Real world data on treatment of pediatric onset multiple sclerosis.

In this issue Saponaro et al. present a longitudinal follow-up study in a real world setting in which they compared the efficacy and safety of disease modifying treatments (DMT) in children with multiple sclerosis (MS) from the French KID-BIOSEP cohort, including children with age of onset under 11 years [ [1] Saponaro A.C. Tully T. Maillart E. Maurey H. Deiva K. KidBiosep cohort study. Treatments of paediatric multiple sclerosis: efficacy and tolerance in a longitudinal follow-up study. Eur. J. Paediatr. Neurol. 2023; 45: 22-28 Abstract Full Text Full Text PDF PubMed Scopus (1) Google Scholar ]. The majority of children in this cohort initially started on traditional first line injectable treatment with interferon-beta. Notably, 77% of the children treated with interferon-beta switched during follow-up to second line treatment, whereas only 15% of the children first treated with newer DMT switched. The mean annualized relapse rate (ARR) dropped from 1,65, or 0,78 after exclusion of the first attack, during the pre-treatment period to 0,45 during treatment with interferon-beta, as was found before [ [2] Krupp L.B. Pohl D. Ghezzi A. Boyko A. Tenembaum S. Chen L. et al. REPLAY Study GroupSubcutaneous interferon β-1a in pediatric patients with multiple sclerosis: regional differences in clinical features, disease management, and treatment outcomes in an international retrospective study. J. Neurol. Sci. 2016; 363: 33-38 Abstract Full Text Full Text PDF PubMed Scopus (14) Google Scholar ]. The mean ARR during treatment with all other, newer DMT (i.e. fingolimod, teriflunomide, dimethyl-fumarate and natalizumab) was lower compared to interferon-beta. Similar results were observed using the number of new T2 lesions as a secondary outcome measure. Although the patient numbers were relatively low, the lowest ARR, number of new T2 lesions and number of new gadolinium enhancing lesions was found during treatment with natalizumab. The frequency and type of adverse events did not essentially differ from previous studies [ 3 Chitnis T. Tenembaum S. Banwell B. Krupp L. Pohl D. Rostasy K. Yeh E.A. Bykova O. Wassmer E. Tardieu M. Kornberg A. Ghezzi A. International Pediatric Multiple Sclerosis Study Group. Consensus statement: evaluation of new and existing therapeutics for pediatric multiple sclerosis. Mult. Scler. 2012; 18: 116-127 Crossref PubMed Scopus (174) Google Scholar , 4 Chitnis T. Arnold D.L. Banwell B. Brück W. Ghezzi A. Giovannoni G. et al. Trial of fingolimod versus interferon beta-1a in pediatric multiple sclerosis. N. Engl. J. Med. 2018; 379: 1017-1027 Crossref PubMed Scopus (181) Google Scholar , 5 Chitnis T. Banwell B. Kappos L. Arnold D.L. Gücüyener K. Deiva K. et al. Safety and efficacy of teriflunomide in paediatric multiple sclerosis (TERIKIDS): a multicentre, double-blind, phase 3, randomised, placebo-controlled trial. Lancet Neurol. 2021; 20: 1001-1011 Abstract Full Text Full Text PDF PubMed Scopus (24) Google Scholar ]. Time children remained on treatment, a potential proxy of both efficacy and safety, was longer in the newer DMT compared to interferon beta.