ArticleThe patient2020
Patient Perspectives on Novel Treatments in Haemophilia: A Qualitative Study.
Article in The patient, 2020. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 20 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
20 citing papers in PubMed, 45 citations in OpenAlex.
- Durability of efficacy, safety, and quality of life 5 years after valoctocogene roxaparvovec gene transfer for severe hemophilia A: final phase 3 GENEr8-1 trial results.Research and practice in thrombosis and haemostasis · 2026Trial
- Blood biodistribution and vector shedding of valoctocogene roxaparvovec in people with severe hemophilia A.Blood advances · 2024Trial
- Clinical immunogenicity outcomes from GENEr8-1, a phase 3 study of valoctocogene roxaparvovec, an AAV5-vectored gene therapy for hemophilia A.Molecular therapy : the journal of the American Society of Gene Therapy · 2024Trial
- 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
- Efficacy, safety, and quality of life 4 years after valoctocogene roxaparvovec gene transfer for severe hemophilia A in the phase 3 GENEr8-1 trial.Research and practice in thrombosis and haemostasis · 2024Article
- Evaluating Gene Therapy as a Potential Paradigm Shift in Treating Severe Hemophilia.BioDrugs : clinical immunotherapeutics, biopharmaceuticals and gene therapy · 2023Review
- Patients' and health care providers' perspectives on quality of hemophilia care in the Netherlands: a questionnaire and interview study.Research and practice in thrombosis and haemostasis · 2023Article
- Valoctocogene roxaparvovec gene transfer in participants with HIV.Blood advances · 2023Article
- How to translate and implement the current science of gene therapy into haemophilia care?Therapeutic advances in hematology · 2023Review
- Patients' view on gene therapy development for lysosomal storage disorders: a qualitative study.Orphanet journal of rare diseases · 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
- Modeling Benefits, Costs, and Affordability of a Novel Gene Therapy in Hemophilia A.HemaSphere · 2022Article
- The Hemophilia Gene Therapy Patient Journey: Questions and Answers for Shared Decision-Making.Patient preference and adherence · 2022Article
- Gene therapy in haemophilia: literature review and regional perspectives for Turkey.Therapeutic advances in hematology · 2022Review
- Patient perspectives regarding gene therapy in haemophilia: Interviews from the PAVING study.Haemophilia : the official journal of the World Federation of Hemophilia · 2021Article
- Patients' Perception of the Impact of Innovation on Hemophilia Care Management Organization: A Qualitative Study Protocol (INNOVHEMO Study).Patient preference and adherence · 2021Article
- Patient and clinician opinions of patient reported outcome measures (PROMs) in the management of patients with rare diseases: a qualitative study.Health and quality of life outcomes · 2020Article
- 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
10 authors at 5 institutions in 1 country.
Funding
No grant is acknowledged in the PubMed record.
Abstract
BACKGROUND AND
objectiveNew treatments for haemophilia are under development or entering the market, including extended half-life products, designer drugs and gene therapy, thereby increasing treatment options for haemophilia. It is currently unknown how people with haemophilia decide whether to switch to a new treatment. Therefore, the objective of this study was to explore what factors may play a role when Dutch patients and parents of boys with moderate or severe haemophilia make decisions about whether to switch to a different treatment, and how disease and treatment characteristics may affect these decisions. This may aid clinical teams in tailored information provision and shared decision making.
methodsWe conducted interviews among adults with moderately severe or severe haemophilia and parents of young boys with severe haemophilia. We aimed to include participants from a variety of backgrounds in terms of involvement in the haemophilia community, age, treatment centre and treatments. Participants were recruited through the Netherlands Haemophilia Society and a haemophilia treatment centre. Semi-structured interviews were recorded and transcribed verbatim. Thematic content analysis was used to analyse the data.
resultsTwelve people with haemophilia and two mothers of boys with haemophilia were included. In general, participants reported to be satisfied with their current treatment. However, they considered ease of use of the medication (fewer injections, easier handling, alternative administration) an added value of new treatments. Participants were aware of the high cost of coagulation factor products and some expressed their concern about the Netherlands Haemophilia Society's long-term willingness to pay for current and novel treatments, especially for increased usage due to high-risk activities. Participants also expressed their concerns about the short- and long-term safety of new treatments and believed the effects of gene therapy were not yet fully understood. Participants expected their treatment team to inform them when a particular new treatment would be suitable for them.
conclusionsWith the number of treatment options set to increase, it is important for healthcare providers to be aware of how patient experiences shape patients' decisions about new therapies.
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.