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Published on: August 9, 2024
Treatment of pediatric multiple sclerosis and variants
1Department of Pediatrics and Pediatric Neurology, Georg-August-University Goettingen, Germany.
Insights
Early treatment with disease-modifying therapies (DMT) benefits pediatric multiple sclerosis (MS) patients. While DMTs approved for adults are increasingly used in children, long-term safety and efficacy data are still needed.
Area of Science:
- Pediatric Neurology
- Neuroimmunology
- Clinical Therapeutics
Background:
- Multiple sclerosis (MS) significantly impacts adult patients, with early treatment improving prognosis.
- Currently, no disease-modifying therapies (DMTs) are specifically approved for pediatric MS.
- Existing guidelines for adult MS therapies do not extend to pediatric populations.
Purpose of the Study:
- To review current knowledge and experiences with DMTs in pediatric MS.
- To summarize safety and tolerability data of DMTs in children and adolescents with MS.
- To provide an overview of symptomatic MS therapies in pediatric patients.
Main Methods:
- Review of recent small cohort studies on DMT safety and tolerability in pediatric MS.
- Analysis of reported side effects for interferon beta (IFNB) and glatiramer acetate (GA) in pediatric patients.
- Synthesis of data on MS variants treated in pediatric centers.
Main Results:
- Reported side effects of IFNB and GA in pediatric MS appear comparable to adult data.
- Long-term tolerability, safety, and efficacy of DMTs in pediatric MS remain largely unestablished.
- Increasing use of adult-approved DMTs in children and adolescents with MS is noted.
Conclusions:
- Early initiation of DMTs is crucial for improving long-term outcomes in pediatric MS.
- Further research is essential to establish long-term safety and efficacy of DMTs in pediatric MS.
- Symptomatic treatments for pediatric MS also require further investigation.
Abstract:
Studies in adult patients with multiple sclerosis (MS) suggest significant benefit of early treatment initiation. However, there are no approved therapies for children and adolescents with MS. For adult MS, tolerability and efficacy of several immunomodulatory and immunosuppressive drugs have been demonstrated. Guidelines for the use of these MS therapies in children do not exist. Several small cohort studies of the safety and tolerability of disease-modifying therapies (DMT) in children and adolescents with MS have been recently reported. The side effects of interferon beta (IFNB) and glatiramer acetate (GA) appear to be similar to those reported by adults. The long-term tolerability and safety have yet to be established and efficacy data have yet to be studied. In view of the potential for significant long-term physical and cognitive disability in children with MS, and recent evidence that initiation of immunomodulatory therapy early in the course of MS improves long-term prognosis, an increasing number of children and adolescents with MS are being offered the DMT approved for adults. This review summarizes current knowledge of DMT in pediatric MS and experience in several centers treating pediatric MS and MS variants such as neuromyelitis optica or Devic disease, Balo concentric sclerosis, Marburg acute MS, and Schilder disease (myelinoclastic diffuse sclerosis). Finally, an overview of symptomatic MS therapies and experiences with these treatments in pediatric patients is provided.
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