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Adherence outcomes of a liquid hydroxyurea delivery program in a pediatric population
Maria A Sacta1,2,3, Supriya Sarvode1,2,4, Amanuel Kehasse5
1Department of Pediatrics, Boston University Chobanian and Avedisian School of Medicine, Boston, Massachusetts, USA.
Pediatric Blood & Cancer
|September 26, 2024
Summary
Home delivery of liquid hydroxyurea (LHU) significantly reduced hospitalizations and acute chest syndrome in pediatric sickle cell disease patients. This program improved medication adherence and access, enhancing patient outcomes.
Area of Science:
- Pediatric Hematology
- Sickle Cell Disease Management
- Pharmacological Interventions
Background:
- Hydroxyurea is underutilized in pediatric sickle cell disease (SCD) patients.
- Access to medication refills and liquid formulations are key barriers to hydroxyurea adherence.
- Home delivery of compounded liquid hydroxyurea (LHU) was explored to address these barriers.
Purpose of the Study:
- To determine the clinical impact of home-delivered compounded LHU on pediatric SCD patients.
- To assess changes in drug adherence and acute care utilization.
- To evaluate the effectiveness of LHU home delivery in improving SCD management.
Main Methods:
- Retrospective cohort study of pediatric patients (<21 years) receiving LHU from March 2016 to July 2020.
- Utilized electronic health records and pharmacy databases.
- Assessed drug adherence via proportion of days covered (PDC) and analyzed acute care utilization rates before and after LHU program enrollment.
Main Results:
- A cohort of 41 patients showed significant reductions in hospitalizations (p=0.01) and acute chest syndrome episodes (p=0.03) after LHU initiation.
- Patients previously on hydroxyurea had lower hospitalization rates, fewer vaso-occlusive events (VOE), and fewer ED visits.
- Median PDC at 1 year post-LHU initiation was 95%.
Conclusions:
- Home delivery of compounded LHU improved access to hydroxyurea for pediatric SCD patients.
- The LHU home delivery program was associated with reduced hospitalizations and acute chest syndrome.
- This delivery model offers cost savings and improved patient outcomes, enhancing SCD care.
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