Palliative Care Needs of Children With Rare Diseases and Their Families

N/AEnrolling By InvitationNCT06938542

Children's National Research Institute480 enrolled

Overview

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.

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.

Eligibility

Sex: ALLMin age: 12 MonthsMax age: 99 YearsHealthy volunteers:

Medical Language ↔ Plain English

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. Exclusion Criteria: * Family caregiver or support person is actively homicidal, suicidal, or psychotic at the time of enrollment.

Outcomes

Primary Outcomes

Family Appraisal of Caregiving for Palliative Care (FACQ-PC)

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.

Time frame: Baseline, 3-, 6-, and 12 month post-intervention

Functional Assessment of Chronic Illness Therapy-Spirituality-12 Version 4 Expanded (FACIT-Sp-EX)

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.

Time frame: Baseline, 3-, 6-, and 12 month post-intervention

Advance Care Plan for Child with Rare Disease Located in the Electronic Health Record (EHR).

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.

Time frame: Baseline and 1 year

Child Healthcare Utilization: initiation of palliative care consultations

Using a standardized data abstraction form to count initiation of palliative care consultations during the study.

Time frame: Baseline, 3-, 6-, 12-month.

Generalized Anxiety Disorder-7 (GAD-7)

Quality of life indicator with respect to emotional health-anxiety symptoms. 7 items. Higher scores indicate greater anxiety.