Long-term hydroxyurea therapy in beta-thalassaemia patients
Article first published online: 26 FEB 2003
European Journal of Haematology
Volume 70, Issue 3, pages 151–155, March 2003
How to Cite
De Paula, E. V., Lima, C. S. P., Arruda, V. R., Alberto, F. L., Saad, S. T. O. and Costa, F. F. (2003), Long-term hydroxyurea therapy in beta-thalassaemia patients. European Journal of Haematology, 70: 151–155. doi: 10.1034/j.1600-0609.2003.00037.x
- Issue published online: 26 FEB 2003
- Article first published online: 26 FEB 2003
- Accepted for publication 21 January 2003
Objective: The study aimed to investigate the use of hydroxyurea (HU) for the treatment of beta-thalassaemia (β-thal) patients.
Methods: We examined the haematological effects of orally administered HU (10–20 mg/kg/d) in 11 patients, including four β-thal major and seven β-thal intermedia patients. Complete blood count and levels of foetal haemoglobin (HbF), liver enzymes and serum creatinine were evaluated before and during HU. Response to therapy was evaluated at 6 months of treatment.
Results: A substantial increase in haemoglobin (Hb) level (4.1 g/dL), leading to complete withdrawal from a regular transfusion programme, was observed in one unique β-thal major patient. In the β-thal intermedia patients, increases in Hb level of 1.3, 1.9 and 2.0 g/dL were observed in three of seven (42.9%) patients during HU therapy. The mean values of Hb, mean corpuscular haemoglobin (MCH), and HbF were higher during HU treatment than baseline values (8.7 vs. 7.7 g/dL, P = 0.05; 26.7 vs. 22.9 pg, P = 0.05; 57.2 vs. 44.9%, P = 0.04; respectively). In contrast, the mean reticulocyte count measured during therapy decreased (97.0 × 109 vs. 632.0 × 109/L, P = 0.03). No correlations were observed between levels of Hb and HbF (r = 0.77, P = 0.10), and levels of Hb and reticulocyte counts (r = 0.26, P = 0.31). No significant toxicity was observed in our patients.
Conclusion: These results suggest that HU may improve Hb levels in β-thal. Thus, we may conclude that a large trial concerning the response to HU in these patients should be carried out to clarify this issue.