Efficacy of hydroxyurea on the rate of blood transfusion requirement and serum ferritin levels in β-thalassemia major
Keywords:Thalassemia major, Hydroxyurea, Blood transfusion, Serum ferritin
Background: Objective: To evaluate the efficacy of hydroxyurea on the rate of blood transfusion requirement and serum ferritin levels in β-thalassemia major patients.
Methods: Design: Comparative trial. Setting: In a tertiary care centre in North India over a period of one year from December 2012 to November 2013. Blood transfusion requirements and serum ferritin levels during ten months prior to the beginning of the study in the same patients were considered as controls. Techniques: This was a hospital based interventional study, and was conducted over a period of 10 months at thalassemia clinic in post graduate department of pediatrics at govt. medical college and hospital, Jammu. Patients of β-thalassemia major attending the clinic, which fulfilled the inclusion criteria were selected as cases as well as controls. An informed written consent was taken from the parents/guardian of the patients to ensure compliance and regular follow up.
Results: The blood transfusion requirements were significantly decreased at 6 and 10 months after starting treatment as compared to previous 10 months. There was a significant decrease in serum ferritin levels also after starting the treatment.
Conclusions: The demonstrable significant decrease in the transfusion requirements as well as improvement in laboratory parameters including serum ferritin levels after initiating treatment with oral hydroxyurea in β-thalassemia major patients apparently suggests that oral hydroxyurea may find place in the management of β-thalassemia major children. However larger trials are warranted to derive optimum dosing schedule and confirm its efficacy so that it can be incorporated into future management guidelines.
Cooley TB, Lee P. A series of cases of splenomegaly in children with anemia and peculiar bone changes. Trans Am Pediatr Soc. 1925;37:29-30.
Greepi E, Heri JF. Moliticofamiliare con aumento della resistance dei grobuli. Minerva Med. 1928;8:1-10.
Steingberg MH, Benz EJ, Adewoye HA, Ebert B. Pathophysiology of human erythrocyte and its hemoglobin. In: Hoffman R, Benz EJ, Shattil SJ, Furie B, Cohen HJ, Silberstein LE et al., eds. Hematology. 4th ed. Philadelphia: Elsvier; 2000: 442-454.
Weatherall DJ. Disorders of globin synthesis: The thalassemias: overview. In: Lichtman MA, Bentler E. Shigsohn U, Kmnshansky K, Okipps TO, eds. William Hematology. 7th ed. New York: McGraw-Hill; 2007: 633-667.
Winichagoon P. Fucharoen S, Chen P. Genetic factors affecting clinical severity of beta thalassemia syndrome. J Pediatr Hematol Oncol. 2000;22:573.
Galanello R, Melis MA, Ruggeri R, Addis M, Scalas MT, Maccioni L, et al. β-thalassemia trait in Sardinia. Hemoglobin. 1979;3:33-46.
Borgne-Pignati C, Cappellini MD, De Sthefano P, Del Vecchio GC, Forni GL, Gamberini MR, et al. Survival and complications in thalassemia. Ann N Y Acad Sci. 2005;1054:40-7.
Zamani F, Shakeri R, Eslami SM, Razavi SM, Ali Basi. Hydroxyurea therapy in 49 patients with major beta-thalassemia. Arch Iran Med. 2009;12:295-7.
Kosaryan M, Koorosh VK, Karami H, Eliteshami S. Effect of hydroxyurea on thalassemia major and thalassemia intermedia in Iranian patients. Pak J Med Sci. 2009;25:74-8.
Chernoff AI. The distribution of thalassemia gene. Ahistorical review. Blood. 1959;14:899.
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. 2004;112:529-30.
Ansari SH, Shambi TS, Siddiqui FJ. Efficacy of hydroxyurea in reduction of pack red cell transfusion requirement among children having beta-thalassemia major: Karachi HU Trial. J Pediatr Hematol Oncol. 2007;29:729-30.
Cooley TB, Lee PA. Series of cases of splenomegaly in children with anemia and peculiar bone changes. Trans Am Pediatr Soc. 1925;37:29-30.
De Baun MR. Hydroxyurea therapy. In: De Baun MR, eds. Nelson Textbook of Paediatrics. 18th ed. Philadelphia: Saunders; 2007: 2018-2038.
Alebouyeh M, Moussavi F, Haddad-Deylami H, Vossough P. Hydroxyurea in the treatment of major beta-thalassemia and importance of genetic screening. Ann Hematol. 2004;83:430-3.