Palliative Care Needs of Children with Rare Diseases and their Families

罕见病儿童及其家人的姑息治疗需求

基本信息

批准号：
10041054
负责人：
Maureen Ellen Lyon
金额：
$ 22.31万
依托单位：
CHILDREN'S RESEARCH INSTITUTE
依托单位国家：
美国
项目类别：
财政年份：
2020
资助国家：
美国
起止时间：
2020-09-09 至 2022-07-31
项目状态：
已结题

来源：
https://reporter.nih.gov/project-details/10041054
关键词：
Abbreviations Address Advance Care Planning Affect Assessment tool Caregivers Caring Child Child Care Child Welfare Child health care Childhood Communication Complex Consultations Decision Making Diagnosis Documentation Electronic Health Record Emergency department visit Emotional Exclusion Family Family Caregiver Family Planning Future Gender Goals HIV Healthcare Hospitalization Hospitals Intervention Knowledge Malignant Childhood Neoplasm Malignant Neoplasms Medical Medical center Metabolic Diseases Modeling Monitor National Institute of Nursing Research Needs Assessment Operative Surgical Procedures Outcome Palliative Care Patients Pediatrics Persons Population Heterogeneity Preparation Probability Process Quality of life Questionnaires Race Randomized Randomized Controlled Trials Rare Diseases Religion and Spirituality Reporting Research Research Personnel Safety Sampling Single-Blind Study Social isolation Techniques Teenagers Testing Theoretical model Uncertainty Vulnerable Populations Well in self acronyms comorbidity disability evidence base experience family caregiving family influence follow-up health care service utilization health disparity improved innovation mortality multi-site trial pediatric patients pilot trial post intervention preference primary outcome psychologic rare genetic disorder research study satisfaction secondary outcome sex skill acquisition treatment as usual trial design

项目摘要

A rare disease is a condition affecting fewer than 200,000 persons. Pediatric patients with rare diseases experience high mortality with 30% not living to see their 5th birthday. Families are likely to be asked to make complex medical decisions for their child. Pediatric advance care planning involves preparation and skill development to help make future medical care choices. Children with rare disorders are a heterogeneous group often with co-morbidities, resulting in their exclusion from research, thereby creating a health disparity for this vulnerable population. Available research on families of children with rare diseases lacks scientific rigor. Although desperately needed, there are few empirically validated interventions to address these issues. We propose to close a gap in our knowledge of families' needs for support in a heterogeneous group of children with rare diseases; and to test an advance care planning intervention. The FAmily CEntered (FACE) pediatric advance care planning intervention, proven successful with cancer and HIV, is adapted to families with children who have rare diseases. Theoretically informed and developed by the PI, Lyon, and colleagues, the proposed intervention will use Respecting Choices Next Steps Pediatric Advance Care Planning™ for families whose child is unable to participate in health care decision-making. Our consultation with families of children with rare disorders and the National Organization for Rare Disorders (NORD) revealed that basic palliative care needs should be addressed first, before an advance care planning intervention. For the study to be able to meet this request, all families randomized to the intervention will first complete the Carer Support Needs Assessment Tool (CSNAT)© which our investigative team adapted for use in pediatrics. In the CSNAT Approach, facilitators assess caregivers' prioritized palliative care needs and develop Shared Action Plans for palliative care support. Thus, we propose an innovative 3-session FACE-Rare intervention, integrating two evidence-based approaches. We will evaluate FACE-Rare using a scientifically rigorous intent-to-treat, single-blinded, randomized controlled trial design. Family/child pairs or dyads (N=30 dyads) will be randomized to FACE-Rare (CSNAT Sessions 1 & 2 plus Respecting Choices Sessions 3) or control (Treatment As Usual) groups. Both groups will receive palliative care information. All families will complete questionnaires at baseline and 3- months follow-up. Investigators will evaluate the initial efficacy of FACE-Rare on family quality of life (psychological, spiritual). We will estimate how religiousness and caregiver appraisal influence families' quality of life. We will also explore health care utilization by the children during the study and family satisfaction. If the aims of this pilot trial are achieved, a future, large, multi-site trial will test the full theoretical model to improve care for children with rare diseases and their families through family engaged pediatric Advance Care Planning. The ultimate goal is to minimize suffering and enhance the quality of life of family caregivers of children with rare diseases; and through this process to improve the palliative care of their children.

罕见病是指影响不到 200,000 名罕见病儿科患者的疾病。死亡率很高，30% 的人可能无法活到 5 岁生日。为孩子做出复杂的医疗决定涉及准备和技能。帮助做出未来医疗护理选择的发展。患有罕见疾病的儿童是异质性的。群体经常患有合并症，导致他们被排除在研究之外，从而造成健康差距对于这一弱势群体，现有的关于罕见病儿童家庭的研究缺乏科学依据。尽管迫切需要，但很少有经过经验验证的干预措施可以解决这些问题。缩小我们对异质儿童群体家庭支持需求的了解差距患有罕见疾病；并测试 Family CEntered (FACE) 儿科干预措施。护理计划干预措施在癌症和艾滋病毒方面已被证明取得了成功，适用于有儿童的家庭由 PI、Lyon 及其同事从理论上了解和开发的人，提出了这一建议。干预措施将使用 Respecting Choices Next Steps Pediatric Advance Care Planning™ 来帮助那些患有以下疾病的家庭：儿童无法参与医疗保健决策。我们与罕见儿童的家庭进行协商。疾病和国家罕见疾病组织 (NORD) 表明，基本的姑息治疗需求为了使研究能够满足这一要求，应在预先护理计划干预之前首先解决这一问题。根据要求，所有随机接受干预的家庭将首先完成护理人员支持需求评估我们的调查团队改编用于儿科的工具 (CSNAT)© 在 CSNAT 方法中，协调员。评估护理人员的优先姑息治疗需求，并制定姑息治疗支持的共同行动计划。因此，我们提出了一种创新的 3 次 FACE-Rare 干预措施，整合了两种基于证据的干预措施我们将使用科学严谨的意向治疗、单盲、评估方法来评估 FACE-Rare。随机对照试验设计。家庭/儿童配对或二人组（N = 30 组）将被随机分配至 FACE-Rare。（CSNAT 会话 1 和 2 加上尊重选择会话 3）或控制组（照常治疗）。小组将收到姑息治疗信息。所有家庭将在基线和 3- 时填写问卷。研究人员将评估 FACE-Rare 对家庭生活质量的初步疗效。（心理、精神）。我们将评估宗教信仰和照顾者评价如何影响家庭质量。我们还将探讨孩子们在学习期间的医疗保健利用情况以及家庭满意度。如果这次试点试验的目标得以实现，未来的大型多地点试验将测试完整的理论模型通过家庭参与的儿科预先护理，改善对罕见病儿童及其家人的护理规划的最终目标是尽量减少家庭照顾者的痛苦并提高其生活质量。患有罕见疾病的儿童；并通过这一过程改善其孩子的姑息治疗。