Journal of Clinical Trials
Open Access
ISSN: 2167-0870
ISSN: 2167-0870
Sheba Medical Center, Derech Sheba, Ramat Gan, Israel
Research Article
Perceptions and Fears of Hemophilia Patients and Caregivers Regarding Participation in Gene Therapy Clinical Trials
Author(s): Brutman Barazani T, Barg AA, Bashari D, Danin-Kreiselman M, Kenet G and Sarina Levy-Mendelovich*
Introduction: As first products of Hemophilia gene therapy were recently approved by the European Commission
(EC) and Food and Drug Administration (FDA) understanding patient’s fears, expectations and acquaintance of
data is of high interest. Our aim was to investigate the knowledge and concerns of our patients and their caregivers
regarding gene therapy and what are the misconceptions that exist.
Methods: A questionnaire including 18 questions assessing demography, hemophilia history/treatment, knowledge,
fears and expectations regarding gene therapy treatment for hemophilia were collected from 100 patients with severe
hemophilia and their caregivers.
Results: One hundred questionnaires were completed by 65 patients and 35 parents (primary caregivers of patients)
with a median age of 36 years (.. View More»
DOI:
10.35248/2167-0870.23.13.534