Haemophilia (05/21/19) Miesbach, W.; O'Mahoney, B.; Key, N.S.; et al.
Although gene therapy could revolutionize treatment for people with hemophilia, reducing their bleeding risk while reducing or abrogating the need for exogenous factor administration, researchers say it is important for both physicians and patients to have sources of clear and reliable information in order to discuss both the risks and benefits of treatment. Research involving adeno-associated viral (AAV) vector-mediated gene therapy has shown improvement in endogenous factor levels over sustained periods, significant reduction in annualized bleed rates, lower exogenous factor usage, and a positive safety profile. However, it is important for physicians to stress that research is ongoing, and evidence gaps remain, such as long-term safety profiles. Furthermore, key patient groups—including children and adolescents, those with liver or kidney dysfunction, and those with a prior history of factor inhibitors or pre-existing neutralizing AAV antibodies—could be excluded under the eligibility criteria for gene therapy trials.