• Wolfgang Miesbach and Robert Klamroth.
• Medical Clinic 2, Institute of Transfusion Medicine, University Hospital, Frankfurt, Germany.
• Patient Prefer Adher. 2020 Jan 1; 14: 767-770.

BackgroundThe phase ½ hemophilia B clinical trial (AMT-060) demonstrated stable endogenous FIX levels after 3.5 years (mean FIX activity between 5.1% and 7.5%) with continued reductions in annualized bleeds to near zero with the higher dose, and a 78-96% reduction by year in exogenous FIX use.ObjectiveThe views of all the three participants from Germany participating in the AMT-060 study have been investigated about their experiences with conventional and gene therapy and the effects of the forms of therapy on everyday life.Patients/MethodsThe patients (aged 33-35 years) performed regular prophylactic replacement with factor IX concentrate prior to the gene therapy, reported 0-7 bleeds in the year prior to the treatment, with Hemophilia Joint Health Scores of 0-8. Following topics have been investigated "dealing with illness", "participation in studies", "perception of conventional therapy", "perception of gene therapy", "significance of participation in gene therapy studies", "therapy of haemophilia after the end of the study".ResultsAll three participants have started to become more active and do more sports. However, they expressed anxiety about not knowing how long the effect would last and they felt that psychological support would be needed if the factor IX level fell back in the future. No patient expressed concern about any long-term potential negative consequences of gene therapy.ConclusionGene therapy has the potential to change the life of patients with haemophilia not only by the reduction of bleeding events but also by the increase of active and sportive activities.© 2020 Miesbach and Klamroth.

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