NIMH Study Finds Anti-Psychotic Medication Useful in Treating Serious Behavioral Problems Among Children with Autism
Bethesda, MD, July 31, 2002 - 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
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 atypical anti-psychotic medications.
The study found risperidone to be significantly more effective than placebo
in improving behavior. Using a stringent definition of improvement, 69 per
cent of the children randomly assigned to risperidone were much or very much
improved at the end of the study, as compared with only 12 per cent 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
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
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
The following are the authors of this report listed by role and study site:
- 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;
- 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;
- 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.
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.
NIMH Press Office
Marilyn Weeks or Rayford Kytle