ArticleOrphanet journal of rare diseases2021
Clinical, humanistic, and economic burden of severe haemophilia B in adults receiving factor IX prophylaxis: findings from the CHESS II real-world burden of illness study in Europe.
Article in Orphanet journal of rare diseases, 2021. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 19 papers.
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Who cites it
19 citing papers in PubMed.
- Social vulnerability, access to care, and outcomes in hemophilia.Blood vessels, thrombosis & hemostasis · 2026Article
- Article
- Burden of Treatment and Disease on People with Hemophilia: Global Real-World Survey.Patient preference and adherence · 2026Article
- Disease burden and unmet medical need in severe hemophilia in Greece: insights from clinicians and patients.Frontiers in public health · 2026Article
- Clinical, humanistic, and economic burden of haemophilia A in China: findings from a real-world survey.Orphanet journal of rare diseases · 2025Article
- Economic Burden of Haemophilia from a Societal Perspective: A Scoping Review.PharmacoEconomics - open · 2025Review
- Development of iPSC-derived FIX-secreting hepatocyte sheet as a novel treatment tool for hemophilia B treatment.Stem cell research & therapy · 2025Article
- The role of public health in rare diseases: hemophilia as an example.Frontiers in public health · 2025Review
- Assessing health care resource use, outcomes, and costs among Medicaid beneficiaries receiving factor IX prophylaxis for hemophilia B.Journal of managed care & specialty pharmacy · 2024Article
- Assessing health plan payer's budget impact of etranacogene dezaparvovec for the treatment of hemophilia B in the United States.Journal of managed care & specialty pharmacy · 2024Article
- Cost-Effectiveness of Recombinant Factor IX Fc Prophylaxis and Recombinant Factor IX On-Demand Treatment in Patients with Haemophilia B Without Inhibitors.Advances in therapy · 2024Article
- Concizumab prophylaxis in persons with hemophilia A or B with inhibitors: patient-reported outcome results from the phase 3 explorer7 study.Research and practice in thrombosis and haemostasis · 2024Article
- Cost-Effectiveness Analysis of Etranacogene Dezaparvovec Versus Extended Half-Life Prophylaxis for Moderate-to-Severe Haemophilia B in Germany.PharmacoEconomics - open · 2024Review
- Economic and Humanistic Burden of Moderate and Severe Hemophilia A and B in Spain: Real-World Evidence Insights from the CHESS II Study.Journal of health economics and outcomes research · 2024Article
- Hemophilia Healing with AAV: Navigating the Frontier of Gene Therapy.Current gene therapy · 2024Review
- Gene Therapy Approaches for the Treatment of Hemophilia B.International journal of molecular sciences · 2023Review
- Current status of treatment and disease burden of a cohort of hemophilia B in China.Frontiers in public health · 2023Article
- Association of factor expression levels with annual bleeding rate in people with haemophilia B.Haemophilia : the official journal of the World Federation of Hemophilia · 2023Article
- HAEMFIX: Impact of Switching From SHL-FIX to EHL-FIX in Patients With Haemophilia B.Haemophilia : the official journal of the World Federation of HemophiliaArticle
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Authors and funding
6 authors.
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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.
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