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

Research output: Contribution to journal › Journal article › Research › peer-review

Standard

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

Harvard

Ghezzi, A, Amato, MP, Edan, G, Hartung, HP, Havrdová, EK, Kappos, L, Montalban, X, Pozzilli, C, Sørensen, PS, Trojano, M, Vermersch, P & Comi, G 2021, 'The introduction of new medications in pediatric multiple sclerosis: Open issues and challenges', Multiple Sclerosis Journal, vol. 27, no. 3, pp. 479-482. https://doi.org/10.1177/1352458520930620

APA

Ghezzi, A., Amato, M. P., Edan, G., Hartung, H. P., Havrdová, E. K., Kappos, L., Montalban, X., Pozzilli, C., Sørensen, P. S., Trojano, M., Vermersch, P., & Comi, G. (2021). The introduction of new medications in pediatric multiple sclerosis: Open issues and challenges. Multiple Sclerosis Journal, 27(3), 479-482. https://doi.org/10.1177/1352458520930620

Vancouver

Ghezzi A, Amato MP, Edan G, Hartung HP, Havrdová EK, Kappos L et al. The introduction of new medications in pediatric multiple sclerosis: Open issues and challenges. Multiple Sclerosis Journal. 2021;27(3):479-482. https://doi.org/10.1177/1352458520930620

Author

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. / The introduction of new medications in pediatric multiple sclerosis : Open issues and challenges. In: Multiple Sclerosis Journal. 2021 ; Vol. 27, No. 3. pp. 479-482.

Bibtex

@article{9c56409d79454796b69f487acd099f1a,
title = "The introduction of new medications in pediatric multiple sclerosis: Open issues and challenges",
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.",
keywords = "clinical trials, disease-modifying drugs, observational studies, Pediatric multiple sclerosis, randomized controlled trials",
author = "Angelo Ghezzi and Amato, {Maria Pia} and Gilles Edan and Hartung, {Hans Peter} and Havrdov{\'a}, {Eva Kubala} and Ludwig Kappos and Xavier Montalban and Carlo Pozzilli and S{\o}rensen, {Per Soelber} and Maria Trojano and Patrich Vermersch and Giancarlo Comi",
note = "Publisher Copyright: {\textcopyright} The Author(s), 2020.",
year = "2021",
doi = "10.1177/1352458520930620",
language = "English",
volume = "27",
pages = "479--482",
journal = "Multiple Sclerosis Journal",
issn = "1352-4585",
publisher = "SAGE Publications",
number = "3",

}

RIS

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