Patient Characteristics Associated with the Use of Newly Approved Disease-Modifying Therapy (DMT) for Sickle Cell Disease in Texas Medicaid
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, Austin, TX, USA, 3Baylor College of Medicine, Medicine-Hematology & Oncology, Houston, TX, USA
1The University of Texas at Austin, Health Outcomes Division, College of Pharmacy, Austin, TX, USA, 2Dell Children’s Hospital, Austin, TX, USA, 3Baylor College of Medicine, Medicine-Hematology & Oncology, Houston, TX, USA
OBJECTIVES: This study aimed to identify patient characteristics associated with the utilization of new DMTs, including L-glutamine, crizanlizumab-tmca, and voxelotor.
METHODS: This study used Texas Medicaid data from 01/2016 to 08/2023. Patients 4-64 years old were included if they had ≥1 inpatient or ≥2 outpatient visits on separate dates with an SCD diagnosis and were continuously enrolled during the 1-year pre-index period. The index date was the date of the first prescription filling for new DMT users and 1 year after the first SCD diagnosis for non-users. Demographic (age, sex, race/ethnicity, metropolitan statistical area) and clinical (hydroxyurea use and number of SCD-related outpatient visits, vaso-occlusive crisis [VOC] events, and red blood cell transfusions within the 1-year pre-index period) characteristics were described and included in the logistic regression model to identify characteristics associated with use of new DMTs.
RESULTS: A total of 483 users of new DMTs (mean [SD] age = 23.4 [12.0], 53.2% female) and 4,125 non-users (mean [SD] age = 25.9 [15.8], 61.0% female) were included. Among new DMT users, 176, 183, and 254 patients were prescribed L-glutamine, crizanlizumab, and voxelotor, respectively. Logistic regression showed that new DMT users were more likely to: 1) be in 13-17,18-26, or 27-34 years of age (Odds Ratio [OR]=2.20, 1,78, and 1.50, 95% CI: 1.57-3.07, 1.28-2.47, and 1.03-2.17, respectively, all p<0.05, reference: 5-12 years); 2) have had more SCD-related outpatient visits (5-9 visits: OR=2.67, 95%CI: 1.77-4.03, p<.0001; ≥10 visits: OR=4.51, 95%CI: 3.00-6.77, p<.0001; reference: 1-4 visits); 3) have pre-index hydroxyurea use (OR=3.64, 95%CI: 2.87-4.61, p<.0001); and 4) have a higher mean number of VOC events (OR=1.08, 95%CI: 1.06-1.11, p<.0001), compared to non-users.
CONCLUSIONS: Age and greater healthcare utilization (i.e., more outpatient visits, more VOC events, and previous use of hydroxyurea) indicating severe conditions were associated with use of newly approved DMTs.
METHODS: This study used Texas Medicaid data from 01/2016 to 08/2023. Patients 4-64 years old were included if they had ≥1 inpatient or ≥2 outpatient visits on separate dates with an SCD diagnosis and were continuously enrolled during the 1-year pre-index period. The index date was the date of the first prescription filling for new DMT users and 1 year after the first SCD diagnosis for non-users. Demographic (age, sex, race/ethnicity, metropolitan statistical area) and clinical (hydroxyurea use and number of SCD-related outpatient visits, vaso-occlusive crisis [VOC] events, and red blood cell transfusions within the 1-year pre-index period) characteristics were described and included in the logistic regression model to identify characteristics associated with use of new DMTs.
RESULTS: A total of 483 users of new DMTs (mean [SD] age = 23.4 [12.0], 53.2% female) and 4,125 non-users (mean [SD] age = 25.9 [15.8], 61.0% female) were included. Among new DMT users, 176, 183, and 254 patients were prescribed L-glutamine, crizanlizumab, and voxelotor, respectively. Logistic regression showed that new DMT users were more likely to: 1) be in 13-17,18-26, or 27-34 years of age (Odds Ratio [OR]=2.20, 1,78, and 1.50, 95% CI: 1.57-3.07, 1.28-2.47, and 1.03-2.17, respectively, all p<0.05, reference: 5-12 years); 2) have had more SCD-related outpatient visits (5-9 visits: OR=2.67, 95%CI: 1.77-4.03, p<.0001; ≥10 visits: OR=4.51, 95%CI: 3.00-6.77, p<.0001; reference: 1-4 visits); 3) have pre-index hydroxyurea use (OR=3.64, 95%CI: 2.87-4.61, p<.0001); and 4) have a higher mean number of VOC events (OR=1.08, 95%CI: 1.06-1.11, p<.0001), compared to non-users.
CONCLUSIONS: Age and greater healthcare utilization (i.e., more outpatient visits, more VOC events, and previous use of hydroxyurea) indicating severe conditions were associated with use of newly approved DMTs.
Conference/Value in Health Info
2025-05, ISPOR 2025, Montréal, Quebec, CA
Value in Health, Volume 28, Issue S1
Code
HSD24
Topic
Health Service Delivery & Process of Care
Disease
SDC: Rare & Orphan Diseases