Abstract Details

Title A Retrospective Study on Functional Outcome Measures in Patients with Stiff-Person Syndrome

Topic Autoimmune Neurology

Presentation(s) P2 - Poster Session 2 (8:00 AM-9:00 AM)

Poster/Presentation
Number 8-002

Objective The objective of this study is to retrospectively analyze change in the timed 25-feet walk (T25FW) and modified Rankin Scale (mRS) for established SPSD patients.

Background Stiff Person Syndrome Spectrum Disorder (SPSD) is a rare autoimmune disorder characterized by progressive muscle stiffness and painful spasms.

Design/Methods We queried Health Data Compass (HDC), from 2012 through 2022 for all patients ≥ 18-year-old with ICD-10 codes pertaining to SPSD in a recent epidemiology to define incidence and prevalence of SPSD (in press). 273 records were reviewed for diagnostic confirmation using the proposed Mayo Clinic and Johns Hopkins clinical criteria. Only antibody-positive patients were included in this retrospective outcome study. All statistical analyses were conducted in R statistical computing software. We grouped all the patients and the measurements together within each year. Linear regression and Mixed Model for Repeated Measures (MMRM) were used to analyze the T25FW change from baseline. MMRM models patient-specific random effects to account for intra-patient variability.

Results 32 patients met inclusion criteria. 18 patients had T25FW data with 69 measurements, and 27 patients had retrospective mRS with 368 measurements. Average baseline T25FW was 9.4 ± 4.0 seconds. The change from baseline in the T25FW in the first year was -1.7 seconds, followed by a gradual increase with a positive change of 2.5 seconds at year 6, modeled by linear regression and MMRM. Average baseline mRS was 2.7 ± 1.3, with no apparent change over an average observation period of 3.3 ± 2.7 year (median = 0).

Conclusions

This retrospective analysis using the T25FW shows that SPSD is a chronic and progressive disease, even under active treatment. T25FW has value in understanding disease severity and progression and may be useful to monitor therapy response. mRS is insensitive to disease progression in mild-moderate disability SPSD patients.

Authors/Disclosures
Mallory Lowe, MD PRESENTER	Dr. Lowe has received research support from Kyverna Therapeutics.
Kelli Money, MD, PhD	Dr. Money has nothing to disclose.
Tyler L. Borko, BA (University of Colorado)	Mr. Borko has nothing to disclose.
Paul D. Crane, MD (University of Colorado)	Dr. Crane has nothing to disclose.
Kunbin Qu, PhD	Dr. Qu has received personal compensation for serving as an employee of Kyverna Therapeutics. Dr. Qu has stock in Kyverna.
Stefan Sillau	Stefan Sillau has nothing to disclose.
Eric Engebretson (University of Colorado Denver)	Mr. Engebretson has nothing to disclose.
Sham Dholakia, MD, PhD	No disclosure on file
James Chung, MD, PhD	Dr. Chung has received personal compensation for serving as an employee of Kyverna Therapeutics. Dr. Chung has stock in Kyverna Therapeutics.
Esther H. Nie, PhD (Stanford Healthcare Center for Academic Medicine (CAM))	Dr. Nie has received personal compensation for serving as an employee of Kyverna Therapeutics. Dr. Nie has received personal compensation in the range of $5,000-$9,999 for serving as a Consultant for Medidata, Acorn AI. The institution of Dr. Nie has received research support from Roche/Genentech.
Kavita Nair, PhD, FAAN (University of Colorado)	Dr. Nair has received personal compensation in the range of $5,000-$9,999 for serving as a Consultant for Bristol Meyers Squibb. Dr. Nair has received personal compensation in the range of $500-$4,999 for serving as a Consultant for Novartis. Dr. Nair has received personal compensation in the range of $10,000-$49,999 for serving as a Consultant for PhRMA Foundation. Dr. Nair has received personal compensation in the range of $10,000-$49,999 for serving as a Consultant for Genentech. Dr. Nair has received personal compensation in the range of $500-$4,999 for serving as a Consultant for TG Therapeutics. Dr. Nair has received personal compensation in the range of $5,000-$9,999 for serving as a Consultant for MJH Lifesciences. Dr. Nair has received personal compensation in the range of $5,000-$9,999 for serving as a Consultant for Sanofi. Dr. Nair has received personal compensation in the range of $500-$4,999 for serving as a Consultant for EMD Serono. Dr. Nair has received personal compensation in the range of $500-$4,999 for serving as a Consultant for Amgen. Dr. Nair has received personal compensation in the range of $500-$4,999 for serving as a Consultant for Anylam . Dr. Nair has received personal compensation in the range of $500-$4,999 for serving as a Consultant for Applied Patient Experience. Dr. Nair has received personal compensation in the range of $500-$4,999 for serving as a Consultant for Esai. Dr. Nair has received personal compensation in the range of $5,000-$9,999 for serving as a Consultant for MHJ Life Sciences. Dr. Nair has received personal compensation in the range of $10,000-$49,999 for serving on a Speakers Bureau for Sanofi-Genzyme. Dr. Nair has received personal compensation in the range of $500-$4,999 for serving on a Speakers Bureau for Amgen. Dr. Nair has received personal compensation in the range of $500-$4,999 for serving as an officer or member of the Board of Directors for ICER. The institution of Dr. Nair has received research support from Genentech. The institution of Dr. Nair has received research support from Novartis. The institution of Dr. Nair has received research support from Genentech. The institution of Dr. Nair has received research support from Phrma Foundation. The institution of Dr. Nair has received research support from Bristol Meyers Squibb. The institution of Dr. Nair has received research support from Novartis. The institution of Dr. Nair has received research support from NINDS. The institution of Dr. Nair has received research support from Sanofi Genzyme. The institution of Dr. Nair has received research support from Kyverna Therapeutcis. Dr. Nair has a non-compensated relationship as a Board Member with Center for Improving Value in Healthcare that is relevant to AAN interests or activities.
Aaron M. Carlson, MD (University of Colorado, School of Medicine, Department of Neurology)	Dr. Carlson has received research support from Horizon Therapeutics (Amgen).
Amanda L. Piquet, MD, FAAN (University of Colorado)	The institution of Dr. Piquet has received personal compensation in the range of $10,000-$49,999 for serving as a Consultant for Genentech/Roche. The institution of Dr. Piquet has received personal compensation in the range of $5,000-$9,999 for serving as a Consultant for Alexion. The institution of Dr. Piquet has received personal compensation in the range of $5,000-$9,999 for serving as a Consultant for Kyverna . The institution of Dr. Piquet 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/Roche. The institution of Dr. Piquet has received personal compensation in the range of $5,000-$9,999 for serving on a Scientific Advisory or Data Safety Monitoring board for Kyverna. The institution of Dr. Piquet has received personal compensation in the range of $10,000-$49,999 for serving on a Scientific Advisory or Data Safety Monitoring board for Alexion. Dr. Piquet has received personal compensation in the range of $10,000-$49,999 for serving as an Expert Witness for Sands Anderson PC. Dr. Piquet has received personal compensation in the range of $5,000-$9,999 for serving as an Expert Witness for Joe Jones Law Firm. Dr. Piquet has received personal compensation in the range of $500-$4,999 for serving as an Expert Witness for Cortez & Associates. Dr. Piquet has received personal compensation in the range of $500-$4,999 for serving as an Expert Witness for Falk Waas. The institution of Dr. Piquet has received research support from Rocky Mountain MS Center. The institution of Dr. Piquet has received research support from Roche/Genentech. The institution of Dr. Piquet has received research support from NYU. The institution of Dr. Piquet has received research support from Anokion. The institution of Dr. Piquet has received research support from UCB . The institution of Dr. Piquet has received research support from Foundation for Sarcoidosis. The institution of Dr. Piquet has received research support from Kyverna . Dr. Piquet has received publishing royalties from a publication relating to health care. Dr. Piquet has received publishing royalties from a publication relating to health care. Dr. Piquet has received personal compensation in the range of $10,000-$49,999 for serving as a Litigative Consultant with US-Dept HHS/DICP. Dr. Piquet has a non-compensated relationship as a Medical Advisory Board Member with Autoimmune Encephalitis Alliance (AEA) that is relevant to AAN interests or activities. Dr. Piquet has a non-compensated relationship as a Medical Advisory Board Member with Stiff Person Syndrome Research Foundation (SPSRF) that is relevant to AAN interests or activities.

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