Document Detail

A two-year pilot trial of hydroxyurea in very young children with sickle-cell anemia.
MedLine Citation:
PMID:  11743503     Owner:  NLM     Status:  MEDLINE    
OBJECTIVE: Hydroxyurea improves hematologic values and decreases vaso-occlusive complications in adults and children with sickle cell anemia (SCA), but has not been tested in infants before the onset of chronic organ dysfunction. We conducted a collaborative pilot trial of hydroxyurea in infants with SCA to assess its (1) feasibility of administration, (2) toxicity, (3) hematologic effects, and (4) effect on spleen function. STUDY DESIGN: Patients with hemoglobin (Hb) SS or Sbeta(0) thalassemia (n = 28, median age 15 months) received hydroxyurea for 2 years at 20 mg/kg/day. Hydroxyurea was temporarily discontinued for predefined toxicity. RESULTS: Seven patients exited the study early: five for noncompliance or refusal to continue, one for mild stroke, and one for fatal splenic sequestration. The predominant toxicity was transient neutropenia, which was usually associated with a viral-like illness. After 2 years of treatment, mean Hb level = 8.8 g/dL and Hb F = 20.3%, both higher than predicted age-specific levels. Radionuclide splenic uptake was absent in 47% of patients at study completion, compared with predicted functional asplenia in 80% of the patients. CONCLUSIONS: Hydroxyurea therapy for infants with SCA is feasible and well tolerated, has hematologic efficacy, and may delay functional asplenia. The potential for hydroxyurea to preserve organ function in SCA should be further evaluated.
W C Wang; L W Wynn; Z R Rogers; J P Scott; P A Lane; R E Ware
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Publication Detail:
Type:  Clinical Trial; Journal Article; Research Support, Non-U.S. Gov't; Research Support, U.S. Gov't, P.H.S.    
Journal Detail:
Title:  The Journal of pediatrics     Volume:  139     ISSN:  0022-3476     ISO Abbreviation:  J. Pediatr.     Publication Date:  2001 Dec 
Date Detail:
Created Date:  2001-12-14     Completed Date:  2002-01-25     Revised Date:  2007-11-14    
Medline Journal Info:
Nlm Unique ID:  0375410     Medline TA:  J Pediatr     Country:  United States    
Other Details:
Languages:  eng     Pagination:  790-6     Citation Subset:  AIM; IM    
Department of Hematology/Oncology, St. Jude Children's Research Hospital, Memphis, Tennessee 38105, USA.
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MeSH Terms
Age Factors
Anemia, Sickle Cell / blood,  complications,  drug therapy*
Antisickling Agents / adverse effects,  therapeutic use*,  toxicity
Blood Cell Count
Child, Preschool
Feasibility Studies
Hematologic Diseases / blood,  chemically induced
Hemoglobins / analysis,  drug effects*
Hydroxyurea / adverse effects,  therapeutic use*,  toxicity
Pilot Projects
Splenic Diseases / blood,  etiology,  prevention & control*
Time Factors
Grant Support
Reg. No./Substance:
0/Antisickling Agents; 0/Hemoglobins; 127-07-1/Hydroxyurea
Comment In:
J Pediatr. 2001 Dec;139(6):763-4   [PMID:  11743496 ]

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

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