Abstract Details

Title Quantitative muscle strength changes in adults with Spinal Muscular Atrophy receiving Nusinersen

Topic Neuromuscular and Clinical Neurophysiology (EMG)

Presentation(s) P5 - Poster Session 5 (11:45 AM-12:45 PM)

Poster/Presentation
Number 11-008

Objective

To observe quantitative isometric strength changes in adults with Spinal Muscular Atrophy (SMA) who are being treated with Nusinerson.

Background

As disease modifying treatments for SMA become increasingly more available, having insight to changes in strength and function of treated patients is a crucial next step in understanding the impact of strength changes with treatment.

Design/Methods

We recruited 42 participants diagnosed with SMA receiving Nusinersen. Quantitative muscle testing was measured using a Microfet handheld dynamometer to measure 4 muscle groups bilaterally: elbow flexion (EF), elbow extension (EE), knee flexion (KF) and knee extension (KE). Data were collected from January 2017 through Feb 2021 during routine clinical evaluations. Descriptive statistics include: Age, gender, SMA type, copy number, disease duration, current functional status, onset age, handedness and motor function assessments. Linear mixed model regression was applied with a random intercept accounting for multiple visits within participants.

Results

Thirty-five participants had at least one year of follow up; 17 had > 2 years. Thirty of those participants had assessments prior to starting treatment as well as at least one post dose measurement of at least 6 months. Strength ranges discriminated between 3 functional groups: non-sitters, sitters and walkers. Pearson correlation coefficients show moderate correlation for sitters with RULM for most muscle groups. For walkers there was a low correlation with strength and RULM but the HFMSE had a strong correlation with KF and moderate correlation with KE.

Conclusions

Our study shows that strength measures are able to discriminate between functional groups. Nusinersen treatment duration is a significant indicator of strength and may positively impact strength changes in adults with SMA particularly walkers. Based on our results, strength assessments are able to determine strength imbalances and recommended to better understand the impact of treatment on motor function. Future analysis will determine strength changes over time.

Authors/Disclosures
PRESENTER	No disclosure on file
Sally Dunaway, DPT (Stanford University)	Dr. Dunaway has received personal compensation in the range of $5,000-$9,999 for serving as a Consultant for Biogen. Dr. Dunaway has received personal compensation in the range of $5,000-$9,999 for serving as a Consultant for Roche/Genentech. Dr. Dunaway has received personal compensation in the range of $5,000-$9,999 for serving as a Consultant for Scholar Rock. Dr. Dunaway has received personal compensation in the range of $10,000-$49,999 for serving as a Consultant for Cure SMA. The institution of Dr. Dunaway has received research support from Cure SMA. The institution of Dr. Dunaway has received research support from Scholar Rock. The institution of Dr. Dunaway has received research support from Ionis Pharmaceuticals.
	No disclosure on file
Connie Wolford, NP (Stanford Healthcare)	Ms. Wolford has received personal compensation in the range of $5,000-$9,999 for serving on a Speakers Bureau for Biogen.
Zihuai He	Zihuai He has nothing to disclose.
John W. Day, MD, PhD (Stanford University School of Medicine)	Dr. Day has received personal compensation in the range of $5,000-$9,999 for serving on a Scientific Advisory or Data Safety Monitoring board for Novartis Gene Therapy. Dr. Day has received personal compensation in the range of $5,000-$9,999 for serving on a Scientific Advisory or Data Safety Monitoring board for Biogen. Dr. Day has received personal compensation in the range of $500-$4,999 for serving on a Scientific Advisory or Data Safety Monitoring board for Roche/Genentech. Dr. Day has received personal compensation in the range of $500-$4,999 for serving on a Scientific Advisory or Data Safety Monitoring board for Sarepta. Dr. Day has received personal compensation in the range of $500-$4,999 for serving on a Scientific Advisory or Data Safety Monitoring board for Avidity. Dr. Day has received personal compensation in the range of $10,000-$49,999 for serving on a Scientific Advisory or Data Safety Monitoring board for PepGen. Dr. Day has received personal compensation in the range of $10,000-$49,999 for serving on a Scientific Advisory or Data Safety Monitoring board for Epirium Bio. Dr. Day has received personal compensation in the range of $5,000-$9,999 for serving on a Scientific Advisory or Data Safety Monitoring board for Solid Biosciences. Dr. Day has received personal compensation in the range of $10,000-$49,999 for serving on a Scientific Advisory or Data Safety Monitoring board for Muscular Dystrophy Association. The institution of Dr. Day has received research support from Astellas Pharma. The institution of Dr. Day has received research support from Novartis Gene Therapy. The institution of Dr. Day has received research support from Biogen. The institution of Dr. Day has received research support from Roche/Genentech. The institution of Dr. Day has received research support from Sanofi/Genzyme. The institution of Dr. Day has received research support from Sarepta. The institution of Dr. Day has received research support from Scholar Rock. The institution of Dr. Day has received research support from AMO Pharma. The institution of Dr. Day has received research support from AnnJi. Dr. Day has received research support from CureSMA. The institution of Dr. Day has received research support from Muscular Dystrophy Association. The institution of Dr. Day has received research support from Ionis Pharmaceuticals. The institution of Dr. Day has received research support from NMD Pharma. The institution of Dr. Day has received research support from SMA Foundation. Dr. Day has received intellectual property interests from a discovery or technology relating to health care.
Tina Duong	Tina Duong has received personal compensation for serving as an employee of Roche. Tina Duong has received personal compensation for serving as an employee of Novartis. Tina Duong has received personal compensation for serving as an employee of Scholar Rock. Tina Duong has received personal compensation for serving as an employee of Genentech. Tina Duong has received personal compensation for serving as an employee of Novartis. Tina Duong has received personal compensation for serving as an employee of Sarepta. Tina Duong has received personal compensation for serving as an employee of Biogen. Tina Duong has received personal compensation for serving as an employee of Audentes. Tina Duong has received personal compensation in the range of $10,000-$49,999 for serving as a Consultant for Roche. Tina Duong has received personal compensation in the range of $0-$499 for serving as a Consultant for Audentes. Tina Duong has received personal compensation in the range of $500-$4,999 for serving on a Scientific Advisory or Data Safety Monitoring board for Roche. Tina Duong has received personal compensation in the range of $500-$4,999 for serving on a Scientific Advisory or Data Safety Monitoring board for Biogen. Tina Duong has received personal compensation in the range of $500-$4,999 for serving on a Scientific Advisory or Data Safety Monitoring board for Novartis. Tina Duong has received personal compensation in the range of $500-$4,999 for serving on a Speakers Bureau for Roche. Tina Duong has received personal compensation in the range of $500-$4,999 for serving on a Speakers Bureau for Genentech. Tina Duong has received personal compensation in the range of $500-$4,999 for serving on a Speakers Bureau for Sarepta. Tina Duong has received personal compensation in the range of $500-$4,999 for serving on a Speakers Bureau for biogen. The institution of Tina Duong has received research support from biogen. The institution of Tina Duong has received research support from scholar rock.

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