Real-World Effectiveness of Ocrelizumab in a Multi-Centre Pediatric-Onset Multiple Sclerosis (POMS) Cohort in the United Kingdom
Omar Abdel-Mannan1, Arman Eshaghi1, Dimitrios Champsas1, Kshitij Mankad2, Wallace Brownlee1, Thomas Rossor4, Sukhvir Wright6, Evangeline Wassmer7, Cheryl Hemingway3, Ming Lim5, Olga Ciccarelli1, Yael Hacohen1
1Queens Square MS Centre, UCL Queen Square Institute of Neurology, 2Neuroradiology, 3Paediatric Neurology, Great Ormond Street Hospital for Children, 4Paediatric Neurology, Evelina London Children's Hospital, 5Evelina London Children's Hospital, 6Birmingham Children’s Hospital, 7Paediatric Neurology, Birmingham Children’s Hospital
Objective:
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.
Background:
There is a growing consensus favouring earlier use of highly effective disease modifying therapies (DMTs) in POMS.
Design/Methods:
We conducted a prospective study including consecutive POMS (<18 years) from three UK tertiary paediatric neurosciences centers who received ocrelizumab.
Results:
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.
Conclusions:
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.