Prescribing Trends of Disease-Modifying Medications in Texas Medicaid Enrollees with Sickle Cell Disease

Author(s)

Yahan Zhang, MS1, Hyeun Ah KANG, MEd, MS, RPh, PhD1, Kristin M. Richards, BS, MS, RPh, PhD1, Alicia Chang, M.D.2, Kenneth A. Lawson, BS, MS, PhD1, Mark Myers Udden, M.D.3;
1The University of Texas at Austin, Health Outcomes Division, College of Pharmacy, Austin, TX, USA, 2Dell Children’s Hospital, Children’s Blood and Cancer Center, Austin, TX, USA, 3Baylor College of Medicine, Medicine-Hematology & Oncology, Houston, TX, USA
OBJECTIVES: Sickle cell disease (SCD) is a rare, inherited blood disorder that affects approximately 100,000 Americans. Patients with SCD suffer from various acute and chronic complications. Disease-modifying therapies (DMTs) could effectively prevent, reduce, and mitigate complications associated with the condition. This study aims to describe the prescribing trends of all types of DMTs for SCD treatment, especially after the approval of three new types of medications, including L-glutamine, crizanlizumab-tmca, and voxelotor, in addition to the traditional medication hydroxyurea.
METHODS: This retrospective study used Texas Medicaid data from 01/2016 to 08/2023. Patients with SCD were included if they had at least 1 inpatient or 2 outpatient visits on separate dates with an SCD diagnosis code. The number of patients who received at least one prescription of each type of medication (L-glutamine, crizanlizumab, voxelotor, hydroxyurea), as well as any type of DMT, were reported by month.
RESULTS: A total of 7,828 SCD patients were identified during the study period (59.6% female). Among them, 2,459 (31.4%), 176 (2.2%), 254 (3.2%), 184 (2.4%), and 2,524 (32.2%) patients had ≥1 prescription filled for hydroxyurea, L-glutamine, voxelotor, crizanlizumab, or any DMTs, respectively. Hydroxyurea remains the most frequently prescribed DMT. The trends in the monthly numbers of patients prescribed DMTs were as follows: hydroxyurea was relatively stable between 2016 and mid-2020, followed by a significant rise and steady increase through 2023; L-glutamine increased slightly after approval and remained stable over time; crizanlizumab and voxelotor increased gradually after approval with crizanlizumab showing a slower rate than voxelotor; and DMTs regardless of type increased after approval of newer types of DMTs.
CONCLUSIONS: Hydroxyurea remains the dominant DMT prescribed. The number of monthly users of L-glutamine, voxelotor, and crizanlizumab increased gradually; however, that number remains low.

Conference/Value in Health Info

2025-05, ISPOR 2025, Montréal, Quebec, CA

Value in Health, Volume 28, Issue S1

Code

PT18

Topic

Health Service Delivery & Process of Care

Disease

SDC: Rare & Orphan Diseases

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