Date of Award


Degree Type


Degree Name

Master of Science in Pharmaceutical Sciences


Biomedical and Pharmaceutical Sciences

First Advisor

Aisling Caffrey


Background: The medication management of individuals with sickle cell disease (SCD) has not been well described in the literature. Further, changes since the release of treatment guidelines in 2014 have not been assessed.

Objective: This study aimed to quantify the real-world medication management of patients with SCD in the United States, including trends in medication utilization over time and since the 2014 treatment guidelines, overall and by patient age, sex, race/ethnicity, and region.

Methods: This retrospective cohort study examined individuals diagnosed with SCD from January 1, 2010, to December 31, 2018, in Optum’s de-identified Clinformatics® Data Mart database. The study population included individuals with at least one inpatient or two outpatient diagnosis claims for SCD (International Classification of Diseases, Ninth Revision, Clinical Modification [ICD-9-CM]: 282.41,282.42, 282.6, 282.60-282.69; ICD-10-CM: D57, D57.X, D57.XX, excluding D57.3 = sickle cell trait) during the study period. Changes in medication utilization over time were assessed with Joinpoint regression to calculate annual percent changes (APCs) and average annual percent change (AAPC). Stratified time trends were evaluated by patient demographics.

Results: Over the 8-year study period, we identified 10,528 individuals with SCD. The mean age of patients with SCD was 39.1 years (standard deviation [SD] 22.5 years). Over half (59.7%) of the study population was female. Throughout the study period, 20.2% of individuals with SCD filled hydroxyurea prescriptions, demonstrating a significant annual increase of 7.5% (p

Conclusion: Medication utilization increased for almost all medications utilized by patients with SCD, which indicates better medication management since the release of the NHLBI SCD treatment guidelines in 2014. However, there is still a need for improvement in the prophylactic utilization of hydroxyurea and penicillin. Future studies need to assess the utilization of recently approved SCD medications (L-glutamine, voxelotor, crizanlizumab), changes in utilization of other medications since the approval of new SCD medications and new clinical guidelines released in 2019, and the relationship between improved medication management and clinical outcomes in patients with SCD.



To view the content in your browser, please download Adobe Reader or, alternately,
you may Download the file to your hard drive.

NOTE: The latest versions of Adobe Reader do not support viewing PDF files within Firefox on Mac OS and if you are using a modern (Intel) Mac, there is no official plugin for viewing PDF files within the browser window.