Abstract
BackgroundReal-world studies of the burden of severe haemophilia B in the context of recent therapeutic advances such as extended half-life (EHL) factor IX (FIX) products are limited. We analysed data from the recent CHESS II study to better understand the clinical, humanistic, and economic burden of severe haemophilia B in Europe. Data from male adults with severe haemophilia B receiving prophylaxis were analysed from the retrospective cross-sectional CHESS II study conducted in Germany, France, Italy, Spain and the United Kingdom. Inhibitors were exclusionary. Patients and physicians completed questionnaires on bleeding, joint status, quality of life, and haemophilia-related direct and indirect costs (2019–2020). All outcomes were summarised using descriptive statistics.ResultsA total of 75 CHESS II patients were eligible and included; 40 patients (53%) provided self-reported outcomes. Mean age was 36.2 years. Approximately half the patients were receiving EHL versus standard half-life (SHL) prophylaxis (44% vs 56%). Most patients reported mild or moderate chronic pain (76%) and had ≥ 2 bleeding events per year (70%), with a mean annualised bleed rate of 2.4. Mean annual total haemophilia-related direct medical cost per patient was €235,723, driven by FIX costs (€232,328 overall, n = 40; €186,528 for SHL, €290,620 for EHL). Mean annual indirect costs (€8,973) were driven by early retirement or work stoppage due to haemophilia. Mean quality of life (EQ-5D) score was 0.67.ConclusionsThese data document a substantial, persistent real-world burden of severe haemophilia B in Europe. Unmet needs persist for these patients, their caregivers, and society.
Highlights
Real-world studies of the burden of severe haemophilia B in the context of recent therapeutic advances such as extended half-life (EHL) factor IX (FIX) products are limited
The holistic burden of haemophilia B among patients with severe disease, in particular, has been underserved in the literature. As these patients are likely to receive prophylactic therapy, we focused this analysis on patients with severe haemophilia B receiving prophylaxis
Demographic characteristics were generally similar between patients receiving standard half-life (SHL) or EHL prophylaxis
Summary
Real-world studies of the burden of severe haemophilia B in the context of recent therapeutic advances such as extended half-life (EHL) factor IX (FIX) products are limited. We analysed data from the recent CHESS II study to better understand the clinical, humanistic, and economic burden of severe haemophilia B in Europe. Data from male adults with severe haemophilia B receiving prophylaxis were analysed from the retrospective crosssectional CHESS II study conducted in Germany, France, Italy, Spain and the United Kingdom. Patients with haemophilia B endure an ongoing clinical burden with substantial impairment of daily activities and health-related quality of life (HRQoL) [2,3,4]. Patients with severe disease (factor IX level ≤ 1% IU/ dL; FIX) experience frequent spontaneous and recurrent joint bleeds that, without appropriate treatment, lead to early arthritis, deformity, and other long-term complications [5,6,7]. Improved outcomes come with an inherent treatment burden, as treatments are administered 2–3 times per week for standard
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