PediQUEST ResPOND: Piloting an intervention to treat recurrent pain in children with severe neurological impairment

项目摘要 在美国，估计有数万名儿童、青少年和年轻人（AYA）患有严重的 神经功能障碍（SNI），这种疾病对少数民族和低收入家庭造成不平等的负担 背景超过60%的儿童患有无法控制的疼痛，这种疼痛通常是持续的，无法识别的， 未经治疗。影响延伸到他们的父母谁通常经历不懈的身体和心理 痛苦目前还没有针对SNI儿童复发性疼痛治疗的严格干预研究。 这种差距可能存在，因为患有SNI的儿童受到污名化，而且往往来自历史上的边缘化 背景，而不是优先在科学研究。为了解决这一不平等问题， 干预发展，研究小组已经调整和完善了以前应用于儿童的干预措施， 癌PediQUEST对神经功能障碍儿童疼痛的反应（PQ-Respond）使用“倒数” 激活”的策略，以克服正常化，接受父母和临床医生，症状困扰， 是严重疾病和专业儿科姑息治疗团队（PPC）整合的不可避免的结果，以应对 孩子的苦恼。具体而言，PQ-ResPOND将结合联合收割机（i）通过每周采集的父母介导的激活， 通过我们的PQ应用程序向父母和临床医生反馈父母报告的儿童疼痛（非交流性儿童疼痛 检查表-修订量表（NCCPC-R））和其他症状（PediQUEST-记忆症状评估量表 适用于患有SNI的儿童），（ii）PPC团队激活初级临床医生：通过PPC的咨询，以及 (iii)PPC团队通过使用PQ-ResPOND检查表进行标准化复发性疼痛方法的培训来激活 （由该研究的跨专业专家小组改编）。总体假设是，PQ-Respond将改善儿童 复发性疼痛和父母的痛苦、负担和激活。本R21提案的总体目标是 一项研究中心II期初步可行性2：1随机对照试验，比较PQ-缓解（干预，n=30）与常规治疗（对照，n=15）， 45例≥1岁的AYA儿童，伴有SNI和复发性疼痛行为。为期2周的导入期将排除以下儿童： 轻度疼痛和调查无应答者。符合随机化标准的儿童的父母将每周回答PQ- 通过PQ应用程序进行12周的响应调查和每月结果调查，以及半结构化离职面谈。 具体目标是1。评估研究的可行性（>60%的入组率和>80%的保留率）， 可接受性（>70%的父母会建议干预，大多数父母和临床医生提供积极的 在定性离职面谈期间的反馈），并估计每月入学率;以及2.估计效应量， 拟议试验结果的可变性：儿童疼痛，父母痛苦和负担（焦虑，抑郁，压力，负担， 整体健康和睡眠质量）和父母激活（使用补充疗法和应对）。结果将告知 大型III期疗效试验的研究设计和样本量计算，程序手册的制定 和保真度监控策略。跨专业研究团队拥有扩展和测试此PPC的专业知识 干预，以减轻疼痛的儿童SNI，一个新的人口是边缘化，污名化，研究不足。

PROJECT SUMMARY In the US, tens of thousands of children, adolescents, and young adults (AYA) are estimated to live with severe neurologic impairment (SNI), a condition that poses an unequal burden on families from minority and low-income backgrounds. Over 60% of these children suffer from uncontrolled pain, which is often persistent, unrecognized, and untreated. The impact extends to their parents who commonly experience unremitting physical and psychological distress. There have been no rigorous intervention studies aimed at treating recurrent pain in children with SNI. This gap may exist because children with SNI are stigmatized and often from historically marginalized backgrounds, and not prioritized in scientific research. To address this inequity and using the ORBIT Model for intervention development, the research team has adapted and refined an intervention previously applied in children with cancer. The PediQUEST Response to Pain Of children with Neurologic Disability (PQ-ResPOND) uses “reciprocal activation” strategies to overcome normalization, the acceptance among parents and clinicians that symptom distress is an unavoidable outcome of serious illness and integration of a specialty pediatric palliative care team (PPC) to respond to child distress. Specifically, PQ-ResPOND will combine (i) parent-mediated activation through weekly collection and feedback to parents and clinicians, via our PQ App, of parent reported child pain (Non-Communicating Children’s Pain Checklist-Revised scale (NCCPC-R)) and other symptoms (PediQUEST-Memorial Symptom Assessment Scale adapted for children with SNI), with (ii) PPC team activation of primary clinicians: through consultation by PPC, and (iii) PPC team activation through training in a standardize recurrent pain approach using the PQ-ResPOND Checklist (adapted by the study’s interprofessional expert panel).The overall hypothesis is that PQ-ResPOND will improve child recurrent pain, and parent distress, burden, and activation. The overall objective of this R21 proposal is to conduct a single site Phase II Pilot Feasibility 2:1 RCT of PQ-ResPOND (intervention, n=30) vs. usual care (control, n=15) in 45 children ≥1 year-old & AYA with SNI and recurrent pain behaviors. A 2-week run-in period will exclude children with mild pain and survey non-responders. Parents of children who meet randomization criteria will answer weekly PQ- ResPOND surveys and monthly outcome surveys over 12 weeks via the PQ App, and a semi-structured exit interview. Specific Aims are to 1. assess the study’s feasibility (>60% enrollment rate and >80% retention), intervention acceptability (>70% parents would recommend the intervention and most parents and clinicians provide positive feedback during qualitative exit interviews), and estimate monthly enrollment rate; and 2. Estimate effect sizes and variability of the proposed trial outcomes: child pain, parent distress and burden (anxiety, depression, stress, burden, overall health, and sleep quality), and parent activation (use of complementary therapies and coping). Results will inform the study design and sample size calculation of a larger Phase III Efficacy trial, development of a manual of procedures and fidelity monitoring strategies. The interprofessional research team has the expertise to expand and test this PPC intervention to ease pain in children with SNI, a novel population that is marginalized, stigmatized, and understudied.