क्लिनिकल परीक्षण जर्नल
खुला एक्सेस
आईएसएसएन: 2167-0870
आईएसएसएन: 2167-0870
Brutman Barazani T, Barg AA, Bashari D, Danin-Kreiselman M, Kenet G, 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 (18-75 years). Five of the respondents have previously received a gene therapy product for hemophilia and thus they answered a modified questionnaire. The greatest concern of the participants was that there is not enough data regarding complications and possible liver damage. Only 50/95 knew that Adeno- Associated Viruses (AAV) modified gene therapy will affect only the patient undergoing this treatment. Surprisingly 21/95 think that the mutation will be corrected and therefore hemophilia will not be transferred to their off-springs following gene therapy. Interestingly, among patients with Hemophilia A, the subgroup of Eemicizumab treated patients had more knowledge and more realistic expectations.
Discussion and conclusion: While most of the patients have information about gene therapy, their understanding is limited. In conclusion, this study highlights the need to work with patients, families and pharma companies in order to create better educational resources regarding gene therapy.