Early and active treatment with fingolimod for pediatric onset multiple sclerosis: the experience of an Italian pediatric center over the past 3 years.

Abstract

Aim: Describing the experience with early and high efficacy disease modifying treatment with fingolimod of a single Pediatric Multiple Sclerosis Center.

Method: We analyzed all pediatric multiple sclerosis (POMS) patients treated with fingolimod between May 2021 and April 2024. The study assessed the efficacy of fingolimod treatment defined as the absence of clinical relapses, disability progression, and new/enlarging or enhancing lesions on MRI. Cognitive profile has also been assessed at symptoms' onset and after one year of treatment.

Results: We included 6 patients (50% males) with average age at symptoms onset of 12.4 years and of 12.7 years at the diagnosis. Treatment with fingolimod was started on average 2.0 months (SD 1.7) after the diagnosis. The average treatment duration was 22.9 (SD 8.2) months and the average follow-up length 26.1 months (SD 8.9). None of the patients had clinical relapses during fingolimod treatment. Two patients showed new asymptomatic neuroradiological lesions after 18 months of treatment. All patients had normal cognitive profiles at first evaluation; four of them were tested after one year of treatment showing stable data.

Interpretation: Fingolimod offers better compliance and significantly improves patients' quality of life compared to injection therapies especially in pediatric population, reducing injection associated anxiety and risk of discontinuation. It appears to be safe and well tolerated and may be used as first line treatment in the highly active and aggressive disease course of pediatric onset multiple sclerosis.