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Current therapeutic options in pediatric multiple sclerosis
1Department of Neurology, Division of Child Neurology, Pediatric MS Center of the JNI, SUNY Buffalo, Women and Children's Hospital of Buffalo, 219 Bryant St., Buffalo, NY, 14222, USA, yehann1@gmail.com.
Insights
Pediatric multiple sclerosis (MS) management involves disease-modifying therapies (DMTs) and symptom control. First-line injectable DMTs are recommended, with oral agents still under investigation for pediatric use.
Area of Science:
- Pediatric Neurology
- Neuroimmunology
- Clinical Therapeutics
Background:
- Relapsing-remitting pediatric multiple sclerosis (MS) requires comprehensive management strategies.
- Current therapies focus on preventing relapses, treating acute attacks, and managing symptoms.
- Limited data exists for novel oral agents in the pediatric MS population.
Purpose of the Study:
- To outline current therapeutic approaches for relapsing-remitting pediatric MS.
- To discuss the selection and sequencing of disease-modifying therapies (DMTs).
- To address the management of acute relapses and associated symptoms.
Main Methods:
- Review of established and emerging treatment guidelines for pediatric MS.
- Analysis of first-line, second-line, and potential future therapeutic options.
- Consideration of adjunctive therapies for symptom management and rehabilitation.
Main Results:
- First-line DMTs for pediatric MS include injectable interferon beta (1a IM, 1a SC, 1b SC) and glatiramer acetate.
- Second-line options like natalizumab, cyclophosphamide, and mitoxantrone require careful consideration due to potential serious side effects.
- Oral agents (fingolimod, cladribine) show promise in adults but lack pediatric data; acute relapses may be treated with pulse methylprednisolone.
Conclusions:
- Therapeutic decisions in pediatric MS should prioritize safety and efficacy, starting with established injectable DMTs.
- Close monitoring for adverse effects is crucial, especially with potent second-line therapies.
- Multidisciplinary care, including neuropsychological and physical therapy, is essential for managing cognitive and physical symptoms.
Opinion Statement:
Therapies for relapsing-remitting pediatric multiple sclerosis (MS) are aimed at preventing relapses (disease modifying therapies), treating acute attacks, and managing disabling cognitive and physical symptoms. Initial disease modifying therapy to prevent relapses should use one of four first-line injectable therapies that are approved for adult relapsing-remitting MS: interferon beta 1a IM, interferon beta 1a SC, interferon beta 1b SC, or glatiramer acetate. If breakthrough disease occurs or the medication is poorly tolerated, the next step should be to try one of the other first-line therapies. If the first-line therapies have been exhausted, second-line therapies such as natalizumab, cyclophosphamide, or mitoxantrone may be considered. One must use caution when choosing these potent therapies, as secondary effects may include serious infection or malignancy. Phase III studies in adult MS have been published on two oral agents, fingolimod and cladribine, and fingolimod has received FDA approval for use in relapsing-remitting MS in adults. These drugs have not been evaluated in the pediatric MS population, nor have any of three other oral agents now in phase III development: laquinimod, BG-12, and teriflunomide. Acute relapses can be treated with pulse methylprednisolone at a dosage of 20 to 30 mg/kg per day (maximum 1 g per day) for 3 to 5 days. If this is ineffective, intravenous immunoglobulin (2 g/kg divided over 2-5 days) or plasmapheresis may be considered. Neuropsychological, physical therapy, and occupational therapy screening should be performed on patients with pediatric MS. Interventions focusing on visual motor integration may be particularly useful in this group Spasticity may be treated with symptomatic therapies, but one must be aware of potential adverse effects of agents such as baclofen and diazepam. Headache, fatigue, anxiety, and depression are frequently seen, and patients may need a psychiatry consultation and counseling.
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