Health care resource utilization and cost burden of hemophilia B in the United States

Hemophilia B is a rare congenital blood disorder characterized by factor IX deficiency. Clinical profiles of hemophilia B range from mild to severe forms of the disease. The objective of this study was to characterize the economic burden associated with differing clinical 3 profiles of hemophilia B from a US health system perspective. Using the IBM MarketScan database (June 2011-February 2019), a claims-based algorithm was developed to identify 4 distinct profiles (mild, moderate, moderate-severe, and severe) in adult males with hemophilia B based on the frequency of hemorrhage events and factor IX replacement claims. Mean annual health care resource use (HRU) and costs were statistically compared between patients with hemophilia B (N - 454) and 1:1 demographic-matched controls (N - 454), both overall and with stratification by clinical profile. Compared with matched controls, patients with hemophilia B had a significantly higher comorbidity burden (Charlson Comorbidity Index, mean +/- standard deviation [SDI: 0.9 = 1.7 vs 0.3 +/- 0.9, P < .001). Across all clinical profiles, patients with hemophilia B had significantly higher HRU vs matched controls (mean = SD: 0.3 +/- 0.6 vs 0.1 +/- 0.3 inpatient admissions; 0.6 +/- 1.2 vs 0.2 = 0.6 emergency department visits; 17.7 +/- 22.9 vs 8.0 = 11.0 outpatient visits; all P < .001). Annual total health care costs per patient among patients with hemophilia B were more than 25-fold higher vs matched controls (mean = SD: $201 635 = $411 530 vs $7879 +/- $29 040, respectively, P < .001). Annual total health care costs per patient increased with increasing severity (mean = SD: mild, $80811 +/- $284313; moderate, $137455 +/- $222021; moderate-severe, $251 619 = $576 886; severe, $632 088 +/- $501 270). The findings of this study highlight the substantial burden of illness associated with hemophilia B.