In the Journals

Hydroxyurea use remains low in children with severe sickle cell disease

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May 18, 2016

Despite limited alternative treatment options, many hospitalized children with clinically severe sickle cell disease still do not use hydroxyurea, according to results of a retrospective analysis.

In 2014, the National Heart, Lung and Blood Institute issued an expert panel guideline recommending hydroxyurea be offered to all children with hemoglobin SS and S beta0 sickle cell disease and that it be considered an option for children with clinically severe hemoglobin SC and S beta+ disease genotypes.

Susan Creary

Susan E. Creary

Although previous data have shown that the length of hospital stay among children and adults with sickle cell disease has decreased significantly since hydroxyurea was approved by the FDA in 1998, the actual impact of hydroxyurea on hospitalized children had not been known.

Susan E. Creary, MD, MSc, assistant professor of pediatric hematology and oncology and a physician in the hematology/oncology/BMT clinic at Nationwide Children’s Hospital at Ohio State University, and colleagues conducted this study to determine the rate of hydroxyurea use in hospitalized children and those with clinically severe disease, as well as to determine if hydroxyurea use improved hospitalization outcomes.

The researchers identified 2,665 patients with sickle cell disease aged 2 to 18 years who were discharged from one of the 42 participating hospitals in the Pediatric Health Information System database between Jan. 2011 and Sept. 2014.

From that cohort, approximately 80% had an inpatient code that indicated they were prescribed hydroxyurea during their admission. That included 81.8% of patients with the SS genotype, 84.2% with the sickle beta thalassemia, 65.5% with SC genotype, and 82.1% of patients with an unspecified genotype.

A significantly greater proportion of hydroxyurea nonusers had a recent ICU admission compared with hydroxyurea users (30.1% vs. 18.7%; P < .001).

Additionally, more nonusers had a history of three or more hospital admissions compared with hydroxyurea users (33.9%; 21.5%; P < .001).

However, after propensity score weighting, researchers observed no significant differences in length of stay, prevalence of admission to the ICU or prevalence of transfusions.

The researchers identified some limitations with their study, specifically the potential for coding errors in the database. Another limitation of the database was the lack of distinction between genotypes, which prevented the researchers from using them in the propensity score weighting.

Additionally, the researchers were unable to ascertain whether hydroxyurea use was initiated during their hospitalization or if it was accidentally not ordered.

Finally, the researchers were unable to measure if hydroxyurea had a constant effect on patients because the database lacks information on dose, therapy duration, response or adherence.

“Hospitalized children with sickle cell disease frequently use hydroxyurea. However, hydroxyurea is not utilized by many children with clinically severe sickle cell disease who have limited other treatment options,” Creary and colleagues wrote.
Our results support consideration of hydroxyurea in children with sickle cell disease to prevent their hospitalization, rather than as a treatment to improve their hospitalization outcomes,” they added. “To improve the care of hospitalized children with sickle cell disease, additional treatments are needed to reduce their length of stay, ICU admissions and need for erythrocyte transfusions.” – by Anthony SanFilippo

Disclosure: The researchers report no relevant financial disclosures.

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