Prioritising high efficacy therapies in children with MS

High-efficacy therapy (HET) as a first-line option in paediatric-onset MS resulted in sustained reduction of clinical and radiological disease activity over 5 years, compared with first choosing moderate-efficacy therapy (MET), in a study led by Dr Nail Benallegue (Nantes University, France). The results of the study prompt the authors to advocate HET as first choice in children with MS.

“The common thing to do in paediatric-onset MS is still to escalate therapy and thus to start with a MET” [1], argued Dr Benallegue. In a retrospective observational study (NCT02889965), Dr Benallegue and colleagues compared the real-world effectiveness of HET and MET on disease activity and evaluated treatment cessation [2]. They used data from 36 French MS expert centres participating in the French MS registry. The study population consisted of 530 treatment-naïve patients <18 years of age with relapsing-remitting MS who initiated MET (n=422) or HET (n=108) between 2010 and 2022. For data analysis, an innovative parametric time-to-event model was used to estimate the dynamics of event rate adjusting for confounders.

The results showed that after a median follow-up of 5.8 years, both HET and MET reduced the risk of first relapse during the first 2 years of treatment. Over a 5-year period, HET reduced this risk of first relapse by 54% compared with MET (adjusted HR 0.46; 95% CI 0.31–0.67; P<0.001). In these first 5 years, the annual relapse risk was 20% in the MET group and 8% in the HET group (see Figure). These results were confirmed by a 66% reduction of new T2 and gadolinium-enhancing lesions with HET at 2 years, measured with MRI in 300 patients (adjusted OR 0.34; 95% CI 0.18–0.66; P=0.001). There was no difference in Expanded Disability Status Scale (EDSS) score progression.

Figure: Primary outcome: time to first relapse [2]



HET, high-efficacy therapy; MET, moderate-efficacy therapy.

MET was associated with a 6-fold higher risk of discontinuation within 2 years (HR 5.97; 95% CI 2.92–12.20), mostly due to inefficacy or intolerance. At 5 years, 86.1% of patients had discontinued MET and 50.7% discontinued HET. Overall, a HET-strategy was sustained in 92 (85.2%) patients; 257 (60.9%) patients on MET switched to HET.

1. Henderson M, et al. JAMA Neurol. 2023;80:860–867.
2. Benallegue N, et al. Highly effective therapies as first-line treatments for pediatric onset multiple sclerosis in a French nationwide cohort. O141, MSMilan 2023, 11–13 October, Milan, Italy.

 

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Posted on 4 December 202325 March 2024