Anakinra Treatment for Refractory Cerebral Autoinflammatory Responses

Abstract BackgroundRefractory cerebral autoinflammatory-autoimmune diseases are often associated with dysregulated innate immunity, which are targeted by anakinra, an interleukin-1 receptor antagonist. Here, we analyzed the therapeutic effect of anakinra in refractory cerebral autoinflammatory response (CAIR).MethodsWe analyzed single institutional patients treated with anakinra for CAIR from January 2017 to May 2021. Anakinra was sympathetically used for patients with intractable CAIR at 100 mg/day subcutaneously. A good response was defined as any improvement of the modified Rankin Scale, Clinical Assessment Scale in Autoimmune Encephalitis, or Expanded Disability Status Scale.ResultsA total of twelve patients with various diagnostic etiologies were treated with anakinra (mean age=45.1; male=7). Four patients showed good responses, and eight patients had unclear responses. Among the good responders, 75% of the patients had pathologically demonstrated CAIR. The two very good responders had primary progressive multiple sclerosis and cerebral granulomatosis with polyangiitis in each, and microglia/macrophage infiltration was prominent in their brain biopsies. No patient had a serious adverse effect.ConclusionsAnakinra may be a therapeutic option for refractory cerebral autoinflammatory diseases with microglia and macrophage infiltrations.