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.

罕见病是影响不到20万人的病情。儿科患者罕见疾病经历高死亡率，30％的人无法忍受他们的5岁生日。可能会要求家庭对孩子的复杂医疗决定。小儿预先护理计划涉及准备和技能开发有助于做出未来的医疗选择。罕见疾病的孩子是异质的经常有合并症的小组，导致他们被排除在研究之外，从而造成了健康差异对于这个脆弱的人群。关于罕见疾病儿童家庭的可用研究缺乏科学严谨。尽管迫切需要，但很少有经过严格验证的干预措施来解决这些问题。我们提议缩小我们对家庭对家庭支持需求的了解的差距患有罕见疾病；并测试预先护理计划干预措施。家庭以（面）为中心预先护理计划干预措施，证明是癌症和艾滋病毒的成功，适用于有孩子的家庭患有罕见疾病的人。 PI，Lyon及其同事的理论知情和开发干预将使用尊重选择的选择下一步儿科预先护理计划™ 孩子无法参加医疗保健决策。我们与罕见儿童家人的磋商疾病和国家稀有疾病组织（NORD）表明，基本的姑息治疗需求在进行预先护理计划干预之前，应首先解决。为了使研究能够满足要求，所有随机进行干预的家庭将首先完成护理人员的支持需求评估工具（CSNAT）©我们的调查团队适用于儿科。在CSNAT方法中，促进者评估护理人员优先的姑息治疗需求，并制定共同的姑息治疗计划。这，我们提出了一种创新的3条面部稀有干预措施，将两个基于证据方法。我们将使用科学严格的意图对治疗，单叶，单盲，评估面部野蛮随机对照试验设计。家庭/儿童对或二元组（n = 30个二元组）将随机分为稀有（CSNAT会议1和2加上尊重选择会话3）或对照组（照常治疗）组。两个都小组将收到姑息治疗信息。所有家庭将在基线和3-- 几个月的随访。调查人员将评估面部稀有效率对家庭生活质量的最初效率（心理，精神）。我们将估计宗教和照顾者评估如何影响家庭的质量生活。我们还将在研究期间探索儿童的医疗保健利用和家庭满意度。如果实现了这项试验试验的目的，那么未来的大型多站点试验将测试完整的理论模型通过家庭订婚的小儿预先护理来改善患有罕见疾病的儿童及其家人的护理规划。最终目标是最大程度地减少苦难并提高家庭护理人员的生活质量患有罕见疾病的儿童；通过这一过程，可以改善孩子的姑息治疗。