Advance Care Planning for Children With Rare Diseases: A Pilot RCT

Maureen E Lyon; Jamie L Fraser; Jessica D Thompkins; Heidi Clark; Nicola Brodie; Kathryn Detwiler; Clarivet Torres; Michael F Guerrera; Tamiko Younge; Samar Aoun; Eduardo A Trujillo Rivera

doi:10.1542/peds.2023-064557

Advance Care Planning for Children With Rare Diseases: A Pilot RCT

Pediatrics. 2024 May 3:e2023064557. doi: 10.1542/peds.2023-064557. Online ahead of print.

Authors

Maureen E Lyon^{1

2}, Jamie L Fraser^{1

2}, Jessica D Thompkins¹, Heidi Clark¹, Nicola Brodie^{1

2}, Kathryn Detwiler¹, Clarivet Torres¹, Michael F Guerrera¹, Tamiko Younge¹, Samar Aoun³, Eduardo A Trujillo Rivera^{1

2}

Affiliations

¹ Children's National Hospital, Washington, District of Columbia.
² Department of Pediatrics, George Washington University School of Medicine and Health Sciences, Washington, District of Columbia.
³ Peron Institute, Palliative Care, The Western University of Australia, Perth, Washington, Australia.

PMID: 38699801
DOI: 10.1542/peds.2023-064557

Abstract

Background and objective: Pediatric rare diseases are often life-limiting conditions and/or require constant caregiving. Investigators assessed the initial efficacy of the FAmily CEntered (FACE) pediatric advance care planning (pACP), FACE-Rare, intervention on families' quality of life.

Methods: A pilot-phase, single-blinded, intent-to-treat, randomized controlled clinical trial enrolled families from 1 pediatric quaternary hospital between 2021 and 2023. Intervention families received 3 weekly 60-minute (FACE-Rare pACP) sessions: (1) Carer Support Needs Assessment Tool or Action Plan, (2) Carer Support Needs Assessment Tol 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, Functional Assessment of Chronic Illness Therapy (FACIT)-Spirituality, and health care utilization. Generalized mixed effect models with γ 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-Rare and 12 to TAU. TAU caregivers reported statistically lower meaning and peace than FACE-Rare caregivers (0.9, P = .03, confidence interval [CI]: 0.75-0.99). Black caregivers reported significantly less caregiver distress (0.7, P = .04, CI: 0.47-0.98) than non-Black caregivers. Poor families reported more anxiety (3.5, P = .002, CI: 1.62-7.94), more caregiver strain (1.2, P = .006, CI: 1.07-1.42); and less family well-being (0.8, P = .02, CI: 0.64-0.95).

Conclusions: FACE®-Rare was feasible, acceptable, safe, and demonstrated initial efficacy, providing greater feelings of meaning and peace to caregivers. Poverty impacted well-being. A multisite trial is needed to determine generalizability.