Faculty Bibliography

Background and aims: Approximately 3-5% of Multiple Sclerosis (MS) cases manifest in childhood and adolescence, characteristically with highly active inflammatory disease course. Paediatric-onset MS (POMS) has an impact on brain integrity and may increase Brain Volume Loss (BVL) above age-expected rates. This study assessed the effect of oral Fingolimod up to 0.5mg daily versus intramuscular interferon (IFN) beta-1a 30mug once weekly on MRI outcomes in POMS patients. Methods: In this double-blind, double-dummy, activecontrolled, multicentre study, patients with POMS (aged 10-<18 years) received either Fingolimod (dose adjusted for body weight; N=107) or IFN beta-1a (N=107) for up to 2 years. MRI was performed at baseline and every 6 months until the End Of The Study (EOS) core phase. Key MRI outcomes were the number of new/newly enlarging T2 (n/ neT2) lesions and Gd-enhancing T1 (Gd+T1) lesions, Annual Rate Of Brain Volume Change (ARBVC), annualised rate of number of new T1 hypointense lesions, change in total T2 Hyperintense Lesion Volume (T2LV) and the number of Combined Unique Active Lesions (CUAL). Results: At the EOS, compared with IFN beta-1a, fingolimod significantly reduced the annualised rate of n/ neT2 lesions (52.6%; p<0.001), number of Gd+T1 lesions per scan (66.0%; p<0.001), ARBVC (-0.48% vs. -0.80%, p=0.014), annualised rate of number of new T1 hypointense lesions (62.8%; p<0.001), T2LV (percent change from baseline: 18.4% vs. 32.4%, p<0.001) and CUAL per scan (60.7%; p<0.001). Conclusion: Fingolimod significantly reduced MRI activity and slowed BVL for up to 2 years vs. IFN beta-1a in paediatric-onset MS