A trial of deferiprone in transfusion-dependent iron overloaded children.
OBJECTIVE
To determine the efficacy and safety of deferiprone.


METHODS
Prospective study.


METHODS
5 paediatric medical units at the Lady Ridgeway Hospital for Children (LRHC), Colombo.


METHODS
Transfusion-dependent iron overloaded children in the age group 2 to 15 years.


METHODS
Patients were given a total daily dose of 75 mg/kg of deferiprone orally in divided doses.


METHODS
Efficacy of deferiprone therapy was assessed by 4-monthly serum ferritin assays using the ELISA technique. Safety of deferiprone therapy was assessed by 4-weekly white cell counts, platelet counts and serum transaminase levels. The Z-test was used to assess the significance of the difference between the mean initial serum ferritin level and the mean subsequent serum ferritin level.


RESULTS
54 patients received deferiprone therapy for a mean duration of 9 +/- 3 months. Initial serum ferritin levels ranged from 1500 to 10,700 ng/ml with a mean of 5743. Subsequent serum ferritin levels, obtained in 48 patients ranged from 740 to 7300 ng/ml with a mean of 3558 (p < 0.001). In 47 of the 48 patients subsequent serum ferritin levels were lower than initial levels. One child developed severe neutropaenia, which reverted to normal on discontinuation of treatment. 11 children developed arthropathy, which responded to ibuprofen therapy combined in some cases with a reduction of the dose of deferiprone to 50 mg/kg/day. Serum transaminase levels were raised in 5 patients but reverted to pretreatment values or lower despite continuation of deferiprone therapy.


CONCLUSIONS
Deferiprone is a safe and effective oral iron-chelating agent which can be used, under strict supervision, in transfusion-dependent iron overloaded children.
Lucas
G N
GN
Lady Ridgeway Hospital for Children, Colombo.
Perera
B J
BJ
Fonseka
E A
EA
De Silva
D D
DD
Fernandopulle
M
M
D000293:Adolescent; D002648:Child; D002675:Child, Preschool; D000077543:Deferiprone; D005293:Ferritins / Q000097:blood; D006801:Humans; D007502:Iron Chelating Agents / Q000008:administration & dosage / Q000627:therapeutic use*; D019190:Iron Overload / Q000188:drug therapy*; D011446:Prospective Studies; D011728:Pyridones / Q000008:administration & dosage / Q000627:therapeutic use*; D013188:Sri Lanka; D065227:Transfusion Reaction; D017086:beta-Thalassemia / Q000628:therapy
D007502:Iron Chelating Agents; D011728:Pyridones; D000077543:Deferiprone; D005293:Ferritins