FACE for Children With Rare Diseases

Not Applicable

Completed

Conditions: Rare Diseases

Interventions: Behavioral: FAmily CEntered (FACE) pACP Intervention

Registration Number: NCT04855734

Lead Sponsor: Children's National Research Institute

Brief Summary: Children with ultra-rare or complex rare diseases are routinely excluded from research studies because of their conditions, creating a health disparity. However, new statistical techniques make it possible to study small samples of heterogeneous populations. We propose to study the palliative care needs of family caregivers of children with ultra-rare diseases and to pilot test a palliative care needs assessment and advance care planning intervention to facilitate discussions about the future medical care choices families are likely to be asked to make for their child.

Detailed Description: 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, Dr. 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.

Recruitment & Eligibility

Status: COMPLETED

Sex: All

Target Recruitment: 42

Inclusion Criteria

Child inclusion criteria are

≥1.0 years and <18.0 years at enrollment;e
unable to participate in end-of-life care decision-making;
have a rare disease as operationally defined (See Human Subjects);
not under a Do Not Resuscitate Order or Allow a Natural Death Order; and
not in the Intensive Care Unit.

Family caregiver inclusion criteria are:

≥ 18.0 years at enrollment;
legal guardian of child and child's caregiver;
can speak and understand English; and
not known to be developmentally delayed.

Exclusion Criteria

(1) Family caregiver is actively homicidal, suicidal, or psychotic at the time of enrollment.

Study & Design

Study Type: INTERVENTIONAL

Study Design: PARALLEL

Arm && Interventions

Group	Intervention	Description
FACE-Rare Intervention	FAmily CEntered (FACE) pACP Intervention	FACE-Rare is a behavioral intervention that combines the CSNAT Pediatric Approach and the Respecting Choices® Next Steps ACP over 3 sessions. Sessions 1\&2: CSNAT is an evidence-based process of family caregiver assessment and support in specialized medical (palliative) care. The CSNAT tool is structured around 16 categories of family caregiver support. With the goal to decrease caregiver burden, this process consists of 5 stages wherein a nurse or practitioner works with the caregiver to create a shared support plan for the child. Session 3: Respecting Choices® Next Steps- This advanced care planning (pACP) conversation engages families in a process for how to make future medical decisions consistent with their goals and values. The interview is structured in 6 stages to achieve 2 main goals: to facilitate conversations with the family about their child's medical condition, history, fears, values, beliefs, and hopes; and to set the stage for the family's future healthcare decisions.

Primary Outcome Measures

Name	Time	Method
Beck Anxiety Inventory	Change from Baseline Anxiety at 3 Months Post-Enrollment	The Beck Anxiety Inventory (BAI) is a 21-item measure of anxiety rated on a 4-point Likert scale of symptoms over the past week. The BAI has demonstrated reliability and validity to assess anxiety in individuals aged 17-80 years. The BAI has good reliability and validity for family caregivers of seriously ill children. It is a measure of Quality of life: emotional health. Higher scores mean more anxiety symptoms. Total score was used in analysis. Scores range from 0-63.
Functional Assessment of Chronic Illness Therapy (FACIT)- Spiritual Well-Being Scale, Version 4	Change from Baseline Spiritual Well-Being at 3 Months Post-Enrollment	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. Higher scores indicate better spiritual well-being.
Advance Care Plan Document for Children With Rare Diseases	Change from Baseline ACP Documentation in the Electronic Health Record at 12 weeks Post-Enrollment	Advanced Care Planning (ACP) Documentation in Electronic Health Records (EHR). Score will be present in EHR or absent from EHR. Evidence of any advance care document (yes/no) will count.
Days in Palliative Care Prior to Death	12 weeks Post-Enrollment	Score is number of days patient was enrolled in a palliative care program in the 30 days prior to death. Applicable only to patients who have deceased since study enrollment. Used to standardize child healthcare utilization from data abstraction based on retrospective medical chart review. Minimum value is 0 with no maximum. Higher score indicates higher level of healthcare utilization.
Hospitalizations	Change from Baseline to Frequency at 12 weeks Post-Enrollment	Used to standardize child healthcare utilization from data abstraction by the research nurse based on medical chart review in Electronic Health Record. Score is recorded number of inpatient hospital admissions for clinical treatment for the child with a rare disease. Minimum value is 0 with no maximum. Higher score indicates higher level of healthcare utilization. Questions was "Hospitalization in the last 3 months?"
Place of Death	12 weeks Post-Enrollment	Location where the patient was pronounced dead, as reported in their EMR. Applicable only to patients who have deceased since study enrollment. Categories are Inpatient hospice setting, Home with hospice, Home without hospice, Hospital ICU, Hospital-Not ICU, Other, or Unknown. Used to standardize child healthcare utilization from data abstraction based on retrospective medical chart review.

Secondary Outcome Measures

Name	Time	Method
Brief-Multidimensional Measure of Religion and Spirituality - Responses to Question 1	Baseline and 3-month follow-up.	Survey responses to: 1. How often do you feel God's presence? On 6-point Likert-scale responses range from every day to never or almost never.
Family Appraisal of Caregiving Questionnaire for Palliative Care for Palliative Care (FACQ-PC)	Change from Baseline appraisal of Caregiving at 12 weeks Post-Enrollment	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. We did not calculate a Total score. We only used the 4 subscale scores. 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 were computed by taking the mean of the items (score range 1-5). Some items were 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.
Brief Multidimensional Measurement of Religiousness/Spirituality (BMMRS-adapted) Survey Responses to Question 2.	Baseline and 3-month follow-up	Responses to survey question 2, How often do you pray privately? 7 point Likert scale ranging from a few times a month to once a day. Higher scores in pray item indicate more prayer.
Hickman Role Stress Decisional Burden Scale	Up to 5 Weeks Post-Enrollment	Visual analogue scale 0-100. "How stressful is it for you to make medical decisions for your child?" 1 item. Higher score means more stress, lower scores mean less stress.