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Palliative Care in Children with Inherited Metabolic Diseases: Why does it matter?

Joana Pereira Mendes1, Andreia Nogueira1, Ema Grilo1

  • 1Paediatric Palliative Care Team, Hospital Pediátrico, Centro Hospitalar e Universitário de Coimbra, Portugal.

Endocrine, Metabolic & Immune Disorders Drug Targets
|November 3, 2023
PubMed

Insights

Paediatric palliative care teams (PPCT) support children with inherited metabolic diseases (IMD) at home and in end-of-life planning. Referral to PPCTs reduced emergency department admissions, offering crucial emotional and bereavement support to families.

Area of Science:

  • Pediatric Palliative Care
  • Inherited Metabolic Diseases
  • Healthcare Resource Utilization

Background:

  • Inherited metabolic diseases (IMD) impose significant burdens on children and families, with challenges in symptom identification, prognostic uncertainty, and bereavement.
  • Literature on the impact of paediatric palliative care (PPC) for these complex cases is limited.

Purpose of the Study:

  • To evaluate children with IMD referred to a PPC team (PPCT).
  • To analyze the impact of PPCT on home care, treatment limitations, hospital resource use, and end-of-life support.

Main Methods:

  • Retrospective cohort study of children with IMD referred to a specialized PPCT from 2016-2022.
  • Assessed clinical data including symptom control, referral timing, follow-up duration, treatment limitations, device dependency, hospital resource use (prior to and after referral), place of death, and end-of-life support.

Main Results:

  • Fifteen children with IMD (median age 7 years) were referred to PPCT; 73% received home PPC and 80% had planned limitations on treatment.
  • A decrease in emergency department admissions was observed post-referral (10 vs 2), though hospital and intensive care admissions remained similar.
  • Six children died during the study period, all with planned treatment limitations, and 67% received PPCT support at end-of-life, with families receiving emotional and bereavement support.

Conclusions:

  • Children with severe, complex IMD require extensive medical support and consume significant healthcare resources.
  • Communication impairments in non-verbal children pose challenges for symptom assessment.
  • PPCT referral facilitated home support, advance care planning, and reduced emergency visits, highlighting the need for a holistic approach to PPC for children with IMD.
Abstract

Keywords:
ChildrenComplexityHome careHospital resourcesInherited metabolic diseasesPaediatric palliative care referral

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