Anti-CD20 therapies outperform platform DMTs in prepubertal MS

Anti-CD20 therapies are more effective than platform disease-modifying treatments (DMTs) in preventing relapses and new MRI lesions in multiple sclerosis (MS) patients under 12 years of age. Patients treated with platform DMTs showed greater deep grey matter volume reduction compared with those treated with anti-CD20 therapies.

Data on the efficacy and safety of high-efficacy DMTs in prepubertal children are minimal. Dr Riccardo Nistri (University College London, UK) stated that this is primarily due to misdiagnosis, difficulties in recruiting young children for trials, safety concerns, and the need for dose adjustments based on weight. Dr Nistri and colleagues conducted a retrospective analysis of prepubertal children with MS treated at 5 highly specialised UK centres between 2015 and 2025 [1]. The study aimed to compare the effects of platform treatments (interferons, glatiramer acetate, and dimethyl fumarate) with those of anti-CD20 therapies (rituximab, ocrelizumab, and ofatumumab). The required follow-up was at least 2 years. The 2 primary endpoints were the annualised relapse rate (ARR) and the annualised rate of new lesions detected on T2-weighted/FLAIR images compared with baseline scan, as well as gadolinium-enhancing lesions.

Of 75 enrolled participants, 65 were included in the analysis; 37 received platform DMTs and 28 received anti-CD20 therapy. Among platform DMTs, interferons were most common (n=33), while ocrelizumab was the predominant anti-CD20 therapy (n=20).

In the anti-CD20 group, both the ARR and the annualised rate of new T2-weighted MRI lesions were significantly lower than in the platform therapy group after 2 years. The ARR was 0.11 versus 0.66, respectively, a between-group difference of 0.55 (95% CI 0.23-0.87; P=0.005). The annualised rate of new T2-weighted MRI lesions was 0.87 and 2.37, a between-group difference of 1.50 (95% CI 0.23-2.77; P=0.01). Platform therapy was also associated with greater deep grey matter volume loss (P=0.026). There were no differences in total brain volume, which Dr Nistri attributed to the relatively short follow-up. Safety was comparable in both groups; no hospitalisations or life-threatening events were reported. A longer follow-up is needed to assess long-term outcomes and safety.

1. Nistri R, et al. Anti-CD20 treatment in pre-pubertal onset paediatric MS patients. O050, ECTRIMS 2025 Congress, 24-26 September 2025, Barcelona, Spain.

Medical writing support was provided by Michiel Tent

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Posted on 26 September 202526 September 2025