镰状细胞病(SCD)是一种具有高发病率和早期死亡率的慢性遗传性疾病;它在美国影响近100,000个人。骨髓移植,唯一的治疗方法,由于许多访问障碍,不到20%的患者可用。基因转移疗法(GTT)已被证明在动物模型中具有治愈性,并被批准在一些中心用于人类的早期研究。GTT将为所有患者提供更容易获得的治疗选择。重要的是了解患者对GTT的看法,以帮助确保人体临床试验的成功。
对年龄较小(18-30岁)和年龄较大(31岁及以上)的SCD患者进行了两个重点小组,以获取患者对GTT的知识和信念的数据。来自这两个焦点小组的数据用于开发GTT教育手册。进行了第三个焦点小组,以获取参与者对教育和手册的可接受性和可行性的反馈。
大多数成年人,尤其是年轻人,对GTT知之甚少,对化疗的副作用表示恐惧和不确定(例如,脱发,不孕症),使用人类免疫缺陷病毒(HIV)衍生的病毒载体,和潜在的癌症风险。参与者希望教育材料完全透明,但建议研究人员不要分享这种媒介与HIV的关系,因为文化上的耻辱,也没有HIV病毒用于GTT媒介。
老年人比年轻人更愿意参与人体临床GTT试验。在招募试验时,研究人员应该开发GTT教育材料,解决参与者对医疗保健系统缺乏信任的问题,文化信仰,与副作用有关的恐惧,并包括视觉插图。使用此类材料将为患有SCD的成年人提供充分评估GTT所需的信息。
Sickle cell disease (SCD) is a chronic genetic disease with high morbidity and early mortality; it affects nearly 100,000 individuals in the USA. Bone marrow transplantation, the only curative treatment, is available to less than 20% of patients because of a number of access barriers. Gene transfer therapy (GTT) has been shown to be curative in animal models and is approved for use in humans for early-phase studies at a few centers. GTT would offer a more accessible treatment option available to all patients. It is important to understand patient perspectives on GTT to help ensure human clinical trial success.
Two focus groups were conducted with younger (18-30 years) and older (31 years and older) adults with SCD to obtain data on patient knowledge and beliefs about GTT. Data from these two focus groups was used to develop a GTT educational brochure. A third focus group was conducted to obtain participant feedback on acceptability and feasibility of education and the brochure.
Most adults, especially young adults, had little knowledge about GTT and expressed fear and uncertainty about the side effects of chemotherapy (e.g., hair loss, infertility), use of a human immunodeficiency virus (HIV)-derived viral vector, and potential for cancer risk. Participants wanted full transparency in educational materials, but advised researchers not to share the vector\'s relation to HIV because of cultural stigma and no HIV virus is used for the GTT vector.
Older adults had more desire to participate in human clinical GTT trials than younger participants. When recruiting for trials, researchers should develop GTT educational materials that address participant lack of trust in the healthcare system, cultural beliefs, fears related to side effects, and include visual illustrations. Use of such materials will provide adults with SCD the information they need to fully evaluate GTT.