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NIMH Study Finds Anti-Psychotic Medication Useful in Treating Serious Behavioral Problems among Children with Autism

Press Release

One of a newer class of anti-psychotic medications was successful and well tolerated for the treatment of serious behavioral disturbances associated with autistic disorder in children ages 5 to 17. The findings of the large, multi-site, eight-week, placebo-controlled clinical trial, which was funded by the National Institute of Mental Health (NIMH), are being published this week in the New England Journal of Medicine.

"Although this study did not attempt to treat the core symptoms of autism, our findings suggest that risperidone can be useful in treating moderate to severe behavior problems that are associated with autism in children," said Lawrence Scahill, Ph.D., principal investigator at the Yale Child Study Center site.

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 behavioral disturbances, such as self-injury, aggression, hyperactivity, and tantrums in response to routine environmental demands. For these disturbances, behavioral therapy and medications are the two main forms of treatment.

In this multi-site study, researchers randomly assigned 101 children and adolescents, 82 boys and 19 girls, age 5 to 17, to receive either placebo or risperidone, one of a new class of anti-psychotic medications called atypical.

The study found risperidone to be significantly more 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.

Risperidone was well tolerated, with few neurological side effects. However, risperidone was associated with a substantial increase in body weight (an average of about a six-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 placebo-controlled study. Concerns about neurological and other side effects of haloperidol cause many clinicians to avoid its use in children.

The atypical anti-psychotic medications 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.

Few studies of atypical anti-psychotic medications have been conducted in children with autism. The primary goal of this study was to evaluate the efficacy and safety of risperidone, the first widely available atypical anti-psychotic medication, in children with autism accompanied by serious behavioral disturbances.

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:

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

Yale University was the coordinating center for the study. Additional funding was provided by the National Center for Research Resources (NCRR) at the National Institutes of Health (NIH).

More information on the trial and other autism research is available on the NIMH Clinical Trials.gov web site at http://www.clinicaltrials.gov/show/nct00005014  and at http://www.clinicaltrials.gov/search/term=autism .

NIMH is one of the 26 components that make up 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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About the National Institute of Mental Health (NIMH): The mission of the NIMH is to transform the understanding and treatment of mental illnesses through basic and clinical research, paving the way for prevention, recovery and cure. For more information, visit the NIMH website.

About the National Institutes of Health (NIH): NIH, the nation's medical research agency, includes 27 Institutes and Centers and is a component of the U.S. Department of Health and Human Services. NIH is the primary federal agency conducting and supporting basic, clinical, and translational medical research, and is investigating the causes, treatments, and cures for both common and rare diseases. For more information about NIH and its programs, visit the NIH website .