Patient Perspectives on Gene Transfer Therapy for Sickle Cell Disease.
Patient Perspectives on Gene Transfer Therapy for Sickle Cell Disease.
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DOI：
10.1007/s12325-017-0587-7
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发表时间：
2017-08
影响因子：
3.8
通讯作者：
Crosby LE
中科院分区：
文献类型：
--
作者：
Strong H;Mitchell MJ;Goldstein-Leever A;Shook L;Malik P;Crosby LE
镰状细胞病（SCD）是一种慢性遗传性疾病，具有高发病率和早期死亡率;在美国影响近10万人。骨髓移植是唯一的治愈性治疗方法，由于存在一些获取障碍，只有不到20%的患者可以获得。基因转移疗法（GTT）已在动物模型中显示出疗效，并在少数中心被批准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。了解患者对GTT的看法非常重要，有助于确保人体临床试验的成功。在年轻（18-30岁）和老年（≥ 31岁）SCD成人中进行了两个焦点组，以获得患者对GTT的知识和信念的数据。来自这两个重点小组的数据被用来编写全球工作队教育手册。第三个重点小组的任务是收集参与者对教育和小册子的可接受性和可行性的反馈意见。大多数成年人，特别是年轻人，对GTT知之甚少，并对化疗的副作用表示恐惧和不确定（例如，脱发、不育）、使用人类免疫缺陷病毒（HIV）衍生的病毒载体以及潜在的癌症风险。与会者希望教育材料完全透明，但建议研究人员不要分享该载体与艾滋病毒的关系，因为文化上的耻辱，没有艾滋病毒被用于GTT载体。老年人比年轻参与者更愿意参与人类临床GTT试验。在招募试验人员时，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，文化信仰，与副作用相关的恐惧，并包括视觉插图。使用这些材料将为SCD成年人提供全面评估GTT所需的信息。
Sickle cell disease (SCD) is a chronic genetic disease with high morbidity and early mortality; affecting nearly 100,000 individuals in the United States. Bone marrow transplantation, the only curative treatment, is available to less than 20% of patients, due to a number of access barriers. Gene transfer therapy (GTT) has 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) 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 due to cultural stigma and no HIVvirus 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.
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影响因子：
1.2
作者：
Patterson, Chavis A.;Barakat, Lamia P.;Hsu, Lewis L.
通讯作者：
Hsu, Lewis L.
接受单倍体相合和匹配相关造血细胞移植治疗镰状细胞病的儿科患者的长期结果和器官功能评估。
DOI：
10.1016/j.bbmt.2013.02.010
发表时间：
2013-05
期刊：
Biology of blood and marrow transplantation : journal of the American Society for Blood and Marrow Transplantation
影响因子：
--
作者：
Dallas MH;Triplett B;Shook DR;Hartford C;Srinivasan A;Laver J;Ware R;Leung W
通讯作者：
Leung W
影响因子：
2.2
作者：
Peters-Lawrence MH;Bell MC;Hsu LL;Osunkwo I;Seaman P;Blackwood M;Guillaume E;Bellevue R;Krishnamurti L;Smith WR;Dampier CD;Minniti CP;Sickle Cell Disease Clinical Research Network (SCDCRN)
通讯作者：
Sickle Cell Disease Clinical Research Network (SCDCRN)
影响因子：
2.6
作者：
Haywood, Carlton, Jr.;Lanzkron, Sophie;Ratanawongsa, Neda;Bediako, Shawn M.;Lattimer-Nelson, Lakshmi;Beach, Mary Catherine
通讯作者：
Beach, Mary Catherine
影响因子：
3.2
作者：
Le, Phu Quoc;Gulbis, Beatrice;Ferster, Alina
通讯作者：
Ferster, Alina