The introduction of new medications in pediatric multiple sclerosis: Open issues and challenges. (March 2021)

• Record Type: Journal Article
• Authors: Ghezzi, Angelo; Amato, Maria Pia; Edan, Gilles; Hartung, Hans-Peter; Havrdová, Eva Kubala; Kappos, Ludwig; Montalban, Xavier; Pozzilli, Carlo; Sorensen, Per Soelber; Trojano, Maria; Vermersch, Patrich; Comi, Giancarlo
• Is Part Of: Multiple sclerosis. Volume 27:Number 3(2021)
• Page Start: 479
• Page End: 482
• Publication Date: 2021-03
• Subjects: Pediatric multiple sclerosis -- clinical trials -- randomized controlled trials -- observational studies -- disease-modifying drugs; Central nervous system -- Diseases -- Periodicals; Myelin sheath -- Diseases -- Periodicals; Inflammation -- Periodicals; Multiple sclerosis -- Periodicals; Central Nervous System Diseases -- Periodicals; Demyelinating Diseases -- Periodicals; Inflammation -- Periodicals; Multiple Sclerosis -- Periodicals; Système nerveux central -- Maladies -- Périodiques; Gaine de myéline -- Maladies -- Périodiques; Inflammation (Pathologie) -- Périodiques; Sclérose en plaques -- Périodiques; Electronic journals; 616.834005
• DOI: 10.1177/1352458520930620
• Languages: English
• ISSNs: 1352-4585

Abstract

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.