ArticlePatient preference and adherence2023
Gene Therapy for Hemophilia A: A Mixed Methods Study of Patient Preferences and Shared Decision-Making.
Article in Patient preference and adherence, 2023. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 2 papers.
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.
The trial behind it
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.
Neither the registry nor the abstract names a trial number. If this is a trial report, that itself is worth knowing.
Who cites it
2 citing papers in PubMed, 15 citations in OpenAlex.
- Patient, consumer and societal values, perceptions and preferences on high-cost gene therapies.Gene therapy · 2026Article
- Construction of a Shared Decision-Making Program for Chronic Pain Management in Adult Patients with Hemophilic Arthropathy.Patient preference and adherence · 2026Article
Corrections and comments
PubMed lists nothing against this paper. Absence here is not a guarantee, only a check that was made.
Authors and funding
2 authors at 2 institutions in 1 country.
Funding
No grant is acknowledged in the PubMed record.
Abstract
Purpose: As gene therapies for hemophilia are incorporated into clinical practice, shared decision-making (SDM) is recommended for implementation. SDM tools may facilitate informed decision-making for gene therapy and other novel therapies. Objective: To inform the development of SDM tools for hemophilia gene therapy. Patients and Methods: Men with severe hemophilia were recruited from the National Hemophilia Foundation (NHF) Community Voices in Research (CVR). Semi-structured interviews were completed, and the interviews were transcribed verbatim for quantitative and qualitative analysis. Results: Twenty-five men with severe hemophilia A participated. All participants reported treatment with prophylaxis, nine (36%) on continuous prophylaxis with clotting factor, one (4%) on intermittent prophylaxis with clotting factor, and 15 (60%) on continuous prophylaxis with emicizumab. Ten (40%) indicated that they are excited about gene therapy, 12 (48%) indicated that they are hopeful about gene therapy, one (4%) indicated that they are worried or scared about gene therapy, and one (4%) indicated that they do not have strong feelings about gene therapy. Participants reported engaging Hemophilia Treatment Center, family, and the hemophilia community in their decision-making process. The most reported information needs are efficacy, safety, cost/insurance, mechanism of action, and follow-up. In addition, key information themes that emerged were patient testimonials, hard data and statistics, and comparison to other products. Twenty-two (88%) indicated that a SDM tool would be useful when discussing gene therapy with their hemophilia team. Two indicated that they do their own research, and the tool would not add anything. One needed more information to provide an answer. Conclusion: These data highlight the utility of a SDM tool for hemophilia gene therapy and key information needs. Data including comparison to other treatments should be provided along with patient testimonials in a transparent format. Patients will engage the Hemophilia Treatment Center, family, and community members in the decision-making process.
Indexed as
Identifiers
What OpenQuestion holds
Registered trials
Read under generation 80e0d062 · epoch 390. Bibliography from PubMed, PubMed Central and OpenAlex; grants from NIH RePORTER; trial links from ClinicalTrials.gov; estimates, votes and beliefs from the OpenQuestion graph.