Efficacy and safety of high dose hydroxyurea in transfusion dependent thalassemic children: a quasi experimental study

Kiran Suthar, Pramod Sharma, Manish Verma, Vishnu Kr. Goyal


Background: This study was conducted to find out whether high dose hydroxyurea is an effective and safe modality, in inducing haemoglobin synthesis to decrease blood transfusion requirement in transfusion dependent thalassemics.

Methods: This quasi experimental un-control before and after comparison study was conducted in Thalassemia Day Care Centre, Department of Pediatrics over a period of six months after obtaining an approval from the Institute’s ethical committee. Fifty transfusions dependent thalassemic children belonging from 2 to 18 yrs were given hydroxyurea in dose of 20mg/kg after getting consent. Pre and post intervention haemoglobin and HbF levels were obtained using Hb electrophoresis by HPLC. Paired t test was applied to find out statistical significance and p value <0.05 was taken as significant.

Results: Significant rise in haemoglobin pre and post intervention (p<0.001) but the rise in HbF was not significant (p=0.110). One patient had bone marrow depression which was reversible with drug withdrawal and one patient had rise in s. creatinine.

Conclusions: High dose hydroxyurea is an effective and safe drug in inducing Hemoglobin synthesis in transfusion dependent thalassemics. 



Haemoglobin F, High dose hydroxyurea, Transfusion dependent thalassemia

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Rund D, Rachmilewitz E Beta-thalassemia. N Engl J Med. 2005;353:1135-46.

Forget BG. The pathophysiology and molecular genetics of beta thalassemia. Mt Sinai J Med. 1993;60:95-103.

Pootrakul P, Sirankapracha P, Hemsorach S, Moungsub W, Kumbunlue R, Piangitjagum A et al. A correlation of erythrokinetics, ineffective erythropoiesis, and erythroid precursor apoptosis in Thai patients with thalassemia. Blood. 2000;96:2606-12.

Cokic VP, Andric SA, Stojilkovic SS, Noguchi CT, Schechter AN. Hydroxyurea nitrosylates and activates soluble guanylyl cyclase in human erythroid cells. Blood. 2008;111(3):1117-23.

Sauvage C, Rouyer-Fessard P, Beuzard Y. Improvement of mouse beta thalassaemia by hydroxyurea. Br J Haematol. 1993;84(3):492-6.

Mabaera R, West RJ, Conine SJ, Macari ER, Boyd CD, Engman CA et al. A cell stress signaling model of fetal hemoglobin induction: what doesn’t kill red blood cells may make them stronger. Exp Hematol. 2008;36(9):1057-72.

Rigano P, Pecoraro A, Calzolari R, Troia A, Acuto S, Renda D et al. Desensitization to hydroxycarbamide following long-term treatment of thalassaemia intermedia as observed in vivo and in primary erythroid cultures from treated patients. Br J Haematol. 2010;151(5):509-15.

DeBaun Michael R, Jones Melissa F, Vichinsky Elliott. 456.1 Sickle cell disease. In Kliegman, Stanton, St. Geme, Schor, Behrman Nelson textbook of pediatrics. 19th edition Philadelphia: Elsevier; 2011:1667.

Ishaq F, Mannan J, Seyal T, Hassan S. Efficacy and side effects of hydroxyurea in patient with thalassemia intermedia. Pak Paed J. 2011;35(1):8-12

Tan X, Patel I, Chang J. Review of the four item Morisky Medication Adherence Scale (MMAS-4) and eight item Morisky Medication Adherence Scale (MMAS-8). Innovations Pharm. 2014;5(3):165.

Thein SL. The emerging role of fetal hemoglobin induction in non-transfusion-dependent thalassemia. Blood Rev. 2012;26:S35-9.

Perrine SP. Fetal globin induction—can it cure β thalassemia? ASH Education Program Book. 2005;2005(1):38-44.

Mancuso A, Maggio A, Renda D, Marzo R, Rigano P. Treatment with hydroxycarbamide for intermedia thalassaemia: decrease of efficacy in some patients during long-term follow up. Br J Haematol. 2006;133(1):105-6.

De Paula EV, Lima CS, Arruda VR, Alberto FL, Saad ST, Costa FF. Long-term hydroxyurea therapy in beta-thalassaemia patients. Eur J Haematol. 2003;70(3):151-5.

Bordbar MR, Silavizadeh S, Haghpanah S, Kamfiroozi R, Bardestani M, Karimi M. Hydroxyurea treatment in transfusion-dependent β-thalassemia patients. Iran Red Crescent Med J. 2014;16(6):e18028.

Koren A, Levin C, Dgany O, Kransnov T, Elhasid R, Zalman L et al. Response to hydroxyurea therapy in beta-thalassemia. Am J Hematol. 2008;83(5):366-70.

Karimi M, Darzi H, Yavarian M. Hematologic and clinical responses of thalassemia intermedia patients to hydroxyurea during 6 years of therapy in Iran. J Pediatr Hematol Oncol. 2005;27(7):380-5.

Bradai M, Pissard S, Abad MT, Dechartres A, Ribeil JA, Landais P et al. Decreased transfusion needs associated with hydroxyurea therapy in Algerian patients with thalassemia major or intermedia. Transfusion. 2007;47(10):1830-6.

Bradai M, Abad MT, Pissard S, Lamraoui F, Skopinski L, de Montalembert M. Hydroxyurea can eliminate transfusion requirements in children with severe beta-thalassemia. Blood. 2003;102(4):1529-30.

Yavarian M, Karimi M, Bakker E, Harteveld CL, Giordano PC. Response to hydroxyurea treatment in Iranian transfusion-dependent beta-thalassemia patients. Haematologica. 2004;89(10):1172-8.