The introduction of new medications in pediatric multiple sclerosis: Open issues and challenges
Language English Country Great Britain, England Media print-electronic
Document type Journal Article, Randomized Controlled Trial
- Keywords
- Pediatric multiple sclerosis, clinical trials, disease-modifying drugs, observational studies, randomized controlled trials,
- MeSH
- Child MeSH
- Adult MeSH
- Fingolimod Hydrochloride therapeutic use MeSH
- Immunosuppressive Agents * therapeutic use MeSH
- Humans MeSH
- Multiple Sclerosis * drug therapy MeSH
- Check Tag
- Child MeSH
- Adult MeSH
- Humans MeSH
- Publication type
- Journal Article MeSH
- Randomized Controlled Trial MeSH
- Names of Substances
- Fingolimod Hydrochloride MeSH
- Immunosuppressive Agents * MeSH
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.
Centro Studi Sclerosi Multipla Ospedale di Gallarate Gallarate Italy
CIC 1414 INSERM Department of Neurology CHU Rennes Rennes France
Department of Basic Medical Sciences Neurosciences and Sense Organs University of Bari Bari Italy
Istituto di Neurologia Sperimentale IRCCS Ospedale San Raffaele Milan Italy
Multiple Sclerosis Center Sant' Andrea Hospital Rome Italy
University of Lille INSERM UMR S1172 CHU Lille FHU Imminent Lille France
References provided by Crossref.org
