Malak AlMojel1, Samar Ahmed2, Jasem Al-Hashel 3, Raed Alroughani4
1Division of Neurology, Amiri Hospital, Kuwait, 2Department of Neurology, Ibn Sina Hospital, Kuwait, 3Division of Neurology, Ibn Sina Hospital, 4Division of Neurology, Amiri Hospital
Objective:
To evaluate the effectiveness and safety of Ocrelizumab treatment for POMS, relapsing phenotype.
Background:
Pediatric-onset multiple sclerosis (POMS) patients tend to have a higher rate of relapses and progression to secondary progressive multiple sclerosis (SPMS) at earlier age compared to adult-onset MS. Although Ocrelizumab, a recombinant humanized anti-CD20 monoclonal IgG1, approved by FDA and EMA for adult patients with multiple sclerosis (AOMS), limited data in the literature is found in regards to its efficacy and safety in POMS subsets.
Design/Methods:
A retrospective study was conducted on POMS patients, where children with the following criteria: 1) Ocrelizumab treatment initiated at an age younger than 18 years old and 2) patients with a follow-up for at least one year were included. The primary end-point was the proportion of relapse-free patients at the end of observational period. The secondary end-points were the change of annualized relapse rate (ARR), Expanded Disability Status Scale (EDSS) score, and proportion of patients with no MRI activity (new/enlarging T2 lesions and gadolinium (Gd) enhancing lesions).
Results:
Of the 24 POMS patients enrolled in the study, 58.3% were females with a mean age of 16.33 years. Prior to the administration of Ocrelizumab, all patients had clinical and radiological activities, of whom 23 were naïve and one had prior therapy. The median duration of follow-up under Ocrelizumab was 15 months. At the end of cohort, 91.7% of patients were relapse-free, with a decrease in the mean ARR from 1.08 to 0.08; p < 0.001. Nonetheless, only one patient (4.2%) had MRI activity during the treatment course. Mean EDSS was stabilized in 75% of patient and improved in 16.7% patients. NEDA-3 was achieved in 22 (91.7%) subjects. None of the patients had serious side effects. One patient experienced moderate infusion reaction.
Conclusions:
Ocrelizumab can be considered a safe and effective treatment for POMS with RRMS.