Phosphorylated neurofilament heavy chain: A biomarker of survival for C9ORF72-associated amyotrophic lateral sclerosis
Document Type
Article
Abstract
As potential treatments for C9ORF72-associated amyotrophic lateral sclerosis (c9ALS) approach clinical trials, the identification of prognostic biomarkers for c9ALS becomes a priority. We show that levels of phosphorylated neurofilament heavy chain (pNFH) in cerebrospinal fluid (CSF) predict disease status and survival in c9ALS patients, and are largely stable over time. Moreover, c9ALS patients exhibit higher pNFH levels, more rapid disease progression, and shorter survival after disease onset than ALS patients without C9ORF72 expansions. These data support the use of CSF pNFH as a prognostic biomarker for clinical trials, which will increase the likelihood of successfully developing a treatment for c9ALS. Ann Neurol 2017;82:139–146.
Publication Date
7-1-2017
Publication Title
Annals of Neurology
ISSN
03645134
E-ISSN
15318249
Volume
82
Issue
1
First Page
139
Last Page
146
PubMed ID
28628244
Digital Object Identifier (DOI)
10.1002/ana.24980
Recommended Citation
Gendron, Tania F.; Daughrity, Lillian M.; Heckman, Michael G.; Diehl, Nancy N.; Wuu, Joanne; Miller, Timothy M.; Pastor, Pau; Trojanowski, John Q.; Grossman, Murray; Berry, James D.; Hu, William T.; Ratti, Antonia; Benatar, Michael; Silani, Vincenzo; Glass, Jonathan D.; Floeter, Mary Kay; Jeromin, Andreas; Boylan, Kevin B.; Petrucelli, Leonard; Prudencio, Mercedes; Caputo, Ana M.; Desaro, Pamela; Palmucci, Carla; Robertson, Amelia; Song, Yuping; Rush, Beth K.; Pedraza, Otto; Lacomis, David; Ladha, Shafeeq; Fournier, Christina N.; McCluskey, Leo F.; and Elman, Lauren B., "Phosphorylated neurofilament heavy chain: A biomarker of survival for C9ORF72-associated amyotrophic lateral sclerosis" (2017). Translational Neuroscience. 1052.
https://scholar.barrowneuro.org/neurobiology/1052