Health care resource utilization and cost burden of hemophilia Bin 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.

Automated summary

The study aimed to characterize the economic burden associated with different clinical profiles of hemophilia B from a US health system perspective. Results showed that patients with hemophilia B had significantly higher healthcare resource use and costs compared to matched controls, with costs increasing as the severity of the condition worsened.