D005260:Female; D005293:Ferritins / Q000097:blood; D006801:Humans; D007223:Infant; D007502:Iron Chelating Agents / Q000008:administration & dosage* / Q000009:adverse effects; D008297:Male; D011446:Prospective Studies; D011728:Pyridones / Q000008:administration & dosage* / Q000009:adverse effects; D013789:Thalassemia / Q000628:therapy
chemical_list: D007502:Iron Chelating Agents; D011728:Pyridones; D000077543:Deferiprone; D005293:Ferritins