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1.
Ceylon Med J ; 2002 Dec; 47(4): 119-21
Article Dans Anglais | IMSEAR | ID: sea-48509

Résumé

OBJECTIVE: To establish efficacy and safety of deferiprone. DESIGN: Prospective study. SETTING: The Lady Ridgeway Hospital for Children, Colombo. PATIENTS: Transfusion-dependent children in the age group 1 to 15 years. INTERVENTION: Patients were given 75 mg/kg/day of deferiprone orally in divided doses. MEASUREMENTS: 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.


Sujets)
Administration par voie orale , Adolescent , Anémie/sang , Transfusion sanguine , Enfant , Enfant d'âge préscolaire , Femelle , Ferritines/sang , Humains , Nourrisson , Agents chélateurs du fer/administration et posologie , Mâle , Études prospectives , Pyridones/administration et posologie , Thalassémie/thérapie
2.
Ceylon Med J ; 2000 Jun; 45(2): 71-4
Article Dans Anglais | IMSEAR | ID: sea-49250

Résumé

OBJECTIVE: To determine the efficacy and safety of deferiprone. DESIGN: Prospective study. SETTING: 5 paediatric medical units at the Lady Ridgeway Hospital for Children (LRHC), Colombo. PATIENTS: Transfusion-dependent iron overloaded children in the age group 2 to 15 years. INTERVENTION: Patients were given a total daily dose of 75 mg/kg of deferiprone orally in divided doses. MEASUREMENTS: 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.


Sujets)
Adolescent , Transfusion sanguine/effets indésirables , Enfant , Enfant d'âge préscolaire , Ferritines/sang , Humains , Agents chélateurs du fer/administration et posologie , Surcharge en fer/traitement médicamenteux , Études prospectives , Pyridones/administration et posologie , Sri Lanka , bêta-Thalassémie/thérapie
7.
Ceylon Med J ; 1976 Jun; 21(2): 132-6
Article Dans Anglais | IMSEAR | ID: sea-48813
8.
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