title: Long-term hydroxyurea therapy in beta-thalassaemia patients.
abstract: 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.
authors.affiliation: Haematology and Haemotherapy Centre, and Department of Internal Medicine, State University of Campinas, Campinas, São Paulo, Brazil.
authors.forename: Erich Vinicius
authors.initials: EV
authors.lastname: de Paula
authors.forename: Carmen Silvia Passos
authors.initials: CS
authors.lastname: Lima
authors.forename: Valder Roberval
authors.initials: VR
authors.lastname: Arruda
authors.forename: Fernando Lopes
authors.initials: FL
authors.lastname: Alberto
authors.forename: Sara Teresinha Ollala
authors.initials: ST
authors.lastname: Saad
authors.forename: Fernando Ferreira
authors.initials: FF
authors.lastname: Costa
mesh_terms: 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*
chemical_list: D006454:Hemoglobins; D005319:Fetal Hemoglobin; D003401:Creatine; D006918:Hydroxyurea