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Long‐term hydroxyurea therapy in beta‐thalassaemia patients
Author(s) -
De Paula Erich Vinicius,
Lima Carmen Silvia Passos,
Arruda Valder Roberval,
Alberto Fernando Lopes,
Saad Sara Teresinha Ollala,
Costa Fernando Ferreira
Publication year - 2003
Publication title -
european journal of haematology
Language(s) - English
Resource type - Journals
SCImago Journal Rank - 0.904
H-Index - 84
eISSN - 1600-0609
pISSN - 0902-4441
DOI - 10.1034/j.1600-0609.2003.00037.x
Subject(s) - medicine , gastroenterology , mean corpuscular volume , reticulocyte , beta (programming language) , creatinine , fetal hemoglobin , hemoglobin , biology , computer science , programming language , pregnancy , biochemistry , fetus , genetics , messenger rna , gene
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 × 10 9 vs. 632.0 × 10 9 /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.