Patterns of Use and Safety of Newer Disease-modifying therapies in Pediatric Multiple Sclerosis in the US (S51.004)

Objective: We aim to characterize the use and safety of newer disease-modifying therapies (DMT) in patients with multiple sclerosis (MS) and clinically isolated syndrome (CIS) treated before 18 years. Background: Treatment of pediatric MS is challenging as conventional first-line injectable agents may be poorly tolerated and may not adequately control the disease, requiring escalation to more potent newer agents, which lack safety and efficacy data in children. Design/Methods: This is a cohort study including children with a final diagnosis of MS or CIS followed at 12 clinics participating in the US Network of Pediatric MS Centers. Demographic and clinical data were collected, including DMT start and stop dates, indication, side effects, and reason for discontinuation. Results: As of August 2017, 1,023 pediatric patients with MS (n=751) or CIS (n=272) had been entered in the database (65% female, mean onset 13.0+/−3.9 years, mean follow up 3.5+/−3.1 years), with 78.4% with MS and 11.4% with CIS receiving a DMT before 18 years. Patients were seen a median of 1.6 times per year. Of those who received a DMT before 18 years, at least one newer DMT was used in 41.9%, including natalizumab (n=101), dimethyl fumarate (n=100), rituximab (n=57), fingolimod (n=37), daclizumab (n=5), and teriflunomide (n=3). The first agent was a newer DMT in 16.8% (36 dimethyl fumarate, 30 natalizumab, 22 rituximab, 14 fingolimod, 2 teriflunomide). Analyses of changes in treatment patterns over the last 10 years are in progress. The short-term side effect profiles of newer DMTs were not different than reported in adults. Conclusions: Newer DMTs are often used in pediatric MS, and have similar short-term side effect profiles to use in adults. These findings may be helpful in guiding pediatric MS management. Studies of efficacy and long-term side effects are needed. Study Supported by: The National Multiple Sclerosis Society (NMSS). Disclosure: Dr. Krysko has nothing to disclose. Dr. Graves has received personal compensation for consulting, serving on a scientific advisory board, speaking, or other activities with Biogen. Dr. Rensel has received personal compensation for consulting, serving on a scientific advisory board, speaking, or other activities with Dr. Mary Rensel serves as a consultant or speaker for Biogen, Teva, Genzyme and Novartis. . Dr. Rensel has received research support from Medimmune. Dr. Aaen has received research support from Dr. Aaen serves as a site PI for a clinical trial sponsored by Biogen. Dr. Benson has nothing to disclose. Dr. Chitnis has received personal compensation for consulting, serving on a scientific advisory board, speaking, or other activities with Bayer, Biogen, Novartis, Sanofi-Genzyme, Roche-Genentech. Dr. Gorman has received research support from Dr. Gorman has received research funding from Biogen for being a clinical trial site. Dr. Goyal has received personal compensation for consulting, serving on a scientific advisory board, speaking, or other activities with Dr. Goyal has received fees for providing consultations on medicolegal cases related to neuroradiology. None involved MS or the subject matter of this abstract. Dr. Goyal holds stock and/or stock options in IBM Stock. Dr. Harris has nothing to disclose. Dr. Krupp has received personal compensation for consulting, serving on a scientific advisory board, speaking, or other activities with as a speaker, consultant and/or participant on an advisory board from Biogen Idec, Novartis, Teva Neurosciences and Multicell; grant support from the National Multiple Sclerosis Society, National Institutes of Health and the Department of Defense. Dr. Krupp has received research support from research support from Novartis, Biogen Idec, Celgene Corporation and Genentech and support from the Lourie Foundation, Slomo and Cindy Silvian Foundation and the Multiple Sclerosis Foundation. Dr Lotze has nothing to disclose. Dr. Mar has nothing to disclose. Dr. Moodley has nothing to disclose. Dr. Rodriguez has nothing to disclose. Dr. Rose has received research support from Biogen. Dr. Schreiner has received research support from Within the last year, Dr. Schreiner participated in two clinical trials funded by Biogen and one clinical trial by MSDx. Dr. Weinstock-Guttman has received personal compensation for consulting, serving on a scientific advisory board, speaking, or other activities with Bianca Weinstock- Guttman received honoraria as a speaker and as a consultant for Biogen Idec, Teva Pharmaceuticals, EMD Serono, Genzyme&Sanofi, Novartis and Acorda. Dr. Weinstock-Guttman has received research support from Dr Weinstock-Guttman received research funds from Biogen Idec, Teva Pharmaceuticals, EMD Serono, Genzyme&Sanofi, Novartis, Acorda. Dr. Waltz has nothing to disclose. Dr. Casper has nothing to disclose. Dr. Waubant has received personal compensation for consulting, serving on a scientific advisory board, speaking, or other activities with She volunteers on an advisory board for a Novartis trial. She is a site PI for clinical trials with Roche and Novartis. Dr. Waubant has received research support from Received funds from NIH, NMSS, PCORI and Race to Erase MS.