Center for Bioethics and Social Sciences in Medicine, Michigan Medicine, US.
Department of Neurology, Michigan Medicine, US.
Tremor Other Hyperkinet Mov (N Y). 2021 Jan 20;11:3. doi: 10.5334/tohm.584.
Promising disease modifying therapies for Huntington's disease are now entering pivotal trials, raising questions of what patients and families consider successful outcomes. Consistent with an ongoing movement to incorporate patient preferences into the development of new therapies, we conducted a pilot study to assess Huntington's disease community views on emerging DMTs to assist in planning large-scale studies of patient preferences.
Semi-structured interviews were conducted with members of the Huntington's community (manifest disease, at-risk, and family/caregivers). Participants were asked which symptoms they believed should be targeted with novel treatments, as well as potential benefits and tradeoffs of delaying symptom onset versus prolonging late-stage disease.
Participants (N = 14) emphasized the need for treatments improving cognitive and/or behavioral symptoms. Many wanted treatments that delayed symptom onset up to 5-10 years, though some considered shorter delays acceptable due to potential value in advancing research to help future generations. Concern regarding potential for prolonging later-stage disease was variable, with some participants uncertain if they would want a treatment that delayed onset but prolonged later-stage disease. Others stated that any delay in onset would be desirable, regardless of potential prolongation of later stage disease.
This study demonstrates a breadth of opinions among the Huntington's disease community surrounding both the benefits and complex tradeoffs that might occur with disease modifying treatments. These preliminary findings will inform future large-scale studies of attitudes toward disease modifying treatments, which may ultimately guide the design and outcome measure selection for clinical trials.
In-depth interviews with the Huntington's disease community were used to explore patient and family preferences regarding potential disease modifying therapies. Many wanted symptom delay of 5-10 years, though some considered shorter delays acceptable for altruistic reasons. Opinions on trade-offs varied, suggesting larger preference studies are needed to inform trial design.
有前景的亨廷顿舞蹈症治疗方法目前正在进入关键性试验阶段，这引发了一个问题，即患者和家属认为什么样的结果才算是成功。为了将患者的偏好纳入新疗法的开发过程中，我们进行了一项试点研究，以评估亨廷顿舞蹈症社区对新兴疾病修饰疗法的看法，为规划大规模的患者偏好研究提供帮助。
对亨廷顿舞蹈症社区的成员（有明显症状、有患病风险和家属/照顾者）进行了半结构式访谈。参与者被问到他们认为哪些症状应该作为新疗法的目标，以及延迟症状发作与延长晚期疾病的潜在获益和权衡。
参与者（N=14）强调需要治疗改善认知和/或行为症状的方法。许多人希望治疗方法能延迟症状发作 5-10 年，但也有一些人认为由于推进研究以帮助后代的潜在价值，较短的延迟也是可以接受的。对于潜在延长晚期疾病的担忧程度不一，一些参与者不确定他们是否会想要一种既能延迟发作又能延长晚期疾病的治疗方法。还有一些人则表示，无论潜在的晚期疾病延长时间如何，任何延迟发作的治疗方法都是可取的。
这项研究展示了亨廷顿舞蹈症社区在疾病修饰治疗的获益和复杂权衡方面的广泛观点。这些初步发现将为未来关于疾病修饰治疗态度的大规模研究提供信息，这可能最终会指导临床试验的设计和结局测量的选择。
对亨廷顿舞蹈症社区进行深入访谈，以探讨患者和家属对潜在疾病修饰疗法的偏好。许多人希望延迟症状发作 5-10 年，但也有一些人出于利他主义的原因认为较短的延迟是可以接受的。对权衡取舍的看法各不相同，这表明需要进行更大规模的偏好研究，以指导试验设计。
