Related Experiment Videos
The Use of Natalizumab in Pediatric Patients With Active Relapsing Multiple Sclerosis: A Prospective Study
Raed Alroughani1, Samar Farouk Ahmed2, Raed Behbehani3
1Division of Neurology, Amiri Hospital, Sharq, Kuwait; Neurology Clinic, Dasman Diabetes Institute, Dasman, Kuwait.
Insights
Natalizumab significantly reduced relapse rates and MRI activity in pediatric multiple sclerosis (MS) patients with aggressive disease. This treatment demonstrated a comparable safety and efficacy profile to that observed in adults with MS.
Area of Science:
- Neurology
- Immunology
Background:
- Pediatric multiple sclerosis (MS) is increasingly recognized.
- Current treatment strategies rely on adult data due to a lack of approved therapies for children.
- Natalizumab is used off-label for pediatric MS, necessitating efficacy and safety evaluation.
Purpose of the Study:
- To evaluate the effectiveness and safety of natalizumab in pediatric patients with MS.
- To assess natalizumab's impact on clinical relapse rates and disability progression.
- To determine the effect of natalizumab on radiological disease activity in pediatric MS.
Main Methods:
- A prospective study of pediatric MS patients treated with natalizumab was conducted using national MS registry data.
- Analysis included relapsing patients with at least one year of follow-up.
- Outcomes measured included annual relapse rate, change in Expanded Disability Status Scale (EDSS), and proportion of patients with MRI activity.
Main Results:
- Thirty-two pediatric MS patients received natalizumab for at least 12 months, with 66% having breakthrough disease on prior therapies.
- Natalizumab significantly reduced the annual relapse rate (1.66 to 0.06, P < 0.001) and improved EDSS scores (3.3 to 2.2, P < 0.001).
- Radiological activity decreased significantly, with the proportion of patients showing gadolinium-enhancing or new T2 lesions dropping from 93.8% to 12.5% (P < 0.001). No major adverse events were reported.
Conclusions:
- Natalizumab effectively reduced clinical and radiological disease activity in a cohort of pediatric MS patients with aggressive or breakthrough disease.
- The drug exhibited a similar clinical efficacy and safety profile in pediatric MS as observed in adult MS populations.
- These findings support the use of natalizumab in pediatric MS patients refractory to first-line treatments.
Background:
Pediatric multiple sclerosis (MS) has been increasingly recognized. In the absence of approved disease-modifying therapies (DMTs) for pediatric patients, clinicians resort to data extrapolated from clinical trials conducted in adults with MS. The objective of this article was to study the effectiveness and safety of natalizumab in with pediatric MS.
Methods:
Patients with pediatric MS (aged less than 18 years) who had been treated with natalizumab were followed up prospectively as part of the national MS registry. Data of relapsing patients who had at least a one-year follow-up were analyzed. The primary outcome measure was the annual relapse rate after natalizumab treatment. Secondary outcomes measures included the mean change in disease progression measured by the expanded disability status scale and the proportion of patients with radiologic activity (gadolinium-enhancing or new T2 lesions) at the last follow-up visit.
Results:
Thirty-two patients with pediatric MS had been treated with natalizumab for at least 12 months, of whom 72% were females. The 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 disease-modifying therapies. The mean number of natalizumab infusions was 34.5 ± 18. The annual relapse rate was significantly reduced (1.66 ± 0.5 vs 0.06 ± 0.25; P < 0.001), whereas the mean expanded disability status 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 magnetic resonance imaging activity was significantly reduced (93.8% versus 12.5%; P < 0.001). No major adverse events were observed.
Conclusion:
In our pediatric MS cohort with aggressive or breakthrough disease, treatment with natalizumab was effective in reducing clinical and radiologic disease activity. Natalizumab has a similar clinical efficacy and safety profile as in adult MS.