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Abstract Details

Real-world Effectiveness of Initial Treatment with Newer versus Injectable Disease-modifying Therapies in Pediatric Multiple Sclerosis
Multiple Sclerosis
S29 - Multiple Sclerosis: Disease-modifying Therapy (1:36 PM-1:48 PM)
004
We assessed real-world effectiveness of initial treatment with newer compared to injectable disease-modifying therapies (DMT) on disease activity in pediatric multiple sclerosis (MS) and clinically isolated syndrome (CIS).
Treatment of pediatric MS is challenging as most DMTs lack randomized controlled trial data in children.

This is a cohort study of children with MS/CIS followed at 12 clinics in the US Network of Pediatric MS Centers, who received initial therapy with newer (fingolimod, dimethyl fumarate, teriflunomide, natalizumab, rituximab, ocrelizumab) or injectable (interferon-beta or glatiramer acetate) DMTs. Propensity scores (PS) were computed with logistic regression to predict newer DMT use, including pre-identified confounders. Relapse rate while on initial DMT was modeled with negative binomial regression, adjusted for PS-quintile. Time to new/enlarging T2 hyperintense and gadolinium-enhancing lesions on MRI brain were modeled with midpoint survival analyses, adjusted for PS-quintile.
741 children (66% female, 15% CIS) began therapy before 18 years, 197 with newer and 544 with injectable DMTs. Those started on newer DMTs were older (15.2 vs. injectable 14.4 years, p=0.001) and less likely to have a monofocal presentation (37% vs. injectable 55%, p<0.001). In PS-quintile adjusted analysis, those started on newer DMTs had lower relapse rate than those on injectables (rate ratio 0.45, 95% CI 0.29-0.70, p<0.001; rate difference 0.27, 95% CI 0.14-0.40, p=0.004). One would need to treat with newer over injectable DMTs for 3.7 person-years to prevent one relapse. Those started on newer DMTs also had lower rate of new/enlarging T2 (HR 0.51, 95% CI 0.36-0.72, p<0.001) and gadolinium-enhancing lesions (HR 0.38, 95% CI 0.23-0.63, p<0.001) than those on injectables.
Initial treatment of pediatric MS/CIS with newer DMTs led to better disease activity control compared to injectables, supporting greater effectiveness of newer therapies. Long-term safety data for newer DMTs is required.
Authors/Disclosures
Kristen M. Krysko, MD (St. Michael's Hospital)
PRESENTER 		Dr. Krysko has received personal compensation in the range of $5,000-$9,999 for serving as a Consultant for Biogen. Dr. Krysko has received personal compensation in the range of $500-$4,999 for serving as a Consultant for Roche. Dr. Krysko has received personal compensation in the range of $5,000-$9,999 for serving as a Consultant for EMD Serono. Dr. Krysko has received personal compensation in the range of $10,000-$49,999 for serving as a Consultant for Novartis. Dr. Krysko has received personal compensation in the range of $500-$4,999 for serving on a Scientific Advisory or Data Safety Monitoring board for Novartis. Dr. Krysko has received personal compensation in the range of $500-$4,999 for serving on a Scientific Advisory or Data Safety Monitoring board for EMD Serono. The institution of Dr. Krysko has received research support from MS Society of Canda. The institution of Dr. Krysko has received research support from Roche.
Jennifer Graves, MD, PhD (UCSD) Dr. Graves has received personal compensation in the range of $500-$4,999 for serving on a Scientific Advisory or Data Safety Monitoring board for TG Therapeutics. Dr. Graves has received personal compensation in the range of $500-$4,999 for serving on a Scientific Advisory or Data Safety Monitoring board for Horizon. Dr. Graves has received personal compensation in the range of $5,000-$9,999 for serving as an Editor, Associate Editor, or Editorial Advisory Board Member for MSJ. The institution of Dr. Graves has received research support from Octave. The institution of Dr. Graves has received research support from Sanofi. The institution of Dr. Graves has received research support from EMD Serono.
Mary R. Rensel, MD, FAAN Dr. Rensel has received personal compensation in the range of $5,000-$9,999 for serving as a Consultant for Biogen. Dr. Rensel has received personal compensation in the range of $500-$4,999 for serving as a Consultant for Cycle Pharma. Dr. Rensel has received personal compensation in the range of $5,000-$9,999 for serving as a Consultant for genentech. Dr. Rensel has received personal compensation in the range of $5,000-$9,999 for serving as a Consultant for Novartis . Dr. Rensel has received personal compensation in the range of $10,000-$49,999 for serving as a Consultant for EMD Serono. Dr. Rensel has received personal compensation in the range of $5,000-$9,999 for serving on a Scientific Advisory or Data Safety Monitoring board for biogen. Dr. Rensel has received personal compensation in the range of $5,000-$9,999 for serving on a Speakers Bureau for Genentech. Dr. Rensel has received personal compensation in the range of $5,000-$9,999 for serving on a Speakers Bureau for TG. Dr. Rensel has received personal compensation in the range of $10,000-$49,999 for serving on a Speakers Bureau for Sanofi. Dr. Rensel has received personal compensation in the range of $10,000-$49,999 for serving on a Speakers Bureau for EMD Serono. Dr. Rensel has received personal compensation in the range of $10,000-$49,999 for serving on a Speakers Bureau for TG. Dr. Rensel has stock in Brain Fresh LLC . The institution of Dr. Rensel has received research support from NMSS. The institution of Dr. Rensel has received research support from Roche- Genentech .
Bianca Weinstock-Guttman, MD (Department of Neurology, University At Buffalo) Dr. Weinstock-Guttman has received personal compensation in the range of $10,000-$49,999 for serving as a Consultant for Biogen. Dr. Weinstock-Guttman has received personal compensation in the range of $5,000-$9,999 for serving as a Consultant for Genentech. Dr. Weinstock-Guttman has received personal compensation in the range of $5,000-$9,999 for serving as a Consultant for Novartis . Dr. Weinstock-Guttman has received personal compensation in the range of $500-$4,999 for serving as a Consultant for EMD Serono. Dr. Weinstock-Guttman has received personal compensation in the range of $500-$4,999 for serving as a Consultant for Abbvie. Dr. Weinstock-Guttman has received personal compensation in the range of $500-$4,999 for serving as a Consultant for Genzyme &Sanofi. Dr. Weinstock-Guttman has received personal compensation in the range of $500-$4,999 for serving as a Consultant for Janssen . Dr. Weinstock-Guttman has received personal compensation in the range of $500-$4,999 for serving as a Consultant for Bayer. Dr. Weinstock-Guttman has received personal compensation in the range of $500-$4,999 for serving as a Consultant for Horizon. Dr. Weinstock-Guttman has received personal compensation in the range of $500-$4,999 for serving on a Scientific Advisory or Data Safety Monitoring board for Genentech. Dr. Weinstock-Guttman has received personal compensation in the range of $0-$499 for serving on a Scientific Advisory or Data Safety Monitoring board for Novartis. Dr. Weinstock-Guttman has received personal compensation in the range of $5,000-$9,999 for serving on a Speakers Bureau for Biogen. Dr. Weinstock-Guttman has received personal compensation in the range of $500-$4,999 for serving on a Speakers Bureau for Janssen. Dr. Weinstock-Guttman has received personal compensation in the range of $0-$499 for serving as a Reviewer with NIH.
Alice Edwards, DO (Neurology Specialists) The institution of Dr. Rutatangwa has received research support from NIH.
Gregory S. Aaen, MD (Loma Linda University School of Medicine) Dr. Aaen has nothing to disclose.
Anita L. Belman, MD, FAAN No disclosure on file
Leslie A. Benson, MD (Children's Hospital Boston) The institution of Dr. Benson has received research support from ROHHAD Fight, Inc. The institution of Dr. Benson has received research support from Shore Foundation. The institution of Dr. Benson has received research support from Alexion Pharmacuticals. The institution of Dr. Benson has received research support from Biogen, Inc. The institution of Dr. Benson has received research support from NIH. Dr. Benson has received personal compensation in the range of $0-$499 for serving as a consultant with MA Department of public health. Dr. Benson has received personal compensation in the range of $5,000-$9,999 for serving as a consultant with National vaccine injury compensation program.
Tanuja Chitnis, MD, FAAN (Brigham and Women's Hospital) Dr. Chitnis has received personal compensation in the range of $10,000-$49,999 for serving as a Consultant for Novartis. Dr. Chitnis has received personal compensation in the range of $5,000-$9,999 for serving as a Consultant for Roche-Genentech. Dr. Chitnis has received personal compensation in the range of $10,000-$49,999 for serving as a Consultant for Siemens. Dr. Chitnis has received personal compensation in the range of $500-$4,999 for serving as a Consultant for Octave Biosciences. Dr. Chitnis has received personal compensation in the range of $500-$4,999 for serving as a Consultant for Sanofi. The institution of Dr. Chitnis has received personal compensation in the range of $500-$4,999 for serving on a Scientific Advisory or Data Safety Monitoring board for Novartis. Dr. Chitnis has received personal compensation in the range of $500-$4,999 for serving on a Speakers Bureau for Academic CME. The institution of Dr. Chitnis has received research support from Novartis. The institution of Dr. Chitnis has received research support from Sanofi. The institution of Dr. Chitnis has received research support from Octave. The institution of Dr. Chitnis has received research support from Genentech-Roche. The institution of Dr. Chitnis has received research support from Tiziana Life Sciences. The institution of Dr. Chitnis has received research support from Bristol-Myers Squibb. The institution of Dr. Chitnis has received research support from Wesley Clover.
Mark Gorman, MD The institution of Dr. Gorman has received research support from Pfizer. The institution of Dr. Gorman has received research support from Roche / Genetech .
Manu S. Goyal, MD Dr. Goyal has received stock or an ownership interest from IBM. The institution of Dr. Goyal has received research support from NIH. Dr. Goyal has received personal compensation in the range of $0-$499 for serving as a Conference attendee with Capital Medical University, Shandong Madic Technology Co Ltd, Tancheng Talent Office.
No disclosure on file
Lauren B. Krupp, MD, FAAN (NYU Langone Medical Center) Dr. krupp has received personal compensation in the range of $500-$4,999 for serving as a Consultant for Bristol Myers Squibb. Dr. krupp has received personal compensation in the range of $500-$4,999 for serving as a Consultant for Celgene. Dr. krupp has received personal compensation in the range of $500-$4,999 for serving as a Consultant for Medscape. Dr. krupp has received personal compensation in the range of $500-$4,999 for serving as a Consultant for EBIX. Dr. krupp has received personal compensation in the range of $500-$4,999 for serving on a Scientific Advisory or Data Safety Monitoring board for Biogen. Dr. krupp has received personal compensation in the range of $500-$4,999 for serving on a Scientific Advisory or Data Safety Monitoring board for Hoffman LaRoche. Dr. krupp has received personal compensation in the range of $5,000-$9,999 for serving as an Expert Witness for MMMK. Dr. krupp has received personal compensation in the range of $500-$4,999 for serving as an Expert Witness for Patrick, Dolan, and Kaufman. Dr. krupp has received intellectual property interests from a discovery or technology relating to health care.
Timothy E. Lotze, MD, FAAN (Texas Children's Hospital) Dr. Lotze has received personal compensation in the range of $5,000-$9,999 for serving as an Expert Witness for Department of Justice VICP. The institution of Dr. Lotze has received research support from NIH. The institution of Dr. Lotze has received research support from National MS Society. The institution of Dr. Lotze has received research support from Sarepta Therapeutics. The institution of Dr. Lotze has received research support from PTC THERAPEUTICS. The institution of Dr. Lotze has received research support from Avexis. Dr. Lotze has received publishing royalties from a publication relating to health care. Dr. Lotze has received publishing royalties from a publication relating to health care.
Soe S. Mar, MD, FAAN (Washington University School of Medicine) The institution of Dr. Mar has received research support from NIH.
Manikum Moodley, MD Dr. Moodley has nothing to disclose.
Jayne Ness, MD (UAB Dept of Pediatrics, Div Pediatric Neurology) An immediate family member of Dr. Ness has received personal compensation in the range of $500-$4,999 for serving as a Consultant for Jan One. The institution of Dr. Ness has received research support from Chugai/Roche. The institution of Dr. Ness has received research support from National Multiple Sclerosis Society. The institution of an immediate family member of Dr. Ness has received research support from NIH. An immediate family member of Dr. Ness has received publishing royalties from a publication relating to health care.
Moses Rodriguez, MD, FAAN (Mayo Clinic Department Of Neurology) Dr. Rodriguez has received intellectual property interests from a discovery or technology relating to health care.
John W. Rose, MD, FAAN (Imaging and Neurosciences Center) The institution of Dr. Rose has received research support from National Multiple Sclerosis Society. The institution of Dr. Rose has received research support from Guthy Jackson Charitable Foundation. The institution of Dr. Rose has received research support from NIH . The institution of Dr. Rose has received research support from VA. The institution of Dr. Rose has received research support from Biogen. The institution of Dr. Rose has received research support from Friends of MS. Dr. Rose has received intellectual property interests from a discovery or technology relating to health care.
Teri Schreiner, MD, MPH, FAAN (University of Colorado/ Children's Hospital of Colorado) The institution of Dr. Schreiner has received personal compensation in the range of $500-$4,999 for serving as a Consultant for CDC. Dr. Schreiner has received personal compensation in the range of $500-$4,999 for serving on a Scientific Advisory or Data Safety Monitoring board for Biogen. The institution of Dr. Schreiner has received research support from Roche Genentech.
Jan-Mendelt Tillema, MD (Mayo Clinic) Dr. Tillema has nothing to disclose.
Michael Waltz No disclosure on file
Theron C. Casper, PhD (University of Utah) Dr. Casper has received personal compensation in the range of $500-$4,999 for serving as a Consultant for Aperta Biosciences. The institution of Dr. Casper has received research support from NIH. The institution of Dr. Casper has received research support from The National Multiple Sclerosis Society. The institution of Dr. Casper has received research support from Roche. The institution of Dr. Casper has received research support from Biogen. The institution of Dr. Casper has received research support from UCB.
Emmanuelle Waubant, MD, PhD, FAAN (USCF MS Center) The institution of Dr. Waubant has received research support from NIH. The institution of Dr. Waubant has received research support from NMSS. The institution of Dr. Waubant has received research support from PCORI. The institution of Dr. Waubant has received research support from Race to Erase MS. The institution of Dr. Waubant has received research support from Roche. The institution of Dr. Waubant has received research support from Department of Defense. Dr. Waubant has received publishing royalties from a publication relating to health care.