Long-term hydroxyurea therapy in beta-thalassaemia patients.
OBJECTIVE
The study aimed to investigate the use of hydroxyurea (HU) for the treatment of beta-thalassaemia (beta-thal) patients.


METHODS
We examined the haematological effects of orally administered HU (10-20 mg/kg/d) in 11 patients, including four beta-thal major and seven beta-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 beta-thal major patient. In the beta-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 x 10(9) vs. 632.0 x 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.


CONCLUSIONS
These results suggest that HU may improve Hb levels in beta-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.
de Paula
Erich Vinicius
EV
Haematology and Haemotherapy Centre, and Department of Internal Medicine, State University of Campinas, Campinas, São Paulo, Brazil.
Lima
Carmen Silvia Passos
CS
Arruda
Valder Roberval
VR
Alberto
Fernando Lopes
FL
Saad
Sara Teresinha Ollala
ST
Costa
Fernando Ferreira
FF
D000293:Adolescent; D000328:Adult; D000368:Aged; D001772:Blood Cell Count; D004796:Clinical Enzyme Tests; D003401:Creatine / Q000097:blood; D004909:Erythrocyte Indices / Q000187:drug effects; D005319:Fetal Hemoglobin / Q000187:drug effects; D006454:Hemoglobins / Q000187:drug effects; D006801:Humans; D006918:Hydroxyurea / Q000008:administration & dosage / Q000627:therapeutic use* / Q000633:toxicity; D008099:Liver / Q000201:enzymology; D008297:Male; D017701:Reticulocyte Count; D017086:beta-Thalassemia / Q000188:drug therapy*
D006454:Hemoglobins; D005319:Fetal Hemoglobin; D003401:Creatine; D006918:Hydroxyurea