Related Experiment Video

Updated: Apr 17, 2026

An In Vitro Model for the Study of Cellular Pathophysiology in Globoid Cell Leukodystrophy
07:45

An In Vitro Model for the Study of Cellular Pathophysiology in Globoid Cell Leukodystrophy

Published on: October 21, 2014

8.5K

Finding comfort in complexity: The role of palliative care in children with leukodystrophy

Cia Bishop1, Vanessa Nguyen2, Tabitha D'Souza3

  • 1Department of Pediatrics, Division of Pediatric Palliative Care, Children's Healthcare of Atlanta, Emory University, Atlanta, GA, USA.

Molecular Genetics and Metabolism
|April 15, 2026
PubMed

Insights

Palliative care consultation can benefit children with leukodystrophy, improving symptom management and advanced care planning. Early integration of neuro-palliative care is crucial for this population.

Area of Science:

  • Pediatric Neurology
  • Palliative Care
  • Neurodegenerative Diseases

Background:

  • Leukodystrophy causes progressive neurological decline in children.
  • Palliative care aids symptom management and advanced care planning for children with severe neurological impairment.

Purpose of the Study:

  • Evaluate the role, timing, and impact of palliative care on end-of-life outcomes for pediatric leukodystrophy patients.

Main Methods:

  • Retrospective chart review of pediatric patients (0-18 years) with leukodystrophy.
  • Data collected from Children's Healthcare of Atlanta between 2016 and 2023.

Main Results:

  • 26% of leukodystrophy patients received palliative care, with a median of 2.4 years from diagnosis to consultation.
  • Among deceased patients, 63% had palliative care, and hospice enrollment was associated with DNR/DNI status and home deaths.
  • Most deaths occurred in the hospital, but 40% of deceased patients died at home.

Conclusions:

  • Goals of care conversations and advanced care planning are beneficial for pediatric leukodystrophy patients and families.
  • There are opportunities to enhance early neuro-palliative integration for children with leukodystrophy.
Abstract

Keywords:
Advance care planningEnd-of-life careHospice careNeuropalliative carePediatric leukodystrophyPediatric palliative carePlace of deathQuality of life

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