MS in Children – An Overlooked Sister of Adult Diseases?

Children versus Adults

Multiple sclerosis (MS) is a chronic autoimmune disease of the central nervous system (CNS). Two components play a major role in the pathogenesis of MS – inflammatory and degenerative. MS primarily affects young adults between the ages of 20 and 40, more frequently women. However, this is not always the case. Worldwide, 2.8 million people suffer from this disease, with at least 30,000 of them not yet having reached adulthood. Most pediatric patients have the relapsing-remitting form of MS (98%) and compared to adult patients, children tend to have a higher frequency of relapses and a larger total lesion volume on MRI. Especially due to the earlier onset of the disease, there is also an earlier transition to secondary progression.

Time is Merciless

Although there are significantly fewer pediatric patients compared to adult patients, this does not diminish the importance of treating this disease. On the contrary – the earlier the disease starts, the longer the potential timeframe of a quality life, which must be fought for with effective treatment. And as is already known from the pathogenesis of MS, the sooner treatment begins, the better its outcomes can be. All currently available disease-modifying drugs (DMDs) are essentially anti-inflammatory, but as the disease progresses, the degenerative component and compartmentalized inflammation behind the closed blood-brain barrier unfortunately start to prevail. Therefore, early initiation of treatment is absolutely crucial to prevent irreversible CNS damage and delay disability. Waiting for effective treatment until adulthood could have very serious consequences.

Hope for Better Times?

Unlike adult patients, only older injectable DMDs – interferons and glatiramer acetate – had long been registered for pediatric patients, and only for children over 12 years of age. However, relatively recently, based on data from a randomized double-blind comparator study (interferon beta), the first oral drug – fingolimod – was approved for children as young as 10 years old. This was then followed last year by another oral DMD indicated for the same age category – teriflunomide. Teriflunomide confirmed its efficacy and safety in the pediatric population in an international double-blind placebo-controlled study in patients aged 10–17 (TERIKIDS). In this clinical study, it reduced the risk of clinical relapse by 34% compared to placebo and significantly (by 55%) reduced the number of new and enlarging T2 lesions per scan.

The mentioned ever-expanding on-label possibilities for influencing MS in pediatric patients bring the promise of more promising tomorrows and effective treatment supported by an increasingly extensive base of quality safety data.

(dos)

Sources:

1. MSIF. Atlas of MS, 3^rd ed. Multiple Sclerosis International Federation, 2020. Available at: www.atlasofms.org
2. Alroughani R., Boyko A. Pediatric multiple sclerosis: a review. BMC Neurol 2018; 18 (1): 27, doi: 10.1186/s12883-018-1026-3.
3. Renoux C., Vukusic S., Confavreux C. The natural history of multiple sclerosis with childhood onset. Clin Neurol Neurosurg 2008; 110 (9): 897−904, doi: 10.1016/j.clineuro.2008.04.009.
4. Pena J. A., Lotze T. E. Pediatric multiple sclerosis: current concepts and consensus definitions. Autoimmune Dis 2013; 2013: 673947, doi: 10.1155/2013/673947.
5. Chitnis T., Banwell B., Kappos L. et al. Safety and efficacy of teriflunomide in paediatric multiple sclerosis (TERIKIDS): a multicentre, double-blind, phase 3, randomised, placebo-controlled trial. Lancet Neurol 2021; 20 (12): 1001−1011, doi: 10.1016/S1474-4422(21)00364-1.
6. Havrdová E., Piťha J. Clinical practice guideline for the diagnosis and treatment of multiple sclerosis and neuromyelitis optica and spectrum disorder. Czech Neurological Society ČLS JEP, 2020. Available at: www.czech-neuro.cz/content/uploads/2020/04/rs_odborna-2.0_final_pub_web-2.pdf