Abstract Details

Title Botulinum Toxin Has a Durable Treatment Effect in Stiff Person Syndrome

Topic Autoimmune Neurology

Presentation(s) P1 - Poster Session 1 (8:00 AM-9:00 AM)

Poster/Presentation
Number 14-009

Objective

To compare clinical outcomes after BoNT injections longitudinally among people with SPS.

Background Stiff person syndrome (SPS) is a disabling, immune-mediated disorder causing progressive rigidity and painful spasms. Botulinum toxin (BoNT) anecdotally improves symptoms in people with SPS. Evidence for safety and efficacy of repeated BoNT injections longitudinally is lacking.

Design/Methods We conducted a retrospective cohort study of 22 seropositive SPS patients who received at least 3 BoNT treatments by a single injector (EM) at Johns Hopkins between August 2018 and January 2023, approximately every 3 months. Muscles were selected based on patient-specific phenomenological presentation. Between visits 1 and 3, we compared: muscles injected, units per muscle, patient-reported response (Likert scale 1-5; higher=better response), time to effect, side effects, systemic therapies used, and timed 25-foot walk. These data points will also be collected for patients who have 8 or more visits, when applicable.

Results Twenty-two SPS patients (21 anti-GAD-65, 1 amphiphysin) were included. The majority were female (68.2%), white (68.2%), had classic features (90.1%), with a median age 53.1 years and median Modified Rankin Score of 2. 86.4% were on immune therapies and all were on additional symptomatic treatments at the time of BoNT treatments. Common muscle groups injected included cranio-cervical, upper trunk/shoulders, lower trunk/hips, and distal legs. Supramaximal doses (up to 795 units) were used without adverse effects. Between the first and third injections, the number of patients reporting improvement increased from 64% to 81%, and the mean Likert rating increased from 3.78 to 4.53. Additional analyses will be done to compare outcomes at later visits for patients with 8 or more visits (n=10).

Conclusions BoNT may be an effective adjunctive symptomatic therapy for people with SPS with targeted muscle selection based on specific phenomenology. Moreover, there may be a durable treatment effect with repeat injections over time.

Authors/Disclosures
Samantha Roman, MD (Johns Hopkins Hospital) PRESENTER	Dr. Roman has nothing to disclose.
Elena Taylor	No disclosure on file
Scott D. Newsome, DO, FAAN (Johns Hopkins Hospital)	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.
Emile S. Moukheiber, MD (Johns Hopkins Hospital)	Dr. Moukheiber has nothing to disclose.

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