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Blood, Vol. 94 No. 5 (September 1), 1999:
pp. 1550-1554
Safety of Hydroxyurea in Children With Sickle Cell Anemia: Results of
the HUG-KIDS Study, a Phase I/II Trial
Thomas R. Kinney,
Ronald W. Helms,
Erin E. O'Branski,
Kwaku Ohene-Frempong,
Winfred Wang,
Charles Daeschner,
Elliott Vichinsky,
Rupa Redding-Lallinger,
Beatrice Gee,
Orah S. Platt, and
Russell E. Ware for the Pediatric Hydroxyurea Group
From Duke Pediatric Sickle Cell Program, Duke Children's Hospital,
Durham, NC; the Department of Biostatistics and Frank Porter Graham
Child Development Center, University of North Carolina at Chapel Hill,
Chapel Hill, NC; Comprehensive Sickle Cell Center, The Children's
Hospital of Philadelphia, Philadelphia, PA; St Jude Children's
Research Hospital, Memphis, TN; Pitt Memorial Hospital, East Carolina
University, Greenville, NC; Sickle Cell Center of Northern California,
Oakland Children's Hospital, Oakland, CA; the University of North
Carolina Pediatric Sickle Cell Program, University of North Carolina at
Chapel Hill, Chapel Hill, NC; and the Department of Medicine,
Children's Hospital, Harvard Medical School, Boston, MA.
Previous studies have determined the short-term
toxicity profile, laboratory changes, and clinical efficacy associated
with hydroxyurea (HU) therapy in adults with sickle cell anemia. The safety and efficacy of this agent in pediatric patients with sickle cell anemia has not been determined. Children with sickle cell anemia,
age 5 to 15 years, were eligible for this multicenter Phase I/II trial.
HU was started at 15 mg/kg/d and escalated to 30 mg/kg/d unless the
patient experienced laboratory toxicity. Patients were monitored by
2-week visits to assess compliance, toxicity, clinical adverse events,
growth parameters, and laboratory efficacy associated with HU
treatment. Eighty-four children were enrolled between December 1994 and
March 1996. Sixty-eight children reached maximum tolerated dose (MTD)
and 52 were treated at MTD for 1 year. Significant hematologic changes
included increases in hemoglobin concentration, mean corpuscular
volume, mean corpuscular hemoglobin, and fetal hemoglobin parameters,
and decreases in white blood cell, neutrophil, platelet, and
reticulocyte counts. Laboratory toxicities typically were mild,
transient, and were reversible upon temporary discontinuation of HU. No
life-threatening clinical adverse events occurred and no child
experienced growth failure. This Phase I/II trial shows that HU therapy
is safe for children with sickle cell anemia when treatment was
directed by a pediatric hematologist. HU in children induces similar
laboratory changes as in adults. Phase III trials to determine if HU
can prevent chronic organ damage in children with sickle cell anemia are warranted.
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A patient on hydroxyurea for sickle cell disease who developed an opportunistic infection
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Blood 2002 100: 363-364.
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