Kristen M Krysko1, Jennifer Graves2, Mary Rensel2, Bianca Weinstock-Guttman2, Gregory Aaen2, Leslie Benson2, Tanuja Chitnis2, Mark Gorman2, Manu Goyal2, Lauren Krupp2, Timothy Lotze2, Soe Mar2, Moses Rodriguez2, John Rose2, Michael Waltz2, T Charles Casper2, Emmanuelle Waubant2. 1. From the Department of Neurology (K.M.K., J.G., E.W.), University of California San Francisco; Department of Neurology (M. Rensel), Cleveland Clinic, OH; Department of Neurology (B.W.-G.), State University of New York at Buffalo; Department of Pediatrics (G.A.), Loma Linda University, CA; Department of Neurology (L.B., M. Gorman), Boston Children's Hospital, MA; Department of Neurology (T.C.), Massachusetts General Hospital, Boston; Department of Neurology (M. Goyal, S.M.), Washington University in Saint Louis, MO; Department of Neurology (L.K.), New York University Langone Medical Center, NY; Department of Neurology (T.L.), Texas Children's Hospital, Houston; Department of Neurology (M. Rodriguez), Mayo Clinic, Rochester, MN; and Department of Neurology (J.R.), Biostatistician II (M.W.), and Department of Pediatrics (T.C.C.), University of Utah, Salt Lake City. Kristen.krysko@ucsf.edu. 2. From the Department of Neurology (K.M.K., J.G., E.W.), University of California San Francisco; Department of Neurology (M. Rensel), Cleveland Clinic, OH; Department of Neurology (B.W.-G.), State University of New York at Buffalo; Department of Pediatrics (G.A.), Loma Linda University, CA; Department of Neurology (L.B., M. Gorman), Boston Children's Hospital, MA; Department of Neurology (T.C.), Massachusetts General Hospital, Boston; Department of Neurology (M. Goyal, S.M.), Washington University in Saint Louis, MO; Department of Neurology (L.K.), New York University Langone Medical Center, NY; Department of Neurology (T.L.), Texas Children's Hospital, Houston; Department of Neurology (M. Rodriguez), Mayo Clinic, Rochester, MN; and Department of Neurology (J.R.), Biostatistician II (M.W.), and Department of Pediatrics (T.C.C.), University of Utah, Salt Lake City.
Abstract
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.
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.
Authors: L A Benson; B C Healy; M P Gorman; N F Baruch; T Gholipour; A Musallam; T Chitnis Journal: Mult Scler Relat Disord Date: 2013-07-12 Impact factor: 4.339
Authors: A Ghezzi; M P Amato; M Capobianco; P Gallo; G Marrosu; V Martinelli; N Milani; C Milanese; L Moiola; F Patti; V Pilato; C Pozzilli; M Trojano; M Zaffaroni; G Comi Journal: Mult Scler Date: 2005-08 Impact factor: 6.312
Authors: Stephen L Hauser; Emmanuelle Waubant; Douglas L Arnold; Timothy Vollmer; Jack Antel; Robert J Fox; Amit Bar-Or; Michael Panzara; Neena Sarkar; Sunil Agarwal; Annette Langer-Gould; Craig H Smith Journal: N Engl J Med Date: 2008-02-14 Impact factor: 91.245
Authors: Angelo Ghezzi; Maria Pia Amato; Pietro Annovazzi; Marco Capobianco; Paolo Gallo; Loredana La Mantia; Maria Giovanna Marrosu; Vittorio Martinelli; Nicoletta Milani; Lucia Moiola; Francesco Patti; Carlo Pozzilli; Maria Trojano; Mauro Zaffaroni; Giancarlo Comi Journal: Neurol Sci Date: 2009-04-22 Impact factor: 3.307
Authors: Jonathan D Santoro; Michael Waltz; Greg Aaen; Anita Belman; Leslie Benson; Mark Gorman; Manu S Goyal; Jennifer S Graves; Yolanda Harris; Lauren Krupp; Timothy Lotze; Soe Mar; Manikum Moodley; Jayne Ness; Mary Rensel; Moses Rodriguez; Teri Schreiner; Jan-Mendelt Tillema; Emmanuelle Waubant; Bianca Weinstock-Guttman; Brigitte F Hurtubise; Shelly Roalstad; John Rose; T Charles Casper; Tanuja Chitnis Journal: Neurology Date: 2020-07-20 Impact factor: 9.910