Palliative Care Needs of Children With Rare Diseases and Their Families

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.

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.

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.

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

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

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.

Using a standardized data abstraction form to count # of days in palliative care before death.

Using a standardized data abstraction form to count # of days hospitalized during the study.

Using a standardized data abstraction form to count # of days used Emergency Department during study.

Using a standardized data abstraction form to count # of days was admitted to ICU during the study.

Using a standardized data abstraction form to count # of surgeries during the study.

Age of family caregiver, support person, and child.

Study specific process measure to assess adverse events and benefit/burden of participation. 13 items. Higher scores indicate greater satisfaction. Items are on a 5-point Likert scale from strongly disagree to strongly agree. 6 items were negative (felt afraid, too much to handle, harmful, angry, sad, hurtful) and 7 items were positive (useful, helpful, load off my mind, satisfied, something I needed to do, courageous, worthwhile). Each subscale is scored separately. Higher score for positive scale was better outcome. Higher score for negative scale was worse outcome.

Visual analogue scale 0-100. "How stressful is it for you to make medical decisions for your child?" 1 item.

Responses to 5 items from our previous research will be used to test moderator effects: "How often do you go to religious services? How often do you feel God's presence? how often do you pray privately? Do you identify as a religious person? Do you identify as a spiritual person?" Responses are on a 5 point likert scale. Higher scores indicated greater attendance at religious services, etc.

NINR/National Institute of Health: "The SDOH are the conditions in which people are born, grow, live, work and age…." 32 items. Social connection score (how often, talk on telephone with family, friends, neighbors; get together; attend church or religious services; belong to clubs or organizations; marital partner status) and 2023 Federal Poverty Level for household income. Health insurance and housing insecurity will be used in secondary analysis.

Urban vs. rural setting. Address of participant is entered into the online analyzer, and it determines if this is a rural or urban setting which will be recorded in the data base.

Measures child and family caregiver palliative care needs: The CAT is an evidence-based, comprehensive tool comprising 12 questions (broad areas of support need), used to identify two domains of unmet support needs, the current caring situation and the health and well-being of the caregiver. There is a traffic light scoring system so caregivers can rate their support need as green (low), amber (medium), and red (high) indicating level of need and potential risk each alert poses to the caregiving situation. Administered only to intervention participants.

Sex of child, family caregiver and support person.

Race of child, family caregiver, and support person

Ethnicity of child, family caregiver, and support person.

Marital status of family caregiver and support person.

Education of family caregiver and support person.

Income above or below the 2025 Federal Poverty Level. Number of persons living in the household and household income will be collected to calculate these data.