ArticleHaemophilia : the official journal of the World Federation of Hemophilia2021
Patient perspectives regarding gene therapy in haemophilia: Interviews from the PAVING study.
Article in Haemophilia : the official journal of the World Federation of Hemophilia, 2021. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 26 papers.
What it found
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The trial behind it
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Who cites it
26 citing papers in PubMed, 42 citations in OpenAlex.
- Pharmacovigilance in Cell and Gene Therapy: Evolving Challenges in Risk Management and Long-Term Follow-Up.Drug safety · 2026Review
- Acceptance of Cell and Gene Therapy Among Patients with Hematological Diseases in China: A Cross-Sectional Study.Patient preference and adherence · 2026Article
- Gene therapy for hemophilia: results of ISTH global survey on current knowledge, attitudes, and preparedness of the hemophilia care team.Therapeutic advances in hematology · 2025Article
- Preferences for gene therapy in Duchenne muscular dystrophy: insights from patient and caregiver interviews and attribute development.Frontiers in pharmacology · 2025Article
- 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
- Ethical aspects of hemophilia gene therapy: a qualitative interview study with stakeholders.Research and practice in thrombosis and haemostasis · 2023Article
- "I just wanted to speak to someone- and there was no one…": using Burden of Treatment Theory to understand the impact of a novel ATMP on early recipients.Orphanet journal of rare diseases · 2023Article
- How to translate and implement the current science of gene therapy into haemophilia care?Therapeutic advances in hematology · 2023Review
- Gene Therapy for Hemophilia A: A Mixed Methods Study of Patient Preferences and Shared Decision-Making.Patient preference and adherence · 2023Article
- Decision-making about gene therapy in transfusion dependent thalassemia.BMC pediatrics · 2022Article
- Examining patient and professional perspectives in the UK for gene therapy in haemophilia.Haemophilia : the official journal of the World Federation of Hemophilia · 2022Article
- The experiences of people with haemophilia and their families of gene therapy in a clinical trial setting: regaining control, the Exigency study.Orphanet journal of rare diseases · 2022Article
- Haemophilia gene therapy: experiences and lessons from treated patients.Orphanet journal of rare diseases · 2022Article
- The Hemophilia Gene Therapy Patient Journey: Questions and Answers for Shared Decision-Making.Patient preference and adherence · 2022Article
- Gene Therapy and Hemophilia: Where Do We Go from Here?Journal of blood medicine · 2022Review
- Article
- Patient preferences for gene therapy in haemophilia: Results from the PAVING threshold technique survey.Haemophilia : the official journal of the World Federation of Hemophilia · 2021Article
- Patient preferences and priorities for haemophilia gene therapy in the US: A discrete choice experiment.Haemophilia : the official journal of the World Federation of Hemophilia · 2021Article
- Gene Therapy in Hemophilia: Recent Advances.International journal of molecular sciences · 2021Review
Corrections and comments
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Authors and funding
9 authors at 4 institutions in 3 countries.
Funding
Abstract
introductionExploring patient perceptions regarding gene therapies may provide insights about their acceptability to patients.
objectiveTo investigate opinions of people with haemophilia (PWH) regarding gene therapies. Moreover, this study aimed to identify patient-relevant attributes (treatment features) that influence PWH's treatment choices.
methodsSemi-structured individual interviews were conducted with Belgian PWH, types A and B. A predefined interview guide included information sections and open, attribute ranking and case questions. Qualitative data were organized using NVivo 12 and analysed following framework analysis. Sum totals of scores obtained in the ranking exercise were calculated per attribute.
resultsIn total, 20 PWH participated in the interviews. Most participants demonstrated a positive attitude towards gene therapy and were very willing (40%; n = 8) or willing (35%; n = 7) to receive this treatment. The following five attributes were identified as most important to PWH in making their choice: annual bleeding rate, factor level, uncertainty of long-term risks, impact on daily life, and probability that prophylaxis can be stopped. While participants were concerned about the uncertainty regarding long-term safety, most participants were less concerned about uncertainty regarding long-term efficacy.
conclusionsThis qualitative study showed that most PWH have a positive attitude towards gene therapy and that besides efficacy, safety and the related uncertainties, also impact on daily life is important to patients. The identified patient-relevant attributes may be used by regulators, health technology assessment bodies and payers in their evaluation of gene therapies for haemophilia. Moreover, they may inform clinical trial design, pay-for-performance schemes and real-world evidence studies.
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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.