Sudhir Gupta, M.D., University of California, Irvine (Bridge Grant)
Autism is a disease of unknown cause. A number of treatment approaches have been used with varied success. A majority of these have been open trials (subject to personal bias). Earlier in an open study we reported that a subset of children with autism show a beneficial effect to the treatment with intravenous immune globulin (IVIG). In order to avoid any bias, we conducted a double-blind, placebo-controlled study to examine the effect of IVIG in autism. Twenty children were enrolled, and the study was divided into phases. In Phase 1, 12 children were randomly assigned with placebo and the other 12 with IVIG. Treatments were given at 4-week intervals for a total of 6 treatments. In Phase 2, those children who were receiving placebo treatments were switched to IVIG treatment, and those receiving IVIG treatment were switched to placebo. During Phase 2, children were treated for 6 treatments as in Phase 1. Psychometric analysis was done using number measurements and a large number of immunological evaluations were also done. As a group, there was no significant difference between the effect of placebo and IVIG. Some children did show improvement on IVIG; however the significance of improvement could not be demonstrated because of the small study size. A number of limitation were noted in the design of the study, which may have resulted in not reflecting true beneficial potential of IVIG in autism.