NIMH Study Finds Anti-Psychotic Medication Useful In Treating Behavioral Disturbance Among Children With Autism

INDIANAPOLIS - Promising results from a multi-center clinical trial for a medication for children with autism will be published Aug. 1 in the New England Journal of Medicine. The Indiana University School of Medicine Department of Psychiatry was one of the five sites testing the drug, which is one of a newer class of anti-psychotic medications.

“The results are encouraging because few medications have shown to be as promising or effective in the treatment of autism,” said Christopher McDougle, M.D., the Albert Eugene Sterne Professor of Clinical Psychiatry and chairman of psychiatry at the IU School of Medicine, a co-author of the NEJM article.

The medication, risperidone, was successful and well tolerated for the treatment of serious behavioral disturbance associated with autistic disorder in children ages 5 to 17. The positive findings are from a multi-site, eight-week, placebo-controlled clinical trial funded by the National Institute of Mental Health (NIMH).

"The findings suggest that risperidone can be useful in treating moderate to severe behavior problems that are associated with autism in children," said Dr. McDougle, who also is executive director of the Institute of Psychiatric Research, and director of the Section of Child Psychiatry at IU.

Autism is a chronic condition that appears in early childhood and is characterized by core symptoms of impaired social relatedness, delayed language and restricted patterns of behavior. It affects as many as 20 children per 10,000. Although the causes of autism are unknown for most cases, available evidence implicates abnormalities in brain development. Twin and family studies indicate a strong genetic contribution.

In addition to core symptoms, children with autism frequently exhibit serious behavior disturbances, such as self-injury, aggression and tantrums in response to routine environmental demands. For these disturbances, behavior therapy and medications are the two main forms of treatment.

In the risperidone clinical trial, researchers randomly assigned 101 participants (82 males and 19 females, age 5 to 17) to receive either placebo or the study medication, one of a new class of anti-psychotics called atypical.

The study found risperidone to be more significantly effective than placebo in improving behavior. Using a stringent definition of improvement, 69 percent of the children randomly assigned to risperidone were much or very much improved at the end of the study, as compared with only 12 percent in the placebo group.

This is the largest positive effect by a medication ever observed in children with autism, claim investigators.

Risperidone was in general well tolerated, with few neurological side effects. However, risperidone was associated with a substantial increase in body weight (an average of about 6 pound increase in the 8-week period).

Several medications have been used previously to treat autism with limited success. To date, only haloperidol has been shown to be superior to placebo for serious behavior problems in more than one study. Concerns about neurological and other side effects of haloperidol cause many clinicians to avoid its use in children.

The atypical anti-psychotics are of great interest in treating children with autism because studies have shown them to be beneficial to adults with schizophrenia, with fewer neurological side effects than other previous medications.

Few studies of atypical anti-psychotics as treatments for children with autism have been published. The primary goal of this study was to evaluate the efficacy and safety of risperidone, the first widely available atypical, in children with autism accompanied by serious behavioral disturbance.

The study was conducted at five sites of the Research Units of Pediatric Psychopharmacology (RUPP) network, which is funded by NIMH. The RUPP network is composed of research units devoted to conducting studies to test the efficacy and safety of medications commonly used by practitioners to treat children and adolescents (off-label use) but not yet adequately tested.

The following are the authors of this report listed by role and study site:

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