Document Detail


Efficacy of hydroxyurea in providing transfusion independence in β-thalassemia.
MedLine Citation:
PMID:  21602718     Owner:  NLM     Status:  MEDLINE    
Abstract/OtherAbstract:
BACKGROUND: Packed red blood cell (PRC) transfusion with iron chelation is the mainstay of treatment for β-thalassemia major. This prospective interventional trial serves as a follow up to our similar earlier study that evaluated the efficacy and safety of hydroxyurea (HU) in minimizing PRC transfusions in patients with β-thalassemia major.
METHODS: One hundred fifty-two patients with β-thalassemia major received HU at a mean dose of 16 mg/kg/d. The results were analyzed at the end of 24 months. Transfusion requirement during the 6 months preceding the study was considered as the control.
RESULTS: One hundred forty-six of 152 patients were evaluated after 24 months of follow up; 6 patients were either lost to follow-up or withdrew consent. Grade 1 myelosuppression was observed in 4 patients and diarrhea in 2 patients. Sixty children (41%) did not require any transfusion after using HU; 57 patients (39%) showed partial response with greater than 50% reduction in PRC transfusion; and 29 patients (20%) were nonresponders with less than 50% reduction in PRC transfusion. The mean volume of PRC transfused was reduced for all patients.
CONCLUSIONS: HU was found to be safe in patients with β-thalassemia major, and resulted in the reduction of transfusion requirement and in an increase in the interval between transfusions.
Authors:
Saqib H Ansari; Tahir S Shamsi; Mushtaq Ashraf; Kousar Perveen; Tasneem Farzana; Muneera Bohray; Sajida Erum; Tabassum Mehboob
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Publication Detail:
Type:  Controlled Clinical Trial; Journal Article    
Journal Detail:
Title:  Journal of pediatric hematology/oncology     Volume:  33     ISSN:  1536-3678     ISO Abbreviation:  J. Pediatr. Hematol. Oncol.     Publication Date:  2011 Jul 
Date Detail:
Created Date:  2011-06-22     Completed Date:  2011-09-01     Revised Date:  2011-10-06    
Medline Journal Info:
Nlm Unique ID:  9505928     Medline TA:  J Pediatr Hematol Oncol     Country:  United States    
Other Details:
Languages:  eng     Pagination:  339-43     Citation Subset:  IM    
Affiliation:
Department of Pediatric Hematology, National Institute of Blood Diseases, Karachi, Pakistan. ansarisaqib@hotmail.com
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MeSH Terms
Descriptor/Qualifier:
Adolescent
Child
Child, Preschool
Erythrocyte Transfusion / utilization*
Female
Follow-Up Studies
Humans
Hydroxyurea / administration & dosage*,  adverse effects
Infant
Male
Nucleic Acid Synthesis Inhibitors / administration & dosage*,  adverse effects
Treatment Outcome
Young Adult
beta-Thalassemia / drug therapy*
Chemical
Reg. No./Substance:
0/Nucleic Acid Synthesis Inhibitors; 127-07-1/Hydroxyurea

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


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