FACE-Rare: Protocol of a randomized controlled trial of a family-centered advance care planning intervention for families of children with rare diseases.

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

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.