@article {117508,
	title = {Response to hydroxycarbamide in pediatric β-thalassemia intermedia: 8 years{\textquoteright} follow-up in Egypt.},
	journal = {Annals of hematology},
	volume = {93},
	year = {2014},
	month = {2014 Dec},
	pages = {2045-50},
	abstract = {<p>Hydroxycarbamide (hydroxyurea or HU) has been shown to increase fetal hemoglobin (HbF) in patients with β-thalassemia intermedia (TI). The reported effects of HU in increasing the total hemoglobin (Hb) have been inconsistent. Studies of long-term therapy with HU in pediatric TI are rather uncommon. A retrospective observational study was carried out to evaluate the clinical responses to HU in Egyptian patients with β-TI. One hundred patients; children (n = 82, mean age 9.9 {\textpm} 4.1 years) and adults (n = 18) were studied for the mean Hb, HbF\%, median serum ferritin, transfusion history, and splenic size before and after HU therapy (mean dose 20.0 {\textpm} 4.2 mg/kg/day, range 10-29 mg/kg/day) over a follow-up period 4 to 96 months (mean 35.4 {\textpm} 19.2 months). Molecular studies were also done for group of patients (n = 42). The overall response rate to HU was 79 \%; 46 \% were minor responders (with a reduction in transfusion rate by 50 \% or more and/or an increase in their total hemoglobin level by 1-2 g/dl) and 33 \% major responders (becoming transfusion-free and/or having an increase in total hemoglobin level by \&gt;2 g/dl). Mean hemoglobin increased among responders from 6.9 {\textpm} 0.9 g/dl to 8.3 {\textpm} 1.4 g/dl (p \&lt; 0.001). A significant rise in mean HbF (27.0 vs. 42.5 \%; p \&lt; 0.011) and a decrease in median serum ferritin (800 vs. 644 ng/ml; p \&lt; 0.001) were also observed among responders (n = 45). Transfusions stopped in 44 \% of pretreatment frequently transfused responders (n = 11/25). Splenic size decreased in 37 \% of patients (n = 30/81). The predominant β-thalassemia mutation was 1-6 (T \&gt; C) in 32/42 (76 \%) of studied patients; 28/32 were responders. Bivariate analysis showed no predictors of response as regards sex, pediatric and adult age, splenic status, or genotype. Hydroxycarbamide is a good therapeutic modality in the management of pediatric as in adult TI patients. It can minimize the need for blood transfusion, concomitant iron overload, and blood-born viral transmission especially in developing countries like Egypt.</p>
},
	keywords = {Adolescent, Adult, Age Factors, beta-Thalassemia, Blood Transfusion, Child, Child, Preschool, Combined Modality Therapy, Drug Evaluation, Egypt, Ferritins, Fetal Hemoglobin, Humans, Hydroxyurea, Iron Overload, neutropenia, Retrospective Studies, Splenectomy, Splenomegaly, Treatment Outcome, Young Adult},
	issn = {1432-0584},
	doi = {10.1007/s00277-014-2154-5},
	author = {Amal El-Beshlawy and Mona El-Ghamrawy and Mona Abou El-Ela and Said, Fadwa and Sonia Adolf and Abdel-Rahman Ahmed Abdel-Razek and Rania Ismail Magdy and Amina Abdel-Salam}
}
