The use of natalizumab in active relapsing pediatric Multiple Sclerosis patients: A prospective study

Publication date: Available online 2 February 2017
Source:Pediatric Neurology
Author(s): R. Alroughani, S.F. Ahmed, R. Behbehani, J. Al-Hashel
BackgroundPediatric MS has been increasingly recognized. In the absence of approved disease modifying therapies (DMTs) for pediatric cohorts, clinicians resort data extrapolated from clinical trials conducted in adult MS patients.ObjectivesTo study the effectiveness and safety of natalizumab in pediatric MS patients.MethodsPediatric (aged < 18 years) MS patients who had been treated with natalizumab were followed prospectively as part of the national MS registry. Data of relapsing patients who had at least 1-year follow-up data were analyzed. The primary outcome measure was the annual relapse rate (ARR) post natalizumab treatment. Secondary outcomes measures included the mean change in disease progression measured by Expanded Disability Status Score (EDSS) and the proportion of patients with radiological activity (gadolinium-enhancing or new T2 lesions) at the last follow-up visit.ResultsThirty-two pediatric MS patients had been treated with natalizumab for at least 12 months; of whom 72% were females. Mean age at onset and disease duration were 14.9 ±2.6 and 5.1 ±3.1 years respectively. Most patients (n=21, 66%) had breakthrough disease on first line DMTs. Mean number of natalizumab infusions was 34.5 ±18. The ARR was significantly reduced (1.66±0.5 vs. 0.06±0.25; p < 0.001) while the mean EDSS score improved (3.3± 1.3 vs. 2.2 ±1.0; p < 0.001) at the last follow-up visits. The proportion of patients with MRI activity was significantly reduced (93.8% vs. 12.5%; p <0.001). No major adverse events were observed.ConclusionIn our cohort of patients with pediatric MS who had aggressive or breakthrough disease, treatment with natalizumab was effective in reducing clinical and radiological disease activity. Natalizumab has a similar clinical efficacy and safety profile as in adult MS.



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