ArticleHaemophilia : the official journal of the World Federation of Hemophilia
Patient and Caregiver Preferences for Long-Term Prophylactic Treatment of Bleeds in Haemophilia: A Discrete-Choice Experiment.
Article in Haemophilia : the official journal of the World Federation of Hemophilia. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 1 paper.
What it found
Each row is one number read from the abstract, on the scale the paper reported it, with its interval. Left of the dashed line favours the treatment, right favours the comparator. Under each row is the sentence it came from. New to these charts? A ten-minute tutorial.
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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Trials whose registry record cites this paper, or whose number appears in the abstract. A trial that started after this paper was published is citing it as background, not reporting it.
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Who cites it
1 citing paper in PubMed.
- Patient and Caregiver Preferences for Long-Term Prophylactic Treatment of Bleeds in Haemophilia: A Discrete-Choice Experiment.Haemophilia : the official journal of the World Federation of HemophiliaArticle
Corrections and comments
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Authors and funding
13 authors.
Funding
Abstract
introductionThe expansion of haemophilia treatment to include non-factor prophylaxis provides new options with various benefits, risks, administration modes, and device types, necessitating trade-offs in decision-making.
aimThis study evaluated prophylactic treatment preferences of people with haemophilia and their caregivers, assessing their willingness to make trade-offs among treatment attributes.
methodsA web-based survey containing a discrete-choice experiment (DCE) was completed by adults (≥ 18 years old) and caregivers of children (aged 8-17 years) with haemophilia in the United States and United Kingdom. Participants were asked to complete 10 choice tasks, choosing their preferred prophylaxis from two hypothetical profiles defined by seven attributes. The primary analysis employed a mixed logit model, and subgroup analyses explored preference heterogeneity.
resultsParticipants were 194 adults with haemophilia (US: 150; UK: 44) and 169 caregivers (US: 150; UK: 19). Avoiding daily treatment administration had the highest attribute importance among both adults and caregivers, followed by changes in annual bleeds. Participants were willing to accept an increased risk of serious side effects or developing inhibitors, or reduced efficacy in preventing annual bleeds, to switch from intravenous to subcutaneous administration. Refrigeration requirements and the need for a second treatment for breakthrough bleeds had lower importance.
conclusionsParticipants highly value avoiding daily administration and reducing bleeds, and they are willing to accept increased risks or reduced efficacy to have subcutaneous rather than intravenous administration. As new treatments become available, understanding these preferences can facilitate shared decision-making in treatment selection and enhance patient-centred drug development and patient communication.
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