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Studies Report that Highly Effective Therapies Control Disease Activity More Effectively in Children and Teens with MS - Updated

April 5, 2024

Originally posted February 21, 2024

Highly effective disease modifying therapies (DMTs) controlled disease activity better than moderately effective DMTs in two studies looking back at the medical records of children or teens with MS. These studies add critical evidence on the benefits of early treatment with highly effective medicines in children and teens, although longer-term studies are important to ensure the safety of this approach.

Background: The U.S. Food and Drug Administration (FDA) has approved many DMTs to treat adults with relapsing forms of MS. One of them – the oral MS therapy Gilenya® (fingolimod) – is also approved for the treatment of children and adolescents 10 years of age or older who have relapsing forms of MS.

Researchers continue to study how best to treat children and teens with MS, and studies have suggested that this population might benefit from early treatment, but research has been lacking about whether to start treatment with more moderately effective therapies with longer safety track records, or the newer, more highly effective therapies that have more potential for adverse side effects. The studies described below list the different types of therapies studied.

It’s important to note that what is considered high, low, or moderate effectiveness varies from study to study and also varies when talking about MS that starts in adults versus MS that begins in children. Get comprehensive information on medications used in MS.

JAMA Neurology Study: This was a retrospective study, meaning that researchers looked back at the medical records of 530 people whose MS began in childhood or adolescence in a database comprising 36 French medical centers. They specifically looked for children with relapsing-remitting MS who had started treatment with a DMT before adulthood and had at least one follow-up visit where clinical information and MRI data were collected.

In this study, the following were considered moderately effective therapies: azathioprine, cyclophosphamide, dimethyl fumarate, glatiramer acetate, interferon beta, methotrexate, mycophenolate mofetil, and teriflunomide. Highly effective therapies were: alemtuzumab, fingolimod, mitoxantrone, cladribine, natalizumab, ocrelizumab, ofatumumab, and rituximab.

Both treatment approaches were found to be effective in the first two years. However, those who started out taking highly effective therapies were 20% less likely to have a relapse within two years, and 54% less likely to have a relapse within 5 years than those taking moderately effective therapies.

Those on moderately effective therapies were more likely to discontinue therapy (nearly 80%, versus 40% in those taking highly effective therapies). This occurred most often because of persistent disease activity or difficulty tolerating the therapy.

Lancet Study: This was a longitudinal study, meaning it looked at medical records over time. The team looked at the records of 5,224 people whose MS symptoms first appeared before age 18, from 41 countries. Records were obtained from the international MSBase registry and the Italian MS and Related Disorders Register. The primary outcome the researchers investigated was the time to any change in disability status.

In this study, the following were considered moderately effective therapies: dimethyl fumarate, glatiramer, acetate, interferon beta, and teriflunomide. Highly effective therapies were: alemtuzumab, cladribine, daclizumab, fingolimod, mitoxantrone, natalizumab, ocrelizumab, rituximab, and autologous hematopoietic stem cell transplantation.

Although both low and high effectiveness treatments reduced the risk of disability worsening, the risk was significantly lower in people taking high effectiveness treatments. This difference was most pronounced if high effectiveness treatments were administered before young people developed moderate or severe disability.
Importantly, the nature of this study did not provide information on safety or tolerability of the therapies or other potential signs of disease activity such as brain MRI scans.

Why do these studies matter? These studies add evidence that initiating MS treatment with highly effective therapies earlier may be more beneficial for children and teens with MS than starting with moderately effective DMTs. Importantly, these are “observational” studies, where researchers looked at medical records. The standard way to prove treatment effectiveness is a randomized trial, in which children would be randomly assigned to receive the treatments and then followed. Treatment decisions remain a careful discussion between health care providers, younger people with MS, and their families.

Learn more…
Get resources for coping with pediatric MS
Register for the virtual program “Pediatric MS: Navigating Your Journey”
Watch a webinar with an expert on pediatric MS

“Highly Effective Therapies as First-Line Treatment for Pediatric-Onset Multiple Sclerosis,” by Nail Benallegue, MD, PhD; Fabien Rollot, MSc; Sandrine Wiertlewski, MD; Romain Casey, PhD; Marc Debouverie, MD, PhD; Anne Kerbrat, MD, PhD; Jérôme De Seze, MD, PhD; Jonathan Ciron, MD; Aurelie Ruet, MD, PhD; Pierre Labauge, MD, PhD; Elisabeth Maillart, MD; Helene Zephir, MD, PhD; Caroline Papeix, MD, PhD; Gilles Defer, MD; Christine Lebrun-Frenay, MD, PhD; Thibault Moreau, MD, PhD; Eric Berger, MD; Bruno Stankoff, MD, PhD; Pierre Clavelou, MD, PhD; Olivier Heinzlef, MD; Jean Pelletier, MD, PhD; Eric Thouvenot, MD, PhD; Abdullatif Al Khedr, MD; Bertrand Bourre, MD; Olivier Casez, MD; Philippe Cabre, MD, PhD; Abir Wahab, MD; Laurent Magy, MD, PhD; Sandra Vukusic, MD, PhD; David-Axel Laplaud, MD, PhD; for the OFSEP (Observatoire Français de la Sclérose en Plaques) Investigators is published in JAMA Neurology (Published online February 12, 2024).

Disease-modifying therapies in managing disability worsening in paediatric-onset multiple sclerosis: a longitudinal analysis of global and national registries” by Sifat Sharmin, PhD, Izanne Roos, PhD, Charles B Malpas, PhD, Pietro Iaffaldano, MD, Marta Simone, MD, Prof Massimo Filippi, MD, Prof Eva Kubala Havrdova, MD, Prof Serkan Ozakbas, MD, Prof Vincenzo Brescia Morra, MD, Raed Alroughani, MD, Mauro Zaffaroni, MD, Prof Francesco Patti, MD, Sara Eichau, MD, Giuseppe Salemi, MD, Alessia Di Sapio, MD, Prof Matilde Inglese, MD, Emilio Portaccio, MD, Prof Maria Trojano, MD, Prof Maria Pia Amato, MD, and Prof Tomas Kalincik, PhD on behalf of theItalian Multiple Sclerosis and Related Disorders Register and MSBase Study Group, is published in The Lancet Child & Adolescent Health (Published:March 25, 2024).

About Multiple Sclerosis

Multiple sclerosis is an unpredictable disease of the central nervous system. Currently there is no cure. Symptoms vary from person to person and may include disabling fatigue, mobility challenges, cognitive changes, and vision issues. An estimated 1 million people live with MS in the United States. Early diagnosis and treatment are critical to minimize disability. Significant progress is being made to achieve a world free of MS.

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