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

Abstract

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.

Efficacy and Safety of Hydroxyurea as Adjuvant Therapy in Pediatric Patients of Transfusion-Dependent Beta-Thalassemia Major at Zhob, Balochistan

“…Sukhar K et al enrolled 50 patients for studying the efficacy of hydroxyurea, however, 15 were dropped because of poor compliance (11), loss on follow-up (2), and side effects (2); forming the sample size of 35; however, they found a significant improvement in post-treatment hemoglobin levels [12]. Kosaryan M et al…”

“…Hydroxyurea showed no serious side effects in almost all the studies conducted so far, except mild liver or renal impairment observed in a couple of researches [12][13][14][15][16][17][18]. It can be used safely in children as a single oral dose per day and monitored with complete blood count, alanine aminotransferase (ALT), and serum creatinine levels regularly.…”

“…Sukhar K et al enrolled 50 patients for studying the efficacy of hydroxyurea, however, 15 were dropped because of poor compliance (11), loss on follow-up (2), and side effects (2); forming the sample size of 35; however, they found a significant improvement in post-treatment hemoglobin levels [ 12 ]. Kosaryan M et al studied 297 patients (248 cases of transfusion-dependent thalassemia major) and 48 cases of thalassemia intermedia); they claimed hydroxyurea to be very beneficial, as transfusions were completely stopped in 44.7% of transfusion-dependent thalassemia-major cases.…”

“…It can be used safely in children as a single oral dose per day and monitored with complete blood count, alanine aminotransferase (ALT), and serum creatinine levels regularly. Withdrawal of this drug can reverse the biochemical alterations that occurred in a few studies [ 12 , 18 ].…”

“…It has been used in a dose of 10 mg/kg to 35 mg/kg as a single daily oral dose for thalassemia and sickle cell disease. The main side effects are liver and kidney dysfunction and bone marrow suppression [ 12 ].…”