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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

Language English Country Great Britain

Document type Journal Article, Randomized Controlled Trial

Persistent link   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.

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$a Disease-modifying drugs (DMDs) for multiple sclerosis (MS) have been evaluated in pediatric patients in observational studies demonstrating a similar, even better clinical effect compared to adults, with a similar safety. Only fingolimod has been tested in a randomized controlled trial (RCT) and is approved for pediatric multiple sclerosis (ped-MS). Numerous methodological, practical, and ethical issues underline that RCTs are difficult to conduct in ped-MS. This also creates a lack of safety information. To facilitate the availability of new agents in ped-MS, we encourage to develop a different approach based on pharmacokinetic/pharmacodynamic studies to yield information on optimal doses and implementation of obligatory registries to obtain information on safety as primary endpoint.
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$a Amato, Maria Pia $u Department of NEUROFARBA, University of Florence, Florence, Italy; IRCCS Fondazione Don Carlo Gnocchi, Florence, Italy
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$a Edan, Gilles $u CIC 1414 INSERM, Department of Neurology, CHU Rennes, Rennes, France
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$a Hartung, Hans-Peter $u Department of Neurology, UKD and Center of Neurology and Neuropsychiatry, Heinrich-Heine-University, Düsseldorf, Germany
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$a Havrdová, Eva Kubala $u Department of Neurology and Center for Clinical Neuroscience, First Medical Faculty, Charles University, Prague, Czech Republic
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$a Kappos, Ludwig $u Neurologic Clinic and Policlinic, Departments of Medicine, Clinical Research, Biomedicine and Biomedical Engineering, University Hospital and University of Basel, Basel, Switzerland
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$a Montalban, Xavier $u St Michael's Hospital, University of Toronto, Toronto, ON, Canada; Department of Neurology, Cemcat, Hospital Vall d'Hebron, Barcelona, Spain
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$a Pozzilli, Carlo $u Multiple Sclerosis Center, Sant' Andrea Hospital, Rome, Italy
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$a Sorensen, Per Soelber $u Department of Neurology, Danish Multiple Sclerosis Center, Copenhagen University and Rigshospitalet, Copenhagen, Denmark
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$a Trojano, Maria $u Department of Basic Medical Sciences, Neurosciences and Sense Organs, University of Bari, Bari, Italy
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$a Vermersch, Patrich $u University of Lille, INSERM UMR-S1172, CHU Lille, FHU Imminent, Lille, France
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$a Comi, Giancarlo $u Istituto di Neurologia Sperimentale (INSPE), IRCCS Ospedale San Raffaele, Milan, Italy
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