ArticlePatient preference and adherence2022
The Hemophilia Gene Therapy Patient Journey: Questions and Answers for Shared Decision-Making.
Article in Patient preference and adherence, 2022. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 14 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
14 citing papers in PubMed, 33 citations in OpenAlex.
- Final Analysis of the Phase 1/2 Trial of Valoctocogene Roxaparvovec for Severe Haemophilia A.Haemophilia : the official journal of the World Federation of HemophiliaTrial
- Patient, consumer and societal values, perceptions and preferences on high-cost gene therapies.Gene therapy · 2026Article
- The State of Shared Decision-Making in Immune Thrombocytopenia: A Narrative Review and Health Care Provider Perspective.Health science reports · 2026Article
- Enhancing Engagement to Support Shared Decision Making Within Haemophilia: Development of Materials for People With Haemophilia, Caregivers and Healthcare Professionals.Haemophilia : the official journal of the World Federation of Hemophilia · 2025Article
- Gene therapy in hemophilia: the dawn of a new era.Research and practice in thrombosis and haemostasis · 2025Review
- Mapping the Patient Experience in a Pediatric Hemophilia Unit: Our Patient Journey.Journal of clinical medicine · 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
- Barriers to gene therapy, understanding the concerns people with haemophilia have: an exigency sub-study.Orphanet journal of rare diseases · 2024Article
- [Gene therapy of Hemophilia: Recommendations from the German, Austrian, and Swiss Society for Thrombosis and Haemostasis Research (GTH)].Hamostaseologie · 2023Article
- Gene Therapy for Hemophilia A: A Mixed Methods Study of Patient Preferences and Shared Decision-Making.Patient preference and adherence · 2023Article
- How to translate and implement the current science of gene therapy into haemophilia care?Therapeutic advances in hematology · 2023Review
- Infrastructural considerations of implementing gene therapy for hemophilia in the Nordic context.Therapeutic advances in hematology · 2023Article
- Supporting Patient-Centred Decisions on Gene Therapy for Haemophilia A: A Conceptual Model.Haemophilia : the official journal of the World Federation of HemophiliaArticle
- The emerging need to manage patient expectations of haemophilia gene therapy amidst media hype in the UK.Therapeutic advances in rare diseaseArticle
Corrections and comments
PubMed lists nothing against this paper. Absence here is not a guarantee, only a check that was made.
Authors and funding
5 authors at 5 institutions in 4 countries.
Funding
No grant is acknowledged in the PubMed record.
Abstract
Purpose: The anticipated emergence of hemophilia gene therapy will present people with hemophilia (PWH) and treating clinicians with increasingly complex treatment options. It will be critical that PWH and their families be empowered to participate fully in decision-making through transparent communication and the development of targeted educational resources. Methods: The Council of Hemophilia Community (CHC) convened across a series of roundtable meetings to define the patient journey for hemophilia gene therapy, and to develop a question-and-answer style resource to guide discussion between healthcare professionals (HCPs) and their patients. Patient groups were also consulted during the development of this tool. Results: The CHC defined 5 key stages in the hemophilia gene therapy patient journey: pre-gene therapy (information-seeking and decision-making), treatment initiation, short- and long-term post-gene therapy follow-up. PWH will have different questions and concerns at each stage of their journey, which should be discussed with their HCP to aid decision-making. The resulting patient journey infographic and Q&A resource (see Supplementary Materials) has been developed for HCPs and PWH to provide a novel and practical roadmap of key issues and considerations throughout all stages. Conclusion: These resources support a collaborative, patient-centric, shared decision-making approach to inform treatment decision discussions between HCPs and PWH. The value of such discussions will be influenced by the language adopted; health literacy is a particularly important consideration, and these discussions should be accessible and tailored to PWH. HCPs and PWH can benefit from awareness of the common questions and uncertainties as they progress together along the patient journey. While the contents of this article are specific to hemophilia gene therapy, the concepts developed here could be adapted to aid patients in other disease states.
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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.