Efficacy and side effects of hydroxyurea in patients with thalasemia intermedia

AUTHORS

Majid Yavarian 1 , * , Nader Fayazi 2 , Nashmil Rostami 3 , Maryam Shamsaei 3 , Mehran Karimi 4

1 Assistant Professor Department of Genetic, Shiraz University of Medical Sciences, Shiraz, Iran.

2 Assistant Professor Department of Internal Medicine, Hormozgan University of Medical Sciences, Bandar Abbas, Iran.

3 General Practitioner, Hormozgan University of Medical Sciences, Bandar Abbas, Iran.

4 Professor Department of Internal Medicine, Shiraz University of Medical Sciences, Shiraz, Iran.

How to Cite: Yavarian M, Fayazi N, Rostami N, Shamsaei M, Karimi M. Efficacy and side effects of hydroxyurea in patients with thalasemia intermedia, Hormozgan Med J. 2007 ; 11(2):e89355.

ARTICLE INFORMATION

Hormozgan Medical Journal: 11 (2); e89355
Published Online: February 19, 2007
Article Type: Research Article
Received: June 18, 2006
Accepted: February 19, 2007

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Abstract

Introduction: The β-thalassaemias represent a heterogenous group of diseases
resulting from decreased β-globin mRNA expression and imbalanced alpha/betaglobin
chain synthesis which are clinically manifested by ineffective erythropoiesis
and excessive hemolysis. Increasing levels of hemoglobin F (HbF) by improving the
balance in globin chain synthesis. Hydroxyurea (HU), as an effective with low
toxicity for activating y-globin gene, has been shown to enhance HbF synthesis.
Methods: To analyze the clinical presentation and possible side effects of HU
treatment 80 thalassemia intermedia patients were selected and followed-up for one
year regularly. All of them started their regular blood transfusion beyond of 2 years
of age and were being treated with 10-12 mg/kg/d HU orally.
Results: The study group consist of 38 men and 42 women. The average age was
16.8(±6.9) years and thalassemia was diagnosed according to the RBC indexes and
Hb electrophoresis. An improvement of erythropoiesis was reflected by an increase
in hemoglobin concentration, the state of energy, fatigability, and mood. About
83% (69) of the patients tolerated the HU well and showed a dramatic response to
the drug. Thirty of 80 transfusion-dependent patients became completely
transfusion-free and 23 cases had one or two transfusions throughout the study.
Vomiting and thrombocytopenia were seen in two patients.
Conclusion: During this close observation, no malignant change was seen in our
patients. HU therapy appears to be safe and effective when administered in
thalassemic patients.

Keywords

Thalassemia – Hydroxyurea – Adverse Effect

© 2007, Hormozgan Medical Journal. This is an open-access article distributed under the terms of the Creative Commons Attribution-NonCommercial 4.0 International License (http://creativecommons.org/licenses/by-nc/4.0/) which permits copy and redistribute the material just in noncommercial usages, provided the original work is properly cited.

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