Understanding Patient Expectations of Treatment Outcomes

人们对肿瘤学1期临床试验的伦理问题提出了重要的关注。在之前的研究中，许多患者报告对1期试验的个人临床益处抱有很高的期望。在导致这项建议的项目(RO1CAIOO77I)中，研究人员检查了当患者报告对收益的高期望时他们的意思是什么，以及这些报告是否反映了对I期试验的理解不足。使用混合方法，研究人员确定了真正理解高期望对每个患者意味着什么的能力的几个限制。在继续他们的工作中，调查人员建议从不同的角度评估对利益的高期望的伦理意义。具体地说，研究人员建议在I期试验中解决对益处的高期望是否对患者有害。许多患者将不会体验到I期试验的良好临床结果。决策情感理论预测，对受益期望较高的患者可能会经历更大的痛苦和对其参与的负面感觉。有里约数据可用来确定，对福利的高期望何时反映了一种有益的应对机制，而不是一种潜在的有害形式的否认。这种知识上的差距带来了严重的困难，因为没有证据可以指导临床医生和研究人员决定是鼓励还是阻止患者表达过高的期望。随着ROICAI 00771的更新，研究人员将收集初步数据，帮助临床医生、研究人员和负责人类受试者保护的人员了解在I期试验中参与者对收益期望过高的后果。研究人员将通过追求3个具体目标来实现这一目标：(1)探索第一阶段试验中经历疾病进展的参与者对受益的期望与后来的身心健康之间的联系。研究人员假设，较高的期望值与抑郁和焦虑的增加更大相关，他们将探索与身体功能的联系。(2)探索在经历疾病进展的I阶段试验的参与者中，对收益的期望与后来对同意过程的看法之间的关联。调查人员假设，较高的期望值与对登记参加试验的决定更多的后悔有关。(3)探索第一阶段研究参与者在第一次疾病重新评估时患有非进展性疾病的期望与后来经历之间的关系。研究人员将在一项前瞻性研究中实现这些目标，在患者参加第一阶段试验后立即对患者进行结构化访谈，并在患者收到他们最初的疾病重新评估结果后一周进行后续访谈。这项研究将产生初步数据，解决在第一阶段肿瘤学试验中对研究参与者福利的理解方面的一个重要差距。如果对良好结果的高度信心会给临床结果不佳的患者带来负面后果，这一发现可能表明，临床医生和研究人员应该努力降低患者的过高期望。然而，如果高期望没有负面影响，临床医生和研究人员可能会考虑是否允许对他们同意的试验表现出明确理解的患者使用这种治疗乐观态度。

Important concerns have been raised about the ethics of phase 1 clinical trials in oncology. In previous research, many patients have reported high expectations of personal clinical benefit from phase 1 trials. In the project leading to this proposal (RO1CAIOO77I), the investigators examined what patients mean when they report high expectations of benefit and whether these reports reflect poor understanding about the phase I trial. Using a mixed-methods approach, the investigators identified several limitations on the ability to truly understand what high expectations mean to every patient. In continuing their work, the investigators propose to evaluate the ethical significance of high expectations of benefit from a different perspective. Specifically, the investigators propose to address whether high expectations of benefit are harmful to patients in phase I trials. Many patients will not experience good clinical outcomes from phase I trials. Decision affect theory predicts that patients with higher expectations of benefit might experience greater distress and negative feelings about their participation. There are rio data available to determine when high expectations of benefit reflect a helpful coping mechanism rather than a potentially harmful form of denial. This gap in knowledge presents serious difficulty, because there is no evidence to guide clinicians and researchers in deciding whether to foster or discourage patients' expressions of high expectations. With a renewal of ROICAI 00771, the investigators will collect preliminary data that will help clinicians, researchers, and persons charged with human subjects protection to understand the consequences of high expectations of benefit among participants in phase I trials. The investigators will achieve this objective by pursuing 3 Specific Aims: (1) To explore associations between expectations of benefit and later physical and mental well-being among participants in phase I trials who experience disease progression. The investigators hypothesize that higher expectations are associated with greater increases in depression and anxiety, and they will explore associations with physical functioning. (2) To explore associations between expectations of benefit and later perceptions of the consent process among participants in phase I trials who experience disease progression. The investigators hypothesize that higher expectations are associated with more regret concerning the decision to enroll in the trial. (3) To explore relationships between expectations and later experiences among phase I research participants who have nonprogressive disease at the time of their first disease reevaluation. The investigators will accomplish these aims in a prospective study using structured interviews of patients immediately after enrollment in a phase 1 trial and follow-up interviews I week after the patients receive the results of their initial disease reevaluations. The study will generate preliminary data that address an important gap in understanding of research participants' welfare in phase 1 oncology trials. If high confidence in a good outcome has negative consequences for patients with poor clinical outcomes, this finding might suggest that clinicians and researchers should try to reduce patients' high expectations. However, if there are no negative effects of high expectations, clinicians and researchers might consider whether to allow such therapeutic optimism in patients who have demonstrated clear understanding of the trial to which they have consented.