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Long-term fingolimod treatment in two pediatric patients with multiple sclerosis
Giovanna Borriello1, Carlo Pozzilli2
1Sant'Andrea Hospital, Rome, Italy. Giovanna.borriello@gmail.com.
Insights
Early treatment with fingolimod is effective for pediatric multiple sclerosis (POMS). Long-term data show sustained clinical benefit and safety in children with POMS, regardless of prior therapy.
Area of Science:
- Neurology
- Pediatric Neurology
- Multiple Sclerosis Research
Background:
- Pediatric-onset multiple sclerosis (POMS) requires early intervention with disease-modifying therapies (DMTs) to mitigate progression.
- The PARADIGMS trial established fingolimod's efficacy over interferon beta-1a in children with POMS over 2 years.
- Long-term safety and efficacy data for fingolimod in pediatric patients were previously limited.
Observation:
- Two cases of POMS patients treated with oral fingolimod beyond 2 years are presented.
- The first patient, treatment-naïve, remained stable for 5 years with no relapses or new lesions.
- The second patient, previously treated with interferon beta-1a, experienced no relapses in over 2 years of fingolimod treatment with stable MRI findings.
Findings:
- Prolonged fingolimod treatment demonstrated sustained clinical benefit in both pediatric patients.
- No relapses or radiological progression were observed during extended fingolimod therapy.
- Fingolimod proved effective as both a first-line and second-line treatment option.
Implications:
- These findings support the long-term safety and efficacy of fingolimod for managing POMS.
- Extended fingolimod therapy can offer sustained disease control in pediatric multiple sclerosis patients.
- This case series contributes valuable real-world evidence for extended fingolimod use in POMS.
Abstract:
Data suggest that patients with pediatric-onset multiple sclerosis (POMS) should initiate treatment with a disease-modifying therapy early to slow progression. The PARADIGMS trial demonstrated that oral fingolimod reduced the annual rate of relapse by 82% compared with intramuscular interferon beta-1a in children with POMS. The PARADIGMS study had a follow-up of 2 years, but no data are available about the safety and efficacy of fingolimod for longer periods in children with POMS. Here we present two cases of children with POMS who achieved sustained clinical benefit from treatment with fingolimod for more than 2 years. The first patient, an 11-year-old male, who participate in the PARADIGMS study, was treatment naïve at the time of fingolimod initiation. His clinical condition remained stable over 5 years of treatment, with no relapses and no radiological lesion progression. The second patient was a female who initiated fingolimod at the age of 12 years, 2 years after her POMS diagnosis and after an 8-month trial of interferon beta-1a. The patient had experienced two relapses during interferon beta-1a but had no relapses in more than 2 years of treatment with fingolimod, and her MRI scans showed no new or active lesions. These data show that prolonged treatment with fingolimod can be safe and effective during long-term treatment as first- or second-line therapy in children with POMS.
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