Hydroxyurea and termination of transfusion therapy in transfusion dependent thalassemics
Sachdeva, Anand; Khanna, Kumar; Verma, C.P.; Kaul, Dinesh and Arya, Subash C. (2002) Hydroxyurea and termination of transfusion therapy in transfusion dependent thalassemics. In: The 44th American Society of Hematology Annual Meeting, 2002, Detroit, USA.
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Aim: Hydroxyurea is one of the agents which can cause fetal chain activation and thus may be useful as a therapy for beta thalassemia. We used hydoxyurea in Beta-thalassemia major patients with the aim of making them transfusion independent. Method: We administered orally hydoxyurea in 70 patients of Beta-thalassemia with dose of 15-20 mg/kg/day. They have been followed up for a period of 4 months - 3 years. The age of the patients ranged from 1.5-25 years (mean age 11.6 yrs) The mean age of presentation with anemia was 4.35 yr. There were 41 males and 29 females. Response was evaluated with the help of lab evaluation of a rise in hemoglobin and HbF levels, MCV, decreasing nucleated RBC's and reticulocyte count. Clinically the response was categorized as complete if the need for transfusion was obviated, partial response if the interval between transfusions increased but not completely abolished and no response if the child had to take transfusions as before. Mutation analysis of all the patients was done and the response was correlated with the mutation of the patient. Results: It was seen that 25/70 (36%) had a complete response, 15/70 (21%) had a partial response and 24/70 (34%) had no response or discontinued treatment due to toxicity. 6/70 (9%) could not be evaluated, as the duration was too short. We will be presenting the data of mutation analysis and the responders. Conclusion: Hydroxyurea remains a good option for patients who have presented with anemia at more than 2 years of age. Long term side effects need to be evaluated.
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