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Clinical trials of disease-modifying agents in pediatric MS

2019; Lippincott Williams & Wilkins; Volume: 92; Issue: 22 Linguagem: Inglês

10.1212/wnl.0000000000007572

1526-632X

Emmanuelle Waubant, Brenda Banwell, Evangeline Wassmer, Maria Pia Sormani, Maria Pia Amato, Rogier Hintzen, Lauren Krupp, Kevin Rostásy, Sílvia Tenembaum, Tanuja Chitnis, Gregory Aaen, Elhachmia Ait Ben Adou, Raed Alroughani, Veronica Gonzalez Alvarez, Maria Anagnostouli, Banu Anlar, Thaís Armangué, Georgina Arrambide, Damiano Baroncini, R Ts Bembeeva, Leslie Benson, Neli Bizjak, Astrid Blaschek, Alexey Boyко, J. Nicholas Brenton, Wolfgang Brück, Bruna Klein da Costa, Dominique Dive, Christiane Elpers, Massimo Filippi, Manuela de Oliveira Fragomeni, Eva Havrdová, Cheryl Hemingway, Barbara Kornek, Kumaran Deiva, Adrian R. Lacy, Zuzana Libá, Ming Lim, Tim Lotze, Jean K. Mah, Naila Makhani, Soe Mar, Kyla A. McKay, Shay Menascu, Lucia Moiola, Patricia Mulero, Moustapha Ndiaye, Rinze F. Neuteboom, Jayne Ness, Enedina Maria Lobato de Oliveira, Scott Otallah, Francesco Patti, José Albino da Paz, Carlos A. Pérez, Daniela Pohl, Anne‐Louise Ponsonby, Mary Rensel, Maria A. Rocca, Nick Rijke, Moses Rodriguez, Ian Rossman, Hiroshi Sakuma, Teri Schreiner, Ángeles Schteinschnaider, E. Morghen Sikes, Isabella Laura Simone, Michael Sweeney, Jan Mendelt Tillema, Regina M. Troxell, Hélène Verhelst, Leidi Vilchez, Liesbeth De Waele, Bianca Weinstock‐Guttman, Colin Wilbur, Mikaeloff Yann, E. Ann Yeh, Dimitrios Zafeiriou,

Viral Infections and Immunology Research

Objective The impetus for this consensus discussion was to recommend clinical trial designs that can deliver high-quality data for effective therapies for pediatric patients, in a reasonable timeframe, with a key focus on short- and long-term safety. Methods The International Pediatric Multiple Sclerosis Study Group convened a meeting of experts to review the advances in the understanding of pediatric-onset multiple sclerosis (MS) and the advent of clinical trials for this population. Results In the last few years, convincing evidence has emerged that the biological processes involved in MS are largely shared across the age span. As such, treatments proven efficacious for the care of adults with MS have a biological rationale for use in pediatric MS given the relapsing-remitting course at onset and high relapse frequency. There are also ethical considerations on conducting clinical trials in this age group including the use of placebo owing to highly active disease. It is imperative to reconsider study design and implementation based on what information is needed. Are studies needed for efficacy or should safety be the primary goal? Further, there have been major recruitment challenges in recently completed and ongoing pediatric MS trials. Phase 3 trials for every newly approved therapy for adult MS in the pediatric MS population are simply not feasible. Conclusions A primary goal is to ensure high-quality evidence-based treatment for children and adolescents with MS, which will improve our understanding of the safety of these agents and remove regulatory or insurance-based limitations in access to treatment.