Document Detail


Hydroxyurea for sickle cell anemia: what have we learned and what questions still remain?
MedLine Citation:
PMID:  21372708     Owner:  NLM     Status:  MEDLINE    
Abstract/OtherAbstract:
PURPOSE OF REVIEW: Sickle cell anemia (SCA) is a well characterized severe hematological disorder with substantial morbidity and early mortality. Hydroxyurea is a potent inducer of fetal hemoglobin, and evidence over the past 25 years has documented its laboratory and clinical efficacy for both adults and children with SCA.
RECENT FINDINGS: The phase III study of hydroxyurea in infants (BABY HUG) has just been completed and preliminary results indicate equivocal benefits for organ protection during the 2-year treatment period, but significant benefits for pain, acute chest syndrome, hospitalizations, and transfusions. Three new reports document the benefits of hydroxyurea on reducing mortality in SCA: two adult trials (LaSHS and MSH) and one pediatric study (Brazilian cohort). Recent results from the HUSTLE protocol suggest minimal genotoxicity or carcinogenicity with long-term hydroxyurea exposure.
SUMMARY: The potential utility of hydroxyurea for all patients with SCA is clear and indisputable. With decades of accumulated evidence and documented efficacy with an acceptable long-term safety profile, it is time to consider hydroxyurea treatment the standard of care for all young patients with SCA. Exporting our knowledge and experience with hydroxyurea to developing nations with large medical burdens from SCA can help relieve global suffering from this condition.
Authors:
Patrick T McGann; Russell E Ware
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Publication Detail:
Type:  Journal Article; Research Support, N.I.H., Extramural; Review    
Journal Detail:
Title:  Current opinion in hematology     Volume:  18     ISSN:  1531-7048     ISO Abbreviation:  Curr. Opin. Hematol.     Publication Date:  2011 May 
Date Detail:
Created Date:  2011-04-14     Completed Date:  2011-10-17     Revised Date:  2013-06-30    
Medline Journal Info:
Nlm Unique ID:  9430802     Medline TA:  Curr Opin Hematol     Country:  United States    
Other Details:
Languages:  eng     Pagination:  158-65     Citation Subset:  IM    
Affiliation:
Department of Hematology, St. Jude Children's Research Hospital, Memphis, Tennessee, USA. patrick.mcgann@stjude.org
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MeSH Terms
Descriptor/Qualifier:
Anemia, Sickle Cell / drug therapy*,  metabolism
Humans
Hydroxyurea / metabolism,  therapeutic use*
Grant Support
ID/Acronym/Agency:
N01-HB07155/HB/NHLBI NIH HHS; R01-HL090941/HL/NHLBI NIH HHS; T32-CA070089/CA/NCI NIH HHS; U01-HL078787/HL/NHLBI NIH HHS; U54 HL070590-07/HL/NHLBI NIH HHS; U54-HL07090/HL/NHLBI NIH HHS
Chemical
Reg. No./Substance:
127-07-1/Hydroxyurea
Comments/Corrections

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