Adherence outcomes of a liquid hydroxyurea delivery program in a pediatric population

Hydroxyurea remains underutilized in the pediatric sickle cell population despite its well-known efficacy in decreasing sickle cell complications and hospitalizations. Access to refills and liquid formulation remains a critical barrier to adherence to hydroxyurea regimens. This study was undertaken to determine the clinical impact of home-delivering compounded liquid hydroxyurea (LHU) to pediatric patients with sickle cell disease.

A retrospective cohort study was conducted using electronic health records and pharmacy databases. Pediatric patients younger than 21 years of age at the time of hydroxyurea initiation from March 2016 to July 2020 who received compounded LHU from Boston Medical Center Pharmacy were included. The primary outcomes of the study were drug adherence (assessed by evaluating the proportion of days covered [PDC]), rates of acute care utilization before and after enrolling in the LHU delivery program.

The final cohort included 41 patients, showing a significant decrease in hospitalizations (p = .01) and acute chest syndrome episodes (p = .03) after the initiation of the LHU delivery program. In comparing hydroxyurea-naïve patients with those previously exposed to hydroxyurea, the latter group had lower hospitalization rates (p = .01), fewer vaso-occlusive event (VOE) episodes (p = .02), and fewer emergency department (ED) visits (p = .01). The median PDC value 1 year post initiation of LHU was 95.

Home delivery of compounded LHU from the pharmacy to pediatric sickle cell disease patients improved access to hydroxyurea, and was linked to reduced hospitalizations and acute chest syndrome episodes. This advancement in cost savings and improved patient outcomes is a significant step forward in pediatric hematology. By overcoming access barriers, home delivery programs can greatly enhance outcomes among pediatric patients with sickle cell disease.