Miesbach, W. orcid.org/0000-0002-4506-0061, O’Mahony, B. orcid.org/0000-0001-9780-6972, Key, N.S. orcid.org/0000-0002-8930-4304 et al. (1 more author) (2019) How to discuss gene therapy for haemophilia? A patient and physician perspective. Haemophilia, 25 (4). pp. 545-557. ISSN 1351-8216
Abstract
Gene therapy has the potential to revolutionise treatment for patients with haemophilia and is close to entering clinical practice. While factor concentrates have improved outcomes, individuals still face a lifetime of injections, pain, progressive joint damage, the potential for inhibitor development and impaired quality of life. Recently published studies in adeno‐associated viral (AAV) vector‐mediated gene therapy have demonstrated improvement in endogenous factor levels over sustained periods, significant reduction in annualised bleed rates, lower exogenous factor usage and thus far a positive safety profile. In making the shared decision to proceed with gene therapy for haemophilia, physicians should make it clear that research is ongoing and that there are remaining evidence gaps, such as long‐term safety profiles and duration of treatment effect. The eligibility criteria for gene therapy trials mean that key patient groups may be excluded, eg children/adolescents, those with liver or kidney dysfunction and those with a prior history of factor inhibitors or pre‐existing neutralising AAV antibodies. Gene therapy offers a life‐changing opportunity for patients to reduce their bleeding risk while also reducing or abrogating the need for exogenous factor administration. Given the expanding evidence base, both physicians and patients will need sources of clear and reliable information to be able to discuss and judge the risks and benefits of treatment.
Metadata
Item Type: | Article |
---|---|
Authors/Creators: |
|
Copyright, Publisher and Additional Information: | © 2019 The Authors. Haemophilia Published by John Wiley & Sons Ltd. This is an open access article under the terms of the Creative Commons Attribution License, http://creativecommons.org/licenses/by/4.0/, which permits use, distribution and reproduction in any medium, provided the original work is properly cited. |
Keywords: | Adeno‐associated virus; factor IX; factor VIII; gene therapy; haemophilia |
Dates: |
|
Institution: | The University of Sheffield |
Academic Units: | The University of Sheffield > Sheffield Teaching Hospitals |
Depositing User: | Symplectic Sheffield |
Date Deposited: | 23 May 2019 15:01 |
Last Modified: | 03 Dec 2021 11:05 |
Status: | Published |
Publisher: | Wiley |
Refereed: | Yes |
Identification Number: | 10.1111/hae.13769 |
Open Archives Initiative ID (OAI ID): | oai:eprints.whiterose.ac.uk:146485 |