Survey of Health Care Provider Understanding of Gene Therapy Research for Inherited Metabolic Disorders
Survey of Health Care Provider Understanding of Gene Therapy Research for Inherited Metabolic Disorders
复制标题
DOI：
10.1016/j.clinthera.2022.07.002
复制
发表时间：
2022-08-01
影响因子：
3.2
通讯作者：
Vockley, Jerry
中科院分区：
文献类型：
--
作者：
Hansen, Joyanna;Ramachandran, Radha;Vockley, Jerry
目的：遗传性代谢紊乱 (IMD) 的治疗传统上依赖于难以维持、昂贵且与社会隔离的饮食干预。 IMD 基因疗法的开发旨在提供足够的基因活性来解决这些疾病的根本原因。本研究对医疗保健提供者 (HCP) 进行了调查，以了解他们对基因治疗技术的熟悉程度，并确定多学科护理团队中不同角色的教育需求。 方法：将基于网络的 26 个问题调查的链接分发给北美和欧洲参与 IMD 患者护理的 HCP。使用描述性统计分析结果。结果：在 590 名调查链接接收者中，64 人完成了调查。其中，35 名 (55%) 受访者是医生，23 名 (36%) 是营养师，3 名 (5%) 是执业护士，2 名 (3%) 是遗传咨询师，1 名 (2%) 是护士。大多数调查受访者（88% [n = 64 中的 56]）表示相信 IMD 的基因治疗将在研究进行后 5 年内实现。尽管几乎所有医生（97% [n = 34 of 35]）都表示了解基因治疗，但营养师的熟悉率较低（57% [n = 13 of 23]）；与同事和患者/护理人员交谈的信心也不一致。几乎所有 HCP 都希望获得有关基因治疗进展的教育，而最喜欢的信息来源是已发表的文献和国会演讲。启示：迫切需要有关基因治疗方式相关主题的教育。所有专业都需要有关基因治疗的专业教育，这对于 IMD 护理中的统一治疗实践非常重要。 （Clin Ther. 2022；44：1045-1056。）(c) 2022 作者。由 Elsevier Inc. 出版。这是一篇遵循 CC BY-NC-ND 许可证的开放获取文章 (http://creativecommons.org/licenses/by-nc-nd/4.0/)
Purpose: The treatment of inherited metabolic disorders (IMDs) has traditionally relied on dietary interventions that are difficult to maintain, expensive, and socially isolating. The development of gene therapy for IMDs aims to provide sufficient gene activity to address the underlying causes of these conditions. This study surveyed health care providers (HCPs) to characterize their familiarity with gene therapy technologies and to identify educational needs across roles in a multidisciplinary care team.Methods: The link to a Web-based, 26-question survey was distributed to HCPs in North America and Europe who were involved in IMD patient care. Results were analyzed using descriptive statistics.Findings: Of the 590 survey link recipients, 64 completed the survey. Of these, 35 (55%) respondents were physicians, 23 (36%) were dietitians, 3 (5%) were nurse practitioners, 2 (3%) were genetic counselors, and 1 (2%) was a nurse. Most survey respondents (88% [n = 56 of 64]) reported the belief that gene therapy for IMDs would be available within 5 years of study conduct. Although nearly all physicians (97% [n = 34 of 35]) expressed awareness of gene therapy, rates of reported familiarity were lower among dietitians (57% [n = 13 of 23]); confidence in conversations with colleagues and patients/caregivers was also discordant. Nearly all HCPs wanted education on gene therapy advancements, and the most preferred informational sources were published literature and congress presentations.Implications: There is an urgent need for education on topics related to gene therapy modalities. Professional education on gene therapies is desired across all specialties and will be important for unified treatment practices in IMD care. (Clin Ther. 2022;44:1045-1056.) (c) 2022 The Author(s). Published by Elsevier Inc. This is an open access article under the CC BY-NC-ND license ( http://creativecommons.org/licenses/by-nc-nd/4.0/ )