EFFICACY AND ADVERSE EFFECTS OF ORAL IRON CHELATOR DEFERIPRONE (L1, 1,2- DIMETHYL-3-HYDROXYPYRID-4-ONE) IN PATIENTS WITH BETA THALASSAEMIA MAJOR IN PAKISTAN
AbstractBackground: Deferiprone (DFP,L1) is a bidentate oral iron chelator which binds to iron in a3:1 ratio. It has the potential advantage of reduced cost and increased compliance. Weconducted a study in order to determine the efficacy and adverse effects of DFP in Pakistanithalassaemic patients. Methods: A group of 26 thalassaemic patients entered the studyduring the period Jan 1999 to Aug 2002. DFP supplied by Lipomed, Switzerland was givenat a daily dose of 75 mg/kg/day (range 50-75 mg/kg/day). After giving informed writtenconsent all the patients were subjected to clinical examination and investigations formonitoring the response. Blood complete picture, liver function tests, blood urea &creatinine, antinuclear factor antibodies (ANF) were tested in all cases before starting DFPtreatment. Results: The patients ages ranged from 11 to 27 years, 16 were male and 10 werefemale. Initial serum ferritin level ranged from 3100-8800 μg/l, mean serum ferritin levelwas 7129±1467 μg/l (95% CI 6536 – 7721 μg/l). ECG and Echocardiography wasperformed in all cases and in 11 cases Cardiac Multigated acquisition (MUGA) Scan wasalso performed and six patients with impaired left ventricular function were identified. Fourpatients were lost to follow up and one patient died due to cardiomyopathy. Among theremaining 21 patients serum ferritin levels dropped to 1900μg/l to 5600μg/l with mean levelof 4288 μg/l (95%CI 3874 – 4702 μg/l), SD 911 μg/l. Significance of difference was (p <0.001) by Paired samples ‘t’ test. Six patients had gastrointestinal symptoms along with twohaving arthropathy. ANF positivity was not detected in any patient while on DFP treatment.Similarly, agranulocytosis was not detected in any patient. Conclusion. Mean serum ferritinlevel estimated at the start of trial was 7129 μg/l. This shows that Pakistani thalassaemicpatients are quite iron overloaded due to socioeconomic reasons that are peculiar to oursetup. In this study DFP was well tolerated and caused fewer side effects. It had much betterpatient compliance and was effective in lowering serum ferritin level in previously mostpoorly chelated patients.Keywords: Thalassaemia, Deferiprone, Transfusion iron overload, Cardiomyopathy,Demography
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