Palliative Care Needs of Children With Rare Diseases and Their Families
Overview
- Phase
- N/A
- Intervention
- Not specified
- Conditions
- Rare Diseases
- Sponsor
- Children's National Research Institute
- Enrollment
- 42
- Locations
- 1
- Primary Endpoint
- Beck Anxiety Inventory
- Status
- Completed
- Last Updated
- 12 months ago
Overview
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.
Investigators
Maureen Lyon
Dr. Maureen Lyon, Ph.D, ABPP
Children's National Research Institute
Eligibility Criteria
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
Exclusion Criteria
- •(1) Family caregiver is actively homicidal, suicidal, or psychotic at the time of enrollment.
Outcomes
Primary Outcomes
Beck Anxiety Inventory
Time Frame: 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
Time Frame: 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
Time Frame: 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
Time Frame: 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
Time Frame: 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
Time Frame: 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 Outcomes
- Brief-Multidimensional Measure of Religion and Spirituality - Responses to Question 1(Baseline and 3-month follow-up.)
- Family Appraisal of Caregiving Questionnaire for Palliative Care for Palliative Care (FACQ-PC)(Change from Baseline appraisal of Caregiving at 12 weeks Post-Enrollment)
- Brief Multidimensional Measurement of Religiousness/Spirituality (BMMRS-adapted) Survey Responses to Question 2.(Baseline and 3-month follow-up)
- Hickman Role Stress Decisional Burden Scale(Up to 5 Weeks Post-Enrollment)