Economic Burden Associated With Managing Patients With Sickle Cell Disease Receiving Frequent Red Blood Cell Transfusions in the United States

OBJECTIVES: Sickle cell disease (SCD) is a hereditary disorder that causes vaso-occlusive crises (VOCs) and complications due to end organ damage. The management of SCD includes frequent red blood cell transfusions (RBCTs) to alleviate symptoms and prevent further SCD-related complications. This study aimed to describe clinical and economic outcomes of patients with SCD receiving frequent RBCTs in the United States.

METHODS: This longitudinal, retrospective analysis used the Merative^TM MarketScan^® Databases to identify patients with ≥1 inpatient or ≥2 outpatient claims for SCD from 1/1/2015‒3/1/2019 who received ≥6 RBCTs during any 12-month period. The index date was the date of the sixth RBCT. Eligible patients were required to have ≥12 months of continuous enrollment pre- and post-index. Patients were followed from index to end of enrollment, death, or end of the study period (February 29, 2020), whichever came first. Demographics were assessed at index. VOC frequency, healthcare resource utilization, and annual costs were descriptively summarized during follow-up.

RESULTS: A total of 27,448 patients with SCD were identified; 919 met the inclusion criteria for SCD with frequent RBCTs. Approximately 61% (n=561) of eligible patients continued to receive >6 RBCTs each year of the follow-up period. The mean age of patients was 19.5 years, and 50.7% were female. Patients experienced a mean of 4.0 VOCs per patient per year (PPPY) and received a mean of 8.3 RBCTs PPPY during follow-up. Patients had a mean of 2.3 inpatient admissions, 83.5 outpatient visits, 4.4 emergency department visits, and 37.4 outpatient prescriptions PPPY. Mean total annual healthcare costs were $106,123 PPPY, including mean inpatient, outpatient, and outpatient pharmacy costs of $48,463, $28,307, and $29,353, respectively.

CONCLUSIONS: Despite utilizing available care with frequent RBCTs, patients with SCD continue to experience frequent VOCs, highlighting the unmet medical needs and substantial economic burden among this patient group.