The NEALS primary lateral sclerosis registry.

Journal: Amyotrophic Lateral Sclerosis & Frontotemporal Degeneration
Abstract

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.

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Authors
Sabrina Paganoni, Fabiola De Marchi, James Chan, Sara Thrower, Nathan Staff, Neil Datta, Yaz Kisanuki, Vivian Drory, Christina Fournier, Erik Pioro, Stephen Goutman, Nazem Atassi, Sarah Caldwell, Timothy Mcdonough, Caroline Gentile, Jianing Liu, Michelle Turner, Carol Denny, Kevin Felice, Misty Green, Stephanie Scarberry, Saad Abu Saleh, Beatrice Nefussy, Debbie Hastings, Sangri Kim, Blake Swihart, Ximena Arcila Londono, Daniel Newman, Michael Silverman, Angela Genge, Kristiana Salmon, Lauren Elman, Leo Mccluskey, Kelly Almasy, Marc Gotkine, Kimberly Goslin, Arlena Cummings, Eli Edwards, Michael Rivner, Kristy Bouchard, Brandy Quarles, Justin Kwan, Matthew Jaffa, Robert Baloh, Peggy Allred, David Walk, Samuel Maiser, Georgios Manousakis, Valerie Ferment, J Americo Fernandes, Pariwat Thaisetthawatkul, Deborah Heimes, Melissa Phillips, Laura Sams, Melissa Kahler, Alecia Corcoran, Daniel Larriviere, Sadie Chotto, Gracy Juba

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