Document Detail

Hydroxyurea increases hemoglobin F levels and improves the effectiveness of erythropoiesis in beta-thalassemia/hemoglobin E disease.
MedLine Citation:
PMID:  8562958     Owner:  NLM     Status:  MEDLINE    
Hydroxyurea (HU) is one of several agents that have been shown to enhance hemoglobin (Hb) F levels in patients with sickle cell disease and may be useful as a therapy for beta-globinopathies. However, limited information exists on the effects of HU in patients with thalassemia. Accordingly, we examined the hematologic effects of orally administered HU in 13 patients with beta-thalassemia/Hb E, including four patients who had been splenectomized. These patients were treated with escalating doses (final range, 10 to 20 mg/kg/d) for 5 months and were observed in the outpatient hematology clinic every 2 to 4 weeks. Complete blood counts including reticulocyte counts, amounts of Hb E and Hb F, G gamma:A gamma and alpha:non-alpha globin biosynthetic ratios were evaluated before and during treatment. Almost all patients responded with an average increase of 33% in Hb F levels, from a mean (+/- SD) of 42% +/- 11% to 56% +/- 8% (P < .0001), and a reciprocal decline in the percentage of Hb E from 59% +/- 9% to 49% +/- 8% (P < .001). Reticulocytosis was decreased from a mean (+/- SD) of 18.0% +/- 15.6% to 11.7% +/- 9.1% (P < .05); there was also a slight (10%) but statistically significant increase in hemoglobin levels and an improved balance in alpha:non-alpha globin chains ratios. The side effects were minimal in most patients, although these patients tended to tolerate a lower dose of HU before significant myelosuppression than has been our previous experience in sickle cell disease. One splenectomized patient died of sepsis during the trial. We conclude that increased Hb F production in beta-thalassemia/Hb E patients, with an improvement in the alpha:non-alpha globin ratios and, probably, the effectiveness of erythropoiesis, can be achieved using HU. Longer trials of HU in this population, including at other doses and in combination with other agents, appear warranted.
S Fucharoen; N Siritanaratkul; P Winichagoon; J Chowthaworn; W Siriboon; W Muangsup; S Chaicharoen; N Poolsup; B Chindavijak; P Pootrakul; A Piankijagum; A N Schechter; G P Rodgers
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Publication Detail:
Type:  Clinical Trial; Journal Article; Research Support, Non-U.S. Gov't    
Journal Detail:
Title:  Blood     Volume:  87     ISSN:  0006-4971     ISO Abbreviation:  Blood     Publication Date:  1996 Feb 
Date Detail:
Created Date:  1996-03-01     Completed Date:  1996-03-01     Revised Date:  2007-11-15    
Medline Journal Info:
Nlm Unique ID:  7603509     Medline TA:  Blood     Country:  UNITED STATES    
Other Details:
Languages:  eng     Pagination:  887-92     Citation Subset:  AIM; IM    
Department of Medicine, Faculty of Medicine, Siriraj Hospital, Bangkok, Thailand.
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MeSH Terms
Blood Transfusion
Combined Modality Therapy
Erythrocyte Count / drug effects
Erythropoiesis / drug effects*
Fetal Hemoglobin / biosynthesis*,  genetics
Gene Expression Regulation / drug effects*
Globins / biosynthesis*,  genetics
Hemoglobin E / genetics*
Hemoglobinuria / genetics,  surgery,  therapy*
Hydroxyurea / pharmacology*
Immunologic Factors / pharmacology*
Middle Aged
beta-Thalassemia / genetics,  surgery,  therapy*
Reg. No./Substance:
0/Immunologic Factors; 127-07-1/Hydroxyurea; 9004-22-2/Globins; 9034-61-1/Hemoglobin E; 9034-63-3/Fetal Hemoglobin

From MEDLINE®/PubMed®, a database of the U.S. National Library of Medicine

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