Hemophilia gene therapy knowledge and perceptions: Results of an international survey

Research and Practice in Thrombosis and Haemostasis

Claire McLintock¹  K. John Pasi²  Alok Srivastava³  Johnny Mahlangu⁴  Thierry VandenDriessche⁵  David Lillicrap⁶  Wendy Scales⁷  Steven W. Pipe⁸  Flora Peyvandi⁹ 

[1] Auckland City Hospital Auckland New Zealand;Barts and the London School of Medicine and Dentistry London UK;Christian Medical College Vellore India;Faculty of the Health Sciences University of the Witwatersrand and NHLS Johannesburg South Africa;Katholieke Universiteit Leuven Vrije University Brussels Brussels Belgium;Queen’s University Kingston ON Canada;The France Foundation Old Lyme CT USA;University of Michigan Ann Arbor MI USA;University of Milan Milan Italy;

关键词: gene therapy;    hemophilia;    knowledge gaps;    physicians;    respondents;    survey;   

DOI  :  10.1002/rth2.12326

来源: DOAJ

【 摘 要 】

Abstract Background Hemophilia gene therapy is a rapidly evolving therapeutic approach in which a number of programs are approaching clinical development completion. Objective The aim of this study was to evaluate knowledge and perceptions of a variety of health care practitioners and scientists about gene therapy for hemophilia. Methods This survey study was conducted February 1 to 18, 2019. Survey participants were members of the ISTH, European Hemophilia Consortium, European Hematology Association, or European Association for Hemophilia and Allied Disorders with valid email contacts. The online survey consisted of 36 questions covering demographic information, perceptions and knowledge of gene therapy for hemophilia, and educational preferences. Survey results were summarized using descriptive statistics. Results Of the 5117 survey recipients, 201 responded from 55 countries (4% response rate). Most respondents (66%) were physicians, and 59% were physicians directly involved in the care of people with hemophilia. Among physician respondents directly involved in hemophilia care, 35% lacked the ability to explain the science of adeno‐associated viral gene therapy for hemophilia, and 40% indicated limited ability or lack of comfort answering patient questions about gene therapy for hemophilia based on clinical trial results to date. Overall, 75% of survey respondents answered 10 single‐answer knowledge questions correctly, 13% incorrectly, and 12% were unsure of the correct answers. Conclusions This survey highlighted knowledge gaps and educational needs related to gene therapy for hemophilia and, along with other inputs, has informed the development of “Gene Therapy in Hemophilia: An ISTH Education Initiative.”

【 授权许可】

Unknown