Palliative care in 9 children with neurodegeneration with brain iron accumulation

Abstract

Aim

Evaluation of pediatric palliative home care of families with children suffering from neurodegeneration with brain iron accumulation (NBIA) and their parents.

Material and methods

The children were treated at home by a multidisciplinary team. Densitometry was used to evaluate the condition of the skeletal system. Botulinum toxin was injected into the muscles in doses between 22 and 50 units/kg. The quality of palliative care was assessed on the basis of a specially designed questionnaire for parents.

Results

The observations were performed on a group of 9 patients with NBIA. On admission, the median age of patients was 9 years (7–14). The average time of palliative home care was 1569 days (34 days–17 years). The median age at death (6 patients) was 11 years (7–15). The botulinum toxin injections gave the following results: reduction of spasticity and dystonia, reduction of spine and chest deformation, relief of pain and suffering, facilitation of rehabilitation and nursing, prevention of permanent contractures, and reduction of excessive salivation. Bone mineral density and bone strength index were reduced. Two patients experienced pathological fracture of the femur. The body mass index at admission varied between 9.8 and 14.9. In 7 cases, introduction of a ketogenic diet resulted in the increase of body mass and height. The ketogenic diet did not worsen the neurological symptoms. The parents positively evaluated the quality of care.

Conclusion

Palliative home care is the optimal form of treatment for children with NBIA.

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Affiliations

Authors

Contributions

Tomasz Dangel: symptom control, ketogenic diet, writing the manuscript

Tomasz Kmieć: neurological consultations and pharmacotherapy

Artur Januszaniec: symptom control, botulinum toxin treatment

Barbara Ważny: obtaining the clinical data, nursing palliative home care

Corresponding author

Correspondence to Tomasz Dangel.

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This article does not contain any studies with human participants performed by any of the authors.

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Informed consent was obtained from all individual participants included in the study.

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The authors declare that they have no conflict of interest.

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Dangel, T., Kmieć, T., Januszaniec, A. et al. Palliative care in 9 children with neurodegeneration with brain iron accumulation. Neurol Sci 41, 653–660 (2020). https://doi.org/10.1007/s10072-019-04099-5

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Keywords

  • NBIA
  • PKAN
  • Hospice
  • Ketogenic diet
  • Botulinum toxin
  • Quality care