Introduction: Sickle cell disease (SCD), a genetic blood disorder, is characterized by significant acute and chronic complications, necessitating intensive disease management. Prevention-focused maintenance therapies and as-needed (acute) symptomatic treatments are the most common disease management strategies. This study assessed the prevalence of SCD, rates of SCD-related complications, and utilization of maintenance and as-needed SCD therapies within a population of patients insured by commercial or Medicare insurance in the United States.

Methods: This retrospective study analyzed deidentified claims data from individuals with employer-sponsored private commercial, Medicare Supplemental, or Medicare Advantage health insurance using the Merative™ MarketScan®Commercial and Medicare databases, covering October 1, 2015, to June 30, 2023. Prevalence of SCD was quantified in the overall eligible population in patients with ≥12 months of continuous medical and pharmacy enrollment during the study period. Patients with SCD were identified using ≥1 inpatient or 2 outpatient claims for ICD-10-CM codes D57 (excluding D57.3 sickle cell trait) 30 to 365 days apart. In patients with SCD, rates of complications and use of SCD-related therapies were assessed during 12-month baseline and 12-month follow-up periods, stratified by age at index (<18, 18-30, 31-64, and ≥65 years).

Results: Of 50,311,522 eligible individuals, 12,422 met criteria for SCD, resulting in a prevalence of 0.08 per 1,000 patient-years in this population. The complications and treatment analyses included 6,449 patients with ≥24 months of continuous enrollment; 59.6% were female, mean (SD) age was 31.0 (19.5) years, and mean (SD) duration of commercial/Medicare enrollment was 4.1 (1.8) years.

Vaso-occlusive crises were reported in 23.3% of patients, with the highest prevalence in patients aged 18 to 30 years (31.3%). Infections (58.2%), anemia (35.8%), cardiovascular events (27.0%) were the most common complications. Patients aged <18 years had the highest prevalence of fever (39.5%) and asthma (21.3%), while those aged ≥65 years demonstrated the highest rates of cardiovascular complications (66.8%), nephropathy (59%) and renal impairment/failure (55.1%).

Pain medication and antibiotic use was high, with 58.4% and 68.4% of patients with ≥1 fill for prescription pain medication and 52.9% and 58.1% of patients with ≥1 fill for antibiotics in the baseline and follow-up periods, respectively.

Use of disease-modifying therapies was limited, with 21.6% and 24.4% of patients using hydroxyurea (HU), 0.5% and 0.8% using L-glutamine, and 12.2% and 15.4% receiving chronic blood transfusions during the baseline and follow-up periods, respectively. Uptake of newer SCD therapies was low, with <1% of patients using crizanlizumab or voxelotor during either period. HU use was most common among patients aged <18 (30.9%) and 18 to 30 (30.0%) years, while chronic blood transfusions were most common among patients aged 18 to 30 years (20.5%). In contrast, therapy utilization was lowest among patients aged ≥65 years (HU, 5.7%; L-glutamine, 0.4%; crizanlizumab, 0.0%; voxelotor, 0.4%; chronic blood transfusions, 11.0%). Among the 2,421 patients who used maintenance therapy, 66.4% were adherent with ≥80% of days covered, indicating suboptimal adherence in 33.6% of patients. The mean (SD) duration of maintenance therapy was 538.5 (598.0) days. Discontinuation of ≥1 component of the therapy regimen was the most common (59.4%) reason for ending the maintenance therapy.

Limitations:This analysis excluded those who were uninsured or covered by Medicaid, which represents a significant portion of people living with SCD. Also, management of SCD varies based on disease severity; neither stratification nor statistical control for severity was incorporated into this analysis as claims data do not provide this information.

Conclusions: Treatment of SCD remains dominated by symptom management rather than disease-modifying therapy, as reflected by high utilization of as-needed medications and low uptake of newer therapies. The barriers to accessing disease-modifying therapies and targeted treatments warrant further investigation. Optimization of SCD management is needed to help slow disease progression and reduce complications. Strategies to improve management may include increasing use of currently available SCD therapies and development of new therapies to address remaining unmet needs.

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2025
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