Medication Utilization Among Children With Sickle Cell Disease in the United States.

Background and objective: The medication management of children with sickle cell disease (SCD), especially since the release of the 2014 National Heart, Lung, and Blood Institute (NHLBI) SCD treatment guidelines, is not well described in the published literature. This study assessed medication utilization trends overall and by patient demographics.

Methods: This retrospective study examined children aged 1-17 years diagnosed with SCD from January 1, 2010 to December 31, 2018, in Optum's de-identified Clinformatics Data Mart database. Changes in medication utilization over time were assessed using Joinpoint regression. Stratified time trends were evaluated by patient age, sex, race/ethnicity, region, and household income level.

Results: Over the 8-year study period, a total of 1868 children with SCD were identified. Hydroxyurea prescriptions increased 8.8%, on average, each year of the study period (p < 0.001), indicating rising adherence to recommended disease-modifying strategies. Shifts in opioid prescribing showed increased oxycodone use (6.3% average annual increase, p < 0.001) and decreased acetaminophen-codeine use (24.3% average annual decrease, p < 0.001). Notably, non-steroidal anti-inflammatory drug (NSAID) use increased by 4.0% annually (p = 0.010), reflecting shifts toward alternative pain management strategies. Significant shifts in medication utilization, overall and by patient demographics, were observed since the 2014 NHLBI SCD treatment guidelines were released.

Conclusions: Our study findings underscore the dynamic nature of SCD management in children, and highlight the need for continuous evaluation of treatment guideline adherence, as well as treatment effectiveness and safety with the newer SCD medications and treatment guidelines, to optimize clinical outcomes among this vulnerable patient population.