How to discuss gene therapy for haemophilia? A patient and physician perspective.

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, su

SUBMITTER: Miesbach W 

PROVIDER: S-EPMC6852207 | biostudies-literature | 2019 Jul

REPOSITORIES: biostudies-literature