Prevalence and Trends of Medication Utilization Among Children With Sickle Cell Disease in the United States: A Retrospective Cohort Study

OBJECTIVES: The medication management of children with sickle cell disease (SCD) has not been well described in the literature. Further, changes in the medication management of children with SCD since the release of National Heart, Lung, and Blood Institute (NHLBI) SCD treatment guidelines in 2014 have not been assessed. This study aimed to quantify the real-world medication management of children with SCD in the United States, including trends in medication utilization over time and since the 2014 NHLBI SCD treatment guidelines, overall and by patient demographics.

METHODS: This retrospective cohort study analyzed Optum's de-identified Clinformatics® Data Mart database, focusing on patients aged between 1-17 years diagnosed with SCD based on the International Classification of Diseases diagnosis codes, from January 1, 2010, to December 31, 2018. Prevalence and changes in medication utilization trends were assessed using Joinpoint regression, calculating annual percent changes (APCs) and average annual percent change (AAPC). Stratified time trends were evaluated by patient age, sex, race/ethnicity, region, and household income level.

RESULTS: Over the 8-year study period, we identified 1,868 children with SCD in the United States with a mean age of 8.8 years (standard deviation [SD] 5 years). Throughout the study period, 29.5% of the study population filled hydroxyurea prescriptions, demonstrating a significant annual increase of 8.8% (p<0.001). For pain management, about 48.3% of the study population were treated with opiate agonists with a significant average annual decrease of 3.2% (p=0.002) while 19.8% were treated with non-steroidal anti-inflammatory drugs, with a significant average annual increase of 4.0% (p=0.010).

CONCLUSIONS: Medication utilization increased for hydroxyurea, oxycodone, and nonsteroidal anti-inflammatory drugs over the study period. However, there is still a need for improvement in the prophylactic utilization of hydroxyurea and penicillin. Future studies need to assess the relationship between improved medication management and clinical outcomes in patients with SCD.