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Response to hydroxycarbamide in pediatric β-thalassemia intermedia: 8 years’ follow-up in Egypt

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An Erratum to this article was published on 14 March 2016

Abstract

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 ± 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 ± 4.2 mg/kg/day, range 10–29 mg/kg/day) over a follow-up period 4 to 96 months (mean 35.4 ± 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 >2 g/dl). Mean hemoglobin increased among responders from 6.9 ± 0.9 g/dl to 8.3 ± 1.4 g/dl (p < 0.001). A significant rise in mean HbF (27.0 vs. 42.5 %; p < 0.011) and a decrease in median serum ferritin (800 vs. 644 ng/ml; p < 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 > 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.

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Authors and Affiliations

  1. Cairo University, Cairo, Egypt

    Amal El-Beshlawy, Mona El-Ghamrawy, Mona Abou EL-Ela, Fadwa Said, Sonia Adolf, Abdel-Rahman Ahmed Abdel-Razek, Rania Ismail Magdy & Amina Abdel-Salam

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  1. Amal El-Beshlawy
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  2. Mona El-Ghamrawy
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  3. Mona Abou EL-Ela
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  8. Amina Abdel-Salam
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Correspondence to Amal El-Beshlawy.

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El-Beshlawy, A., El-Ghamrawy, M., EL-Ela, M.A. et al. Response to hydroxycarbamide in pediatric β-thalassemia intermedia: 8 years’ follow-up in Egypt. Ann Hematol 93, 2045–2050 (2014). https://doi.org/10.1007/s00277-014-2154-5

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  • DOI: https://doi.org/10.1007/s00277-014-2154-5

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