The NEALS primary lateral sclerosis registry

On behalf of the NEALS PLS Registry Study Group

Research output: Contribution to journalArticlepeer-review

Abstract

Background and objective: Primary lateral sclerosis (PLS) is a neurodegenerative disease characterized by progressive upper motor neuron dysfunction. Because PLS patients represent only 1 to 4% of patients with adult motor neuron diseases, there is limited information about the disease’s natural history. The objective of this study was to establish a large multicenter retrospective longitudinal registry of PLS patients seen at Northeast ALS Consortium (NEALS) sites to better characterize the natural progression of PLS. Methods: Clinical characteristics, electrophysiological findings, laboratory values, disease-related symptoms, and medications for symptom management were collected from PLS patients seen between 2000 and 2015. Results: The NEALS registry included data from 250 PLS patients. Median follow-up time was 3 years. The mean rate of functional decline measured by ALSFRS-R total score was −1.6 points/year (SE:0.24, n = 124); the mean annual decline in vital capacity was −3%/year (SE:0.55, n = 126). During the observational period, 18 patients died, 17 patients had a feeding tube placed and 7 required permanent assistive ventilation. Conclusions: The NEALS PLS Registry represents the largest available aggregation of longitudinal clinical data from PLS patients and provides a description of expected natural disease progression. Data from the registry will be available to the PLS community and can be leveraged to plan future clinical trials in this rare disease.

Original languageEnglish (US)
Pages (from-to)74-81
Number of pages8
JournalAmyotrophic Lateral Sclerosis and Frontotemporal Degeneration
Volume21
Issue numberS1
DOIs
StatePublished - 2020

Keywords

  • PLS
  • disability
  • outcome measures
  • survival
  • upper motor neurons

ASJC Scopus subject areas

  • Neurology
  • Clinical Neurology

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