An exploration of why men with severe haemophilia might not want gene therapy: The exigency study

Abstract

Introduction For many people with haemophilia (PwH) gene therapy offers a potential functional cure. However, some have stated that they do not wish to have gene therapy either now or in the future. Aim This sub‐study, part of the larger Exigency programme, assesses the attitudes, views and understanding of those who do not wish to undergo gene therapy. Methods Participants were approached via social media and word of mouth referral and invited to participate in a focus group or individual interview to discuss their views. Interviews were recorded, transcribed verbatim and analysed thematically. Results Ten adult men with severe haemophilia (eight haemophilia A and two haemophilia B), mean age 34.3 years, participated in a 1‐h focus group (n = 9) or interview (n = 1). All were on prophylaxis. None reported significant treatment burden, and all had annual bleeding rates of less than five in the previous 12 months. Four major themes emerged: self‐identity and its loss, lack of long‐term safety and efficacy data, ongoing concerns about past viral infection, and lack of current treatment burden. Conclusion There are many concerns about gene therapy, including eligibility, effectiveness and safety, which may result in individuals declining it as a therapy. These concerns may recede as more data are published. This study reveals a psychological dynamic around self‐identity and belonging for PwH. The nature of this dynamic is poorly understood and needs exploration to facilitate support for those making decisions about gene therapy.

The experiences of people with haemophilia and their families of gene therapy in a clinical trial setting: regaining control, the Exigency study

“…The Exigency programme [ 19 , 35 ] has been carried out in a high-income country where PwH have good access to intensive treatment. The concerns and issues raised may differ from those of low- and middle-income countries, or the emphasis placed on them may be different.…”

Gene Therapy for Hemophilia A: A Mixed Methods Study of Patient Preferences and Shared Decision-Making

“…Athale published SDM tools, including clinician and patient facing documents, related to key treatment decisions related to prophylaxis and immune tolerance induction. 10 To our knowledge there are no other published SDM tools for hemophilia, but there is a call to action for education and SDM tools and assurance of informed consent and pre-GT education 2,[4][5][6][7]11,12 in anticipation of commercial approval of GT for hemophilia. In a qualitative study of men who underwent GT, the men reported that they were unprepared for the side effects of immunosuppression as well as the follow-up requirements, 6 and in another study participants noted that patients need to be better prepared.…”

“…The "Exigency programme" in the United Kingdom was designed to explore knowledge, expectations, and GT among stakeholders including parents of children with hemophilia and men with hemophilia who have not wished to pursue GT or have had GT. [5][6][7] These qualitative studies provide important data regarding information needs for a PwH considering GT. Wang et al developed a clinician-oriented shared decision-making Q&A resource based on roundtable discussions with health care professionals and patients in collaboration with the Bayer Global and European Patient Councils for Hemophilia and the Bayer Hemophilia Employee Council.…”

“I didn’t know women could have haemophilia”: A qualitative case study

“…This case study emerged from the ongoing Exigency programme [16][17][18] 1). Other than parents of children with haemophilia and partners of men with haemophilia, women were excluded from the Exigency programme.…”

“…She says the exclusion of women in this way has to change because it feels like they are being "punished for having children or wanting to have children or having periods". The right to choose to have a treatment or not, as some men with haemophilia are choosing not to with gene therapy [17] , should be fundamental. To have those decisions enforced because of a lack of available safety data resulting from exclusion from clinical trials would appear to be unjust.…”