Rituximab is Associated with Limiting Progression in Individuals with Stiff Person Syndrome Spectrum Disorders (S40.002)

Abstract

<h3>Objective:</h3> To assess if rituximab minimizes disease progression in people with Stiff Person Syndrome Spectrum Disorders (SPSD). <h3>Background:</h3> Rituximab has been used to treat many autoimmune conditions including SPSD. However, there is a lack of long-term data assessing rituximab in a large cohort of people with SPSD. We previously analyzed the effects of rituximab in a small group of people with SPSD and focused on improvement of function. The purpose of this study was to evaluate the effects of rituximab in a larger cohort of patients over a longer duration of time and assess how this therapy minimizes disease progression. <h3>Design/Methods:</h3> Demographic and clinical data from 235 SPSD patients evaluated at the Johns Hopkins SPS Center were obtained as part of an ongoing longitudinal observational study. Data analysis was performed in patients who met the following inclusion criteria: confirmed diagnosis of SPSD, exposure to rituximab therapy, and &gt;1 follow-up visit with adequate clinical information for outcome measure comparisons. The main outcomes were change in relevant symptoms, objective findings, and global score (combined subjective and objective data). <h3>Results:</h3> Of 69 SPSD patients with rituximab exposure (81% female, age M±SD=49y±13.6y), data analysis was performed in 63 patients who met the inclusion criteria. The median (IQR) years between symptom onset and rituximab exposure was 9.17y (4.46y, 13.26y). At baseline, 97% self-reported gait difficulties, 88% had objective gait abnormalities, and mean (SD) modified Rankin score was 3.0 (0.83). The median and maximum follow-up times were 1.1y and 9.4y, respectively. Based on global scores over the follow-up period, 37 (58.7%) improved, 21 (33.3%) stabilized, and 5 (7.9%) worsened after rituximab exposure. <h3>Conclusions:</h3> The findings of this study suggest that many patients with SPSD may experience plateauing of worsening disability with chronic rituximab treatment. Specific factors associated with treatment response remain unclear and warrant further studies. <b>Disclosure:</b> The institution of Dr. Harrison has received research support from National MS Society. Miss Hu has nothing to disclose. Dr. Aljarallah has received personal compensation in the range of $0-$499 for serving on a Scientific Advisory or Data Safety Monitoring board for Novartis. Dr. Aljarallah has received personal compensation in the range of $0-$499 for serving on a Scientific Advisory or Data Safety Monitoring board for Biogen. Dr. Aljarallah has received personal compensation in the range of $0-$499 for serving on a Speakers Bureau for Sanofi Genzyme. The institution of Dr. Wang has received research support from Genentech. The institution of Dr. Fitzgerald has received research support from NIH. The institution of Dr. Fitzgerald has received research support from National MS Society. Dr. Newsome has received personal compensation in the range of $500-$4,999 for serving as a Consultant for Autobahn. 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 Biogen. 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 EMD Serono. 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 Greenwich Biosciences. 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 Bristol Myers Squibb. 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 Horizon 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 The Stiff Person Syndrome Research Foundation. Dr. Newsome has received personal compensation in the range of $5,000-$9,999 for serving as a Clinical adjudication committee member for clinical trial with medDay Pharmaceuticals. 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.