Social and Behavioral Research Branch, National Human Genome Research Institute, Bethesda, MD, USA.
Center for Newborn Screening, Ethics, and Disability Studies, RTI International, Research Triangle Park, NC, USA.
J Intellect Disabil Res. 2019 Aug;63(8):926-935. doi: 10.1111/jir.12605. Epub 2019 Feb 12.
The objective of this research was to understand parental proxy decision-making for drug trial participation for children with Fragile X syndrome (FXS). Specifically, we aimed to capture preferences, motivations, influencing factors and barriers related to trial involvement among trial joiners and decliners and describe ease of trial decision-making and decisional regret.
Interviews were conducted with parents from two groups: those who chose to enrol their child with FXS in a trial (N = 16; Joiners) and those who declined trial participation (N = 15; Decliners). Data were coded and interpreted through inductive content analysis.
Prominent decisional factors included attitudes about medicating FXS symptoms, potential for direct benefit (primarily evaluated through the degree of match between target outcomes and child symptomatology and drug mechanism), logistical convenience and perceived risks of side effects. The ultimate motivation for participation was potential for direct benefit. None of the parents reported decisional regret, and ease of decision-making ranged from easy to difficult for our participants.
Therapeutic optimism was high among those who elected participation. Parents may benefit from an explanation of the rationale behind chosen outcome variables and may be more interested in trials that target or measure as an exploratory outcome the symptoms they find most concerning. Our findings reinforce the need for future trials to reduce participant inconvenience. Our results contrast with what has previously been observed in parents of children with life-threatening conditions; parents of children with FXS may be more trial risk averse and find trial decisions to be harder. Parents of children with FXS considering trials may benefit from a decisional intervention aimed at deliberating motivations and barriers.
本研究旨在了解脆性 X 综合征 (FXS) 患儿父母作为代理人参与药物试验的决策过程。具体而言，我们旨在捕捉试验参与者和拒绝者与试验参与相关的偏好、动机、影响因素和障碍，并描述试验决策的难易程度和决策后悔感。
对两组父母进行了访谈：一组是选择让其患有 FXS 的孩子参加试验的父母（N=16；参加者），另一组是拒绝参加试验的父母（N=15；拒绝者）。通过归纳内容分析法对数据进行编码和解释。
主要的决策因素包括对治疗 FXS 症状的态度、潜在的直接获益（主要通过目标结果与儿童症状和药物机制的匹配程度来评估）、便利程度和潜在的副作用风险。参与的最终动机是潜在的直接获益。没有父母报告决策后悔感，我们的参与者对决策的难易程度从容易到困难不等。
选择参与的父母表现出很高的治疗乐观态度。父母可能受益于对选择的结果变量背后的基本原理的解释，并且可能对针对他们最关心的症状作为探索性结果进行目标或测量的试验更感兴趣。我们的研究结果强化了未来试验减少参与者不便的必要性。我们的研究结果与之前在患有危及生命疾病的儿童的父母中观察到的结果形成对比；患有 FXS 的儿童的父母可能更不愿意冒险参与试验，并且发现试验决策更难。考虑参与试验的 FXS 儿童的父母可能受益于一项旨在权衡动机和障碍的决策干预措施。
