An exploration of why men with severe haemophilia might not want gene therapy : The exigency study
© 2021 John Wiley & Sons Ltd..
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.
| Medienart: |
E-Artikel |
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| Erscheinungsjahr: |
2021 |
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| Erschienen: |
2021 |
| Enthalten in: |
Zur Gesamtaufnahme - volume:27 |
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| Enthalten in: |
Haemophilia : the official journal of the World Federation of Hemophilia - 27(2021), 5 vom: 06. Sept., Seite 760-768 |
| Sprache: |
Englisch |
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| Beteiligte Personen: |
Fletcher, Simon [VerfasserIn] |
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| Links: |
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| Themen: |
Decision making |
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| Anmerkungen: |
Date Completed 24.09.2021 Date Revised 24.09.2021 published: Print-Electronic Citation Status MEDLINE |
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| doi: |
10.1111/hae.14378 |
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| funding: |
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| Förderinstitution / Projekttitel: |
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| PPN (Katalog-ID): |
NLM328106356 |
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| 500 | |a Date Revised 24.09.2021 | ||
| 500 | |a published: Print-Electronic | ||
| 500 | |a Citation Status MEDLINE | ||
| 520 | |a © 2021 John Wiley & Sons Ltd. | ||
| 520 | |a 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 | ||
| 520 | |a 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 | ||
| 520 | |a 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 | ||
| 520 | |a 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 | ||
| 520 | |a 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 | ||
| 650 | 4 | |a Journal Article | |
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| 650 | 4 | |a haemophilia | |
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| 650 | 4 | |a treatment burden | |
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| 700 | 1 | |a Holland, Michael |e verfasserin |4 aut | |
| 700 | 1 | |a Chaplin, Steve |e verfasserin |4 aut | |
| 700 | 1 | |a Khair, Kate |e verfasserin |4 aut | |
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