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Abstract Details

A Multicenter, Real-world Natural History Study on Timed 25-foot Walk Outcome Measures in Stiff Person Syndrome
Autoimmune Neurology
P4 - Poster Session 4 (8:00 AM-9:00 AM)
2-003
To evaluate longitudinal changes in timed-25-foot walk (T25FW) among patients with stiff person syndrome (SPS).
SPS is a rare autoimmune neurologic disease characterized by muscle stiffness and spasms, with progressive ambulatory dysfunction. Currently, there are no FDA-approved treatments for SPS, and a large subset of patients have an inadequate response to the use of off-label therapies, such as intravenous immunoglobulin (IVIg), as the disease progresses. T25FW is a validated tool to assess walking ability in other neurologic disorders and has been used to capture functional gait impairment in SPS. Additionally, T25FW is the primary endpoint of KYSA-8, the ongoing pivotal trial of KYV-101 CD19 CAR T-cell therapy in SPS. Herein, we present a large, multicenter, observational study to assess T25FW in patients with SPS.
Demographics, clinical characteristics, and relevant outcomes assessments were obtained for patients with a primary diagnosis of SPS from the University of Colorado and Johns Hopkins between 2012-2025.
The cohort included 183 patients with SPS. The median age at symptom onset was 44 years (range, 14-79), and the majority were white (75%) and female (68%). Classic SPS was the most common phenotype (71%); 29% had SPS-plus defined by classic features with cerebellar and/or brainstem involvement. GAD65, glycine receptor, and amphiphysin autoantibody positivity were detected in 66%, 9%, and 2% of patients, respectively. A full analysis of demographics, disease characteristics, treatment history, use of ambulatory devices, modified Rankin scale, and T25FW will be presented.
This study represents the largest real-world natural history study to date examining the impact of SPS on walking speed. T25FW appears to be a clinically meaningful outcome measure in SPS, capturing key aspects of functional impairment. Ambulatory outcome measures may support the development of new therapies, including KYV-101, addressing a critical unmet need for patients with SPS.
Authors/Disclosures
Scott D. Newsome, DO, FAAN (Johns Hopkins Hospital)
PRESENTER 		Dr. Newsome has received personal compensation in the range of $10,000-$49,999 for serving on a Scientific Advisory or Data Safety Monitoring board for Genentech. Dr. Newsome has received personal compensation in the range of $500-$4,999 for serving on a Scientific Advisory or Data Safety Monitoring board for Novartis. Dr. Newsome has received personal compensation in the range of $500-$4,999 for serving on a Scientific Advisory or Data Safety Monitoring board for TG Therapeutics. The institution of Dr. Newsome has received research support from Biogen. The institution of Dr. Newsome has received research support from Genentech/Roche. The institution of Dr. Newsome has received research support from Department of Defense. The institution of Dr. Newsome has received research support from Patient Centered Outcomes Research Institute. The institution of Dr. Newsome has received research support from National MS Society. The institution of Dr. Newsome has received research support from Lundbeck. The institution of Dr. Newsome has received research support from Sanofi. The institution of Dr. Newsome has received research support from Kyverna Therapeutics. Dr. Newsome has received personal compensation in the range of $10,000-$49,999 for serving as a Lead PI for Clinical Trial with Roche.
Mallory Lowe, MD Dr. Lowe has received research support from Kyverna Therapeutics.
Hanyeh Afshar Ms. Afshar has nothing to disclose.
Kelli Money, MD, PhD Dr. Money has nothing to disclose.
Asli Buyukkurt, MD Dr. Buyukkurt has nothing to disclose.
Herbert Chen Herbert Chen has nothing to disclose.
Barrett Crawford Mr. Crawford has nothing to disclose.
Sadie Eggmann Sadie Eggmann has nothing to disclose.
Aisha Elfasi, MD Dr. Elfasi has nothing to disclose.
Angeliki Filippatou, MD (Johns Hopkins University School of Medicine) Dr. Filippatou has nothing to disclose.
Munther Queisi, MD Dr. Queisi has nothing to disclose.
Daniela Riveros Acosta, MD Dr. Riveros Acosta has nothing to disclose.
Ruth Andrea Salazar Camelo, MD (Johns Hopkins School of Medicine) Dr. Salazar Camelo has nothing to disclose.
Jadyn Zook, PRA Ms. Zook has nothing to disclose.
Norma Guzman-Becerra, MSc Mrs. Guzman-Becerra has nothing to disclose.
Jennifer Leslie Dr. Leslie has received personal compensation for serving as an employee of Kyverna Therapeutics. Dr. Leslie has stock in Kyverna Therapeutics.
Skye Geherin Skye Geherin has received personal compensation for serving as an employee of Kyverna Therapeutics. Skye Geherin has received personal compensation for serving as an employee of Ascendis Pharma.
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.