The NEALS primary lateral sclerosis registry

On behalf of the NEALS PLS Registry Study Group

doi:10.1080/21678421.2020.1804591

The NEALS primary lateral sclerosis registry

On behalf of the NEALS PLS Registry Study Group

Neurology

Research output: Contribution to journal › Article › peer-review

8 Scopus citations

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 language	English (US)
Pages (from-to)	74-81
Number of pages	8
Journal	Amyotrophic Lateral Sclerosis and Frontotemporal Degeneration
Volume	21
Issue number	S1
DOIs	https://doi.org/10.1080/21678421.2020.1804591
State	Published - 2020

Funding

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. 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. 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). 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. 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. Stephen A. Goutman receives funding from NIH/NIEHS (K23ES027221), CDC/ATSDR, Target ALS, the ALS Association.

Keywords

PLS
disability
outcome measures
survival
upper motor neurons

ASJC Scopus subject areas

Neurology
Clinical Neurology

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10.1080/21678421.2020.1804591

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@article{4deae23417c24d0da581b62027a30353,

title = "The NEALS primary lateral sclerosis registry",

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{\textquoteright}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.",

keywords = "PLS, disability, outcome measures, survival, upper motor neurons",

author = "{On behalf of the NEALS PLS Registry Study Group} and Sabrina Paganoni and {De Marchi}, Fabiola and James Chan and Thrower, {Sara K.} and Staff, {Nathan P.} and Neil Datta and Kisanuki, {Yaz Y.} and Vivian Drory and Christina Fournier and Pioro, {Erik P.} and Goutman, {Stephen A.} and Nazem Atassi and Maryangel Jeon and Sarah Caldwell and Timothy Mcdonough and Caroline Gentile and Jianing Liu and Michelle Turner and Carol Denny and Kevin Felice and Misty Green and Stephanie Scarberry and Saad Abu-Saleh and Beatrice Nefussy and Debbie Hastings and Sangri Kim and Blake Swihart and Ximena Arcila-Londono and Newman, {Daniel S.} and Michael Silverman and Angela Genge and Kristiana Salmon and Lauren Elman and Leo Mccluskey and Kelly Almasy and Marc Gotkine and Kimberly Goslin and Arlena Cummings and Edwards, {Eli K.} and Michael Rivner and Kristy Bouchard and Brandy Quarles and Justin Kwan and Matthew Jaffa and Robert Baloh and Peggy Allred and David Walk and Samuel Maiser and Georgios Manousakis and Valerie Ferment",

note = "Publisher Copyright: {\textcopyright} 2020 World Federation of Neurology on behalf of the Research Group on Motor Neuron Diseases.",

year = "2020",

doi = "10.1080/21678421.2020.1804591",

language = "English (US)",

volume = "21",

pages = "74--81",

journal = "Amyotrophic Lateral Sclerosis and Frontotemporal Degeneration",

issn = "2167-8421",

publisher = "Informa Healthcare",

number = "S1",

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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 - Manousakis, Georgios

AU - Ferment, Valerie

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

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DO - 10.1080/21678421.2020.1804591

M3 - Article

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AN - SCOPUS:85096366444

SN - 2167-8421

VL - 21

SP - 74

EP - 81

JO - Amyotrophic Lateral Sclerosis and Frontotemporal Degeneration

JF - Amyotrophic Lateral Sclerosis and Frontotemporal Degeneration

IS - S1

ER -

The NEALS primary lateral sclerosis registry

Abstract

Funding

Keywords

ASJC Scopus subject areas

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