Motivations and Decision-Making of Adult Sickle Cell Patients in High-Risk Clinical Research.
Motivations and Decision-Making of Adult Sickle Cell Patients in High-Risk Clinical Research.
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DOI：
10.1016/j.bbmt.2020.03.014
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发表时间：
2020-06
期刊：
影响因子：
--
通讯作者：
Grady C
中科院分区：
文献类型：
--
作者：
Cho HL;Kim SYH;Fitzhugh C;Hsieh M;Tisdale J;Grady C
镰状细胞病（SCD）的基因治疗或干细胞移植（PBSCT）的潜在治愈性但高风险试验为SCD成人带来了新的机会，其中许多人经历了重大的疾病负担和并发症，治疗选择很少，以及护理方面的耻辱和差异。我们探讨了这些试验的参与者和拒绝者的动机和决策过程。半结构化访谈，有目的的样本20名入学者和6名下降。访谈探讨了参与者的SCD经验，动机和决策有关的试验参与，研究相关信息的理解，和回顾性反思。访谈采用内容分析法。大多数人指出了研究的目的、风险和参与的不确定性。参与者和下降者都描述了研究风险和潜在益处（特别是治愈的前景）的深思熟虑的权衡，其中很大程度上考虑了他们的SCD状态，经历和对更好生活的渴望。尽管受到精神/宗教的影响以及家人和朋友的支持，所有人都将参与的决定描述为自己的决定。在一些患者中，试验定义的主要结局状态与患者感知的结局不匹配。有负面经历的患者表示希望在知情同意过程中更加强调风险和可能的结果。这组患有SCD的成年人在决定基因治疗或PBSCT试验时经过深思熟虑。通过强调“成功的”科学成果仍然可能涉及并发症或症状，并通过向前研究参与者推荐和预期讨论来促进未来参与者的决策。
Potentially curative but high-risk trials of gene therapy or stem cell transplantation (PBSCT) for Sickle Cell Disease (SCD) pose new opportunities for adults with SCD, many of whom experience significant disease burden and complications with few treatment options, as well as stigma and disparities in care. We explored motivations and decision-making processes of enrollees and decliners of such trials. Semi-structured interviews with a purposive sample of 20 enrollees and 6 decliners. Interviews explored participants’ SCD experiences, motivations and decision-making about trial participation, understanding of research-related information, and retrospective reflections. Interviews were analyzed with content analysis. Most identified the purpose of research, risks, and uncertainties of participation. Both enrollees and decliners described deliberative weighing of study risks and potential benefits (especially the prospect of a cure), with heavy factoring of their SCD status, experiences, and desire for a better life. Despite the influence of spirituality/religion and support of family and friends, all described the decision about participation as their own. In some patients, the primary outcome status defined by the trial did not match the patients’ perceived outcomes. Patients with negative experiences expressed a desire for greater emphasis on risks and possible outcomes during informed consent. This cohort of adults with SCD were thoughtfully deliberative in their decisions about gene therapy or PBSCT trials. Future participants’ decision-making may be enhanced by emphasizing that ‘successful’ scientific outcomes can still involve complications or symptoms and be facilitated by referrals to former research participants and anticipatory discussions.
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