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The Patient Experience of Gene Therapy for Hemophilia: Qualitative Interviews with Trial Patients

Authors Miesbach W, Klamroth R

Received 23 November 2019

Accepted for publication 12 March 2020

Published 22 April 2020 Volume 2020:14 Pages 767—770


Checked for plagiarism Yes

Review by Single anonymous peer review

Peer reviewer comments 2

Editor who approved publication: Dr Johnny Chen

Wolfgang Miesbach,1 Robert Klamroth2

1Medical Clinic 2, Institute of Transfusion Medicine, University Hospital, Frankfurt, Germany; 2Department for Internal Medicine, Vascular Medicine and Coagulation Disorders, Vivantes Klinikum im Friedrichshain, Berlin D - 10249, Germany

Correspondence: Wolfgang Miesbach
Medical Clinic 2, Institute of Transfusion Medicine, University Hospital, Frankfurt, Germany
Tel +49 69 6301-5051

Background: The 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.
Objective: The 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/Methods: The 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”.
Results: All 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.
Conclusion: Gene 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.

Keywords: gene therapy, haemophilia, patient experience, patient interview

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