OBJECTIVES: Endari (L-glutamine), Adakveo (crizanlizumab-tmca), and Oxbryta (voxelotor) are newly approved disease-modifying treatments (DMTs) for sickle cell disease (SCD). This study aimed to identify demographic and clinical characteristics associated with the utilization of the newly approved DMTs.

METHODS: This retrospective study used the 2016-2021 Meartive™ MarketScan Commercial Database. Subjects were identified if they were 5-64 years old, had ≥1 inpatient or ≥2 outpatient visits on separate dates with an SCD diagnosis, had no cancer diagnosis, had pharmacy coverage during the study period, and were continuously enrolled for 1 year before the index date. For DMT users, the index date was the date of the first prescription fill, while for non-users, it was 1 year after the first SCD diagnosis. Descriptive analysis and logistic regression were conducted to identify demographic (age, sex, region, metropolitan statistical area) or clinical (the number of SCD-related outpatient visits and use of hydroxyurea within the 1-year pre-index period) characteristics that are associated with the use of new DMTs.

RESULTS: Among 5138 subjects included (mean [SD] age = 30.10 [16.06], 57.6% female), 285 (5.55%) were DMT users (i.e., Endari, Adakveo, or Oxbryta). The logistic regression showed that the DMT users were more likely to: 1) be in 13-17 (Odds Ratio [OR]=2.38, 95% CI: 1.48-3.83, p=0.0004), 18-26 (OR=1.57, 95% CI: 1.01-2.46, p=0.0475), and 27-34 (OR=1.73, 95% CI: 1.08-2.77, p=0.0232) age groups (reference: 5-12 years);2) have had more SCD-related outpatient visits (5-9 visits: OR=3.02, 95% CI: 1.93-4.72, p<.0001; ≥10 visits: OR=5.66, 95% CI: 3.68-8.70, p<.0001; reference: 1-4 visits); and 3) have used hydroxyurea (OR=3.01, 95% CI: 2.32-3.92, p<.0001) in the pre-index period, compared to the non-users.

CONCLUSIONS: At the patient level, the use of the newly approved DMTs was associated with age (being adolescents or young adults) and previous greater healthcare utilization.