Palliative care in 9 children with neurodegeneration with brain iron accumulation

Neurol Sci. 2020 Mar;41(3):653-660. doi: 10.1007/s10072-019-04099-5. Epub 2019 Nov 22.


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.

Keywords: Botulinum toxin; Hospice; Ketogenic diet; NBIA; PKAN; Quality care.

Publication types

  • Observational Study

MeSH terms

  • Adolescent
  • Bone Diseases / drug therapy
  • Bone Diseases / etiology
  • Botulinum Toxins / pharmacology
  • Child
  • Dystonia / drug therapy
  • Dystonia / etiology
  • Female
  • Home Care Services*
  • Humans
  • Iron Metabolism Disorders / complications
  • Iron Metabolism Disorders / drug therapy
  • Iron Metabolism Disorders / nursing
  • Iron Metabolism Disorders / therapy*
  • Male
  • Muscle Spasticity / drug therapy
  • Muscle Spasticity / etiology
  • Musculoskeletal Pain / drug therapy
  • Musculoskeletal Pain / etiology
  • Neuroaxonal Dystrophies / complications
  • Neuroaxonal Dystrophies / drug therapy
  • Neuroaxonal Dystrophies / nursing
  • Neuroaxonal Dystrophies / therapy*
  • Neuromuscular Agents / pharmacology*
  • Palliative Care / methods*


  • Neuromuscular Agents
  • Botulinum Toxins

Supplementary concepts

  • Neurodegeneration with brain iron accumulation (NBIA)