Real-World Effectiveness of Ocrelizumab in a Multi-Centre Pediatric-Onset Multiple Sclerosis (POMS) Cohort in the United Kingdom (S31.007)

Abstract

<h3>Objective:</h3> The aim of this study was to evaluate the efficacy and safety of ocrelizumab treatment for pediatric-onset Multiple Sclerosis (POMS) in a real-world clinical setting. <h3>Background:</h3> There is a growing consensus favouring earlier use of highly effective disease modifying therapies (DMTs) in POMS. <h3>Design/Methods:</h3> We conducted a prospective study including consecutive POMS (&lt;18 years) from three UK tertiary paediatric neurosciences centers who received ocrelizumab. <h3>Results:</h3> We included a total of 60 POMS patients; 49 female (81.7%), 41 non-white (68.3%), median age 14.6 yrs (IQR 13.3, 15.5). Median follow-up period was 1.0 yr (range, 0.1–2.6). Forty-three patients (71.7%) had ocrelizumab as their first DMT. The median number of relapses per patient pre-treatment was 2 (range 1–5). Two patients relapsed within one month of starting ocrelizumab and 1 patient had an optic neuritis 6-months post ocrelizumab initiation. During the follow-up period, a median of 2 (range 1–5) repeated MRI scans were performed (total scans, n=140). Forty out of 43 (93.0%) patients achieved no evidence of disease activity (NEDA-3) at 12-months follow-up; two patients had one new brain lesion each at 6-months and a different patient had a relapse (optic neuritis) without new brain lesions at 6-months post treatment. Median EDSS score remained stable during follow-up; 1.0 at baseline and follow-up (p=0.21). In addition, there was no change in cognitive measures at follow-up; baseline SDMT mean score 46 vs follow-up SDMT mean score 48 (p=0.39). The most common adverse events reported were infusion-related-reactions (33/60, 55%), all of which were grade 1 or 2. Serious adverse events were recorded in one patient with enterovirus meningitis, who made a full recovery. <h3>Conclusions:</h3> This study confirms that ocrelizumab, which is proven efficacious for adults with MS, is also effective in POMS with a comparable safety profile. Assessment for long-term efficacy and safety is ongoing. <b>Disclosure:</b> Dr. Abdel-Mannan has nothing to disclose. Arman Eshaghi has nothing to disclose. Dr. Champsas has nothing to disclose. Kshitij Mankad has nothing to disclose. Dr. Brownlee has received personal compensation in the range of $500-$4,999 for serving as a Consultant for Roche. Dr. Brownlee has received personal compensation in the range of $500-$4,999 for serving as a Consultant for Biogen. Dr. Brownlee has received personal compensation in the range of $500-$4,999 for serving as a Consultant for EMD Serono. Dr. Brownlee has received personal compensation in the range of $500-$4,999 for serving as a Consultant for Sanofi. Dr. Brownlee has received personal compensation in the range of $500-$4,999 for serving as a Consultant for Novartis. Dr. Brownlee has received personal compensation in the range of $500-$4,999 for serving as a Consultant for Janssen. Dr. Brownlee has received personal compensation in the range of $500-$4,999 for serving as an Editor, Associate Editor, or Editorial Advisory Board Member for Sage. Dr. Rossor has nothing to disclose. Sukhvir Wright has nothing to disclose. Dr. Wassmer has received personal compensation in the range of $500-$4,999 for serving on a Speakers Bureau for Biogen. The institution of Dr. Wassmer has received research support from Birmingham Children’s Hospital Charity Research Foundation. The institution of Cheryl Hemingway has received personal compensation in the range of $0-$499 for serving as a Consultant for Biogen. The institution of Cheryl Hemingway has received personal compensation in the range of $0-$499 for serving as a Consultant for Novartis. The institution of Cheryl Hemingway has received personal compensation in the range of $500-$4,999 for serving on a Scientific Advisory or Data Safety Monitoring board for UCB. The institution of Cheryl Hemingway has received personal compensation in the range of $500-$4,999 for serving on a Speakers Bureau for Sanofi. The institution of Cheryl Hemingway has received research support from Medical research Committee. Ming Lim has nothing to disclose. Prof. Ciccarelli has received personal compensation in the range of $500-$4,999 for serving on a Scientific Advisory or Data Safety Monitoring board for Novartis. Prof. Ciccarelli has received personal compensation in the range of $0-$499 for serving on a Speakers Bureau for Merck. Prof. Ciccarelli has received personal compensation in the range of $10,000-$49,999 for serving as an Editor, Associate Editor, or Editorial Advisory Board Member for NEUROLOGY Journal. Dr. Hacohen has nothing to disclose.