PediQUEST: Improving Children's Quality of Life through e-PROMS and Palliative Care

摘要 在任何时候，数以万计的儿童都生活在严重的疾病中，他们和他们的家人忍受着高度的 痛苦的程度。虽然国家建议对重症患者进行早期姑息治疗(PC) 为了减轻儿童的痛苦，很少有随机对照试验(RCT)评估PC是否改善了儿童 和家庭结果。在先前的工作中，研究人员开发了儿科生活质量和评估 症状技术(PediQUEST/PQ)，一个收集电子患者报告结果的软件(e-PROMS) 并生成反馈报告。PQ对儿童症状和健康相关问题的反馈效果 家庭和提供者的生活质量(HRQOL)是在三个地点的随机对照试验中测试的，在建立良好的儿科 PC研究网络，在104名患有晚期癌症的2岁儿童中进行了≥调查。这项研究证明了 在这些患者群体中进行多点随机对照试验的可行性，以及获得儿童自我报告的能力。发现 还包括(A)高度的儿童症状痛苦，(B)更严重的症状痛苦与更严重的联系 高质量生活质量，(C)与儿童痛苦有关的高父母心理困扰，和(D)改善儿童高质量生活质量的趋势 介入臂。随后的形成性研究发现了改善症状管理的障碍 包括家庭和提供者“使痛苦正常化”，限制非药物治疗策略的使用，以及 个人电脑的早期参与有限。在Wilson-Cleary的HRQOL模型的基础上，根据这些结果，PI和 研究小组开发了PQ反应(对儿科肿瘤症状体验的反应)。干预措施 包括增强型PQ系统(基于Web)，以及PC咨询团队的早期集成(响应 团队)。这种双重策略寻求使用PQ反馈来标准化症状困扰的报告，并且提供者的 对这种痛苦的反应，经过培训，在评估和与家庭互动中包括一系列步骤 包括治疗和预防策略以及关于症状控制的家庭激活。PQ的试点工作 Response认为它是可行的，受到家庭和肿瘤学家的好评，并具有潜在的有效性。因此，凌驾于 本研究的目的是在四个大型儿科肿瘤学中心进行PQ反应与常规护理的随机对照试验 在2岁晚期癌症儿童中，≥(n=136)。假设包括a)接受干预的儿童 将具有更好(更高)的HRQOL分数b)干预组中孩子的父母将报告更好的状态焦虑， 抑郁与HRQOL和c)干预组家庭将表现出更高的激活水平。家长和 双臂中的儿童(如果≥为5岁)将回答为期16周的每周PQ调查；父母焦虑、抑郁和 HRQOL将每4周评估一次；父母激活每8周一次。假说将用混合语言进行检验 效果模型。该研究将提供关于早期PC集成、使用e-PROM和家长的结果的数据 儿科的激活策略，这是姑息治疗研究中的重要知识差距，并且是一致的 在NINR的战略计划中定义了三个关键主题：症状科学，加强自我管理，以及 姑息治疗。

ABSTRACT At any one time, tens of thousands of children are living with serious illness and they and their families endure a high degree of suffering. While national recommendations call for early palliative care (PC) integration for seriously ill children to ease suffering, very few randomized controlled trials (RCTs) have evaluated whether PC improves child and family outcomes. In prior work, the investigators developed the Pediatric Quality of Life and Evaluation of Symptoms Technology (PediQUEST/PQ), a software that collects electronic Patient-Reported Outcomes (e-PROMS) and generates feedback reports. The effect of using PQ to provide feedback on child's symptoms and health related quality of life (HRQoL) to families and providers was tested in a three-site RCT, within the well established Pediatric PC Research Network, among 104 children ≥2 years old with advanced cancer. The study demonstrated the feasibility of conducting a multisite RCT in this patient population, and an ability to obtain child self-report. Findings also included (a) high levels of child symptom distress, (b) association between higher symptom distress and worse HRQoL, (c) high parental psychological distress linked to child suffering, and (d) trends towards better HRQoL in the intervention arm. Subsequent formative research led to identification of barriers to improving symptom management including families and providers “normalizing distress”, limited use of non-pharmacological treatment strategies, and limited early involvement of PC. Based on Wilson-Cleary's HRQoL model, and informed by these results, the PI and research team developed PQ Response (Response to Pediatric Oncology Symptom Experience). The intervention includes an enhanced PQ system (web-based), coupled with early integration of a PC consulting team (Response team). This dual strategy seeks to standardize the report of symptom distress using PQ feedback, and the providers' response to such distress, trained to include a series of steps in their assessment and interaction with the family including treatment and preventive strategies and family activation regarding symptom control. Pilot work for PQ Response found it feasible, well received by families and oncologists, and potentially effective. Thus, the over-riding aim of this study is to conduct a RCT of PQ Response versus usual care at four large pediatric oncology centers among children ≥2 years old with advanced cancer (n=136). Hypotheses include a) children receiving the intervention will have better (higher) HRQoL scores b) parents of children in the intervention group will report better state-anxiety, depression and HRQoL and c) intervention group families will demonstrate higher levels of activation. Parents and children (if ≥5 years old) in both arms will answer weekly PQ- Surveys for 16 weeks; parent anxiety, depression, and HRQoL will be assessed every 4-weeks; and parent activation every 8 weeks. Hypotheses will be tested using mixed effects models. The study will provide data on outcomes of early PC integration, use of e-PROMS and parent activation strategies in pediatrics, which are important knowledge gaps in palliative care research, and is well aligned with three key themes defined in NINR's strategic plan: science of symptoms, increased self-management, and palliative care.