Early use of high efficacy therapies in pediatric forms of relapsing-remitting multiple sclerosis: A real-life observational study.

Background: Pediatric forms of multiple sclerosis are more active than those in adults. Yet, the effectiveness of different therapeutic approaches is not well studied in this population. Our objective was to compare the effectiveness of the early use of high efficacy therapies (HETs) with the effectiveness of moderate efficacy therapies (METs) in children with MS.
Methods: This observational study included patients diagnosed with pediatric MS, at 4 hospital centers in France, during a 10-year period. METs included: interferon β-1a, glatiramer acetate, dimethyl fumarate, teriflunomide; HETs included: fingolimod, natalizumab, ocrelizumab, alemtuzumab. The primary endpoint was the occurrence of a new relapse, the secondary endpoint was EDSS worsening.
Results: Sixty-four patients were included in the analysis (80% women; mean age 15.5 years, 81% treated with MET) with a median follow-up of 22.5 months. At baseline, 52 patients were on MET (interferon β-1a, glatiramer acetate, dimethyl fumarate, teriflunomide) and 12 patients were on HET (natalizumab, ocrelizumab). The cumulative probability of being relapse-free at 6.5 years was 23.3% on MET, vs 90.9% on HET (p = 0.013). The cumulative probability of no EDSS worsening did not differ between the 2 groups.
Conclusion: Patients starting with METs had much higher clinical disease activity than those starting early with HETs. Rapid initiation of more aggressive treatment may allow better disease control; however, the data on EDSS worsening are not conclusive.
Competing Interests: Declaration of Competing Interest A. Moreau reports no disclosure relevant to the manuscript; I. Kolitsi reports no disclosure relevant to the manuscript; L. Kremer reports no disclosure relevant to the manuscript; M.-C. Fleury reports no disclosure relevant to the manuscript; L. Lanotte reports no disclosure relevant to the manuscript; F. Sellal reports no disclosure relevant to the manuscript; C. Gaultier reports no disclosure relevant to the manuscript; G. Ahle reports grants from Biogen, grants from Novartis, grants from Roche, grants from Sanofi, grants from Abbvie, grants from Pfizer, grants from CSL Behring, outside the submitted work; S. Courtois reports no disclosure relevant to the manuscript; A. Fickl reports no disclosure relevant to the manuscript; S. Mostoufizadeh reports no disclosure relevant to the manuscript; C. Dentel reports no disclosure relevant to the manuscript; N. Collongues reports no disclosure relevant to the manuscript; J. de Seze reports no disclosure relevant to the manuscript; K. Bigaut has received lecturing fees and travel grants from Biogen, Celgene-BMS, Novartis, Roche, and Sanofi-Genzyme.
(Copyright © 2023. Published by Elsevier B.V.)