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Abstract Details

Quality of Life in Adults with Spinal Muscular Atrophy
Neuromuscular and Clinical Neurophysiology (EMG)
P7 - Poster Session 7 (5:30 PM-6:30 PM)
1-010

1. To characterize factors influencing health-related quality of life (Qol). 2. To track changes in Qol overtime after treatment with disease-modifying therapy in a cohort of adult patients with Spinal Muscular Atrophy (SMA).
There is paucity of data on the assessment of health-related quality-of-life in adults with SMA. Short Form-36 (SF-36) is a commonly used instrument previously validated for use in neuromuscular disorders, including in adult patients with SMA.

As part of an ongoing trial in adults with SMA, we assessed Qol at baseline and at follow-up visits (2, 6, 10, 14 and 18 months) using SF-36. The Physical Component Summary (PCS) and Mental Component Summary (MCS) scores standardized to a US reference population were calculated.
We analyzed data from 32 subjects (17 women, 15 men, mean age 39 ±13; range, 18-65). 13 subjects were ambulatory and 18 were non-ambulatory (SMA FRS scores were 31.2±3.3 and 9.9±10.1).  Compared to the non-ambulatory subjects, the ambulatory subjects have higher PCS (42.4±1.5 versus 35.2±1.4; p<0.001) but lower MCS scores (50.9±2.0 versus 59.6±1.4; p<0.001). Patients with a bodily pain sub score below 75 – indicating pain worse than “very mild” and “a little bit of interference” – had significantly lower average scores on the PCS (36.3±6.4 vs. 42.0±4.8; p 0.009) but not on the MCS (54.6±8.2 vs. 57.0±6.6; p 0.216).

Ambulation ability and pain intensity significantly impact how adult SMA patients view their physical QOL. Interestingly, the presence of pain is not detrimental to mental quality of life. A lower mental QOL in ambulatory patients underscores the need for mental health screening regardless of level of disability.  Data on effect nusinersen treatment on Qol will be presented at the meeting.

Authors/Disclosures
Carlos Enrique A. Bermudez
PRESENTER 		No disclosure on file
Samuel Frank, MD (Providence Mission Heritage Medical Group) Dr. Frank has nothing to disclose.
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.
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.
No disclosure on file
No disclosure on file
No disclosure on file
No disclosure on file
No disclosure on file
Sarah Heintzman, NP (The Ohio State University) Ms. Heintzman has nothing to disclose.
No disclosure on file
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.