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


Background: 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


Ahmed S, Saleem M, Modell B, Petrou M. Screening

extended families for genetic hemoglobin disorders in

Pakistan. N Engl J Med 2002;347:1162-8.

Ahmed S, Petrou M, Saleem M. Molecular genetics of

beta-thalassaemia in Pakistan: a basis for prenatal

diagnosis. Br J Haematol 1996 Sep;94(3):476-82.

Khattak MF, Saleem M. Prevalence of heterozygous

beta thalassaemia in northern areas of Pakistan. J Pak

Med Assoc 1992; 42:32-4.

Alwan A, Modell B. Recommendations for introducing

genetics services in developing countries. Nat Rev

Genet 2003; 4: 61-8.

Al-Refaie FN, De Silva CE, Wonke B, Hoffbrand AV.

Changes in transferrin saturation after treatment with the

oral iron chelator deferiprone in patient with iron

overload. J Clin Pathol; 1995; 48: 110-4.

Al-Rafaie FN, Sheppard LN, Nortey P, Wonke B,

Hoffbrand AV. Pharmacokinetics of the oral iron

chelator deferiprone (L1) in patients with iron overload.

Br J Haematol 1995; 89: 403-8.

Kontoghiorghes GJ, Bartlett AN, Hoffbrand AV,

Golddard JG, Shepard L, Barr J, et al. Long term trail

with the oral iron chelator 1,2-dimethyl-3-hydroxypyrid-

-one (L1). Iron chelation and metabolic studies. Br J

Haematol 1990; 76;295-300.

Porter JB. Practical management of iron overload. Br J

Haematol; 2001; 115:239-52.

Wood JC, Tyszka JM, Carson S, Nelson MD, Coates

TD. Myocardial iron loading in transfusion dependent

thalassemia and sickle cell disease. Blood. 2004;


Hoffbrand AV, Cohen A, Hershko C. Role of

deferiprone in chelation therapy for transfusional iron

overload. Blood 2003;102: 17-24.

Bhatti FA, Amin M, Saleem M. Prevalence of antibody

to hepatitis C virus in Pakistani thalassaemics by particle

agglutination test utilizing C-200 and C22-3 viral

antigen coated practical. J Pak Med Assoc 1995; 45

(10): 269-71.

Bergeron RJ, Streiff RR, Weigand J, Luchetta G, Creary

EA, Peter HH. A comparison of the iron chelating

properties of 1,2-dimethyl-3-hydroxypyrid-4-one, 1,2-

diethyl-3-hydroxypyrid-4-one and deferoxamine. Blood

; 79: 1882-90.

Liu ZD, Liu DY, Lu SL, Hider R. Synthesis,

physicochemical properties and biological evaluation of

aromatic ester prodrugs of 1-(2’-hydroxyethyl)-2-ethyl-

-hydroxypyridin-4-one (CP102): orally active iron

chelators with clinical potential. J Pharm Pharmacol

; 51:555-64.

Al-Refaie FN, Hershko C, Hoffbrand AV, Kosaryan M,

Olivieri, NF, Tondury P, et al. Results of long term

deferiprone (L1) therapy: a report by the International

Study Group on Oral Iron Chelators. Br J Haematol

; 91: 224-9.

Modell B, Khan M, Darlison M. Survival in betathalassaemia major in the UK: data from the UK

Thalassaemia Register. Lancet 2000;355:2051-2.

Agarwal MB, Gupte, SS, Viswanathan C, Vasandani D,

Ramanathan J, Desal N. Long-term assessment of

efficacy and safety of L1, an oral iron chelator, in

transfusion dependent thalassaemia: Indian trail. Br J

Haematol 1992; 82: 460-6.

Refaie FN, Wonke B, Hoffbrand AV, Wickens, DG,

Nortey P, Kontoghiorghes GJ. Efficacy and possible

adverse effects of the oral iron chelator 1,2 dimethyl-3-

hydroxypyrid-4 one (L1) in thalassemia major. Blood

; 80: 593-9.

Olivieri NF, Koren G, Hermann C, Bentur Y, Chung D,

Klein J, et al. Comparison of oral iron chelator L1 and

desferrioxamine in iron loaded patients. Lancet 1990;


Stella M, Pinzello G, Maggio A. Iron chelation with oral

deferiprone in patients with thalassaemia. New Eng J

Med 1998; 339: 172-4.

Piga A, Gaglioti C, Fogliacco E, Tricta F. Comparative

effects of deferiprone and deferoxamine on survival and

cardiac disease in patients with thalassemia major: a

retrospective analysis. Haematologica 2003;88: 489-96.

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