The NEALS primary lateral sclerosis registry. (2nd November 2020)

• Record Type: Journal Article
• Authors: Paganoni, Sabrina; De Marchi, Fabiola; Chan, James; Thrower, Sara K.; Staff, Nathan P.; Datta, Neil; Kisanuki, Yaz Y.; Drory, Vivian; Fournier, Christina; Pioro, Erik P.; Goutman, Stephen A.; Atassi, Nazem; Jeon, Maryangel; Caldwell, Sarah; Mcdonough, Timothy; Gentile, Caroline; Liu, Jianing; Turner, Michelle; Denny, Carol; Felice, Kevin; Green, Misty; Scarberry, Stephanie; Abu-Saleh, Saad; Nefussy, Beatrice; Hastings, Debbie; Kim, Sangri; Swihart, Blake; Arcila-Londono, Ximena; Newman, Daniel S.; Silverman, Michael; Genge, Angela; Salmon, Kristiana; Elman, Lauren; Mccluskey, Leo; Almasy, Kelly; Gotkine, Marc; Goslin, Kimberly; Cummings, Arlena; Edwards, Eli K.; Rivner, Michael; Bouchard, Kristy; Quarles, Brandy; Kwan, Justin; Jaffa, Matthew; Baloh, Robert; Allred, Peggy; Walk, David; Maiser, Samuel; Manousakis, Georgios; Ferment, Valerie; Fernandes, J. Americo M.; Thaisetthawatkul, Pariwat; Heimes, Deborah; Phillips, Melissa; Sams, Laura; Kahler, Melissa; Corcoran, Alecia; Larriviere, Daniel G.; Chotto, Sadie; Juba, Gracy;
• Is Part Of: Amyotrophic lateral sclerosis and frontotemporal degeneration. Volume 21(2020)Supplement 1
• Page Start: 74
• Page End: 81
• Publication Date: 2020-11-02
• Subjects: PLS -- upper motor neurons -- outcome measures -- survival -- disability; 616.839
• DOI: 10.1080/21678421.2020.1804591
• Languages: English
• ISSNs: 2167-8421

Abstract

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.