Nadia Waheed, Shafqut Ali, Muhammad Asghar Butt


Background: Thalassemia major is the most common genetic disorder in Pakistan. The study was done to compare the efficacy and safety of the deferiprone with deferrioxamine for the treatment of iron overload in children with thalassemia major. Methods: This randomized controlled trail was conducted at thalassemia blood transfusion unit of Allied Hospital, Faisalabad (AHF)/District Headquarter Hospital (DHQ), Faisalabad. Thalassemia-Unit Hilal-e-Ahmar, Alizeb Foundation and Blood Bank Services Faisalabad from November 2010 to December 2011.Children with beta thalassemia major of age more than 2 years and less than 16 years with transfusion iron over load were randomly allocated to one of the two groups each comprising of 67 patients. One group received deferiprone given at a daily dose of 75 mg/kg in three divided doses orally while the other group received deferrioxamine at dose 50 mg/kg/24 hrs for 5 days/week as parental infusion. Changes in the serum ferritin level were assessed. Cardiac function and toxicity were also examined. Results: Serum ferritin was significantly reduced after 1 year in both treatment arms (p=0.01). Neutropenia observed in 13 (19.40%) non-splenectomized patients taking deferiprone. Transient elevations in ALT were observed in 3 (4.47%) children taking deferiprone. Left ventricular ejection fraction (LVEF) remained in normal range in both treatment arm but has decreased significantly in Deferrioxamine group compliance. Compliance was better in deferiprone as compared to deferrioxamine. Discontinuing percentage 2 (3%) vs 9 (13.43%). Conclusion: Deferiprone is a highly efficacious and safe chelation therapy for patients with thalassemia major who are non-compliant to Deferrioxamine. Deferiprone have an efficacy profile comparable to standard Deferrioxamine.

Keywords: Thalassemia deferiprone, deferrioxamine iron over load

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Malik S, Syed S, Ahmad N. Complications in transfusion dependent patients of B-thalassemia major. Pak J Med Sci 2009;25:678–82.

Arif F, Fayyaz J, Hamid A. Awareness among parents of children with thalassemia major. J Pak Med Asso 2008;58:621–4.

Khan F. Thalassemia: still a challeng. Gomal J Med Sci 2006;4:47–8.

Michael R, DeBaun MR, Vichinsky E. Hemoglobinopathies. In: Kliegman RM, Behrman RE, Jenson HB, Stanton BF, editors. Nelson book of pediatrics 18th ed. Philadelphia: Saunders Elsevir; 2008.p. 2025–38.

Porter JB. Pathophysiology of transfusional Iron overload: contrasting patterns in thalassemia major and sickle cell disease.. Hemoglobin 2009;33:S37–45.

Kohgo Y, Ikuta K, Ohlake T, Torimoto Y, Kato J. Body iron metabolism and pathophysiology of iron overload. Int J Hematol 2008;88:7–15.

Moorjani JD, Issac C. Neurotic Manifestations in adolescents with thalassemia Major. Indian J Pediatr 2006;73:603–7.

Peng CT, Wu KH, Wu SF, Liang DC, Yang CP, Jang RC, et al. Deferiprone or deferoxamine vs. combination therapy in patients with beta-thalassemia major: a case study inTaiwan. Hemoglobin 2006;30:125–30.

Neufeld EJ. Oral chelators deferisirox and deferipron for transfusional iron overlaoad in thalassemia Major. Blood 2006;107:3436–41.

Angelucci E, Brittenham GM, McLaren CE, Ripalti M, Baronciani D, Giardini C, et al. Hepatic iron concentration and total body iron stores in thalassemia major. N Engl J Med 2000;343:327–31.

Zanninelli G, Breur W, Cabantchik ZI. Daily labile plasma iron as an indicator of chelator activity in thalassaemia major patient. Br J Haematol 2009;147:744–51.

Fischer R, Harmatz P, Nielsen P. Does liver biopsy overestimate liver iron concentration? Blood. 2006;108:1775-6.

Schrier SL, Angelucci E. New strategies in the treatment of thalassemias. Annu Rev Med 2005;56:157–71.

Ceci A, Baiardi P, Felisi M, Cappellini MD, Carnelli V, De Sanstis V, et al. The safety and effectiveness of deferiprone in a large-scale, 3-year study in Italian patients. Br J Haematol 2002;118:330–6.

Taher A, Chamoun FM, Koussa S, Saad MA, Khoriaty AI, Neeman, R, et al. Efficacy and side effects of deferiprone (L1) in thalassemia patients not compliant with desferrioxamine. Acta Haematol 1999;101:173–7.

Sajjad R, Ghani F, Adil S, Khurshid M. Oral iron chelation therapy with Deferiprone in patients with Thalassemia Major. J Pak Med Assoc 2009;59:388–90.

Cohen AR, Galanello R, Piga A, Dipalma A, Vullo C, Tricta F. Safety profile of oral iron chelator deferiprone: a multicenter study. Br J Haematol 2000;108:305–12.

Galanello R, Kattamis A, Piga A, Fischer R, Leoni G, Ladis V, et al. A prospective randomized controlled trial on the safety and efficacy of alternating deferoxamine and deferiprone in the treatment of iron over load in patients with thalassemia. Haematologica 2006;91:1241–3.

Borgna-Pignatti C, Cappellini MD, De Stefano P, Del Vecchio GC, Forni GL, Gamberini MR, Ghilardi R, et al. Cardiac morbidity and mortality in deferoxamine or deferiprone treated patients with thalassemia major. Blood 2006;107:3733–7.

Aydinok Y, ulger Z, Deniz N, terzi A, certiner N, Ellis G, Zimmermann A, Manz C. A Randomized Controlled 1-year study of daily deferiprone plus twice weekly deferrioxamine compared with daily deferiprone monotherapy in patients with thalassemia major. Haematologica 2007;92:1599–1606.

Hoffbrand AV, Cohen A, Hershko C. Role of deferiprone in chelation therapy for transfusional iron overload. Blood 2003;102:17–24.

Gomber S, Saxena R, Madan N. Comparative efficacy of Deferrioxamine, deferiprone and in combination on iron chelation in thalassemic children. Indian Pediatr 2004;41:21–7.

Ha SY, Chik KW, Ling, SC, Lee AC, Luk CW, Lam CW, et al A randomized controlled study evaluating the safety and efficacy of deferiprone treatment in thalassemia major patients from Hong Kong. Hemoglobin 2006;30:263–74.

Raphael JL, ,Bernhardt MB, Mahoney DH, Mueller BU. Oral Iron Chelation and treatment of iron overload in a Pediatric hematology center. Pediatr Blood Cancer 2009;52:616–20


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