The introduction of new medications in pediatric multiple sclerosis: Open issues and challenges
Research output: Contribution to journal › Journal article › Research › peer-review
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The introduction of new medications in pediatric multiple sclerosis : Open issues and challenges. / Ghezzi, Angelo; Amato, Maria Pia; Edan, Gilles; Hartung, Hans Peter; Havrdová, Eva Kubala; Kappos, Ludwig; Montalban, Xavier; Pozzilli, Carlo; Sørensen, Per Soelber; Trojano, Maria; Vermersch, Patrich; Comi, Giancarlo.
In: Multiple Sclerosis Journal, Vol. 27, No. 3, 2021, p. 479-482.Research output: Contribution to journal › Journal article › Research › peer-review
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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 - Sørensen, Per Soelber
AU - Trojano, Maria
AU - Vermersch, Patrich
AU - Comi, Giancarlo
N1 - Publisher Copyright: © The Author(s), 2020.
PY - 2021
Y1 - 2021
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
U2 - 10.1177/1352458520930620
DO - 10.1177/1352458520930620
M3 - Journal article
C2 - 32539596
AN - SCOPUS:85101168647
VL - 27
SP - 479
EP - 482
JO - Multiple Sclerosis Journal
JF - Multiple Sclerosis Journal
SN - 1352-4585
IS - 3
ER -
ID: 303772425