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Updated: May 16, 2025

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Medication Utilization Among Children With Sickle Cell Disease in the United States
Abiodun John Ologunowa1, Kelly L Matson1,2, Jung Eun Lee3
1Department of Pharmacy Practice and Clinical Research, College of Pharmacy, University of Rhode Island, Kingston, Rhode Island, USA.
Insights
Medication use in children with sickle cell disease (SCD) has changed significantly, with increased hydroxyurea and NSAID prescriptions, and shifts in opioid use. These trends reflect evolving treatment guidelines and management strategies for SCD in pediatric patients.
Area of Science:
- Pediatric Hematology
- Pharmacology
- Public Health
Background:
- Medication management for pediatric sickle cell disease (SCD) is not well-documented, particularly after the 2014 NHLBI guidelines.
- Understanding medication utilization trends is crucial for optimizing care in this population.
Purpose of the Study:
- To assess medication utilization trends in children with SCD.
- To analyze these trends by patient demographics following the 2014 NHLBI SCD treatment guidelines.
Main Methods:
- Retrospective analysis of de-identified electronic health data from 2010-2018.
- Included children aged 1-17 years diagnosed with SCD.
- Joinpoint regression used to assess changes in medication use over time, stratified by demographics.
Main Results:
- Hydroxyurea prescriptions increased annually (8.8%), indicating better adherence to disease-modifying strategies.
- Opioid prescribing shifted towards oxycodone and away from acetaminophen-codeine.
- NSAID use increased annually (4.0%), suggesting a move towards alternative pain management.
Conclusions:
- Pediatric SCD management is dynamic, with observed shifts in medication use since 2014.
- Continuous evaluation of guideline adherence, medication effectiveness, and safety is needed.
- Optimizing clinical outcomes requires ongoing assessment of treatment strategies for children with SCD.
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.
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