Trial Outcomes & Findings for FACE for Children With Rare Diseases (NCT NCT04855734)

Last Updated: 2025-05-04

Results Overview

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.

Recruitment status

COMPLETED

Study phase

Target enrollment

42 participants

Primary outcome timeframe

Change from Baseline Anxiety at 3 Months Post-Enrollment

Results posted on

2025-05-04

Participant Flow

Family caregivers were recruited from outpatient clinics at Children's National Hospital, a quaternary pediatric hospital. There were 21 children and 21 primary family caregivers enrolled in the study. Randomization was at the level of the dyad (child with a rare disease and the primary family caregiver). Thus, 42 participants (21 child/family dyads) completed the baseline assessment and were then randomized. The flow chart is reported as adolescent/family dyads.

Participant milestones

Participant milestones
Measure	FACE-Rare 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. FAmily CEntered (FACE) pACP Intervention: The (approximately) weekly 3-session FACE-Rare intervention of approximately 45-60 minutes each is comprised of the CSNAT approach \[Sessions 1 \& 2\] and Respecting Choices \[Session 3\].	Treatment-as-Usual (TAU) Control To minimize the burden to families, we have chosen a Treatment-as-Usual (TAU) comparison condition, where patients will receive their normal standard of care. Both study arms will receive palliative (specialized medical) care information at enrollment and complete questionnaires before and after the intervention or TAU period. Current practice for minors with life-limiting illnesses is to defer initial discussions of advanced care planning (pACP) until a medical crisis, so this is what the TAU control arm condition will consist of.
Overall Study STARTED	18	24
Overall Study Children	9	12
Overall Study Caregivers	9	12
Overall Study COMPLETED	16	22
Overall Study NOT COMPLETED	2	2

Reasons for withdrawal

Reasons for withdrawal
Measure	FACE-Rare 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. FAmily CEntered (FACE) pACP Intervention: The (approximately) weekly 3-session FACE-Rare intervention of approximately 45-60 minutes each is comprised of the CSNAT approach \[Sessions 1 \& 2\] and Respecting Choices \[Session 3\].	Treatment-as-Usual (TAU) Control To minimize the burden to families, we have chosen a Treatment-as-Usual (TAU) comparison condition, where patients will receive their normal standard of care. Both study arms will receive palliative (specialized medical) care information at enrollment and complete questionnaires before and after the intervention or TAU period. Current practice for minors with life-limiting illnesses is to defer initial discussions of advanced care planning (pACP) until a medical crisis, so this is what the TAU control arm condition will consist of.
Overall Study Lost to Follow-up	2	2

Baseline Characteristics

Baseline analysis was at level of participant with separate analysis for children with rare diseases and the primary family caregiver.

Baseline characteristics by cohort

PRIMARY outcome

Timeframe: Change from Baseline Anxiety at 3 Months Post-Enrollment

Population: Data analysis was with the family caregivers.

Outcome measures

Outcome measures
Measure	FACE-Rare Intervention n=9 Participants 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. FAmily CEntered (FACE) pACP Intervention: The (approximately) weekly 3-session FACE-Rare intervention of approximately 45-60 minutes each is comprised of the CSNAT approach \[Sessions 1 \& 2\] and Respecting Choices \[Session 3\].	Treatment-as-Usual (TAU) Control n=12 Participants To minimize the burden to families, we have chosen a Treatment-as-Usual (TAU) comparison condition, where patients will receive their normal standard of care. Both study arms will receive palliative (specialized medical) care information at enrollment and complete questionnaires before and after the intervention or TAU period. Current practice for minors with life-limiting illnesses is to defer initial discussions of advanced care planning (pACP) until a medical crisis, so this is what the TAU control arm condition will consist of.
Beck Anxiety Inventory Baseline Total score	10.4 score on a scale Standard Deviation 8.5	9.5 score on a scale Standard Deviation 9.5
Beck Anxiety Inventory 3 month follow-up Total Score	11.1 score on a scale Standard Deviation 8.0	8.5 score on a scale Standard Deviation 9.6

PRIMARY outcome

Timeframe: Change from Baseline Spiritual Well-Being at 3 Months Post-Enrollment

Population: Family caregivers of children with rare diseases.

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.

Outcome measures

Outcome measures
Measure	FACE-Rare Intervention n=9 Participants 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. FAmily CEntered (FACE) pACP Intervention: The (approximately) weekly 3-session FACE-Rare intervention of approximately 45-60 minutes each is comprised of the CSNAT approach \[Sessions 1 \& 2\] and Respecting Choices \[Session 3\].	Treatment-as-Usual (TAU) Control n=12 Participants To minimize the burden to families, we have chosen a Treatment-as-Usual (TAU) comparison condition, where patients will receive their normal standard of care. Both study arms will receive palliative (specialized medical) care information at enrollment and complete questionnaires before and after the intervention or TAU period. Current practice for minors with life-limiting illnesses is to defer initial discussions of advanced care planning (pACP) until a medical crisis, so this is what the TAU control arm condition will consist of.
Functional Assessment of Chronic Illness Therapy (FACIT)- Spiritual Well-Being Scale, Version 4 Baseline Meaning/Peace	24.0 score on a scale Interval 23.0 to 27.0	25.0 score on a scale Interval 23.5 to 27.0
Functional Assessment of Chronic Illness Therapy (FACIT)- Spiritual Well-Being Scale, Version 4 3 month Meaning/Peace	26.0 score on a scale Interval 24.0 to 30.0	26.0 score on a scale Interval 21.8 to 29.0
Functional Assessment of Chronic Illness Therapy (FACIT)- Spiritual Well-Being Scale, Version 4 Baseline Faith	11.0 score on a scale Interval 10.0 to 14.0	6.0 score on a scale Interval 3.8 to 7.5
Functional Assessment of Chronic Illness Therapy (FACIT)- Spiritual Well-Being Scale, Version 4 3 month Faith	9.0 score on a scale Interval 7.0 to 14.0	7.0 score on a scale Interval 4.8 to 11.0

PRIMARY outcome

Timeframe: Change from Baseline ACP Documentation in the Electronic Health Record at 12 weeks Post-Enrollment

Population: Advance care plan for child documented in the Electronic Health Record.

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.

Outcome measures

Outcome measures
Measure	FACE-Rare Intervention n=9 Participants 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. FAmily CEntered (FACE) pACP Intervention: The (approximately) weekly 3-session FACE-Rare intervention of approximately 45-60 minutes each is comprised of the CSNAT approach \[Sessions 1 \& 2\] and Respecting Choices \[Session 3\].	Treatment-as-Usual (TAU) Control n=12 Participants To minimize the burden to families, we have chosen a Treatment-as-Usual (TAU) comparison condition, where patients will receive their normal standard of care. Both study arms will receive palliative (specialized medical) care information at enrollment and complete questionnaires before and after the intervention or TAU period. Current practice for minors with life-limiting illnesses is to defer initial discussions of advanced care planning (pACP) until a medical crisis, so this is what the TAU control arm condition will consist of.
Advance Care Plan Document for Children With Rare Diseases Baseline · No	8 Participants	12 Participants
Advance Care Plan Document for Children With Rare Diseases Baseline · Yes	1 Participants	0 Participants
Advance Care Plan Document for Children With Rare Diseases 3-month follow-up · No	6 Participants	12 Participants
Advance Care Plan Document for Children With Rare Diseases 3-month follow-up · Yes	3 Participants	0 Participants

PRIMARY outcome

Timeframe: 12 weeks Post-Enrollment

Population: Child with rare disease. No child with a rare disease died during the study, so data for Days in Palliative Care Prior to death was not collected.

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.

Outcome measures

Outcome data not reported

PRIMARY outcome

Timeframe: Change from Baseline to Frequency at 12 weeks Post-Enrollment

Population: Children with rare diseases.

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?"

Outcome measures

Outcome measures
Measure	FACE-Rare Intervention n=9 Participants 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. FAmily CEntered (FACE) pACP Intervention: The (approximately) weekly 3-session FACE-Rare intervention of approximately 45-60 minutes each is comprised of the CSNAT approach \[Sessions 1 \& 2\] and Respecting Choices \[Session 3\].	Treatment-as-Usual (TAU) Control n=12 Participants To minimize the burden to families, we have chosen a Treatment-as-Usual (TAU) comparison condition, where patients will receive their normal standard of care. Both study arms will receive palliative (specialized medical) care information at enrollment and complete questionnaires before and after the intervention or TAU period. Current practice for minors with life-limiting illnesses is to defer initial discussions of advanced care planning (pACP) until a medical crisis, so this is what the TAU control arm condition will consist of.
Hospitalizations Baseline · No	7 Participants	7 Participants
Hospitalizations Baseline · Yes	2 Participants	5 Participants
Hospitalizations 3-month follow-up · No	7 Participants	8 Participants
Hospitalizations 3-month follow-up · Yes	2 Participants	4 Participants

PRIMARY outcome

Timeframe: 12 weeks Post-Enrollment

Population: Child with a rare disease. No child with a rare disease died during the study, so Place of Death was not collected.

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.

Outcome measures

Outcome data not reported

SECONDARY outcome

Timeframe: Baseline and 3-month follow-up.

Population: Primary family caregivers of children with rare diseases.

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.

Outcome measures

Outcome measures
Measure	FACE-Rare Intervention n=9 Participants 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. FAmily CEntered (FACE) pACP Intervention: The (approximately) weekly 3-session FACE-Rare intervention of approximately 45-60 minutes each is comprised of the CSNAT approach \[Sessions 1 \& 2\] and Respecting Choices \[Session 3\].	Treatment-as-Usual (TAU) Control n=12 Participants To minimize the burden to families, we have chosen a Treatment-as-Usual (TAU) comparison condition, where patients will receive their normal standard of care. Both study arms will receive palliative (specialized medical) care information at enrollment and complete questionnaires before and after the intervention or TAU period. Current practice for minors with life-limiting illnesses is to defer initial discussions of advanced care planning (pACP) until a medical crisis, so this is what the TAU control arm condition will consist of.
Brief-Multidimensional Measure of Religion and Spirituality - Responses to Question 1 3-month follow up -How often do you feel God's presence? · Never or almost never	2 Participants	0 Participants
Brief-Multidimensional Measure of Religion and Spirituality - Responses to Question 1 Baseline How often do you feel God's presence? · Declined	1 Participants	0 Participants
Brief-Multidimensional Measure of Religion and Spirituality - Responses to Question 1 Baseline How often do you feel God's presence? · Every day	3 Participants	1 Participants
Brief-Multidimensional Measure of Religion and Spirituality - Responses to Question 1 Baseline How often do you feel God's presence? · Most days	1 Participants	3 Participants
Brief-Multidimensional Measure of Religion and Spirituality - Responses to Question 1 Baseline How often do you feel God's presence? · Once in a while	3 Participants	5 Participants
Brief-Multidimensional Measure of Religion and Spirituality - Responses to Question 1 Baseline How often do you feel God's presence? · Some days	1 Participants	2 Participants
Brief-Multidimensional Measure of Religion and Spirituality - Responses to Question 1 Baseline How often do you feel God's presence? · Many times a day.	0 Participants	0 Participants
Brief-Multidimensional Measure of Religion and Spirituality - Responses to Question 1 Baseline How often do you feel God's presence? · Never or almost never	0 Participants	1 Participants
Brief-Multidimensional Measure of Religion and Spirituality - Responses to Question 1 3-month follow up -How often do you feel God's presence? · Declined	0 Participants	0 Participants
Brief-Multidimensional Measure of Religion and Spirituality - Responses to Question 1 3-month follow up -How often do you feel God's presence? · Every day	2 Participants	4 Participants
Brief-Multidimensional Measure of Religion and Spirituality - Responses to Question 1 3-month follow up -How often do you feel God's presence? · Most days	2 Participants	2 Participants
Brief-Multidimensional Measure of Religion and Spirituality - Responses to Question 1 3-month follow up -How often do you feel God's presence? · Once in a while	1 Participants	5 Participants
Brief-Multidimensional Measure of Religion and Spirituality - Responses to Question 1 3-month follow up -How often do you feel God's presence? · Some days	1 Participants	0 Participants
Brief-Multidimensional Measure of Religion and Spirituality - Responses to Question 1 3-month follow up -How often do you feel God's presence? · Many times a day.	1 Participants	1 Participants

SECONDARY outcome

Timeframe: Change from Baseline appraisal of Caregiving at 12 weeks Post-Enrollment

Population: Primary family caregivers of children with a rare disease.

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.

Outcome measures

Outcome measures
Measure	FACE-Rare Intervention n=9 Participants 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. FAmily CEntered (FACE) pACP Intervention: The (approximately) weekly 3-session FACE-Rare intervention of approximately 45-60 minutes each is comprised of the CSNAT approach \[Sessions 1 \& 2\] and Respecting Choices \[Session 3\].	Treatment-as-Usual (TAU) Control n=12 Participants To minimize the burden to families, we have chosen a Treatment-as-Usual (TAU) comparison condition, where patients will receive their normal standard of care. Both study arms will receive palliative (specialized medical) care information at enrollment and complete questionnaires before and after the intervention or TAU period. Current practice for minors with life-limiting illnesses is to defer initial discussions of advanced care planning (pACP) until a medical crisis, so this is what the TAU control arm condition will consist of.
Family Appraisal of Caregiving Questionnaire for Palliative Care for Palliative Care (FACQ-PC) Caregiver strain subscale baseline	3.3 score on a scale Interval 2.9 to 3.6	3.0 score on a scale Interval 2.4 to 3.7
Family Appraisal of Caregiving Questionnaire for Palliative Care for Palliative Care (FACQ-PC) Positive caregiving appraisals Baseline	4.7 score on a scale Interval 4.1 to 4.9	4.6 score on a scale Interval 4.3 to 4.7
Family Appraisal of Caregiving Questionnaire for Palliative Care for Palliative Care (FACQ-PC) Caregiver Distress Baseline	3.3 score on a scale Interval 2.8 to 3.8	3.5 score on a scale Interval 2.9 to 4.0
Family Appraisal of Caregiving Questionnaire for Palliative Care for Palliative Care (FACQ-PC) Family well-being baseline	4.2 score on a scale Interval 3.5 to 4.7	4.2 score on a scale Interval 3.9 to 4.5
Family Appraisal of Caregiving Questionnaire for Palliative Care for Palliative Care (FACQ-PC) caregiver strain 3 Month follow up	3.5 score on a scale Interval 2.8 to 3.8	2.9 score on a scale Interval 2.5 to 3.9
Family Appraisal of Caregiving Questionnaire for Palliative Care for Palliative Care (FACQ-PC) Positive caregiving appraisals 3 month follow up	4.7 score on a scale Interval 4.3 to 5.0	4.6 score on a scale Interval 4.5 to 4.9
Family Appraisal of Caregiving Questionnaire for Palliative Care for Palliative Care (FACQ-PC) Caregiver distress 3 month follow up	2.8 score on a scale Interval 2.5 to 3.3	3.4 score on a scale Interval 2.3 to 3.8
Family Appraisal of Caregiving Questionnaire for Palliative Care for Palliative Care (FACQ-PC) Family well-being 3 month follow up	4.5 score on a scale Interval 3.8 to 4.5	4.2 score on a scale Interval 4.0 to 4.4

SECONDARY outcome

Timeframe: Up to 5 Weeks Post-Enrollment

Population: Primary caregiver of child with rare disease.

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.

Outcome measures

Outcome measures
Measure	FACE-Rare Intervention n=9 Participants 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. FAmily CEntered (FACE) pACP Intervention: The (approximately) weekly 3-session FACE-Rare intervention of approximately 45-60 minutes each is comprised of the CSNAT approach \[Sessions 1 \& 2\] and Respecting Choices \[Session 3\].	Treatment-as-Usual (TAU) Control n=12 Participants To minimize the burden to families, we have chosen a Treatment-as-Usual (TAU) comparison condition, where patients will receive their normal standard of care. Both study arms will receive palliative (specialized medical) care information at enrollment and complete questionnaires before and after the intervention or TAU period. Current practice for minors with life-limiting illnesses is to defer initial discussions of advanced care planning (pACP) until a medical crisis, so this is what the TAU control arm condition will consist of.
Hickman Role Stress Decisional Burden Scale Baseline	75 raw score units on a scale Interval 47.0 to 90.0	71.0 raw score units on a scale Interval 39.3 to 88.0
Hickman Role Stress Decisional Burden Scale 3-month follow-up	75.0 raw score units on a scale Interval 75.0 to 85.0	72.5 raw score units on a scale Interval 50.0 to 81.3

SECONDARY outcome

Timeframe: Baseline and 3-month follow-up

Population: Family caregivers of children with rare diseases.

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.

Outcome measures

Outcome measures
Measure	FACE-Rare Intervention n=9 Participants 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. FAmily CEntered (FACE) pACP Intervention: The (approximately) weekly 3-session FACE-Rare intervention of approximately 45-60 minutes each is comprised of the CSNAT approach \[Sessions 1 \& 2\] and Respecting Choices \[Session 3\].	Treatment-as-Usual (TAU) Control n=12 Participants To minimize the burden to families, we have chosen a Treatment-as-Usual (TAU) comparison condition, where patients will receive their normal standard of care. Both study arms will receive palliative (specialized medical) care information at enrollment and complete questionnaires before and after the intervention or TAU period. Current practice for minors with life-limiting illnesses is to defer initial discussions of advanced care planning (pACP) until a medical crisis, so this is what the TAU control arm condition will consist of.
Brief Multidimensional Measurement of Religiousness/Spirituality (BMMRS-adapted) Survey Responses to Question 2. 3 month follow-up How often do you pray privately? · A few times a month	2 Participants	2 Participants
Brief Multidimensional Measurement of Religiousness/Spirituality (BMMRS-adapted) Survey Responses to Question 2. Baseline How often do you pray privately? · A few times a month	2 Participants	2 Participants
Brief Multidimensional Measurement of Religiousness/Spirituality (BMMRS-adapted) Survey Responses to Question 2. Baseline How often do you pray privately? · A few times a week	1 Participants	3 Participants
Brief Multidimensional Measurement of Religiousness/Spirituality (BMMRS-adapted) Survey Responses to Question 2. Baseline How often do you pray privately? · More than once a day	3 Participants	3 Participants
Brief Multidimensional Measurement of Religiousness/Spirituality (BMMRS-adapted) Survey Responses to Question 2. Baseline How often do you pray privately? · Never	1 Participants	2 Participants
Brief Multidimensional Measurement of Religiousness/Spirituality (BMMRS-adapted) Survey Responses to Question 2. Baseline How often do you pray privately? · Once a month	2 Participants	0 Participants
Brief Multidimensional Measurement of Religiousness/Spirituality (BMMRS-adapted) Survey Responses to Question 2. Baseline How often do you pray privately? · Less than once a month	0 Participants	1 Participants
Brief Multidimensional Measurement of Religiousness/Spirituality (BMMRS-adapted) Survey Responses to Question 2. Baseline How often do you pray privately? · Once a day	0 Participants	1 Participants
Brief Multidimensional Measurement of Religiousness/Spirituality (BMMRS-adapted) Survey Responses to Question 2. 3 month follow-up How often do you pray privately? · A few times a week	2 Participants	4 Participants
Brief Multidimensional Measurement of Religiousness/Spirituality (BMMRS-adapted) Survey Responses to Question 2. 3 month follow-up How often do you pray privately? · More than once a day	3 Participants	3 Participants
Brief Multidimensional Measurement of Religiousness/Spirituality (BMMRS-adapted) Survey Responses to Question 2. 3 month follow-up How often do you pray privately? · Never	1 Participants	1 Participants
Brief Multidimensional Measurement of Religiousness/Spirituality (BMMRS-adapted) Survey Responses to Question 2. 3 month follow-up How often do you pray privately? · Once a month	1 Participants	0 Participants
Brief Multidimensional Measurement of Religiousness/Spirituality (BMMRS-adapted) Survey Responses to Question 2. 3 month follow-up How often do you pray privately? · Less than once a month	0 Participants	1 Participants
Brief Multidimensional Measurement of Religiousness/Spirituality (BMMRS-adapted) Survey Responses to Question 2. 3 month follow-up How often do you pray privately? · Once a day	0 Participants	1 Participants

OTHER_PRE_SPECIFIED outcome

Timeframe: Up to 5 Weeks Post-Baseline

Population: Family caregivers of children with rare diseases

Items for this 13-item assessment of family satisfaction with the intervention were developed using input from the community about the emotional aspects of participation. 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 item was scored separately. Higher score for positive scale was better outcome. Higher score for negative scale was worse outcome. Study-specific process measure to assess adverse events and benefit/burden of participation. 12 items Total.

Outcome measures

Outcome measures
Measure	FACE-Rare Intervention n=9 Participants 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. FAmily CEntered (FACE) pACP Intervention: The (approximately) weekly 3-session FACE-Rare intervention of approximately 45-60 minutes each is comprised of the CSNAT approach \[Sessions 1 \& 2\] and Respecting Choices \[Session 3\].	Treatment-as-Usual (TAU) Control n=12 Participants To minimize the burden to families, we have chosen a Treatment-as-Usual (TAU) comparison condition, where patients will receive their normal standard of care. Both study arms will receive palliative (specialized medical) care information at enrollment and complete questionnaires before and after the intervention or TAU period. Current practice for minors with life-limiting illnesses is to defer initial discussions of advanced care planning (pACP) until a medical crisis, so this is what the TAU control arm condition will consist of.
Satisfaction Questionnaire (Process Measure) 1. It was useful · Strongly disagree	0 Participants	1 Participants
Satisfaction Questionnaire (Process Measure) 1. It was useful · Disagree	0 Participants	2 Participants
Satisfaction Questionnaire (Process Measure) 1. It was useful · Neither agree or disagree.	3 Participants	4 Participants
Satisfaction Questionnaire (Process Measure) 1. It was useful · Agree	3 Participants	4 Participants
Satisfaction Questionnaire (Process Measure) 1. It was useful · Strongly agree.	3 Participants	1 Participants
Satisfaction Questionnaire (Process Measure) 2. It was helpful · Strongly disagree	0 Participants	1 Participants
Satisfaction Questionnaire (Process Measure) 2. It was helpful · Disagree	0 Participants	2 Participants
Satisfaction Questionnaire (Process Measure) 2. It was helpful · Neither agree or disagree.	3 Participants	4 Participants
Satisfaction Questionnaire (Process Measure) 2. It was helpful · Agree	4 Participants	4 Participants
Satisfaction Questionnaire (Process Measure) 2. It was helpful · Strongly agree.	2 Participants	1 Participants
Satisfaction Questionnaire (Process Measure) 3. I felt scared or afraid · Strongly disagree	4 Participants	9 Participants
Satisfaction Questionnaire (Process Measure) 3. I felt scared or afraid · Disagree	3 Participants	2 Participants
Satisfaction Questionnaire (Process Measure) 3. I felt scared or afraid · Neither agree or disagree.	2 Participants	0 Participants
Satisfaction Questionnaire (Process Measure) 3. I felt scared or afraid · Agree	0 Participants	1 Participants
Satisfaction Questionnaire (Process Measure) 3. I felt scared or afraid · Strongly agree.	0 Participants	0 Participants
Satisfaction Questionnaire (Process Measure) 4. It felt like a load off my mind. · Strongly disagree	0 Participants	1 Participants
Satisfaction Questionnaire (Process Measure) 4. It felt like a load off my mind. · Disagree	3 Participants	3 Participants
Satisfaction Questionnaire (Process Measure) 4. It felt like a load off my mind. · Neither agree or disagree.	2 Participants	7 Participants
Satisfaction Questionnaire (Process Measure) 4. It felt like a load off my mind. · Agree	4 Participants	1 Participants
Satisfaction Questionnaire (Process Measure) 4. It felt like a load off my mind. · Strongly agree.	0 Participants	0 Participants
Satisfaction Questionnaire (Process Measure) 5. It was too much to handle. · Strongly disagree	3 Participants	5 Participants
Satisfaction Questionnaire (Process Measure) 5. It was too much to handle. · Disagree	4 Participants	5 Participants
Satisfaction Questionnaire (Process Measure) 5. It was too much to handle. · Neither agree or disagree.	0 Participants	1 Participants
Satisfaction Questionnaire (Process Measure) 5. It was too much to handle. · Agree	2 Participants	1 Participants
Satisfaction Questionnaire (Process Measure) 5. It was too much to handle. · Strongly agree.	0 Participants	0 Participants
Satisfaction Questionnaire (Process Measure) 6. I felt satisfied. · Strongly disagree	0 Participants	0 Participants
Satisfaction Questionnaire (Process Measure) 6. I felt satisfied. · Disagree	1 Participants	2 Participants
Satisfaction Questionnaire (Process Measure) 6. I felt satisfied. · Neither agree or disagree.	2 Participants	4 Participants
Satisfaction Questionnaire (Process Measure) 6. I felt satisfied. · Agree	6 Participants	4 Participants
Satisfaction Questionnaire (Process Measure) 6. I felt satisfied. · Strongly agree.	0 Participants	2 Participants
Satisfaction Questionnaire (Process Measure) 7. It was harmful. · Strongly disagree	6 Participants	10 Participants
Satisfaction Questionnaire (Process Measure) 7. It was harmful. · Disagree	3 Participants	1 Participants
Satisfaction Questionnaire (Process Measure) 7. It was harmful. · Neither agree or disagree.	0 Participants	1 Participants
Satisfaction Questionnaire (Process Measure) 7. It was harmful. · Agree	0 Participants	0 Participants
Satisfaction Questionnaire (Process Measure) 7. It was harmful. · Strongly agree.	0 Participants	0 Participants
Satisfaction Questionnaire (Process Measure) 8. I felt angry. · Strongly disagree	6 Participants	10 Participants
Satisfaction Questionnaire (Process Measure) 8. I felt angry. · Disagree	3 Participants	1 Participants
Satisfaction Questionnaire (Process Measure) 8. I felt angry. · Neither agree or disagree.	0 Participants	1 Participants
Satisfaction Questionnaire (Process Measure) 8. I felt angry. · Agree	0 Participants	0 Participants
Satisfaction Questionnaire (Process Measure) 8. I felt angry. · Strongly agree.	0 Participants	0 Participants
Satisfaction Questionnaire (Process Measure) 9. It was something I needed to do. · Strongly disagree	0 Participants	1 Participants
Satisfaction Questionnaire (Process Measure) 9. It was something I needed to do. · Disagree	1 Participants	0 Participants
Satisfaction Questionnaire (Process Measure) 9. It was something I needed to do. · Neither agree or disagree.	1 Participants	5 Participants
Satisfaction Questionnaire (Process Measure) 9. It was something I needed to do. · Agree	4 Participants	4 Participants
Satisfaction Questionnaire (Process Measure) 9. It was something I needed to do. · Strongly agree.	3 Participants	2 Participants
Satisfaction Questionnaire (Process Measure) 10. I felt sad. · Strongly disagree	1 Participants	7 Participants
Satisfaction Questionnaire (Process Measure) 10. I felt sad. · Disagree	2 Participants	3 Participants
Satisfaction Questionnaire (Process Measure) 10. I felt sad. · Neither agree or disagree.	0 Participants	1 Participants
Satisfaction Questionnaire (Process Measure) 10. I felt sad. · Agree	5 Participants	1 Participants
Satisfaction Questionnaire (Process Measure) 10. I felt sad. · Strongly agree.	1 Participants	0 Participants
Satisfaction Questionnaire (Process Measure) 111. I felt courageous. · Strongly disagree	1 Participants	1 Participants
Satisfaction Questionnaire (Process Measure) 111. I felt courageous. · Disagree	2 Participants	2 Participants
Satisfaction Questionnaire (Process Measure) 111. I felt courageous. · Neither agree or disagree.	3 Participants	6 Participants
Satisfaction Questionnaire (Process Measure) 111. I felt courageous. · Agree	3 Participants	1 Participants
Satisfaction Questionnaire (Process Measure) 111. I felt courageous. · Strongly agree.	0 Participants	2 Participants
Satisfaction Questionnaire (Process Measure) 12. It felt harmful. · Strongly disagree	5 Participants	9 Participants
Satisfaction Questionnaire (Process Measure) 12. It felt harmful. · Disagree	4 Participants	1 Participants
Satisfaction Questionnaire (Process Measure) 12. It felt harmful. · Neither agree or disagree.	0 Participants	2 Participants
Satisfaction Questionnaire (Process Measure) 12. It felt harmful. · Agree	0 Participants	0 Participants
Satisfaction Questionnaire (Process Measure) 12. It felt harmful. · Strongly agree.	0 Participants	0 Participants
Satisfaction Questionnaire (Process Measure) 13. It was worthwhile. · Strongly disagree	0 Participants	1 Participants
Satisfaction Questionnaire (Process Measure) 13. It was worthwhile. · Disagree	0 Participants	0 Participants
Satisfaction Questionnaire (Process Measure) 13. It was worthwhile. · Neither agree or disagree.	0 Participants	2 Participants
Satisfaction Questionnaire (Process Measure) 13. It was worthwhile. · Agree	7 Participants	7 Participants
Satisfaction Questionnaire (Process Measure) 13. It was worthwhile. · Strongly agree.	2 Participants	2 Participants

OTHER_PRE_SPECIFIED outcome

Timeframe: Up to 5 Weeks Post-Baseline

Population: Family caregivers of children living with rare diseases.

This is a process, not an outcome, measure. The participant is asked to rate how good the facilitator was for each of 4 communication skills on a scale from 0, definitely no, to 5, definitely yes. Higher scores mean better communication. Minimum score was 4. Maximum score was 20. We did not calculate a total score, as this was a process measure to assess the facilitator's communication and to provide feedback to the facilitator.

Outcome measures

Outcome measures
Measure	FACE-Rare Intervention n=9 Participants 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. FAmily CEntered (FACE) pACP Intervention: The (approximately) weekly 3-session FACE-Rare intervention of approximately 45-60 minutes each is comprised of the CSNAT approach \[Sessions 1 \& 2\] and Respecting Choices \[Session 3\].	Treatment-as-Usual (TAU) Control n=12 Participants To minimize the burden to families, we have chosen a Treatment-as-Usual (TAU) comparison condition, where patients will receive their normal standard of care. Both study arms will receive palliative (specialized medical) care information at enrollment and complete questionnaires before and after the intervention or TAU period. Current practice for minors with life-limiting illnesses is to defer initial discussions of advanced care planning (pACP) until a medical crisis, so this is what the TAU control arm condition will consist of.
Quality of Communication Questionnaire 1. Do you think that your attitudes are known by the interviewer? · Definitely yes	4 Participants	1 Participants
Quality of Communication Questionnaire 1. Do you think that your attitudes are known by the interviewer? · Do Not Know	1 Participants	5 Participants
Quality of Communication Questionnaire 1. Do you think that your attitudes are known by the interviewer? · Probably yes	4 Participants	3 Participants
Quality of Communication Questionnaire 1. Do you think that your attitudes are known by the interviewer? · Declined	0 Participants	1 Participants
Quality of Communication Questionnaire 1. Do you think that your attitudes are known by the interviewer? · Definitely no	0 Participants	1 Participants
Quality of Communication Questionnaire 1. Do you think that your attitudes are known by the interviewer? · Missing	0 Participants	1 Participants
Quality of Communication Questionnaire 2. Did you feel that the interviewer cared about you as a person? · Definitely yes	8 Participants	6 Participants
Quality of Communication Questionnaire 2. Did you feel that the interviewer cared about you as a person? · Do Not Know	0 Participants	1 Participants
Quality of Communication Questionnaire 2. Did you feel that the interviewer cared about you as a person? · Probably yes	1 Participants	3 Participants
Quality of Communication Questionnaire 2. Did you feel that the interviewer cared about you as a person? · Declined	0 Participants	1 Participants
Quality of Communication Questionnaire 2. Did you feel that the interviewer cared about you as a person? · Definitely no	0 Participants	0 Participants
Quality of Communication Questionnaire 2. Did you feel that the interviewer cared about you as a person? · Missing	0 Participants	1 Participants
Quality of Communication Questionnaire 3. Did you feel that the interviewer listened to what you said? · Definitely yes	9 Participants	8 Participants
Quality of Communication Questionnaire 3. Did you feel that the interviewer listened to what you said? · Do Not Know	0 Participants	0 Participants
Quality of Communication Questionnaire 3. Did you feel that the interviewer listened to what you said? · Probably yes	0 Participants	2 Participants
Quality of Communication Questionnaire 3. Did you feel that the interviewer listened to what you said? · Declined	0 Participants	1 Participants
Quality of Communication Questionnaire 3. Did you feel that the interviewer listened to what you said? · Definitely no	0 Participants	0 Participants
Quality of Communication Questionnaire 3. Did you feel that the interviewer listened to what you said? · Missing	0 Participants	1 Participants
Quality of Communication Questionnaire 4. Did you feel that the interviewer gave you enough attention? · Definitely yes	9 Participants	10 Participants
Quality of Communication Questionnaire 4. Did you feel that the interviewer gave you enough attention? · Do Not Know	0 Participants	0 Participants
Quality of Communication Questionnaire 4. Did you feel that the interviewer gave you enough attention? · Probably yes	0 Participants	0 Participants
Quality of Communication Questionnaire 4. Did you feel that the interviewer gave you enough attention? · Declined	0 Participants	1 Participants
Quality of Communication Questionnaire 4. Did you feel that the interviewer gave you enough attention? · Definitely no	0 Participants	0 Participants
Quality of Communication Questionnaire 4. Did you feel that the interviewer gave you enough attention? · Missing	0 Participants	1 Participants

OTHER_PRE_SPECIFIED outcome

Timeframe: Baseline to 3 month follow-up

Population: Family caregivers of children with rare diseases.

Responses to "How often do you go to religious services?" Responses ranged from "once or twice a year" to "more than once a week." Higher scores indicated greater attendance at religious services. Responses were used as a covariate in the initial Generalized linear mixed effect models. Prior research had indicated that this variable moderated quality of life outcomes. Higher scores indicate more attendance at religious services.

Outcome measures

Outcome measures
Measure	FACE-Rare Intervention n=9 Participants 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. FAmily CEntered (FACE) pACP Intervention: The (approximately) weekly 3-session FACE-Rare intervention of approximately 45-60 minutes each is comprised of the CSNAT approach \[Sessions 1 \& 2\] and Respecting Choices \[Session 3\].	Treatment-as-Usual (TAU) Control n=12 Participants To minimize the burden to families, we have chosen a Treatment-as-Usual (TAU) comparison condition, where patients will receive their normal standard of care. Both study arms will receive palliative (specialized medical) care information at enrollment and complete questionnaires before and after the intervention or TAU period. Current practice for minors with life-limiting illnesses is to defer initial discussions of advanced care planning (pACP) until a medical crisis, so this is what the TAU control arm condition will consist of.
Brief Multidimensional Measure of Religiousness/Spirituality (Brief MMRS) "How Often do You go to Religious Services?" How often do you go to religious services? 3 month follow up · Every week or more often	0 Participants	3 Participants
Brief Multidimensional Measure of Religiousness/Spirituality (Brief MMRS) "How Often do You go to Religious Services?" How often do you go to religious services? baseline · Every month or so	1 Participants	2 Participants
Brief Multidimensional Measure of Religiousness/Spirituality (Brief MMRS) "How Often do You go to Religious Services?" How often do you go to religious services? baseline · Never	3 Participants	3 Participants
Brief Multidimensional Measure of Religiousness/Spirituality (Brief MMRS) "How Often do You go to Religious Services?" How often do you go to religious services? baseline · Once or twice a month	2 Participants	1 Participants
Brief Multidimensional Measure of Religiousness/Spirituality (Brief MMRS) "How Often do You go to Religious Services?" How often do you go to religious services? baseline · Once or twice a year	3 Participants	3 Participants
Brief Multidimensional Measure of Religiousness/Spirituality (Brief MMRS) "How Often do You go to Religious Services?" How often do you go to religious services? baseline · Every week or more often	0 Participants	2 Participants
Brief Multidimensional Measure of Religiousness/Spirituality (Brief MMRS) "How Often do You go to Religious Services?" How often do you go to religious services? baseline · More than once a week	0 Participants	1 Participants
Brief Multidimensional Measure of Religiousness/Spirituality (Brief MMRS) "How Often do You go to Religious Services?" How often do you go to religious services? 3 month follow up · Every month or so	2 Participants	1 Participants
Brief Multidimensional Measure of Religiousness/Spirituality (Brief MMRS) "How Often do You go to Religious Services?" How often do you go to religious services? 3 month follow up · Never	3 Participants	4 Participants
Brief Multidimensional Measure of Religiousness/Spirituality (Brief MMRS) "How Often do You go to Religious Services?" How often do you go to religious services? 3 month follow up · Once or twice a month	3 Participants	1 Participants
Brief Multidimensional Measure of Religiousness/Spirituality (Brief MMRS) "How Often do You go to Religious Services?" How often do you go to religious services? 3 month follow up · Once or twice a year	1 Participants	3 Participants
Brief Multidimensional Measure of Religiousness/Spirituality (Brief MMRS) "How Often do You go to Religious Services?" How often do you go to religious services? 3 month follow up · More than once a week	0 Participants	0 Participants

OTHER_PRE_SPECIFIED outcome

Timeframe: Baseline and 3 month follow-up

Population: Family caregivers of children with rare diseases.

Responses to "To what extent do you consider yourself a religious person?" Responses ranged from "moderately religious" to "not religious at all." Higher scores indicated higher religiousness. Responses were collected as a covariate in the initial Generalized linear mixed effect models. Prior research had indicated that this variable moderated quality of life outcomes. Higher scores in the religious indicate self-perception as a more religious person.

Outcome measures

Outcome measures
Measure	FACE-Rare Intervention n=9 Participants 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. FAmily CEntered (FACE) pACP Intervention: The (approximately) weekly 3-session FACE-Rare intervention of approximately 45-60 minutes each is comprised of the CSNAT approach \[Sessions 1 \& 2\] and Respecting Choices \[Session 3\].	Treatment-as-Usual (TAU) Control n=12 Participants To minimize the burden to families, we have chosen a Treatment-as-Usual (TAU) comparison condition, where patients will receive their normal standard of care. Both study arms will receive palliative (specialized medical) care information at enrollment and complete questionnaires before and after the intervention or TAU period. Current practice for minors with life-limiting illnesses is to defer initial discussions of advanced care planning (pACP) until a medical crisis, so this is what the TAU control arm condition will consist of.
Brief Multidimensional Measure of Religiousness/Spirituality (Brief MMRS): "To What Extent do You Consider Yourself a Religious Person?" To what extent do you consider yourself a religious person? Baseline · Moderately religious	3 Participants	4 Participants
Brief Multidimensional Measure of Religiousness/Spirituality (Brief MMRS): "To What Extent do You Consider Yourself a Religious Person?" To what extent do you consider yourself a religious person? Baseline · Not religious at all	3 Participants	2 Participants
Brief Multidimensional Measure of Religiousness/Spirituality (Brief MMRS): "To What Extent do You Consider Yourself a Religious Person?" To what extent do you consider yourself a religious person? Baseline · Slightly religious	1 Participants	4 Participants
Brief Multidimensional Measure of Religiousness/Spirituality (Brief MMRS): "To What Extent do You Consider Yourself a Religious Person?" To what extent do you consider yourself a religious person? Baseline · Very religious	2 Participants	2 Participants
Brief Multidimensional Measure of Religiousness/Spirituality (Brief MMRS): "To What Extent do You Consider Yourself a Religious Person?" To what extent do you consider yourself a religious person? 3 month follow up · Moderately religious	2 Participants	2 Participants
Brief Multidimensional Measure of Religiousness/Spirituality (Brief MMRS): "To What Extent do You Consider Yourself a Religious Person?" To what extent do you consider yourself a religious person? 3 month follow up · Not religious at all	2 Participants	3 Participants
Brief Multidimensional Measure of Religiousness/Spirituality (Brief MMRS): "To What Extent do You Consider Yourself a Religious Person?" To what extent do you consider yourself a religious person? 3 month follow up · Slightly religious	3 Participants	6 Participants
Brief Multidimensional Measure of Religiousness/Spirituality (Brief MMRS): "To What Extent do You Consider Yourself a Religious Person?" To what extent do you consider yourself a religious person? 3 month follow up · Very religious	2 Participants	1 Participants

OTHER_PRE_SPECIFIED outcome

Timeframe: Baseline to 3 month outcomes.

Population: Family caregivers of children with rare diseases.

Responses to "To What Extent do You Consider Yourself a spiritual person?" Responses ranged from "not spiritual at all" to "very spiritual." Higher score meant more spiritual. Reported are frequency distributions and percentages. A higher score on the spiritual person item indicates self-perception as more spiritual.

Outcome measures

Outcome measures
Measure	FACE-Rare Intervention n=9 Participants 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. FAmily CEntered (FACE) pACP Intervention: The (approximately) weekly 3-session FACE-Rare intervention of approximately 45-60 minutes each is comprised of the CSNAT approach \[Sessions 1 \& 2\] and Respecting Choices \[Session 3\].	Treatment-as-Usual (TAU) Control n=12 Participants To minimize the burden to families, we have chosen a Treatment-as-Usual (TAU) comparison condition, where patients will receive their normal standard of care. Both study arms will receive palliative (specialized medical) care information at enrollment and complete questionnaires before and after the intervention or TAU period. Current practice for minors with life-limiting illnesses is to defer initial discussions of advanced care planning (pACP) until a medical crisis, so this is what the TAU control arm condition will consist of.
Brief Multidimensional Measurement of Religiousness/Spirituality (BMMRS-adapted): To What Extent do You Consider Yourself a Spiritual Person? To what extent do you consider yourself a spiritual person? Baseline · Moderately spiritual	3 Participants	7 Participants
Brief Multidimensional Measurement of Religiousness/Spirituality (BMMRS-adapted): To What Extent do You Consider Yourself a Spiritual Person? To what extent do you consider yourself a spiritual person? Baseline · Not spiritual at all	1 Participants	0 Participants
Brief Multidimensional Measurement of Religiousness/Spirituality (BMMRS-adapted): To What Extent do You Consider Yourself a Spiritual Person? To what extent do you consider yourself a spiritual person? Baseline · Slightly spiritual	2 Participants	5 Participants
Brief Multidimensional Measurement of Religiousness/Spirituality (BMMRS-adapted): To What Extent do You Consider Yourself a Spiritual Person? To what extent do you consider yourself a spiritual person? Baseline · Very spiritual	3 Participants	0 Participants
Brief Multidimensional Measurement of Religiousness/Spirituality (BMMRS-adapted): To What Extent do You Consider Yourself a Spiritual Person? To what extent do you consider yourself a spiritual person? 3 month follow up · Moderately spiritual	2 Participants	4 Participants
Brief Multidimensional Measurement of Religiousness/Spirituality (BMMRS-adapted): To What Extent do You Consider Yourself a Spiritual Person? To what extent do you consider yourself a spiritual person? 3 month follow up · Not spiritual at all	1 Participants	0 Participants
Brief Multidimensional Measurement of Religiousness/Spirituality (BMMRS-adapted): To What Extent do You Consider Yourself a Spiritual Person? To what extent do you consider yourself a spiritual person? 3 month follow up · Slightly spiritual	3 Participants	4 Participants
Brief Multidimensional Measurement of Religiousness/Spirituality (BMMRS-adapted): To What Extent do You Consider Yourself a Spiritual Person? To what extent do you consider yourself a spiritual person? 3 month follow up · Very spiritual	3 Participants	4 Participants

Adverse Events

FACE-Rare Intervention-Children

Serious events: 0 serious events

Other events: 0 other events

Deaths: 0 deaths

Treatment-as-Usual (TAU) Control- Children

Serious events: 0 serious events

Other events: 0 other events

Deaths: 0 deaths

FACE-Rare Intervention-Family Caregiver

Serious events: 0 serious events

Other events: 0 other events

Deaths: 0 deaths

TAU-control-Family Caregiver

Serious events: 0 serious events

Other events: 0 other events

Deaths: 0 deaths

Serious adverse events

Adverse event data not reported

Other adverse events

Adverse event data not reported

Additional Information

Dr. Maureen E. Lyon, Clinical Health Psychologist, Professor of Pediatrics

Children's National Hospital

Phone: 703-346-2873

Email: [email protected]

Results disclosure agreements

Principal investigator is a sponsor employee
Publication restrictions are in place

Measure	FACE-Rare Intervention n=18 Participants 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. FAmily CEntered (FACE) pACP Intervention: The (approximately) weekly 3-session FACE-Rare intervention of approximately 45-60 minutes each is comprised of the CSNAT approach \[Sessions 1 \& 2\] and Respecting Choices \[Session 3\].	Treatment-as-Usual (TAU) Control n=24 Participants To minimize the burden to families, we have chosen a Treatment-as-Usual (TAU) comparison condition, where patients will receive their normal standard of care. Both study arms will receive palliative (specialized medical) care information at enrollment and complete questionnaires before and after the intervention or TAU period. Current practice for minors with life-limiting illnesses is to defer initial discussions of advanced care planning (pACP) until a medical crisis, so this is what the TAU control arm condition will consist of.	Total n=42 Participants Total of all reporting groups
Age, Categorical Child with rare disease · <=18 years	9 Participants n=9 Participants • Baseline analysis was at level of participant with separate analysis for children with rare diseases and the primary family caregiver.	12 Participants n=12 Participants • Baseline analysis was at level of participant with separate analysis for children with rare diseases and the primary family caregiver.	21 Participants n=21 Participants • Baseline analysis was at level of participant with separate analysis for children with rare diseases and the primary family caregiver.
Age, Categorical Child with rare disease · Between 18 and 65 years	0 Participants n=9 Participants • Baseline analysis was at level of participant with separate analysis for children with rare diseases and the primary family caregiver.	0 Participants n=12 Participants • Baseline analysis was at level of participant with separate analysis for children with rare diseases and the primary family caregiver.	0 Participants n=21 Participants • Baseline analysis was at level of participant with separate analysis for children with rare diseases and the primary family caregiver.
Age, Categorical Child with rare disease · >=65 years	0 Participants n=9 Participants • Baseline analysis was at level of participant with separate analysis for children with rare diseases and the primary family caregiver.	0 Participants n=12 Participants • Baseline analysis was at level of participant with separate analysis for children with rare diseases and the primary family caregiver.	0 Participants n=21 Participants • Baseline analysis was at level of participant with separate analysis for children with rare diseases and the primary family caregiver.
Age, Categorical Primary family caregiver · <=18 years	0 Participants n=9 Participants • Baseline analysis was at level of participant with separate analysis for children with rare diseases and the primary family caregiver.	0 Participants n=12 Participants • Baseline analysis was at level of participant with separate analysis for children with rare diseases and the primary family caregiver.	0 Participants n=21 Participants • Baseline analysis was at level of participant with separate analysis for children with rare diseases and the primary family caregiver.
Age, Categorical Primary family caregiver · Between 18 and 65 years	9 Participants n=9 Participants • Baseline analysis was at level of participant with separate analysis for children with rare diseases and the primary family caregiver.	12 Participants n=12 Participants • Baseline analysis was at level of participant with separate analysis for children with rare diseases and the primary family caregiver.	21 Participants n=21 Participants • Baseline analysis was at level of participant with separate analysis for children with rare diseases and the primary family caregiver.
Age, Categorical Primary family caregiver · >=65 years	0 Participants n=9 Participants • Baseline analysis was at level of participant with separate analysis for children with rare diseases and the primary family caregiver.	0 Participants n=12 Participants • Baseline analysis was at level of participant with separate analysis for children with rare diseases and the primary family caregiver.	0 Participants n=21 Participants • Baseline analysis was at level of participant with separate analysis for children with rare diseases and the primary family caregiver.
Sex: Female, Male Child with rare disease · Female	5 Participants n=9 Participants • Sex as a biological variable was analyzed as a separate variable for child and primary family caregiver.	6 Participants n=12 Participants • Sex as a biological variable was analyzed as a separate variable for child and primary family caregiver.	11 Participants n=21 Participants • Sex as a biological variable was analyzed as a separate variable for child and primary family caregiver.
Sex: Female, Male Child with rare disease · Male	4 Participants n=9 Participants • Sex as a biological variable was analyzed as a separate variable for child and primary family caregiver.	6 Participants n=12 Participants • Sex as a biological variable was analyzed as a separate variable for child and primary family caregiver.	10 Participants n=21 Participants • Sex as a biological variable was analyzed as a separate variable for child and primary family caregiver.
Sex: Female, Male Primary family caregiver · Female	7 Participants n=9 Participants • Sex as a biological variable was analyzed as a separate variable for child and primary family caregiver.	10 Participants n=12 Participants • Sex as a biological variable was analyzed as a separate variable for child and primary family caregiver.	17 Participants n=21 Participants • Sex as a biological variable was analyzed as a separate variable for child and primary family caregiver.
Sex: Female, Male Primary family caregiver · Male	2 Participants n=9 Participants • Sex as a biological variable was analyzed as a separate variable for child and primary family caregiver.	2 Participants n=12 Participants • Sex as a biological variable was analyzed as a separate variable for child and primary family caregiver.	4 Participants n=21 Participants • Sex as a biological variable was analyzed as a separate variable for child and primary family caregiver.
Race (NIH/OMB) Child with rare disease · American Indian or Alaska Native	0 Participants n=9 Participants • Analysis of the population was at the level of the individual and not the dyad. We analyzed separately age data for child from age data for primary family caregiver.	0 Participants n=12 Participants • Analysis of the population was at the level of the individual and not the dyad. We analyzed separately age data for child from age data for primary family caregiver.	0 Participants n=21 Participants • Analysis of the population was at the level of the individual and not the dyad. We analyzed separately age data for child from age data for primary family caregiver.
Race (NIH/OMB) Child with rare disease · Asian	0 Participants n=9 Participants • Analysis of the population was at the level of the individual and not the dyad. We analyzed separately age data for child from age data for primary family caregiver.	0 Participants n=12 Participants • Analysis of the population was at the level of the individual and not the dyad. We analyzed separately age data for child from age data for primary family caregiver.	0 Participants n=21 Participants • Analysis of the population was at the level of the individual and not the dyad. We analyzed separately age data for child from age data for primary family caregiver.
Race (NIH/OMB) Child with rare disease · Native Hawaiian or Other Pacific Islander	0 Participants n=9 Participants • Analysis of the population was at the level of the individual and not the dyad. We analyzed separately age data for child from age data for primary family caregiver.	0 Participants n=12 Participants • Analysis of the population was at the level of the individual and not the dyad. We analyzed separately age data for child from age data for primary family caregiver.	0 Participants n=21 Participants • Analysis of the population was at the level of the individual and not the dyad. We analyzed separately age data for child from age data for primary family caregiver.
Race (NIH/OMB) Child with rare disease · Black or African American	4 Participants n=9 Participants • Analysis of the population was at the level of the individual and not the dyad. We analyzed separately age data for child from age data for primary family caregiver.	1 Participants n=12 Participants • Analysis of the population was at the level of the individual and not the dyad. We analyzed separately age data for child from age data for primary family caregiver.	5 Participants n=21 Participants • Analysis of the population was at the level of the individual and not the dyad. We analyzed separately age data for child from age data for primary family caregiver.
Race (NIH/OMB) Child with rare disease · White	3 Participants n=9 Participants • Analysis of the population was at the level of the individual and not the dyad. We analyzed separately age data for child from age data for primary family caregiver.	10 Participants n=12 Participants • Analysis of the population was at the level of the individual and not the dyad. We analyzed separately age data for child from age data for primary family caregiver.	13 Participants n=21 Participants • Analysis of the population was at the level of the individual and not the dyad. We analyzed separately age data for child from age data for primary family caregiver.
Race (NIH/OMB) Child with rare disease · More than one race	2 Participants n=9 Participants • Analysis of the population was at the level of the individual and not the dyad. We analyzed separately age data for child from age data for primary family caregiver.	1 Participants n=12 Participants • Analysis of the population was at the level of the individual and not the dyad. We analyzed separately age data for child from age data for primary family caregiver.	3 Participants n=21 Participants • Analysis of the population was at the level of the individual and not the dyad. We analyzed separately age data for child from age data for primary family caregiver.
Race (NIH/OMB) Child with rare disease · Unknown or Not Reported	0 Participants n=9 Participants • Analysis of the population was at the level of the individual and not the dyad. We analyzed separately age data for child from age data for primary family caregiver.	0 Participants n=12 Participants • Analysis of the population was at the level of the individual and not the dyad. We analyzed separately age data for child from age data for primary family caregiver.	0 Participants n=21 Participants • Analysis of the population was at the level of the individual and not the dyad. We analyzed separately age data for child from age data for primary family caregiver.
Race (NIH/OMB) Primary family caregiver · American Indian or Alaska Native	0 Participants n=9 Participants • Analysis of the population was at the level of the individual and not the dyad. We analyzed separately age data for child from age data for primary family caregiver.	0 Participants n=12 Participants • Analysis of the population was at the level of the individual and not the dyad. We analyzed separately age data for child from age data for primary family caregiver.	0 Participants n=21 Participants • Analysis of the population was at the level of the individual and not the dyad. We analyzed separately age data for child from age data for primary family caregiver.
Race (NIH/OMB) Primary family caregiver · Asian	0 Participants n=9 Participants • Analysis of the population was at the level of the individual and not the dyad. We analyzed separately age data for child from age data for primary family caregiver.	0 Participants n=12 Participants • Analysis of the population was at the level of the individual and not the dyad. We analyzed separately age data for child from age data for primary family caregiver.	0 Participants n=21 Participants • Analysis of the population was at the level of the individual and not the dyad. We analyzed separately age data for child from age data for primary family caregiver.
Race (NIH/OMB) Primary family caregiver · Native Hawaiian or Other Pacific Islander	0 Participants n=9 Participants • Analysis of the population was at the level of the individual and not the dyad. We analyzed separately age data for child from age data for primary family caregiver.	0 Participants n=12 Participants • Analysis of the population was at the level of the individual and not the dyad. We analyzed separately age data for child from age data for primary family caregiver.	0 Participants n=21 Participants • Analysis of the population was at the level of the individual and not the dyad. We analyzed separately age data for child from age data for primary family caregiver.
Race (NIH/OMB) Primary family caregiver · Black or African American	3 Participants n=9 Participants • Analysis of the population was at the level of the individual and not the dyad. We analyzed separately age data for child from age data for primary family caregiver.	1 Participants n=12 Participants • Analysis of the population was at the level of the individual and not the dyad. We analyzed separately age data for child from age data for primary family caregiver.	4 Participants n=21 Participants • Analysis of the population was at the level of the individual and not the dyad. We analyzed separately age data for child from age data for primary family caregiver.
Race (NIH/OMB) Primary family caregiver · White	5 Participants n=9 Participants • Analysis of the population was at the level of the individual and not the dyad. We analyzed separately age data for child from age data for primary family caregiver.	10 Participants n=12 Participants • Analysis of the population was at the level of the individual and not the dyad. We analyzed separately age data for child from age data for primary family caregiver.	15 Participants n=21 Participants • Analysis of the population was at the level of the individual and not the dyad. We analyzed separately age data for child from age data for primary family caregiver.
Race (NIH/OMB) Primary family caregiver · More than one race	1 Participants n=9 Participants • Analysis of the population was at the level of the individual and not the dyad. We analyzed separately age data for child from age data for primary family caregiver.	1 Participants n=12 Participants • Analysis of the population was at the level of the individual and not the dyad. We analyzed separately age data for child from age data for primary family caregiver.	2 Participants n=21 Participants • Analysis of the population was at the level of the individual and not the dyad. We analyzed separately age data for child from age data for primary family caregiver.
Race (NIH/OMB) Primary family caregiver · Unknown or Not Reported	0 Participants n=9 Participants • Analysis of the population was at the level of the individual and not the dyad. We analyzed separately age data for child from age data for primary family caregiver.	0 Participants n=12 Participants • Analysis of the population was at the level of the individual and not the dyad. We analyzed separately age data for child from age data for primary family caregiver.	0 Participants n=21 Participants • Analysis of the population was at the level of the individual and not the dyad. We analyzed separately age data for child from age data for primary family caregiver.
Region of Enrollment United States	18 participants n=18 Participants	24 participants n=24 Participants	42 participants n=42 Participants