Neuropathy in ARSACS is demyelinating but without typical nerve enlargement in nerve ultrasound

J Neurol. 2024 May;271(5):2494-2502. doi: 10.1007/s00415-023-12159-2. Epub 2024 Jan 23.

Abstract

Background: To specify peripheral nerve affection in autosomal recessive spastic ataxia of Charlevoix-Saguenay (ARSACS) by correlating high-resolution nerve ultrasound and nerve conduction studies.

Methods: We assessed a cohort of 11 ARSACS patients with standardized nerve conduction studies and high-resolution ultrasound of peripheral nerves and compared nerve ultrasound findings to a healthy control group matched for age, sex, size and weight.

Results: Mean age of patients was 39.0 (± 14.1) years and disease duration at assessment 30.6 (± 12.5) years. All patients presented with a spasticity, ataxia and peripheral neuropathy. Neuropathy appeared to be primarily demyelinating in 9/11 cases and was not classifiable in 2/11 cases due to not evocable potentials. Nerve ultrasound revealed a normal ultrasound pattern sum score (UPSS) in each ARSACS patient and no significant nerve enlargement compared to the control group.

Conclusions: Peripheral neuropathy in ARSACS showed primarily demyelinating rather than axonal characteristics and presented without nerve enlargement. As demyelinating neuropathies do commonly present enlarged nerves we recommend further genetic testing of the SACS gene in patients who present with this combination of demyelinating neuropathy without nerve enlargement. ARSACS cases that initially presented only with neuropathy without spasticity or ataxia and therefore were misdiagnosed as Charcot-Marie-Tooth disease are supporting this suggestion.

Keywords: ARSACS; Ataxia; Neuropathy; UPSS; Ultrasound.

MeSH terms

  • Adult
  • Cohort Studies
  • Demyelinating Diseases* / diagnostic imaging
  • Female
  • Humans
  • Male
  • Middle Aged
  • Muscle Spasticity* / diagnostic imaging
  • Muscle Spasticity* / etiology
  • Muscle Spasticity* / physiopathology
  • Neural Conduction* / physiology
  • Peripheral Nerves / diagnostic imaging
  • Peripheral Nerves / pathology
  • Peripheral Nervous System Diseases / diagnosis
  • Peripheral Nervous System Diseases / diagnostic imaging
  • Peripheral Nervous System Diseases / pathology
  • Peripheral Nervous System Diseases / physiopathology
  • Spinocerebellar Ataxias* / complications
  • Spinocerebellar Ataxias* / congenital*
  • Spinocerebellar Ataxias* / diagnostic imaging
  • Ultrasonography*
  • Young Adult

Supplementary concepts

  • Spastic ataxia Charlevoix-Saguenay type