ORIGINAL RESEARCH article
Front. Pharmacol.
Sec. Pharmacoepidemiology
HYDROXYUREA UTILIZATION AMONG INDIVIDUALS WITH SICKLE CELL DISEASE IN TENNESSEE: A POOLED ANALYSIS OF CLAIMS DATA
Provisionally accepted
Ayesha Mukhopadhyay1
Matthew Smeltzer1
Judith Dudley2
Sherif Badawy3Joacy G Mathias4
Allison Plaxco1Meredith A Ray1Andrew D. Wiese2Walter I Stevens1James G Gurney1Chinonyelum Nwosu5Jerlym S Porter5
Lisa Klesges6
Jane Hankins5William O Cooper2*- 1The University of Memphis, Memphis, United States
- 2Vanderbilt University Medical Center, Nashville, United States
- 3Ann & Robert H Lurie Children's Hospital of Chicago, Chicago, United States
- 4University of North Carolina Asheville, Asheville, United States
- 5St Jude Children's Research Hospital, Memphis, United States
- 6Washington University in St Louis School of Medicine, St. Louis, United States
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Abstract Importance: Hydroxyurea reduces severe disease among individuals living with sickle cell disease (SCD). These individuals experience high acute care utilization, but their patterns of hydroxyurea utilization association with healthcare utilization are not well investigated. Objective: To determine the association between hydroxyurea use and healthcare utilization among individuals with SCD in Tennessee (TN). Design: We conducted a population-based, retrospective cohort study of individuals with SCD using secondary data analysis of Tennessee Medicaid, Medicare, and Blue Cross Blue Shield of Tennessee (BCBS-TN). Participants: 4,901 individuals with SCD were included. Exposure: Hydroxyurea adherence was estimated using medication possession ratio (MPR). Main Outcomes and Measures: Incidence rate ratios of hospitalizations, emergency department visits, and mortality were calculated using Negative Binomial Models. Results: The prevalence of hydroxyurea prescription dispensation for the state was low (21% for TennCare, 21% for Medicare, and 17% for BCBS-TN). In TennCare and BCBS-TN those younger than 18 had more hydroxyurea utilization and individuals with HbSS or HbSβ0thalassemia filled more hydroxyurea than those with other subtypes (30.5% in TennCare and 23.4% in BCBS-TN). Medication possession ratio (MPR) for the entire state was 19.7%. There was a dose-response relationship between hydroxyurea adherence and incidence of acute healthcare utilization except in 18–25-year-olds. We also found lower mortality in those with higher hydroxyurea adherence. Conclusions and Relevance: In our pooled state-wide analysis, hydroxyurea MPR was low. Higher hydroxyurea use was associated with decreased acute healthcare utilization and lower
Keywords: Sickle cell anemia, Hydroxycarbamide, adherence, Compliance, Hydroxyurea
Received: 27 Aug 2025; Accepted: 18 Nov 2025.
Copyright: © 2025 Mukhopadhyay, Smeltzer, Dudley, Badawy, Mathias, Plaxco, Ray, Wiese, Stevens, Gurney, Nwosu, Porter, Klesges, Hankins and Cooper. This is an open-access article distributed under the terms of the Creative Commons Attribution License (CC BY). The use, distribution or reproduction in other forums is permitted, provided the original author(s) or licensor are credited and that the original publication in this journal is cited, in accordance with accepted academic practice. No use, distribution or reproduction is permitted which does not comply with these terms.
* Correspondence: William O Cooper, william.cooper@vumc.org
Disclaimer: All claims expressed in this article are solely those of the authors and do not necessarily represent those of their affiliated organizations, or those of the publisher, the editors and the reviewers. Any product that may be evaluated in this article or claim that may be made by its manufacturer is not guaranteed or endorsed by the publisher.