TY - JOUR
T1 - A two-year pilot trial of hydroxyurea in very young children with sickle-cell anemia
AU - Wang, Winfred C.
AU - Wynn, Lynn W.
AU - Rogers, Zora R.
AU - Scott, J. Paul
AU - Lane, Peter A.
AU - Ware, Russell E.
N1 - Funding Information:
Supported in part by the American Lebanese Syrian Associated Charities and by General Clinical Research Center grant Nos. M01RR00058 and M01RR00069, National Center for Research Resources, National Institutes of Health. Submitted for publication Mar 7, 2001; revision received May 16, 2001; accepted Aug 14, 2001. Reprint requests: Winfred C. Wang, MD, Department of Hematology/Oncology, St. Jude Children’s Research Hospital, Room R-6002, 332 N Lauderdale, Memphis, TN 38105. Copyright © 2001 by Mosby, Inc. 0022-3476/2001/$35.00 + 0 9/21/119590 doi:10.1067/mpd.2001.119590
PY - 2001
Y1 - 2001
N2 - 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.
AB - 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.
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U2 - 10.1067/mpd.2001.119590
DO - 10.1067/mpd.2001.119590
M3 - Article
C2 - 11743503
AN - SCOPUS:0035666461
SN - 0022-3476
VL - 139
SP - 790
EP - 796
JO - Journal of Pediatrics
JF - Journal of Pediatrics
IS - 6
ER -