ArticlePatient preference and adherence2021
Preferences and Health-Related Quality-of-Life Related to Disease and Treatment Features for Patients with Hemophilia A in a Canadian General Population Sample.
Article in Patient preference and adherence, 2021. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 11 papers.
What it found
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The abstract states no effect estimate the extractor could read, or names no intervention and outcome on the map, so this paper lights no cell and moves no belief. It is still indexed, cited and linked below.
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Who cites it
11 citing papers in PubMed, 21 citations in OpenAlex.
- 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
- Cost Effectiveness of Efanesoctocog Alfa Versus Factor VIII Extended Half-Life in Adolescent and Adult Patients with Hemophilia A in the USA.PharmacoEconomics · 2026Article
- Concizumab prophylaxis in a 2-year-old patient with severe hemophilia B and inhibitor: a case report.Frontiers in pharmacology · 2026Article
- Normalization in hemophilia: conceptual foundations and clinical implications.Research and practice in thrombosis and haemostasis · 2025Review
- 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
- Concizumab, a Non-Replacement Therapy for Persons with Hemophilia with Inhibitors.Journal of clinical medicine · 2025Review
- Rationale for Long-acting Growth Hormone Therapy and Future AspectsJournal of clinical research in pediatric endocrinology · 2025Review
- A time trade-off study in the UK, Canada and the US to estimate utilities associated with the treatment of haemophilia.Health and quality of life outcomes · 2024Article
- Cost-Effectiveness Analysis of Etranacogene Dezaparvovec Versus Extended Half-Life Prophylaxis for Moderate-to-Severe Haemophilia B in Germany.PharmacoEconomics - open · 2024Review
- Cost-effectiveness of ropeginterferon alfa-2b-njft for the treatment of polycythemia vera.Journal of comparative effectiveness research · 2023Article
- Medication Adherence for Haemophilia Patients: Outcome of Prophylaxis Treatment Intervention.Healthcare (Basel, Switzerland) · 2021Article
Corrections and comments
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Authors and funding
8 authors at 5 institutions in 2 countries.
Funding
No grant is acknowledged in the PubMed record.
Abstract
backgroundCurrent treatments for hemophilia A in Canada include on-demand treatment as bleeds occur and regular intravenous prophylactic factor VIII (FVIII) infusions. The subcutaneous therapy emicizumab was recently approved for treatment of hemophilia A. The objective of this study was to estimate utility values associated with hemophilia A health and treatment states from a Canadian societal perspective, including preferences related to treatment efficacy and frequency and route of administration.
methodsA vignette-based time trade-off (TTO) utilities elicitation was undertaken in Canadian adults to compare preferences for six hemophilia health states describing prophylactic and on-demand treatment, with varying bleed rates and frequency of treatment administration. A convenience sample was recruited via market research panels and snowball sampling. Health state/vignette definitions were informed by clinical experience, clinical trial results regarding bleed frequency, and validated by qualitative interviews of hemophilia patients and caregivers (n=10). Utilities were estimated via an online, trained interviewer-guided, vignette-based TTO exercise, where respondents valuated health states describing hemophilia patients (adults or children) receiving subcutaneous prophylaxis, intravenous prophylaxis, and on-demand treatments. Analyses included a descriptive analysis by health state; a mixed-effects analysis of utility values adjusted for subcutaneous vs infusion-based therapies and number of bleeds; and for prophylactic regimens, an analysis of utilities by frequency of infusions or injections.
resultsTTO interviews were conducted with 82 respondents. Mean utilities [95% CI] were highest for subcutaneous prophylaxis (0.90 [0.87-0.93]), followed by intravenous prophylaxis (0.81 [0.78-0.85]), and on-demand treatment (0.70 [0.65-0.76]). In regression analysis, subcutaneous treatment health states were associated with a utility increment of +0.1112. Additional bleeds and more frequent infusions were associated with lower utility values (-0.0027 per bleed and -0.0003 per infusion).
conclusionSubcutaneous prophylaxis is associated with higher utility values compared to intravenous prophylactic and on-demand treatment, while increased bleeds and infusions are associated with reduced utility.
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