Newer disease-modifying therapies (DMTs), including dimethyl fumarate, fingolimod, natalizumab, rituximab, and teriflunomide, appear to have similar short-term safety, tolerability, and side effect profiles when used in both children and adults with multiple sclerosis (MS) or clinically isolated syndrome (CIS), according to a study published in Neurology.
Children with MS (n=748) or CIS (n=271) who were followed at a total of 12 outpatient practices in the US Network of Pediatric MS Centers were enrolled. Study researchers analyzed newer DMT use, including dose, duration, safety, tolerability, and side effects. Newer DMT agents, including alemtuzumab, daclizumab, dimethyl fumarate, fingolimod, teriflunomide, natalizumab, and rituximab, were defined as therapies with increased use in adult patients with MS or those approved by the FDA after 2005.
In the overall cohort, the mean age of MS or CIS onset was 13.0 ± 3.9 years, the median visits per year was 1.6 visits, and the mean follow-up period was 3.5 ± 3.1 years. Approximately 78% and 11% of patients with MS and CIS, respectively, received a DMT before 18 years of age. In 42% of patients, ≥1 newer DMT was prescribed. These DMTs included teriflunomide (n=3), daclizumab (n=5), fingolimod (n=37), rituximab (n=57), natalizumab (n=101), and dimethyl fumarate (n=102). Only 17% of the cohort was prescribed a newer DMT, including dimethyl fumarate (n = 36), natalizumab (n = 30), rituximab (n = 22), fingolimod (n = 14), teriflunomide (n = 2).
Children with MS were more likely to receive a newer DMT prior to turning 18 years old compared with patients with CIS (odds ratio [OR] 3.8; 95% CI, 1.3–11.3; P =.017). Additionally, patients with a more recent onset of disease (ie, for 2014-2017 vs 2005 and earlier) were more likely to receive a newer DMT before 18 years of age (OR 2.9; 95% CI, 1.0–8.5; P =.0001). There was no observable difference between pediatric patients and adults in terms of the short-term side effect profiles of the newer DMTs.
Limitations of the study include the lack of long-term safety data for newer DMTs as well as the lack of data on reasoning behind DMT discontinuation in this cohort.
“Development of shared treatment strategies across multiple centers may allow systematic evaluation of the risks and benefits of newer DMTs in children in a real-word setting,” the investigators concluded. “Additional study of long-term side effects and safety of these agents in children during development are needed and will be possible as longer follow-up becomes available.”
Krysko KM, Graves J, Rensel M, et al; for the US Network of Pediatric MS Centers. Use of newer disease-modifying therapies in pediatric multiple sclerosis in the US. Neurology. 2018;91(19):e1778-e1787.