The introduction of new medications in pediatric multiple sclerosis: Open issues and challenges

Angelo Ghezzi*, Maria Pia Amato, Gilles Edan, Hans Peter Hartung, Eva Kubala Havrdová, Ludwig Kappos, Xavier Montalban, Carlo Pozzilli, Per Soelber Sorensen, Maria Trojano, Patrich Vermersch, Giancarlo Comi

*Corresponding author for this work

Research output: Contribution to journalArticleResearchpeer-review

3 Citations (Scopus)


Disease-modifying drugs (DMDs) for multiple sclerosis (MS) have been evaluated in pediatric patients in observational studies demonstrating a similar, even better clinical effect compared to adults, with a similar safety. Only fingolimod has been tested in a randomized controlled trial (RCT) and is approved for pediatric multiple sclerosis (ped-MS). Numerous methodological, practical, and ethical issues underline that RCTs are difficult to conduct in ped-MS. This also creates a lack of safety information. To facilitate the availability of new agents in ped-MS, we encourage to develop a different approach based on pharmacokinetic/pharmacodynamic studies to yield information on optimal doses and implementation of obligatory registries to obtain information on safety as primary endpoint.

Original languageEnglish
Pages (from-to)479-482
Number of pages4
JournalMultiple Sclerosis Journal
Issue number3
Publication statusPublished - Mar 2021


  • clinical trials
  • disease-modifying drugs
  • observational studies
  • Pediatric multiple sclerosis
  • randomized controlled trials


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