ArticleHaemophilia : the official journal of the World Federation of Hemophilia2021
Evidence of a disability paradox in patient-reported outcomes in haemophilia.
Article in Haemophilia : the official journal of the World Federation of Hemophilia, 2021. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 26 papers, 2 of them syntheses that pooled it.
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The trial behind it
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Who cites it
26 citing papers in PubMed, 2 syntheses or guidelines pooled it.
- The Evolving Landscape of Discrete Choice Experiments in Health Economics: A Systematic Review.PharmacoEconomics · 2025Pooled it
- Discrete Choice Experiments in Health State Valuation: A Systematic Review of Progress and New Trends.Applied health economics and health policy · 2023Pooled it
- Durability of efficacy, safety, and quality of life 5 years after valoctocogene roxaparvovec gene transfer for severe hemophilia A: final phase 3 GENEr8-1 trial results.Research and practice in thrombosis and haemostasis · 2026Trial
- Improvements in health-related quality of life in patients with transfusion-dependent β-thalassemia after exagamglogene autotemcel.Blood advances · 2025Trial
- Estimating the Impact of Hemophilia A Treatment Administration on Patient Health Utility: Combining a Discrete Choice Experiment with Time Trade-Off Method.Advances in therapy · 2026Article
- Normalization of Haemostasis in People with Haemophilia A: Expert Consensus on Unmet Needs and a Framework for Advancing Towards Health Equity.TH open : companion journal to thrombosis and haemostasis · 2026Article
- Disability, Quality of Life, and Vitalism in End-of-Life Care.HEC forum : an interdisciplinary journal on hospitals' ethical and legal issues · 2025Article
- Treatment and Disease Burden in a Cohort of People With Haemophilia Without Inhibitors in the United States.Haemophilia : the official journal of the World Federation of Hemophilia · 2025Article
- The association of factor VIII activity levels with bleeding and quality of life in haemophilia a: findings from the European CHESS II study.Orphanet journal of rare diseases · 2025Article
- Evaluating the Cost-Effectiveness of Etranacogene Dezaparvovec Gene Therapy for Hemophilia B Treatment in the USA.Applied health economics and health policy · 2025Article
- Retrospective analysis of clinical data from 171 low-income patients with hemophilia in Shandong Province.Scientific reports · 2025Article
- Clinical and Humanistic Burden of Non-inhibitor Haemophilia A in Five European Countries: Insights from the CHESS II Study.Advances in therapy · 2024Article
- Exploring the relationship between condition severity and health-related quality of life in people with haemophilia A across Europe: a multivariable analysis of data from the CHESS II study.Health and quality of life outcomes · 2024Article
- Appropriateness of the EQ-5D-5L in capturing health-related quality of life in individuals with transfusion-dependent β-thalassemia: a mixed methods study.Health and quality of life outcomes · 2024Article
- A 360-degree perspective on adeno-associated virus (AAV)-based gene therapy for haemophilia: Insights from the physician, the nurse and the patient.Orphanet journal of rare diseases · 2024Review
- Emicizumab is well tolerated and effective in people with congenital hemophilia A regardless of age, severity of disease, or inhibitor status: a scoping review.Research and practice in thrombosis and haemostasis · 2024Article
- Preferences for potential benefits and risks for gene therapy in the treatment of sickle cell disease.Blood advances · 2023Article
- Current status of treatment and disease burden of a cohort of hemophilia B in China.Frontiers in public health · 2023Article
- Quality of life in children and adolescents with hemophilia A: A systematic review and meta-analysis.Research and practice in thrombosis and haemostasis · 2023Article
- Assessing the value of orphan drugs using conventional cost-effectiveness analysis: Is it fit for purpose?Orphanet journal of rare diseases · 2022Review
Corrections and comments
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Authors and funding
11 authors.
Funding
Abstract
introductionPeople with inherited and long-term conditions such as haemophilia have been shown to adapt to their levels of disability, often reporting better quality of life (QoL) than expected from the general population (the disability paradox).
aimTo investigate the disability paradox in people with haemophilia in the United States by examining preference differences in health state valuations versus the general population.
methodsWe conducted a discrete choice experiment including duration to capture valuations of health states based on patient-reported preferences. Participants indicated their preferences for hypothetical health states using the EQ-5D-5L, where each participant completed 15 of the 120 choice tasks. Response inconsistencies were evaluated with dominated and repeated scenarios. Conditional-logit regressions with random sampling of the general population responses were used to match the sample of patients with haemophilia. We compared model estimates and derived preferences associated with EQ-5D-5L health states.
resultsAfter removing respondents with response inconsistencies, 1327/2138 (62%) participants remained (177/283 haemophilia; 1150/1900 general population). Patients with haemophilia indicated higher preference value for 99% of EQ-5D-5L health states compared to the general population (when matched on age and gender). The mean health state valuation difference of 0.17 indicated a meaningful difference compared to a minimal clinically important difference threshold of 0.07. Results were consistent by haemophilia type and severity.
conclusionOur findings indicated the presence of a disability paradox among patients with haemophilia, who reported higher health states than the general population, suggesting the impact of haemophilia may be underestimated if general population value sets are used.
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