ArticleHaemophilia : the official journal of the World Federation of Hemophilia
Perceptions and Recommendations Regarding Haemophilia B Gene Therapy: A Multistakeholder View From Patients, Caregivers and Healthcare Professionals.
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
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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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Who cites it
1 citing paper in PubMed.
- Microarray patches as a potential alternative to injectable fertility medications: a qualitative study of patients' and healthcare professionals' perceptions.International journal of clinical pharmacy · 2026Article
Corrections and comments
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Authors and funding
8 authors.
Funding
Abstract
backgroundGene therapy for Haemophilia B has received FDA approval, offering patients a transformative therapeutic option. However, effective communication about the benefits, risks, long-term efficacy and follow-up of gene therapy remains essential for informed decision-making. This study aimed to explore the diverse expectations, concerns and perspectives of patients with Haemophilia B, their caregivers and healthcare professionals (HCPs) regarding gene therapy and to identify strategies for improving communication.
methodsA prospective qualitative study was conducted using semi-structured interviews with male patients aged ≥12 years with moderate or severe Haemophilia B (factor level ≤ 2%), their caregivers and HCPs (physicians, nurses, social workers, advanced practice providers and pharmacists). Interviews were audio-recorded, transcribed and analysed thematically.
resultsThirty participants were interviewed, including 15 patients (mean age, 21.1 years), caregivers and 15 HCPs across the United States. Patients and caregivers emphasised five themes: (1) current challenges; (2) hope and optimism; (3) concerns and skepticism; (4) the complex emotional challenges of decision-making and (5) preferences for transparent, patient-friendly communication. HCPs identified four complementary themes: (1) variable patient knowledge; (2) the need for transparency in discussing 'curative' language; (3) factors influencing treatment decisions (trust, prior experiences and financial concerns) and (4) strategies to improve communication (clear language, visuals and testimonials).
conclusionStakeholders view gene therapy as both promising and uncertain. Targeted educational interventions, transparent communication and patient-centred decision discussions are essential to fill knowledge gaps and support informed consent in this transformative era of treatment for Haemophilia B.
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Registered trials
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