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. 2020 Jul;88(1):42-55.
doi: 10.1002/ana.25737. Epub 2020 May 14.

Real-World Effectiveness of Initial Disease-Modifying Therapies in Pediatric Multiple Sclerosis

Kristen M Krysko  1 , Jennifer S Graves  1   2 , Mary Rensel  3 , Bianca Weinstock-Guttman  4 , Alice Rutatangwa  1 , Gregory Aaen  5 , Anita Belman  6 , Leslie Benson  7 , Tanuja Chitnis  8 , Mark Gorman  7 , Manu S Goyal  9 , Yolanda Harris  10 , Lauren Krupp  6 , Timothy Lotze  11 , Soe Mar  12 , Manikum Moodley  13 , Jayne Ness  14 , Moses Rodriguez  15 , John Rose  16 , Teri Schreiner  17 , Jan-Mendelt Tillema  15 , Michael Waltz  18 , T Charles Casper  18 , Emmanuelle Waubant  1 ; US Network of Pediatric MS Centers
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Real-World Effectiveness of Initial Disease-Modifying Therapies in Pediatric Multiple Sclerosis

Kristen M Krysko et al. Ann Neurol. 2020 Jul.

Abstract

Objective: To assess real-world effectiveness of initial treatment with newer compared to injectable disease-modifying therapies (DMTs) on disease activity in pediatric multiple sclerosis (MS) and clinically isolated syndrome (CIS).

Methods: 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-β, glatiramer acetate) DMTs. Propensity scores (PSs) were computed, including preidentified 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 brain magnetic resonance imaging were modeled with midpoint survival analyses, adjusted for PS-quintile.

Results: A total of 741 children 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. In PS-quintile-adjusted analysis, those on newer DMTs had a lower relapse rate than those on injectables (rate ratio = 0.45, 95% confidence interval (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 rather than injectable DMTs for 3.7 person-years to prevent 1 relapse. Those started on newer DMTs had a lower rate of new/enlarging T2 (hazard ratio [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.

Interpretation: 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 are required. ANN NEUROL 2020 ANN NEUROL 2020;88:42-55.

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References

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