Effect of Family Centered Pediatric Advance Care Planning for Children with Rare Diseases on Meaning/Peace (CO101B)

Outcomes 1. Know how advance care planning with families of children with rare diseases can improve family caregiver outcomes.2. Understand the impact of race and social determinants of health on family caregiver appraisal of their caregiving and the impact of poverty on family well-being, caregiver distress, and anxiety. Key Message We pilot tested an intervention designed to support the palliative care needs of families of children with ultra-rare disorders. Families randomized to the FACE®-Rare pediatric advance care planning intervention reported significantly greater meaning/peace/purpose, a measure of spiritual well-being, than controls. Importance Pediatric patients with rare genetic and metabolic conditions typically experience severe disability and high mortality. These children remain understudied and underserved. Few empirically validated interventions exist to support their family caregivers with respect to palliative care needs or end-of-life decision-making. The goal of this intervention was to close this gap in supportive services. Objectives Pediatric patients with rare conditions typically experience severe disability and high mortality. Investigators assessed the initial efficacy of the FAmily CEntered (FACE) pediatric Advance Care Planning (pACP), FACE-Rare, intervention on families’ quality of life.We hypothesized that, compared to Treatment as Usual (TAU), FACE-Rare family caregivers would report significantly better quality of life (QoL) assessed by emotional, spiritual, family caregiving appraisals compared to controls. We also explored the initial efficacy of FACE-Rare on child healthcare utilization (hospitalization, surgeries, and ER visits etc.). Scientific Methods Utilized A pilot-phase, single-blinded, intent-to-treat, randomized controlled clinical trial enrolled families from one pediatric quaternary hospital between 2021 and 2023. Intervention families received three weekly 60-minute (FACE-Rare pACP) sessions: (1) Carer Support Needs Assessment Tool/Action Plan (CSNAT), (2) CSNAT Action Plan Review, and (3) Pediatric Next Steps: Respecting Choices pACP. Controls received Treatment as Usual (TAU). Outcome measures were Beck Anxiety Inventory, Family Appraisal of Caregiving, FACIT-Spirituality, and health care utilization. Generalized Mixed Effect models with Gamma response assessed the intervention effect at 3-month follow-up. Results Children (n=21) were aged 1 to 10 years, 48% male, 24% Black; and 100% technology dependent. Primary family caregivers (n=21) were aged 30 to 43 years, 19% male, 19% Black; and 27% household income below the Federal Poverty Level. Dyads underwent 1:1 randomization: 9 to FACE-TC and 12 to TAU. TAU caregivers reported statistically lower Meaning/Peace than FACE-Rare caregivers (0.86, p=0.032, CI:0.75,0.99). Black caregivers reported significantly less Caregiver Distress (0.68, p=0.038, CI:0.47- 0.98) than non-Black caregivers. Poor families reported more Anxiety (3.51, p=0.002, CI:1.62,7.94), more Caregiver Strain (1.2, p=0.006, CI:1.07,1.42); and less Family Well-Being (0.78, p=0.015, CI:0.64,0.95). Conclusion(s) FACE®-Rare was feasible, acceptable, safe, and demonstrated initial efficacy, providing greater feelings of meaning and peace to caregivers. Poverty impacted well-being. A multi-site trial is needed to determine generalizability. Impact FACE®-Rare is feasible, acceptable, safe, and demonstrates initial efficacy. This promising intervention improved life purpose, meaning, and peace for family caregivers. Poverty impacted QoL. Clinicians caring for children with potentially life-limiting illnesses may want to create space for these conversations but may not have adequate training. Further research is needed to determine where along the continuum of care these conversations are most valuable and accessible. Future studies should examine if meaning and purpose predict health outcomes for family caregivers of children with rare diseases, as well as to determine the generalizability of the FACE Rare intervention and its potential to support health equity. Keywords Advanced care planning/shared decision making/Under served, under resourced populations