| Hemoglobin switching protocols in thalassemia. Experience with sodium phenylbutyrate and hydroxyurea. | |
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MedLine Citation:
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PMID: 9668530 Owner: NLM Status: MEDLINE |
Abstract/OtherAbstract:
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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. |
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Authors:
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G J Dover |
Publication Detail:
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Type: Journal Article; Research Support, Non-U.S. Gov't; Research Support, U.S. Gov't, P.H.S.; Review |
Journal Detail:
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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:
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Created Date: 1998-08-12 Completed Date: 1998-08-12 Revised Date: 2007-11-14 |
Medline Journal Info:
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Nlm Unique ID: 7506858 Medline TA: Ann N Y Acad Sci Country: UNITED STATES |
Other Details:
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Languages: eng Pagination: 80-6 Citation Subset: IM |
Affiliation:
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Department of Pediatrics, Johns Hopkins University School of Medicine, Baltimore, Maryland 21287, USA. gdover@welchlink.welch.jhu.edu |
Export Citation:
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| MeSH Terms | |
Descriptor/Qualifier:
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Antisickling Agents
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therapeutic use* Blood Transfusion Erythropoietin / blood Fetal Hemoglobin / biosynthesis* Globins / biosynthesis* Humans Hydroxyurea / therapeutic use* Phenylbutyrates / therapeutic use* Transcription, Genetic / drug effects beta-Thalassemia / blood, drug therapy* |
| Grant Support | |
ID/Acronym/Agency:
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R01-H28028//PHS HHS |
| Chemical | |
Reg. No./Substance:
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0/Antisickling Agents; 0/Phenylbutyrates; 11096-26-7/Erythropoietin; 127-07-1/Hydroxyurea; 9004-22-2/Globins; 9034-63-3/Fetal Hemoglobin |
From MEDLINE®/PubMed®, a database of the U.S. National Library of Medicine
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