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Publications

Journal Article Epublish

PubMed: 40236792 DOI: 10.2147/CEOR.S511996

Clinical and Economic Burden of Managing Patients with Sickle Cell Disease Receiving Frequent Red Blood Cell Transfusions in the United States.

Clinicoecon Outcomes Res 17:303-313

Authors: Chuka Udeze, Michelle Jerry, Kristin A Evans, Nanxin Li, Siddharth Jain, Biree Andemariam

Affiliations

  • Health Economics and Outcomes Research, Vertex Pharmaceuticals Incorporated, Boston, MA, USA.
  • Research and Analytic Services, Merative, Ann Arbor, MI, USA.
  • Medicine, University of Connecticut Health, Farmington, CT, USA.

Abstract

Purpose

Standard of care for patients with sickle cell disease (SCD) includes red blood cell transfusions (RBCTs). Data on clinical and economic outcomes of patients with SCD receiving frequent RBCTs are limited.

Materials and Methods

This longitudinal, retrospective, claims-based analysis used the Merative™ MarketScan Commercial, Medicare, and Multi-State Medicaid databases. Patients with SCD (identified using ICD-9/10 codes) receiving frequent RBCTs (≥6 RBCTs during any 12-month period) between January 1, 2015, and March 1, 2019, were included. 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. Clinical complications, all-cause healthcare resource utilization (HCRU), and healthcare costs were descriptively summarized during follow-up.

Results

A total of 919 patients with SCD receiving frequent RBCTs met the eligibility criteria for inclusion. Patients experienced a mean of 4.0 vaso-occlusive crises (VOCs) per patient per year (PPPY) and received a mean of 8.3 RBCTs PPPY during follow-up. The most common clinical complications were iron overload (77%), infections (66%), and cerebrovascular disease (48%). Patients had a mean of 2.3 inpatient admissions, 83.5 outpatient visits, and 37.4 outpatient prescriptions PPPY during follow-up. Mean total annual healthcare costs were 106,123 PPPY, including mean inpatient, outpatient medical, and outpatient pharmacy costs of 48,463, 28,307, and 29,353, respectively. Compared to those with <2 baseline VOCs, patients with ≥2 baseline VOCs had more HCRU and higher annual healthcare costs.

Conclusion

Despite utilizing available care with frequent RBCTs, patients with SCD experienced a variety of disease and transfusion-related complications, including frequent VOCs and iron overload, which led to substantial HCRU and costs. These findings highlight the need for novel therapies for this patient group.

MeSH

Cells, Financial Stress, Health Care Costs, Erythrocytes, Blood, Anemia, Sickle Cell, Erythrocyte Transfusion, Iron Overload, Blood Cells, Therapeutics, Outpatients, Vaso-Occlusive Crises