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Article Dans Une Revue Multiple Sclerosis Journal Année : 2021

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

Résumé

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.
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Dates et versions

hal-02887839 , version 1 (02-07-2020)

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Angelo Ghezzi, Maria Pia Amato, Gilles Edan, Hans-Peter Hartung, Eva Kubala Havrdová, et al.. The introduction of new medications in pediatric multiple sclerosis: Open issues and challenges. Multiple Sclerosis Journal, 2021, 27 (3), pp.479-482. ⟨10.1177/1352458520930620⟩. ⟨hal-02887839⟩
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