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.

Related Concept Videos

Drugs for Treatment of Crohn's Disease in IBD Using Immunomodulatory Agents01:29

Drugs for Treatment of Crohn's Disease in IBD Using Immunomodulatory Agents

Crohn's disease is an inflammatory bowel disorder marked by chronic inflammation of the GI tract. Various treatment strategies for Crohn's disease are employed, such as immunomodulatory agents, glucocorticoids, and biologics or anti-TNF therapy. Azathioprine (Imuran), a commonly used immunomodulatory drug for Crohn's disease, is converted in the body to mercaptopurine, which inhibits purine biosynthesis and cell proliferation. Both are utilized in severe cases of Inflammatory Bowel...
308
Drugs for Treatment of Crohn's Disease in IBD Using Glucocorticoids01:21

Drugs for Treatment of Crohn's Disease in IBD Using Glucocorticoids

Glucocorticoids, a class of anti-inflammatory drugs, are pivotal in treating moderate to severe Crohn's disease by inducing remission. They exhibit their anti-inflammatory action by inhibiting the production of inflammatory cytokines such as tumor necrosis factor (TNF)-α, interleukin (IL)-1, and chemokines like IL-8. In addition, they reduce the expression of inflammatory cell adhesion molecules and inhibit gene transcription of nitric oxide synthase, phospholipase A2, cyclooxygenase-2...
282
Drugs for Treatment of Crohn's Disease in IBD Using Biologic Agents: Anti-TNF01:24

Drugs for Treatment of Crohn's Disease in IBD Using Biologic Agents: Anti-TNF

Tumor Necrosis Factor (TNF), a proinflammatory cytokine, contributes significantly to the inflammation seen in Crohn's disease. It exists as soluble TNF and membrane-bound TNF, with actions mediated through TNF receptors (TNFR). TNFR activation leads to the release of proinflammatory cytokines, T-cell activation, collagen production, and leukocyte migration, all contributing to inflammation in Crohn's disease. Anti-TNF monoclonal antibodies, namely infliximab (Remicade), adalimumab...
285
Myasthenia Gravis: Overview and Treatment01:20

Myasthenia Gravis: Overview and Treatment

Myasthenia gravis is a neuromuscular transmission disorder characterized by weakness and increased fatigability of skeletal muscles. It is an autoimmune disease affecting approximately one in 2000 people, where antibodies against the α1 subunit of nicotinic acetylcholine receptors are produced.
These antibodies interfere with the function of the nicotinic receptors in three ways: by binding to the receptor and disrupting acetylcholine binding; by causing cross-linking of receptors which...
2.4K
Drug Dosing: Infants and Children01:29

Drug Dosing: Infants and Children

Pediatric patient dosages diverge from adults due to disparities in body surface area, total body water, and extracellular fluid per kilogram of body weight. The dosing regimen considers the variations in pharmacokinetics and pharmacology across distinct age groups, encompassing preterm newborns, infants, young children, older children, and adolescents. Calculation of pediatric patient doses is predicated on determining body surface area, which exhibits a superior correlation with the child's...
87