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

Multiple Sclerosis : Clinical and Laboratory Research
Angelo GhezziGiancarlo Comi

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.

References

Dec 8, 2011·Multiple Sclerosis : Clinical and Laboratory Research·T ChitnisUNKNOWN International Pediatric Multiple Sclerosis Study Group
Oct 2, 2012·European Journal of Paediatric Neurology : EJPN : Official Journal of the European Paediatric Neurology Society·Carmen Arnal-GarciaCelia Oreja-Guevara
Feb 20, 2013·JAMA Neurology·Barbara KornekAntonios Bayas
Sep 27, 2015·BMC Neurology·Angelo GhezziUNKNOWN MS Study Group-Italian Society of Neurology
Aug 31, 2016·Neurology·Amit Bar-OrTanuja Chitnis
Sep 22, 2017·Multiple Sclerosis : Clinical and Laboratory Research·Peter HuppkeJutta Gärtner
Sep 13, 2018·The New England Journal of Medicine·Tanuja ChitnisUNKNOWN PARADIGMS Study Group
Oct 20, 2018·Neurology·Kristen M KryskoUNKNOWN US Network of Pediatric MS Centers
Apr 5, 2019·Multiple Sclerosis : Clinical and Laboratory Research·Frederik BartelsKevin Rostásy
Jul 30, 2019·Neurology. Neuroimmunology and Neuroinflammation·Angelo GhezziPaolo Gallo
Feb 16, 2020·Lancet Neurology·Tobias DerfussLudwig Kappos
Apr 9, 2020·Annals of Neurology·Kristen M KryskoUNKNOWN US Network of Pediatric MS Centers

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Clinical Trials Mentioned

NCT04075266

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