Hydroxyurea Remains Underused in Children With Sickle Cell Disease

By Reuters Staff

June 24, 2019

NEW YORK (Reuters Health) - Despite recent increases in use of hydroxyurea for sickle cell disease (SCD), only about one in four children with SCD received hydroxyurea in 2015, according to an analysis of Medicaid data.

Recent studies have clearly demonstrated a benefit of hydroxyurea in SCD. And guidelines put forth by the National Heart, Lung, and Blood Institute (NHLBI) in 2014 recommend hydroxyurea be offered to children aged 9 months or older with sickle cell anemia regardless of clinical severity and referred for specialist evaluation in other forms of SCD if pain interferes with quality of life.

To investigate usage patterns, a team led by Dr. David Brousseau of the Medical College of Wisconsin, in Milwaukee, analyzed data from Truven Health Analytics-IBM Watson Health MarketScan Medicaid database from 2009 to 2015. An average of 5,138 children with SCD were included in each year of the analysis.

Hydroxyurea use increased from 14.3% in 2009 to 28.2% in 2015 (P<0.001), the researchers report in a paper online today in Pediatrics.

However, despite the increase, almost two-thirds of children with sickle cell anemia and three-quarters of those with SCD did not fill a single prescription for hydroxyurea in 2015, they say.

"Although it is difficult to determine the percentage of children with SCD who should receive hydroxyurea, the small increase in the rate of prescribing in our study does not match the increase in evidence of the effectiveness of hydroxyurea," the researchers write.

During the study period, there was only a slight decrease in overall acute-care-visit rates across the sickle cell population, from 1.20 acute care visits per person-year in 2009 to 1.04 in 2015. The drop in acute-care visits was exclusively in the youngest age group (1 to 6 years) and oldest age group (17 to 19 years).

"There has been a small increase in hydroxyurea use but no consistent pattern of decreased emergency department or hospital use, suggesting that the benefits of hydroxyurea for preventing acute pain in SCD are not translating to the community," the researchers say.

"These findings illustrate the ongoing barriers to hydroxyurea use in a population-based cohort of Medicaid-enrolled children with SCD and the need to better understand how to translate the benefits to children managed in clinical trials to nonacademic settings," they conclude.

Dr. Brousseau did not respond to a request for comment by press time.

The study had no specific funding and the authors have declared no relevant conflicts of interest.

SOURCE: https://bit.ly/2RBcyhX

Pediatrics 2019