Department of Pediatrics, University of Alberta, 3-62A Heritage Medical Research Centre (HMRC), 11207 - 87 Ave NW, Edmonton, AB, T6G 2S2, Canada.
Faculty of Health Sciences, Simon Fraser University, Blusson Hall 11328, 8888 University Drive, Burnaby, BC, V5A 1S6, Canada.
BMC Pediatr. 2020 Mar 16;20(1):123. doi: 10.1186/s12887-020-2014-5.
Parents of children living with chronic but manageable conditions hope for improved therapies or cures, including Advanced Therapy Medicinal Products (ATMPs). Multiple pediatric clinical trials for ATMPs are underway, but the risk profile of ATMPs for chronic conditions is largely unknown and likely different than for terminal pediatric illnesses. Applying Protection Motivation Theory modified to the context of pediatric ATMP clinical trial enrollment, our study analyses information needs of parents of children living with chronic manageable conditions: Type 1 Diabetes (T1D) or Inherited Retinal Diseases (IRD).
We conducted semi-structured interviews with 15 parents of children living with T1D and 14 parents of children living with an IRD about: a) family background and the diagnostic experience; b) awareness of gene and stem cell therapy research and clinical trials for T1D and IRD; c) information sources on trials and responses to that information; d) attitudes to trial participation, including internationally; e) understanding of trial purpose and process; and f) any experiences with trial participation. We then discussed a pediatric ATMP clinical trial information sheet, which we developed with experts. We applied directed qualitative content analysis, based on PMT, to examine the information preferences of parents in deciding whether to enrol their children in stem cell or gene therapy clinical trials.
Parents balanced trial risks against their child's ability to cope with the chronic condition. The better the child's ability to cope with vision impairment or insulin management, the less likely parents were to assume trial risks. Conversely, if the child struggled with his/her vision loss, parents were more likely to be interested in trial participation, but only if the risks were low and likelihood for potential benefit was high.
Fear of adverse events as part of threat appraisal was the predominant consideration for parents in considering whether to enroll their child living with a manageable, chronic condition in a pediatric clinical trial of an ATMP. This consideration outweighed potential benefits and severity of their child's condition. Parents called for available safety data and fulsome communication processes that would enable them to make informed decisions about clinical trial enrolment on behalf of their children.
患有慢性但可控制疾病的儿童的父母希望获得更好的治疗方法或治愈方法，包括先进的治疗性药物产品（ATMP）。目前正在进行多项针对 ATMP 的儿科临床试验，但 ATMP 治疗慢性疾病的风险概况在很大程度上尚不清楚，而且可能与儿科终末期疾病的风险概况不同。本研究应用经改良后适用于儿科 ATMP 临床试验入组情境的保护动机理论，分析了患有 1 型糖尿病（T1D）或遗传性视网膜疾病（IRD）的儿童的父母的信息需求。
我们对 15 名患有 T1D 的儿童的父母和 14 名患有 IRD 的儿童的父母进行了半结构化访谈，内容涉及：a）家庭背景和诊断经历；b）对 T1D 和 IRD 的基因和干细胞治疗研究及临床试验的认识；c）关于试验的信息来源以及对这些信息的反应；d）对参与试验的态度，包括国际上的态度；e）对试验目的和过程的理解；以及 f）任何参与试验的经历。然后，我们讨论了我们与专家共同制定的儿科 ATMP 临床试验信息表。我们应用基于 PMT 的定向定性内容分析来检查父母在决定是否让孩子参加干细胞或基因治疗临床试验时的信息偏好。
父母权衡了试验风险与孩子应对慢性疾病的能力。孩子应对视力损害或胰岛素管理的能力越强，父母承担试验风险的可能性就越低。相反，如果孩子的视力受损，父母更有可能对参与试验感兴趣，但前提是风险较低且潜在获益的可能性较高。
在考虑是否让患有可控制慢性疾病的孩子参加儿科 ATMP 临床试验时，父母主要考虑的是作为威胁评估一部分的不良事件风险。这一考虑因素超过了潜在获益和孩子病情的严重程度。父母呼吁提供可用的安全性数据和全面的沟通流程，以便他们能够代表孩子做出有关临床试验入组的知情决策。
