Improved Social and Behavioral Scores Following IVIG Therapy Suggest a Neuroimmune Etiology in Some Children with Autism

A number of case reports have documented the efficacy of intravenous immune globulin (IVIG) in children with autoimmune encephalopathy (AIE), and several studies have described improvements in autism-related characteristics following IVIG therapy. Now, in the largest case series of children with autism spectrum disorder (ASD) treated with IVIG, researchers identified brain-targeted autoantibodies in children with ASD. In the study, 82 children were screened for specific blood autoantibodies or other markers associated with AIE of which 49 of those whose autism-related behavioral and other symptoms were recommended a trial of IVIG.

Thirty-one children received varying monthly doses of IVIG, with the majority receiving 2.0 grams per kilogram of body weight or more. The majority of parents (90 percent) reported some improvement, with 71 percent reporting improvements in two or more symptoms. Statistically significant improvement was documented for the subset of patients whose caregivers completed the Aberrant Behavior Checklist (ABC) and/or Social Responsiveness Scale (SRS) questionnaires. The antidopamine D2L receptor antibody, anti-tubulin antibody and ratio of antidopamine D2L to D1 receptor antibodies were related to changes in the ABC.Changes in theCunningham autoantibody panel predicted SRS, ABC and parent survey-based treatment responses with good accuracy. Adverse effects of IVIG administration were common (62 percent) but mostly limited to the treatment period; only two (6 percent) patients discontinued IVIG because of adverse effects.

The investigators believe findings from this open-label case series provides evidence supporting a neuroimmune subgroup in patients with ASD.