Document Detail

Hemoglobin switching protocols in thalassemia. Experience with sodium phenylbutyrate and hydroxyurea.
MedLine Citation:
PMID:  9668530     Owner:  NLM     Status:  MEDLINE    
Homozygous beta thalassemia affects thousands of people around the world. Current management of this condition includes regular transfusion of red cells, which leads to transfusional iron overload requiring chelation therapy: increasing hemoglobin levels while decreasing or eliminating iron overload is therefore a major therapeutic goal in the treatment of thalassemia. Bone marrow transplantation may achieve this goal, but it is not an option for most patients. This study reports on efforts to increase gamma-globin transcription and HbF production using sodium phenylbutyrate (SPB) and hydroxyurea (HU). It was found that 36% (4/11) of all patients or 50% (4/8) of non-transfused patients responded to SPB (increase in Hb levels of 1 g/dL). A positive correlation between baseline serum erythropoietin level and likelihood of response to SPB was observed. Since HU may also increase HbF production, evaluation of combination therapy with these drugs is underway and preliminary results are reported.
G J Dover
Publication Detail:
Type:  Journal Article; Research Support, Non-U.S. Gov't; Research Support, U.S. Gov't, P.H.S.; Review    
Journal Detail:
Title:  Annals of the New York Academy of Sciences     Volume:  850     ISSN:  0077-8923     ISO Abbreviation:  Ann. N. Y. Acad. Sci.     Publication Date:  1998 Jun 
Date Detail:
Created Date:  1998-08-12     Completed Date:  1998-08-12     Revised Date:  2007-11-14    
Medline Journal Info:
Nlm Unique ID:  7506858     Medline TA:  Ann N Y Acad Sci     Country:  UNITED STATES    
Other Details:
Languages:  eng     Pagination:  80-6     Citation Subset:  IM    
Department of Pediatrics, Johns Hopkins University School of Medicine, Baltimore, Maryland 21287, USA.
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MeSH Terms
Antisickling Agents / therapeutic use*
Blood Transfusion
Erythropoietin / blood
Fetal Hemoglobin / biosynthesis*
Globins / biosynthesis*
Hydroxyurea / therapeutic use*
Phenylbutyrates / therapeutic use*
Transcription, Genetic / drug effects
beta-Thalassemia / blood,  drug therapy*
Grant Support
R01-H28028//PHS HHS
Reg. No./Substance:
0/Antisickling Agents; 0/Phenylbutyrates; 11096-26-7/Erythropoietin; 127-07-1/Hydroxyurea; 9004-22-2/Globins; 9034-63-3/Fetal Hemoglobin

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