Press Releases

NIMH Study Finds Additional Benefits of Antipsychotic Medication For Treating Behavioral Disturbance Among Children With Autism

New Haven, CT — July 5, 2005

Risperidone, one of a newer class of antipsychotic medications, was successful and well tolerated for up to 6 months in the treatment of children with autism accompanied by serious behavioral problems. In addition, the investigators observed a decrease in repetitive behavior. The study also showed that it was not necessary to increase the medication dose to maintain these improvements over the 6 month observation period. These findings were published this week in the American Journal of Psychiatry and a companion article in the June issue of the same Journal.

The findings extend results from an earlier clinical trial conducted by the same investigators (Research Units on Pediatric Psychopharmacology Autism Network, New England Journal of Medicine, 2002).

"These findings break new ground in the treatment of autism. For the first time, we have data on both the short- and longer-term benefits of medication for children with autism. The reduction in these serious behavioral problems may set the stage for the successful application of educational efforts in these children,” noted Lawrence Scahill, MSN, PhD who was the principal investigator at the Yale site. Dr. Scahill (in photo above), holds a joint appointment at the Yale School of Nursing and the Yale Child Study Center.

Autism is a chronic condition that appears in early childhood characterized by core symptoms of impaired social interaction, delayed language, and repetitive 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 often exhibit serious behavioral problems including 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 this multisite study, the researchers randomly assigned 101 subjects, 82 males and 19 females, age 5 to 17, to receive either placebo or risperidone. Sixty three subjects who improved after 8 weeks of treatment were followed for another 6 months. After 6 months of treatment, children were randomly assigned to stay on risperidone or to gradual withdrawal of the medication. During the discontinuation phase, clinicians and families were blind to treatment condition, which means that they did not know whether the specific child continued medication or was assigned to gradual withdrawal. The children were monitored weekly for the return of behavioral problems. Gradual discontinuation did indeed result in rapid return of target symptoms in most, but not all, cases.

This study is noteworthy in that it examined a question that many parents ask: “How long will my child have to be on medication?” The study results suggest that it is not unreasonable to consider discontinuation in a child with autism who has achieved enduring benefit after 6 months of treatment.

Risperidone was well tolerated, with no serious side effects, although weight gain was a concern in some cases.

In clinical practice, several medications are used in the treatment of autism, but few medications have been carefully studied. To date, only haloperidol has been shown to be superior to placebo for serious behavior problems in more than one placebo-controlled study. Concerns about neurological and other side effects of haloperidol cause many clinicians to avoid its use in children.

The atypical antipsychotics are of great interest in treating children with autism because studies have shown them to be beneficial in adults with schizophrenia, with fewer neurological side effects than the older medications.

This study is the largest study of an atypical antipsychotic medication in children with autism. The primary goal of this study was to evaluate the short- and longer-term efficacy and safety of risperidone in children with autism accompanied by serious behavioral problems.

The study was conducted by the Research Units of Pediatric Psychopharmacology (RUPP) Autism Network, which is funded by NIMH. This five site consortium is dedicated to the design and conduct of trials that provide clear direction to parents and clinicians on the risks and benefits of medications for children and adolescents with autism.

Authors of this report are listed by role and study site:

• Yale University, Principal Investigator, Lawrence Scahill, M.S.N., Ph.D., Co-Investigators Andres Martin, M.D., Kathleen Koenig, M.S.N., Fred Volkmar, M.D., Deirdre Carroll, M.S.N., Allison Lancor, B.S.
• University of California at Los Angeles, Principal Investigator James T. McCracken, M.D., Co-Investigators James McGough, M.D., Bhavik Shah, M.D., Pegeen Cronin, Ph.D., Daniel Hong, M.A.
• Ohio State University, Principal Investigator Michael G. Aman, Ph.D., Co-Investigators L. Eugene Arnold, M.ED., M.D., Ronald Lindsay, M.D., Patricia Nash, M.D., Jill Hollway, B.A.
• Indiana University, Principal Investigator Christopher J. McDougle, M.D., Co-Investigators David Posey, M.D., Naomi Swiezy, Ph.D., Arlene Kohn, B.A.
• Kennedy Krieger Institute, Principal Investigator Elaine Tierney, M.D., Co-Investigators Jaswinder Ghuman, M.D., Nilda M. Gonzalez, M.D., Marco Grados, M.D.
• National Institute of Mental Health, Principal Investigator Benedetto Vitiello, M.D., Co-Investigator Louise Ritz, M.B.A.
• Columbia University, Statistician, Mark Davies, M.P.H.
• Coordinating Center, Yale University
• Nathan Kline Institute, Data Management, James Robinson, M.E.D., Don McMahon, M.S.

NIMH is one of the 26 components that make up the National Institutes of Health (NIH), the Federal Government's primary agency for biomedical and behavioral research. NIH is part of the U.S. Department of Health and Human Services.

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