TY - JOUR
T1 - The introduction of new medications in pediatric multiple sclerosis
T2 - Open issues and challenges
AU - Ghezzi, Angelo
AU - Amato, Maria Pia
AU - Edan, Gilles
AU - Hartung, Hans Peter
AU - Havrdová, Eva Kubala
AU - Kappos, Ludwig
AU - Montalban, Xavier
AU - Pozzilli, Carlo
AU - Sorensen, Per Soelber
AU - Trojano, Maria
AU - Vermersch, Patrich
AU - Comi, Giancarlo
N1 - Publisher Copyright:
© The Author(s), 2020.
PY - 2021/3
Y1 - 2021/3
N2 - 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.
AB - 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.
KW - clinical trials
KW - disease-modifying drugs
KW - observational studies
KW - Pediatric multiple sclerosis
KW - randomized controlled trials
UR - http://www.scopus.com/inward/record.url?scp=85101168647&partnerID=8YFLogxK
U2 - 10.1177/1352458520930620
DO - 10.1177/1352458520930620
M3 - Article
C2 - 32539596
AN - SCOPUS:85101168647
SN - 1352-4585
VL - 27
SP - 479
EP - 482
JO - Multiple Sclerosis Journal
JF - Multiple Sclerosis Journal
IS - 3
ER -