Improvements made in recent years in the treatment and management of pediatric-onset multiple sclerosis (MS) have resulted in substantial reductions in persistent disability in patients with MS, according to study results published in JAMA Neurology.

This was a retrospective, multicenter, observational study in which study investigators analyzed data reported in the Italian MS registry. They focused their attention on 3198 patients with pediatric-onset MS (mean age at onset, 15.2 years) who were younger than 18 years of age at time of MS diagnosis and had a disease duration of at least 3 years.

Study investigators compared several different MS diagnostic epochs (<1993, 1993-1999, 2000-2006, and 2007-2013) regarding the time it took to reach disability milestones. The disability milestones included Expanded Disability Status Scale (EDSS) scores of 4.0 and 6.0.


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In the overall study cohort, the median time to diagnosis was 3.2 years. Additionally, the median annualized relapse rate in the first year from onset was 1.3 compared with 0.6 in the first 3 years. Over the mean follow-up period of 21.8 years, the median survival times to reach disability milestones of 4.0 and 6.0 were 31.7 and 40.5 years, respectively.

Study investigators reported a gradual decrease in the cumulative risk of reaching EDSS score 4.0 (hazard ratio [HR], 0.70; 95% CI, 0.58-0.83 in 1993-1999; HR, 0.48; 95% CI, 0.38-0.60 in 2000-2006; and HR, 0.44; 95% CI, 0.32-0.59 in 2007-2013) and EDSS score 6.0 (HR, 0.72; 95% CI, 0.57-0.90 in 1993-1999; HR, 0.44; 95% CI, 0.33-0.60 in 2000-2006; and HR, 0.30; 0.20-0.46 in 2007-2013).

A higher proportion of patients with pediatric onset MS were treated with disease-modifying therapies (DMTs) at later diagnosis epochs. These DMTs were also administered earlier and for longer periods of time compared with previous epochs.

Limitations of this study included the lack of magnetic resonance imaging data, the retrospective design, and the lack of cognitive assessment data.

Study researchers concluded that their findings are likely due to “improvement in therapeutic and managing standards” and suggested that “an increase of approved DMTs before age 18 years and upgrades in drug safety may lead to a further improvement of prognosis in this population” in the coming years.

Disclosure: Several study authors declared affiliations with the pharmaceutical industry. Please see the original reference for a full list of authors’ disclosures.

Reference

Baroncini D, Simone M, Iaffaldano P, et al. Risk of persistent disability in patients with pediatric-onset multiple sclerosis. Published online May 3, 2021. JAMA Neurol. doi:10.1001/jamaneurol.2021.1008