A two-year pilot trial of hydroxyurea in very young children with sickle-cell anemia

Winfred C. Wang, Lynn W. Wynn, Zora R. Rogers, J. Paul Scott, Peter A. Lane, Russell E. Ware

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120 Citations (Scopus)

Abstract

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 Sβ0 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.

Original languageEnglish (US)
Pages (from-to)790-796
Number of pages7
JournalJournal of Pediatrics
Volume139
Issue number6
DOIs
StatePublished - 2001

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Hydroxyurea
Sickle Cell Anemia
Sickle Hemoglobin
Fetal Hemoglobin
Thalassemia
Neutropenia
Radioisotopes
Hemoglobins
Spleen
Stroke
Therapeutics

ASJC Scopus subject areas

  • Pediatrics, Perinatology, and Child Health

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A two-year pilot trial of hydroxyurea in very young children with sickle-cell anemia. / Wang, Winfred C.; Wynn, Lynn W.; Rogers, Zora R.; Scott, J. Paul; Lane, Peter A.; Ware, Russell E.

In: Journal of Pediatrics, Vol. 139, No. 6, 2001, p. 790-796.

Research output: Contribution to journalArticle

Wang, Winfred C. ; Wynn, Lynn W. ; Rogers, Zora R. ; Scott, J. Paul ; Lane, Peter A. ; Ware, Russell E. / A two-year pilot trial of hydroxyurea in very young children with sickle-cell anemia. In: Journal of Pediatrics. 2001 ; Vol. 139, No. 6. pp. 790-796.
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