Palliative Care Needs of Children With Rare Diseases and Their Families
试验速览
- 阶段
- 不适用
- 状态
- Enrolling By Invitation
- 发起方
- 入组人数
- 480
- 试验地点
- 1
- 主要终点
- Advance Care Plan for Child with Rare Disease Located in the Electronic Health Record (EHR).
研究概览
简要总结
The palliative care needs of family caregivers of children with rare diseases and their children are largely unmet, including the need for support to prepare for future medical decision making. This trial will test the FACE-Rare intervention to see if investigators can identify and meet those needs; and if FACE-Rare effects family caregivers' quality of life and child healthcare utilization. Finally, investigators will determine if the intersectionality of child-sex, family-race, Federal poverty level, and social connection influences family quality of life and child health care utilization longitudinally.
详细描述
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, resulting in their exclusion from research. 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. Investigators 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 is adapted to families with children who have rare diseases. Theoretically informed and developed and adapted by the principal investigator and key stakeholders, the proposed intervention will use Respecting Choices Next Steps Pediatric ACP™ 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, prior to 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)© adapted by investigators for use in pediatrics. In the CSNAT Approach, facilitators assess caregivers' prioritized palliative care needs and develop Shared Action Plans for increasing informal social support. Thus, investigators propose an innovative 3-session FACE-Rare intervention, integrating two evidence-based approaches. Investigators will evaluate FACE-Rare using a scientifically rigorous intent-to-treat, assessor-blinded, longitudinal, prospective, three-site, randomized controlled trial design. Family/child triads (N=160) will be randomized to FACE-Rare (CSNAT Sessions 1 & 2 plus Respecting Choices Sessions 3) or an enhanced information Treatment as Usual control group. All families will complete questionnaires at baseline and follow-up at 3-, 6- and 12 months. Investigators will evaluate the effect of FACE-Rare on family quality of life (caregiver appraisal, psychological, spiritual). Investigators will assess the palliative care needs of families at four time points. Investigators will determine the intersectionality of child-sex, family-race, and household income on family caregiver quality of life and child healthcare utilization. Investigators will explore the influence of urban vs. rural setting and religious coping on quality-of-life outcomes. Investigators will use advanced statistical methods informed by statistical advice from rare disease investigators for clinical trials in small populations.
研究设计
- 研究类型
- Interventional
- 分配方式
- Randomized
- 干预模型
- Parallel
- 主要目的
- Supportive Care
- 盲法
- Single (Outcomes Assessor)
入排标准
- 年龄范围
- 12 Months 至 99 Years（Child, Adult, Older Adult）
- 性别
- All
- 接受健康志愿者
- 是
入选标准
- •Child inclusion criteria:
- •≥1.0 years and <18.0 years at enrollment.
- •Unable to participate in end-of-life care decision-making.
- •Has a rare disease as operationally defined by NIH's Genetic and Rare Diseases Information Center (GARD).
- •Not under a Do Not Resuscitate Order or Allow a Natural Death Order.
- •Not in the Intensive Care Unit.
- •Family caregiver inclusion criteria:
- •> 18.0 years at enrollment.
- •Child's family caregiver/legal guardian.
- •Not known to be developmentally delayed.
- •Support person inclusion criteria:
- •> 18.0 years at enrollment.
- •Chosen by family caregiver.
- •Not known to be developmentally delayed.
排除标准
- •Family caregiver or support person is actively homicidal, suicidal, or psychotic at the time of enrollment.
研究组 & 干预措施
FACE-Rare
FACE-Rare (CSNAT-P Sessions 1 & 2 plus Next Steps: Respecting Choices Sessions 3). The CSNAT-Pediatric intervention consists of two assessment visits with the facilitator, 2-8 weeks apart, comprising conversations about sources for support in a tertiary children's hospital. The adapted Next Steps: Respecting Choices pediatric Advance Care Planning conversation engages families in a process for how to make future medical decisions consistent with the families' goals and values. The Respecting Choices structured and facilitated conversation has five stages. Stage 1: Assesses the family's understanding of illness. Stage 2: Explores experiences with hospitalization. Stage 3: Explores goals of care. Stage 4: Creates an Advance Care Plan; Stage 5: Questions for providers are written down. Stage 6: Follow-up plan and referrals, as needed.
干预措施: Family Centered pediatric palliative care for family caregivers of children with rare diseases. (Behavioral)
Enhanced Treatment As Usual (TAU)
Treatment as Usual Control (TAU): To minimize the burden to families, we have chosen an enhanced (palliative care information and resources) TAU comparison condition.
干预措施: Family Centered pediatric palliative care for family caregivers of children with rare diseases. (Behavioral)
结局指标
主要结局
Advance Care Plan for Child with Rare Disease Located in the Electronic Health Record (EHR).
时间窗: Baseline and 1 year
Documentation of an advance care plan for child with rare disease in the Electronic Health Record and decisional preference - to continue all treatments, to continue all treatments with exceptions noted, to provide comfort care only.
Child Healthcare Utilization: initiation of palliative care consultations
时间窗: Baseline, 3-, 6-, 12-month.
Using a standardized data abstraction form to count initiation of palliative care consultations during the study.
Generalized Anxiety Disorder-7 (GAD-7)
时间窗: Baseline, 3-, 6-, 12-month
Quality of life indicator with respect to emotional health-anxiety symptoms. 7 items. Higher scores indicate greater anxiety.
Child Healthcare Utilization: # of days in palliative care before death.
时间窗: Baseline, 3-, 6-, 12-month
Using a standardized data abstraction form to count # of days in palliative care before death.
Child Healthcare Utilization: # of Emergency Department visits during study participation
时间窗: Baseline, 3-, 6-, 12-month
Using a standardized data abstraction form to count # of days used Emergency Department during study.
Child Healthcare Utilization: # of days in the Intensive Care Unit (ICU)
时间窗: Baseline, 3-, 6-, 12-month
Using a standardized data abstraction form to count # of days was admitted to ICU during the study.
Child Healthcare Utilization: # of surgeries
时间窗: Baseline, 3-, 6-, 12-month
Using a standardized data abstraction form to count # of surgeries during the study.
Child Healthcare Utilization: # of hospitalizations during study participation
时间窗: Baseline, 3-, 6-, 12-month
Using a standardized data abstraction form to count # of days hospitalized during the study.
Family Appraisal of Caregiving for Palliative Care (FACQ-PC)
时间窗: Baseline, 3-, 6-, and 12 month post-intervention
The FACQ-PC is a 25-item measure consists of four theoretically derived subscales: (i) caregiver strain, (ii) positive caregiving appraisals, (iii) caregiver distress, and (iv) family well-being. Scores are from 5 = strongly agree to 1 = strongly disagree. Investigators will not calculate a Total score. On the subscale scores for positive caregiving appraisals and family well-being, higher scores mean better outcomes, i.e. greater positive caregiving appraisals or family well-being. On the subscale scores for caregiver strain and caregiver distress, higher scores mean worse outcomes, i.e. greater caregiver strain or caregiver distress. The FACQ-PC subscale scores will be computed by taking the mean of the items (score range 1-5). Some items are reverse scored, depending on how the item is phrased, so that higher scores = higher amount of the subscale being measured. So the minimum value for each subscale is 1 and the maximum value for each subscale is 5.
Functional Assessment of Chronic Illness Therapy-Spirituality-12 Version 4 Expanded (FACIT-Sp-EX)
时间窗: Baseline, 3-, 6-, and 12 month post-intervention
Assessed construct of spiritual well-being. Two subscales Meaning/Peace (7 items) and Faith (5 items) and Total score (12 items) were calculated. on a 5-point Likert scale from 0=not at all to 5=very much. Some items are reverse scored. See www.facit.org Meaning/Peace subscale score range from minimum value of 0 to maximum value of 32. Higher scores indicate better meaning/peace. Faith subscale score range from 0 minimum value to maximum value of 16. Higher scores indicate better meaning/peace. Total score range is from 0 minimum value to maximum value of 92. Higher scores indicate better spiritual well-being.
Patient Health Questionaire-9 (PHQ-9)
时间窗: Baseline, 3-, 6-, 12-month
Quality of life indicator with respect to emotional health-depressive symptoms. 9 items. Higer scores indicate higher symptoms of depression. A yes response to question 9 (self-harm) will trigger a referral.
次要结局
未报告次要终点
研究者
Maureen Lyon
Clinical Psychologist, Tenured Professor of Pediatrics
Children's National Research Institute