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

Muhammad Ayyub, Waqar Ali, Masood Anwar, Amin Waqar, M. Naeem Khan, Aamir Ijaz, Tassawar Hussain, Shujaat Hussain

Abstract


Background: Deferiprone (DFP,L1) is a bidentate oral iron chelator which binds to iron in a
3:1 ratio. It has the potential advantage of reduced cost and increased compliance. We
conducted a study in order to determine the efficacy and adverse effects of DFP in Pakistani
thalassaemic patients. Methods: A group of 26 thalassaemic patients entered the study
during the period Jan 1999 to Aug 2002. DFP supplied by Lipomed, Switzerland was given
at a daily dose of 75 mg/kg/day (range 50-75 mg/kg/day). After giving informed written
consent all the patients were subjected to clinical examination and investigations for
monitoring the response. Blood complete picture, liver function tests, blood urea &
creatinine, antinuclear factor antibodies (ANF) were tested in all cases before starting DFP
treatment. Results: The patients ages ranged from 11 to 27 years, 16 were male and 10 were
female. Initial serum ferritin level ranged from 3100-8800 μg/l, mean serum ferritin level
was 7129±1467 μg/l (95% CI 6536 – 7721 μg/l). ECG and Echocardiography was
performed in all cases and in 11 cases Cardiac Multigated acquisition (MUGA) Scan was
also performed and six patients with impaired left ventricular function were identified. Four
patients were lost to follow up and one patient died due to cardiomyopathy. Among the
remaining 21 patients serum ferritin levels dropped to 1900μg/l to 5600μg/l with mean level
of 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 two
having arthropathy. ANF positivity was not detected in any patient while on DFP treatment.
Similarly, agranulocytosis was not detected in any patient. Conclusion. Mean serum ferritin
level estimated at the start of trial was 7129 μg/l. This shows that Pakistani thalassaemic
patients are quite iron overloaded due to socioeconomic reasons that are peculiar to our
setup. In this study DFP was well tolerated and caused fewer side effects. It had much better
patient compliance and was effective in lowering serum ferritin level in previously most
poorly chelated patients.
Keywords: Thalassaemia, Deferiprone, Transfusion iron overload, Cardiomyopathy,
Demography

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