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

A. Ghezzi, MP. Amato, G. Edan, HP. Hartung, EK. Havrdová, L. Kappos, X. Montalban, C. Pozzilli, PS. Sorensen, M. Trojano, P. Vermersch, G. Comi

. 2021 ; 27 (3) : 479-482. [pub] 20200616

Jazyk angličtina Země Velká Británie

Typ dokumentu časopisecké články, randomizované kontrolované studie

Perzistentní odkaz   https://www.medvik.cz/link/bmc21026035

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.

Citace poskytuje Crossref.org

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$a Hartung, Hans-Peter $u Department of Neurology, UKD and Center of Neurology and Neuropsychiatry, Heinrich-Heine-University, Düsseldorf, Germany
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