Intrathecal Steroids: A More Targeted Approach to Central Nervous System Inflammation

ObjectiveDescribe the use of intrathecal (IT) steroid treatment in a cohort of pediatric neuroinflammatory diseases (PND). BackgroundIntravenous (IV) corticosteroids are a commonly used first line treatment in PND but some individuals have an inadequate response to treatment and require escalating immunotherapies. With this comes not only the risks and side effects of corticosteroids, but also that of further immunosuppressive treatments. Importantly, prior models have demonstrated poor central nervous system (CNS) penetrance of IV corticosteroids, which has led to use of intrathecal steroid treatments in some with refractory disease. Design/MethodsRetrospective chart review was performed of pediatric patients with neuroinflammatory diseases who were refractory to treatments and subsequently treated with IT dexamethasone. ResultsFour patients with PND who received greater than two doses of IT dexamethasone were identified. Diagnoses included new onset refractory status epilepticus (NORSE) secondary to anti-GAD encephalitis, febrile infection related epilepsy syndrome (FIRES), and lupus myelitis. All patients demonstrated at least one paraclinical marker of CNS inflammation on initial cerebral spinal fluid studies which included cell count, protein, neopterin, cytokines, and antibody levels. On subsequent sampling, all patients showed decreased neuroinflammatory markers with sequential administrations of IT dexamethasone. Additionally, in those with FIRES (2) and NORSE (1), patients demonstrated the ability to wean anesthetic infusions with ongoing seizure control without increase in maintenance anti-seizure medications. In a cumulative of 20 dose administrations, no adverse events were noted related to the medication administration and no reported infections. ConclusionsThis preliminary data demonstrates effectiveness of IT dexamethasone treatment in PND at decreasing neuroinflammation without adverse events. More data is needed to determine if this treatment may provide improved clinical outcomes in specific clinical circumstances. IT treatments may be an attractive option for patients who have other risk factors related to systemic immunosuppressive treatments or have shown inadequate response to standard of care therapies.