Abstract
BackgroundHemophilia B is a rare congenital bleeding disorder that has a significant negative impact on patients’ functionality and health-related quality of life. The standard of care for severe hemophilia B in the United States is prophylactic factor IX replacement therapy, which incurs substantial costs for this lifelong condition. Accurate estimates of the burden of hemophilia B are important for population health management and policy decisions, but have only recently accounted for current management strategies. The ‘Cost of Severe Hemophilia across the US: a Socioeconomic Survey’ (CHESS US) is a cross-sectional database of medical record abstractions and physician-reported information, completed by hematologists and care providers. CHESS US+ is a complementary database of completed questionnaires from patients with hemophilia. Together, CHESS US and CHESS US+ provide contemporary, comprehensive information on the burden of severe hemophilia from the provider and patient perspectives. We used the CHESS US and CHESS US+ data to analyze the clinical, humanistic, and economic burden of hemophilia B for patients treated with factor IX prophylaxis between 2017 and 2019 in the US.ResultsWe conducted analysis to assess clinical burden and direct medical costs from 44 patient records in CHESS US, and of direct non-medical costs, indirect costs, and humanistic burden (using the EQ-5D-5L) from 57 patients in CHESS US+. The mean annual bleed rate was 1.73 (standard deviation, 1.39); approximately 9% of patients experienced a bleed-related hospitalization during the 12-month study period. Nearly all patients (85%) reported chronic pain, and the mean EQ-5D-5L utility value was 0.76 (0.24). The mean annual direct medical cost was $614,886, driven by factor IX treatment (mean annual cost, $611,971). Subgroup analyses showed mean annual costs of $397,491 and $788,491 for standard and extended half-life factor IX treatment, respectively. The mean annual non-medical direct costs and indirect costs of hemophilia B were $2,371 and $6,931.ConclusionsThis analysis of patient records and patient-reported outcomes from CHESS US and CHESS US+ provides updated information on the considerable clinical, humanistic, and economic burden of hemophilia B in the US. Substantial unmet needs remain to improve patient care with sustainable population health strategies.
Highlights
Hemophilia B is a rare congenital bleeding disorder that has a significant negative impact on patients’ functionality and health-related quality of life
Prophylactic administration of factor IX (FIX) replacement therapy is the standard of care for severe hemophilia B in the US [17,18,19,20], and has helped improve life expectancy to approach that of the general population [21]
Bleeding-related health resource use was accompanied by chronic pain and compromised employment due to hemophilia, along with non-medical and indirect costs related to self-management and use of professional or informal caregivers
Summary
Hemophilia B is a rare congenital bleeding disorder that has a significant negative impact on patients’ functionality and health-related quality of life. The standard of care for severe hemophilia B in the United States is prophylactic factor IX replacement therapy, which incurs substantial costs for this lifelong condition. Hemophilia B is known to cause substantial functional limitations and reduced health-related quality of life (HRQoL) [12,13,14] related to bleeding and joint damage, and other disease-related complications [15, 16]. Prophylactic administration of FIX replacement therapy is the standard of care for severe hemophilia B in the US [17,18,19,20], and has helped improve life expectancy to approach that of the general population [21]. Extended half-life (EHL) and standard half-life (SHL) formulations are administered often, every 1–2 weeks and 2–3 times per week, respectively, which incurs a treatment burden on patients [22, 23] and a notable economic burden to healthcare systems and society [24,25,26,27]
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