An Update on the Treatment of Pediatric Autoimmune Encephalitis

Autoimmune encephalitis (AE) is an increasingly recognized etiology for neuropsychiatric deficits that are highly responsive to immunotherapy. As a result, rheumatologists are often called upon to help with the diagnosis and treatment of these conditions. The purpose of this review is to provide an update on the pharmacologic treatment of AE.

To date, there are no prospective randomized placebo-controlled trials to guide treatment recommendations for AE. First-line therapies include corticosteroids, intravenous immunoglobulin, and plasma exchange. Second-line therapies include rituximab and cyclophosphamide (CYC), as well as mycophenolate mofetil and azathioprine. For patients refractory to both first- and second-line therapy, there is emerging evidence for the interleukin-6 (IL-6) inhibitor tocilizumab, the proteasome inhibitor bortezomib, and low-dose IL-2. Early treatment initiation and treatment escalation in patients with refractory disease improve outcomes. Given the delayed time between dosing and treatment effects of second-line agents, continuing first-line treatment until the patients shows improvement is recommended.

Although AE can present with dramatic, life-threatening neuropsychiatric deficits, the potential for recovery with prompt treatment is remarkable. First- and second-line therapies for AE lead to clinical improvement in the majority of patients, including full recoveries in many. Early treatment and escalation to second-line therapy in those with refractory disease improves patient outcomes. Novel treatments including IL-6 blockade and proteasome inhibitors have shown promising results in patients with refractory disease.