Publication
FACE-Rare: Protocol of a randomized controlled trial of a family-centered advance care planning intervention for families of children with rare diseases
Yu J, Thompkins J, Friebert S, Grossoehme D, Baker J, Gordish-Dressman H, Lyon M · Contemporary Clinical Trials · 2026
Background: Children with rare diseases and their family caregivers must manage extraordinary care needs, significant symptom burdens, and uncertain prognoses. Pediatric Advanced Care Planning (pACP) is a promising communication-based approach to better support these families. However, empirical evidence regarding the efficacy of pACP among this population is lacking. In response, our team developed and successfully pilot-tested FACE-Rare (FAmily-CEntered Advance Care Planning - Rare Disorders). Methods & design: This manuscript describes the protocol for a randomized controlled trial evaluating FACE-Rare's efficacy for improving family quality of life (QOL). We aim to enroll 160 families (child-family caregiver dyads/triads) from three pediatric health systems across the United States. Families will be randomized 1:1 to FACE-Rare or an enhanced treatment as usual control. FACE-Rare is delivered by trained facilitators over three 60-min sessions either virtually (videoconference) or in-person. FACE-Rare utilizes the Carer's Alert Thermometer to identify and address a family's palliative care needs and the Respecting Choices: Next Steps pACP interview to facilitate future medical decision-making. Our primary outcome is family caregiver QOL (caregiver appraisal, emotional distress, and existential well-being). Secondary outcomes include families' palliative care needs and social connection. An exploratory aim seeks to describe the interactions between sociodemographic characteristics (e.g., household income) and family caregiver QOL. Conclusion(s): This is the first randomized controlled trial testing the efficacy of a pACP intervention tailored for families of children with rare diseases. Findings will provide critical evidence about the impact of pACP on family caregiver QOL and medical decision-making for this underserved population.
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