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Children With Rare Diseases and Their Families Appreciate Advance Care Planning

May 3, 2024

Although each rare disease affects fewer than 200,000 people in the US, there are approximately 10,000 rare diseases; thus, in aggregate, rare diseases are not that rare.

Although those with rare diseases often have shorter life spans, discussion of end-of-life care is often not usual care for these patients and their families.

Maureen Lyon, PhD, and colleagues at Children’s National Hospital and the Western University of Australia conducted a pilot randomized controlled trial of a pediatric advance care planning (pACP) intervention for children and their families. The intervention was called FAmily CEntered (FACE) pediatric advance care planning for children with rare diseases (FACE-Rare) and was adapted from evidence-based interventions. The authors’ findings are being early released this week in a Pediatrics article entitled, “Advance Care Planning for Children with Rare Diseases: A Pilot RCT” (10.1542/peds.2023-064557).

Twenty-one children (median age 3 years; 100% were technology-dependent) and their families were randomized to receive either usual care or the FACE-Rare intervention, which consisted of 3–4 45-minute sessions over 3–4 weeks:

  • Session 1: Carer Support Needs Assessment Tool/Action Plan: What would allow the caregiver(s) to better care for the child? What would provide more support for the caregiver(s)? The family, with the help of a facilitator, developed a Shared Action Plan that incorporated both of these priorities and were asked to use the Shared Action Plan during the following week.
  • Session 2: Shared Action Plan review: What was the family able to accomplish? What obstacles did they encounter? The family was provided with additional resources and came up with questions to ask the child’s clinician at the next medical visit.
  • Session 3: Pediatric Next Steps: Respecting Choices pACP: What did the family understand about the child’s diagnosis? What were their fears and hopes? What future medical decisions would be consistent with their goals and values?
  • Session 4 (which was sometimes combined with Session 3): Developing an Advance Care Plan for future medical decisions that are consistent with the family’s goals and values.

Although the sample size was small, the FACE-Rare intervention was feasible, safe, and well received by families. It was also effective in improving feelings of meaning and peace in caregivers.

Even though these types of conversations are difficult, parents appreciate them and find them helpful. If you care for children with any type of chronic, potentially lifespan-limiting illness, read this article to learn more about the importance of these conversations.

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