Immunotherapy for autoimmune autonomic ganglionopathy

Purpose of review: To provide an update on recent advances in the treatment of autoimmune autonomic ganglionopathy (AAG). AAG is an immune-mediated disorder characterized by prominent and selective involvement of autonomic nerve fibers or ganglia. Treatment with intravenous immunoglobulin (IVIG) or plasma exchange (PE) has been reported to be effective in single case reports and recent case series. This review summarizes current treatment options including IVIG, PE, and immunosuppressants agents, alone or in combination. Results: Controlled clinical trials of immunotherapy are not available and experience with immunotherapy is confined to single case reports. Some patients with AAG seem to respond to IVIg or PE as initial therapy but often require subsequent immunosuppressive treatment to sustain or advance the improvement. Preliminary studies suggest that the most severely affected patients may need a protracted and combined treatment program to achieve clinical improvement. Summary: Several recent studies suggest that IVIg, PE, and immunosuppressant agents are effective treatment for AAG. Current available treatments used as monotherapy or in combination may be efficacious and the specific regimen may depend on the severity of autonomic failure. There is preliminary evidence that novel immunosuppressant agents such as Mycophenolate mofetil (MMF) and Rituximab may be effective in certain patients who are unresponsive to IVIG or PE.