The NEALS primary lateral sclerosis registry.

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.

Speckle tracking as a method to measure hemidiaphragm excursion.

INTRODUCTION: Diaphragm excursion measured via ultrasound may be an important imaging outcome measure of respiratory function. We developed a new method for measuring diaphragm movement and compared it to the more traditional M-mode method. METHODS: Ultrasound images of the right and left hemidiaphragms were collected to compare speckle tracking and M-mode measurements of diaphragm excursion. Speckle tracking was performed using EchoInsight (Epsilon Imaging, Ann Arbor, Michigan). RESULTS: Six healthy subjects without a history of pulmonary diseases were included in this proof-of-concept study. Speckle tracking of the diaphragm is technically possible. Unlike M-mode, speckle tracking carries the advantage of reliable visualization and measurement of the left hemidiaphragm. CONCLUSIONS: Speckle tracking accounted for diaphragm movement simultaneously in the cephalocaudad and mediolateral directions, unlike M-mode, which is 1-dimensional. Diaphragm speckle tracking may represent a novel, more robust method for measuring diaphragm excursion, especially for the left hemidiaphragm. Muscle Nerve 55: 125-127, 2017.