A two-year pilot trial of hydroxyurea in very young children with sickle-cell anemia. J Pediatr 2001 Dec;139(6):790-6
Date
12/18/2001Pubmed ID
11743503DOI
10.1067/mpd.2001.119590Scopus ID
2-s2.0-0035666461 (requires institutional sign-in at Scopus site) 153 CitationsAbstract
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.
Author List
Wang WC, Wynn LW, Rogers ZR, Scott JP, Lane PA, Ware REMESH terms used to index this publication - Major topics in bold
Age FactorsAnemia, Sickle Cell
Antisickling Agents
Blood Cell Count
Child, Preschool
Feasibility Studies
Female
Hematologic Diseases
Hemoglobins
Humans
Hydroxyurea
Infant
Male
Pilot Projects
Splenic Diseases
Time Factors
