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.

中文翻译：

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保护动机理论在儿科慢性病临床试验入组中的应用


患有慢性但可控制疾病的儿童的父母希望获得改进的疗法或治愈方法，包括高级治疗药物产品 (ATMP)。 ATMP 的多项儿科临床试验正在进行中，但 ATMP 对慢性病的风险状况很大程度上未知，并且可能与末期儿科疾病不同。我们的研究将修改后的保护动机理论应用于儿科 ATMP 临床试验招募的背景，分析了患有慢性可控疾病（1 型糖尿病 (T1D) 或遗传性视网膜疾病 (IRD)）的儿童父母的信息需求。我们对 15 名 T1D 儿童的家长和 14 名 IRD 儿童的家长进行了半结构化访谈，内容涉及：a) 家庭背景和诊断经验； b) 对 T1D 和 IRD 的基因和干细胞治疗研究和临床试验的认识； c) 有关试验的信息来源以及对该信息的反应； d) 对审判参与的态度，包括国际审判； e) 了解试验目的和过程； f) 参与试验的任何经验。然后，我们讨论了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。父母平衡了试验风险和孩子应对慢性病的能力。孩子应对视力障碍或胰岛素管理的能力越好，父母承担试验风险的可能性就越小。 相反，如果孩子患有视力丧失问题，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。作为威胁评估的一部分，对不良事件的恐惧是父母在考虑是否让患有可控制的慢性病的孩子参加 ATMP 儿科临床试验时的主要考虑因素。这种考虑超过了潜在的好处和孩子病情的严重程度。家长们呼吁提供可用的安全数据和充分的沟通流程，使他们能够代表孩子就临床试验入组做出明智的决定。