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Hydroxycarbamide Effective for Infant Sickle-Cell Anemia

Last Updated: May 13, 2011.

Hydroxycarbamide therapy may be safe and effective for treating infants with sickle-cell anemia, according to a study published in the May 14 issue of The Lancet.


Treatment may reduce pain and dactylitis, increase hemoglobin and fetal hemoglobin levels

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FRIDAY, May 13 (HealthDay News) -- Hydroxycarbamide therapy may be safe and effective for treating infants with sickle-cell anemia, according to a study published in the May 14 issue of The Lancet.

Winfred C. Wang, M.D., from St. Jude Children's Research Hospital in Memphis, Tenn., and colleagues examined the impact of hydroxycarbamide therapy on organ dysfunction and adverse clinical events in the treatment of infants with sickle-cell anemia. Data were collected from 193 children aged 9 to 18 months, who had hemoglobin SS or hemoglobin Sβ0thalassemia and were randomly allocated to receive liquid hydroxycarbamide (20 mg/kg per day) (96 children) or placebo (97 children) for two years. Splenic function and renal function were the primary study end points. Additional assessments were carried out at study visits every two to four weeks and included blood counts, fetal hemoglobin concentration, chemistry profiles, and neurodevelopment.

The investigators found that there were no significant differences between the treatment and placebo groups in the primary end points. Pain was significantly reduced in the hydroxycarbamide group, with 177 events in 62 patients compared to 375 events in 75 patients in the placebo group. There were also fewer dactylitis events and some evidence of reduced acute chest syndrome, hospitalization rates, and transfusion in the hydroxycarbamide group. Hemoglobin and fetal hemoglobin increased and white count decreased in the hydroxycarbamide group. The only adverse effect associated with treatment was mild to moderate neutropenia.

"On the basis of the safety and efficacy data from this trial, hydroxycarbamide can now be considered for all very young children with sickle-cell anemia," the authors write.

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