RT Journal Article
SR Electronic
T1 Use of newer disease-modifying therapies in pediatric multiple sclerosis in the US
JF Neurology
JO Neurology
FD Lippincott Williams & Wilkins
SP e1778
OP e1787
DO 10.1212/WNL.0000000000006471
VO 91
IS 19
A1 Krysko, Kristen M.
A1 Graves, Jennifer
A1 Rensel, Mary
A1 Weinstock-Guttman, Bianca
A1 Aaen, Gregory
A1 Benson, Leslie
A1 Chitnis, Tanuja
A1 Gorman, Mark
A1 Goyal, Manu
A1 Krupp, Lauren
A1 Lotze, Timothy
A1 Mar, Soe
A1 Rodriguez, Moses
A1 Rose, John
A1 Waltz, Michael
A1 Charles Casper, T.
A1 Waubant, Emmanuelle
YR 2018
UL http://n.neurology.org/content/91/19/e1778.abstract
AB Objective To characterize the use and safety of newer disease-modifying therapies (DMTs) in children with multiple sclerosis (MS) and clinically isolated syndrome (CIS) treated under 18 years of age.Methods This is a cohort study including children with MS or CIS followed at 12 outpatient practices participating in the US Network of Pediatric MS Centers. DMT use, including duration, dose, and side effects, was analyzed. Newer DMTs were defined as agents receiving Food and Drug Administration approval or with increased use in adult MS after 2005.Results As of July 2017, 1,019 pediatric patients with MS (n = 748) or CIS (n = 271) were enrolled (65% female, mean onset 13.0 ± 3.9 years, mean follow-up 3.5 ± 3.1 years, median 1.6 visits per year). Of these, 78% (n = 587) with MS and 11% (n = 31) with CIS received DMT before 18 years of age. This consisted of at least one newer DMT in 42%, including dimethyl fumarate (n = 102), natalizumab (n = 101), rituximab (n = 57), fingolimod (n = 37), daclizumab (n = 5), and teriflunomide (n = 3). Among 17%, the initial DMT prescribed was a newer agent (36 dimethyl fumarate, 30 natalizumab, 22 rituximab, 14 fingolimod, 2 teriflunomide). Over the last 10 years, the use of newer agents has increased, particularly in those ≥12 years and to lesser extent in those <12 years. The short-term side effect profiles of newer DMTs did not differ from those reported in adults.Conclusion Newer DMTs are often used in pediatric MS, and have similar short-term safety, tolerability, and side effect profiles as in adults. These findings may help inform pediatric MS management.CI=confidence interval; CIS=clinically isolated syndrome; DMT=disease-modifying therapy; EDSS=Expanded Disability Status Scale; FDA=Food and Drug Administration; JCV=JC virus; MS=multiple sclerosis; OR=odds ratio