ArticleOrphanet journal of rare diseases2022
The experiences of people with haemophilia and their families of gene therapy in a clinical trial setting: regaining control, the Exigency study.
Article in Orphanet journal of rare diseases, 2022. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 18 papers, 1 of them a synthesis that pooled it.
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
18 citing papers in PubMed, 1 synthesis or guideline pooled it, 49 citations in OpenAlex.
- Contributions from medical geneticists in clinical trials of genetic therapies: A points to consider statement of the American College of Medical Genetics and Genomics (ACMG).Genetics in medicine : official journal of the American College of Medical Genetics · 2023Guideline
- Patient, consumer and societal values, perceptions and preferences on high-cost gene therapies.Gene therapy · 2026Article
- Getting closer to hemophilia gene therapy for all?Blood advances · 2025Article
- Gene therapy in hemophilia: the dawn of a new era.Research and practice in thrombosis and haemostasis · 2025Review
- UKHCDO gene therapy taskforce: Guidance for implementation of haemophilia gene therapy into routine clinical practice for adults.Haemophilia : the official journal of the World Federation of Hemophilia · 2025Article
- A novel gene editing lexicon strategy for the haemophilia community: Research plan for development and preliminary results.Haemophilia : the official journal of the World Federation of Hemophilia · 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
- Educational needs of patients, families, and healthcare professionals to support the patient journey in haemophilia gene therapy in the UK.Orphanet journal of rare diseases · 2023Article
- Ethical aspects of hemophilia gene therapy: a qualitative interview study with stakeholders.Research and practice in thrombosis and haemostasis · 2023Article
- Depression and anxiety among hemophilia patients enrolled in clinical trials: a multi-center cohort study.Annals of hematology · 2023Article
- Gene Therapy for Hemophilia A: A Mixed Methods Study of Patient Preferences and Shared Decision-Making.Patient preference and adherence · 2023Article
- Patients' view on gene therapy development for lysosomal storage disorders: a qualitative study.Orphanet journal of rare diseases · 2022Article
- Haemophilia gene therapy: experiences and lessons from treated patients.Orphanet journal of rare diseases · 2022Article
- A Comprehensive Approach: Addressing Psychological Needs in Gene Therapy for Haemophilia.Haemophilia : the official journal of the World Federation of HemophiliaReview
- Defining the Role of Nurses in Gene Therapy for Haemophilia.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
- Perceptions and Recommendations Regarding Haemophilia B Gene Therapy: A Multistakeholder View From Patients, Caregivers and Healthcare Professionals.Haemophilia : the official journal of the World Federation of HemophiliaArticle
Corrections and comments
- Commented on by
Authors and funding
5 authors at 1 institution in 1 country.
Funding
No grant is acknowledged in the PubMed record.
Abstract
backgroundGene therapy has the potential to change the life experience of people with haemophilia and family members. Few studies have sought to explore the impact of gene therapy on both individuals and families. The aim of this study was to capture real-life experiences of gene therapy in People with haemophilia and their families.
resultsSixteen participants with severe haemophilia (11 haemophilia A, five haemophilia B), mean age 41.4 years (range 23-75 years), took part in a single qualitative interview; ten were accompanied by a family member. Mean time since transfection was 3.56 years (range 1-10 years). Participants saw their involvement in gene therapy as a positive experience, freeing them from the personal burden of haemophilia and furthering treatment options for the wider haemophilia community. However, participants reported being unprepared for the side effects of immunosuppression. Some also reported feeling unsupported and having little control over what was happening as their factor levels became the focus of the process.
conclusionThe results suggest that strategies need to be put into place to enable PwH fully to understand the process of gene therapy, and thereby make an informed choice as to whether it is a treatment they might wish for themselves. These include early and ongoing education, increased provision of psychosocial support and ongoing qualitative research.
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.