Hemophilia gene therapy knowledge and perceptions: Results of an international survey
Hemophilia gene therapy knowledge and perceptions: Results of an international survey
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DOI：
10.1002/rth2.12326
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发表时间：
2020-03-19
影响因子：
4.6
通讯作者：
VandenDriessche, Thierry
中科院分区：
文献类型：
--
作者：
Peyvandi, Flora;Lillicrap, David;VandenDriessche, Thierry
背景血友病基因治疗是一种快速发展的治疗方法，许多项目正在接近临床开发完成。目的本研究的目的是评估各种医疗保健从业者和科学家对血友病基因治疗的知识和看法。方法本调查研究于2019年2月1日至18日进行。调查参与者是ISTH、欧洲血友病联盟、欧洲血液学协会或欧洲血友病及相关疾病协会的成员，并具有有效的电子邮件联系方式。在线调查包括36个问题，涵盖人口统计学信息，对血友病基因治疗的看法和知识以及教育偏好。调查结果进行了总结，使用描述性statistics.Results的5117调查收件人，201回答来自55个国家（4%的响应率）。大多数受访者（66%）是医生，59%是直接参与血友病患者护理的医生。在直接参与血友病治疗的医生受访者中，35%缺乏解释血友病腺相关病毒基因治疗科学的能力，40%表示根据迄今为止的临床试验结果，回答血友病基因治疗患者问题的能力有限或缺乏安慰。总体而言，75%的调查受访者回答10个单一答案的知识问题正确，13%不正确，12%不确定的正确答案。结论本次调查突出了知识差距和血友病基因治疗相关的教育需求，沿着其他投入，已通知发展“血友病基因治疗：ISTH教育倡议。"
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."