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Abstract Details

Neurofilament Levels in CSF and Serum in an Adult SMA Cohort treated with Nusinersen
Neuromuscular and Clinical Neurophysiology (EMG)
Neuromuscular and Clinical Neurophysiology (EMG) Posters (7:00 AM-5:00 PM)
056
NA
Nusinersen is approved for all types of spinal muscular atrophy (SMA), however biomarkers are needed to better monitor drug efficacy and disease progression in adults with SMA, a less well studied population.  Neurofilaments (Nfs, specifically Nf-light [NfL] and Nf-heavy [NfH]), are markers of axonal degeneration and promising biomarkers in pediatric SMA, but may be less informative in adult SMA. We prospectively investigated Nfs in ambulatory and non-ambulatory SMA adults.

NfH and NfL levels were quantified using single molecular array (simoa) in CSF of 33 adult SMA patients (SMN copy number 3-4) before treatment and at maintenance doses (6 months). Blood samples were collected from 11 of these patients. CSF NfL and NfH from SMA patients were compared to CSF samples from age-matched controls (n = 6) without neurological disease. Pearson correlation coefficients were performed between Nfs and other functional outcome measures as well as between NFs levels in serum and CSF samples.

Cross-sectional analyses showed an increase in CSF NfL with age in patients (p=0.0082) and controls (p=0.002), but no significant changes in NfL or NfH over time in CSF or serum. CSF NfL showed positive correlation with Revised Upper Limb Module and Maximal Voluntary Isometric Contraction Testing (r=0.581, p=0.01, and r=0.499, p=0.03, respectively). CSF and serum NfH showed strong correlations (r=0.741, p=0.01) and NfL (r=0.670, p=0.04).
Nf levels were similar between SMA and controls and showed no change with nusinersen treatment. Our data also showed an age-related increase in CSF NfL in patients and controls and a positive correlation between serum and CSF samples. Our findings are consistent with previous reports and suggest a lack of active disease activity in adult SMA individuals.
Authors/Disclosures
Kelly Rich, MS, CGC
PRESENTER 		Ms. Rich has nothing to disclose.
No disclosure on file
Sarah Heintzman, NP (The Ohio State University) Ms. Heintzman has nothing to disclose.
Megan G. Pino Ms. Pino has nothing to disclose.
Mehmet E. Yalvac, PhD (The Ohio State Univ. Dept. of Neurology) Dr. Yalvac has nothing to disclose.
William D. Arnold, MD Dr. Arnold has received personal compensation in the range of $5,000-$9,999 for serving on a Scientific Advisory or Data Safety Monitoring board for Genentech. Dr. Arnold has received personal compensation in the range of $5,000-$9,999 for serving on a Scientific Advisory or Data Safety Monitoring board for La Hoffmann Roche. Dr. Arnold has received personal compensation in the range of $500-$4,999 for serving on a Scientific Advisory or Data Safety Monitoring board for Cadent Therapeutics . Dr. Arnold has received personal compensation in the range of $0-$499 for serving on a Scientific Advisory or Data Safety Monitoring board for Novartis. The institution of Dr. Arnold has received research support from NIH. The institution of Dr. Arnold has received research support from NMD Pharma. The institution of Dr. Arnold has received research support from Gilead Sciences. The institution of Dr. Arnold has received research support from CureSMA. Dr. Arnold has received intellectual property interests from a discovery or technology relating to health care.
Bakri Elsheikh, MD, FÂ鶹´«Ã½Ó³»­ (The Ohio State University Wexner Medical Center) Dr. Elsheikh has received personal compensation in the range of $500-$4,999 for serving as a Consultant for Biogen . Dr. Elsheikh has received personal compensation in the range of $500-$4,999 for serving as a Consultant for Argnex . The institution of Dr. Elsheikh has received research support from Biogen. The institution of Dr. Elsheikh has received research support from Cure SMA.
Stephen J. Kolb, MD, PhD (The Ohio State University) Dr. Kolb has received personal compensation in the range of $5,000-$9,999 for serving as a Consultant for AveXis. Dr. Kolb has received personal compensation in the range of $0-$499 for serving on a Scientific Advisory or Data Safety Monitoring board for CureSMA. The institution of Dr. Kolb has received research support from NIH. The institution of Dr. Kolb has received research support from AveXis. The institution of Dr. Kolb has received research support from NIH.