TY - JOUR
T1 - The NEALS primary lateral sclerosis registry
AU - On behalf of the NEALS PLS Registry Study Group
AU - Paganoni, Sabrina
AU - De Marchi, Fabiola
AU - Chan, James
AU - Thrower, Sara K.
AU - Staff, Nathan P.
AU - Datta, Neil
AU - Kisanuki, Yaz Y.
AU - Drory, Vivian
AU - Fournier, Christina
AU - Pioro, Erik P.
AU - Goutman, Stephen A.
AU - Atassi, Nazem
AU - Jeon, Maryangel
AU - Caldwell, Sarah
AU - Mcdonough, Timothy
AU - Gentile, Caroline
AU - Liu, Jianing
AU - Turner, Michelle
AU - Denny, Carol
AU - Felice, Kevin
AU - Green, Misty
AU - Scarberry, Stephanie
AU - Abu-Saleh, Saad
AU - Nefussy, Beatrice
AU - Hastings, Debbie
AU - Kim, Sangri
AU - Swihart, Blake
AU - Arcila-Londono, Ximena
AU - Newman, Daniel S.
AU - Silverman, Michael
AU - Genge, Angela
AU - Salmon, Kristiana
AU - Elman, Lauren
AU - Mccluskey, Leo
AU - Almasy, Kelly
AU - Gotkine, Marc
AU - Goslin, Kimberly
AU - Cummings, Arlena
AU - Edwards, Eli K.
AU - Rivner, Michael
AU - Bouchard, Kristy
AU - Quarles, Brandy
AU - Kwan, Justin
AU - Jaffa, Matthew
AU - Baloh, Robert
AU - Allred, Peggy
AU - Walk, David
AU - Maiser, Samuel
AU - Fernandes, J. Americo M.
AU - Thaisetthawatkul, Pariwat
N1 - Funding Information:
Sabrina Paganoni has received research funding from Target ALS, Harvard NeuroDiscovery Center, MGH ALS Therapy Fund, the Salah Foundation, the Spastic Paraplegia Foundation, Amylyx Therapeutics, Revalesio Inc., the ALS Association, ALS Finding a Cure, the American Academy of Neurology.
Funding Information:
Erik P. Pioro has received clinical trial and research funding from NIH/CDC, ALS Association, Iron Horse Diagnostics, Inc., and serves as consultant to Avanir Pharmaceuticals, Inc., Cytokinetics, Inc., ITF Pharma, Inc., MT Pharma America, Inc., and Otsuka America, Inc.
Funding Information:
Christina Fournier received research support from Mallinckrodt, Amylyx Pharmaceuticals, Neuraltus Pharmaceuticals, and Cytokinetics. Dr. Fournier received research funding from the U.S. Department of Veterans Affairs Office of Research and Development (IK2CX001595-02).
Funding Information:
Nathan P. Staff has received research funding from National Institutes of Health (CA 211887), Minnesota Regenerative Medicine Partnership (RMM 11215 CT002), Target ALS Foundation; serves as co-investigator on clinical trials by Brainstorm Therapeutics, Orion Pharmaceuticals.
Funding Information:
This study was supported in part by the Fund for Primary Lateral Sclerosis Research and Care at Massachusetts General Hospital. The authors thank all study participants and their families and caregivers for their dedication and contribution to MND research.
Funding Information:
Stephen A. Goutman receives funding from NIH/NIEHS (K23ES027221), CDC/ATSDR, Target ALS, the ALS Association.
Publisher Copyright:
© 2020 World Federation of Neurology on behalf of the Research Group on Motor Neuron Diseases.
PY - 2020
Y1 - 2020
N2 - 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.
AB - 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.
KW - PLS
KW - disability
KW - outcome measures
KW - survival
KW - upper motor neurons
UR - http://www.scopus.com/inward/record.url?scp=85096366444&partnerID=8YFLogxK
UR - http://www.scopus.com/inward/citedby.url?scp=85096366444&partnerID=8YFLogxK
U2 - 10.1080/21678421.2020.1804591
DO - 10.1080/21678421.2020.1804591
M3 - Article
C2 - 32915077
AN - SCOPUS:85096366444
VL - 21
SP - 74
EP - 81
JO - Amyotrophic Lateral Sclerosis and Other Motor Neuron Disorders
JF - Amyotrophic Lateral Sclerosis and Other Motor Neuron Disorders
SN - 2167-8421
IS - S1
ER -