New disease-modifying therapies show promise in pediatric multiple sclerosis

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According to the results of a study published in the European Journal of Pediatric Neurology.

The current single-center retrospective study was conducted in people with MS who started before the age of 18 – that is, POMS – who were diagnosed according to the criteria of the International Pediatric Multiple Sclerosis Study Group. In most cases, POMS usually presents as relapsing-remitting MS.

Immunomodulating agents or DMTs used to prevent relapses and treat disability in people with PCOS differ from those used in patients with adult-onset MS (AOMS). In patients with PCOS, injectable DMT (iDMT), interferon-β (IFN-β), and glatiramer acetate (GA) are considered first-line treatments. The majority of the 13 new agents approved by the European Medicines Agency and the United States Food and Drug Administration for the treatment of AOM over the past 2 decades are used off-label in people with POMS, only fingolimod and teriflunomide having been approved for the treatment of children in Europe. Among the nDMTs, teriflunomide can be considered first-line; second-line fingolimod; and rituximab, natalizumab, ocrelizumab, and alemtuzumab as second- or third-line treatments.


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In the present study, researchers sought to assess the effects of treatment with nDMTs versus iDMTs in a cohort of patients with POMS. Clinical records of all patients with POMS who had received nDMTs as escalation therapy or initial treatment and had 12 months or more of follow-up in the pediatric neurology or neurology departments of Ihsan Dogramaci Children’s Hospital at Hacettepe University in Ankara, Turkey, between 2010 and 2020 were reviewed. Relapses were defined as neurological symptoms that lasted ≥ 24 hours.

All participants were divided into 2 groups: (1) Group A: patients who started iDMT and then switched to nDMT; and (2) Group B: patients who were treated with nDMTs from the start. Annualized relapse rate (ARR), presenting symptoms, recent lesion burden on the Expanded Disability Status Scale (EDSS) and presence of contrast lesions (CE) on magnetic resonance imaging (MRI) were compared in the 2 groups.

A total of 43 patients were recruited into the study – 33 people in group A and 10 people in group B. In both groups, the ratio of women to men, age at onset of disease, duration since disease onset and duration of nDMT reception were similar. In group A, initial disease involvement was primarily brainstem and cerebellum, while in group B initial disease involvement was sensory, brainstem/cerebellar, and optic nerve.

The most commonly used nDMTs were fingolimod in 51.5% (17 of 33) of group A participants, versus teriflunomide in 60.0% (6 of 10) of group B participants. was 2.0 in group A versus 1.5 in group B (P > 0.05), which decreased to a median ARR of 1.0 with iDMT use in group A and to a median of 0 with nDMT use in both groups (P <.001>

In group A, the mean follow-up was 6.7 ± 5.0 years (range, 1 to 19 years; median, 6 years), compared to a mean follow-up of 3.9 ± 3.7 years (range, 1 to 12 years; median, 2 years) in Group B. Based on the latest available follow-up information, the median EDSS scores were 1 in Group A and 0 in Group B. Additionally, although the While ARR increased and MRI lesion burden increased progressively in groups during follow-up, the rate of participants with CE lesions decreased in group B.

A major limitation of the current study was the size of the series, which precluded subgroup analyses. An additional limitation was the lack of cognitive assessment and the inability to make an effective statistical comparison with group A due to the small number of patients in group B.

The researchers concluded that “a switch between nDMTs at any given time can be anticipated in series such as ours where the use of potent nDMTs such as natalizumab and ocrelizumab is rare and expected to increase with the acquisition of more experience and changes in regulations over time.”

Disclosure: None of the study authors declared any affiliation with any biotechnology, pharmaceutical and/or device companies.

Reference

Solmaz I, Acar Ozen P, Parlak S, Tuncer A, Anlar B. New disease-modifying treatments in pediatric multiple sclerosis: single center experience. Eur J Paediatr Neurol. Published online June 23, 2022. doi:10.1016/j.ejpn.2022.06.013

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