A PLACEBO-CONTROLLED TRIAL OF ORAL HUMAN IMMUNOGLOBULIN FOR GASTROINTESTINAL DYSFUNCTION IN CHILDREN WITH AUTISTIC DISORDER

Background: Persistent gastrointestinal (GI) symptoms have been reported in subgroups of children with autistic disorder (AD), although it is unknown if a causal relationship exists between GI dysfunction and the expression of symptoms of autism. Currently, there is no specific treatment for these patients. Intravenous immunoglobulin (IVIG) is already approved in the US for the treatment of a range of immune-mediated conditions based on its anti-inflammatory and immunological properties. In an open-label prospective study of the use of oral human immunoglobulin (IGOH), GI symptoms along with behavioral symptoms in children with AD improved. Those results supported the need for a controlled study.

Objectives: We investigated whether a 12-week treatment with IGOH would improve GI symptoms in children aged 2–18 years with AD.

Methods: A randomized, double-blind, placebo-controlled, parallel-group, dose-ranging study (IGOH 140, 420, or 840 mg/day) was conducted with 100 subjects completing the study.

Results: Analysis of primary and secondary endpoints revealed no difference across treatment groups in GI symptoms (p =.52), parent ratings of problem behaviors (p >.20), or parent or physician-assessments of global improvements. IGOH was well-tolerated and there were no treatment-emergent serious adverse events.

Conclusions: This study challenges some assertions regarding the etiology of autism and emphasizes the need for rigorous scientific investigation.