Hydroxyurea decreased hospitalization, ER visits in children with sickle cell disease

ORLANDO — Hydroxyura has been shown to be effective in reducing complications and death in children with sickle cell disease, according to findings presented at a press conference here.

These data are the first to show an association between hydroxyurea and reduced mortality in children with the disease, Clarisse Lobo, MD, senior study author and medical director at the Instituto Estadual de Hematologia Arthur de Siqueira Cavalcanti (HEMORIO) in Rio de Janeiro, Brazil, said.

Hydroxyurea treatment in children for SCD is currently off-label in the US.

Researchers compared 10-year clinical and survival data from the institution among children treated with hydroxyurea vs. untreated children. Since 2000, of 1,643 children with SCD between the ages of 3 and 18 years, 224 were treated with hydroxyurea. The median age at treatment initiation was 6 years.

The median hydroxyurea dose was 15 mg/kg/day; median treatment duration was 1.9 years. Of those treated, the incidence of complications from SCD significantly declined in the first year. Researchers noted significant reductions in hospitalizations (67.9%; P=.002), ER visits (48.7%; P=<.001) and transfusions (36.3%; P=.001) in children receiving hydroxyurea treatment. No serious adverse events were noted.

Cumulative OS was 70.1%. At 10 years of age, survival was 99.4% in treated children vs. 97.4% in untreated children. Older age resulted in a greater difference in survival rate: at 17.9 years, the rate was 97.4% in treated children vs. 66.3% in untreated children.

Forty-six deaths occurred among all patients in the group; 44 in untreated children and two in treated children, both of those attributable to acute chest syndrome. Children who did not receive hydroxyurea treatment were 4.6 times more likely to die than those who received treatment (P=.03).

“These data demonstrate that hydroxyurea appears to be effective in reducing the incidence of acute events, such as hospitalization and transfusions in children with sickle cell disease, but most importantly, it can prevent death,” Lobo said in a press release. “Although these data were based on patients in Brazil, hematologists across the globe cans use these results as they approach the treatment of their pediatric patients who suffer from sickle cell disease.”

For more information:

Lobo C. #843. Presented at: The 52^nd ASH Annual Meeting and Exposition; Dec. 4-7, 2010; Orlando, Fl.

[The study] underscores there are some benefits in terms of reducing hospitalizations and ER visits. The issue of mortality is one we don’t have a lot of information about in children...we’re on the right path to encouraging non-academic providers to consider this as standard of care for their patients.

- Alexis Thompson, MD, PhD

Director of hematology services at Children’s Memorial Hospital and associate professor of pediatrics,

Northwestern University Feinberg School of Medicine in Chicago, IL

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