Kennedy Krieger Institute and NIMH Study Finds an Anti-Psychotic Medication Useful in Treating Behavioral Disturbances in Children with Autism

August 1, 2002
Findings Published in the New England Journal of Medicine

BALTIMORE, MD - Researchers at Kennedy Krieger Institute/Johns Hopkins Hospital and seven other research facilities around the country have found that one of a newer class of anti-psychotic medications was successful and well-tolerated for the treatment of serious behavioral disturbances associated with autism in children ages 5 to 17. The findings of the eight-week, placebo-controlled, clinical trial were published today in the New England Journal of Medicine. "Risperidone, one of a new class of anti-psychotics called atypical neuroleptic, can significantly decrease many of the severe behavioral disturbances that may, at times, be seen with individuals who have autism," said Elaine Tierney, M.D., director of the Department of Psychiatry at Kennedy Krieger Institute. "While many individuals with autism do not have behavioral problems, such as aggression and self-injury, risperidone might help those who have such difficulties." Autism is a chronic condition that appears in early childhood and is characterized by core symptoms, including impaired social relatedness, delayed language and restricted patterns of behavior. It affects as many as one in 500 children. Although the causes of autism are unknown in most cases, available evidence implicates abnormalities in brain development. Twin and family studies indicate a strong genetic contribution. Approximately 5 to 10 percent of children with autism have severe behavior problems, which can include self-injury, aggression and tantrums, in response to routine environmental demands. At Kennedy Krieger, approximately half of the children with autism who are treated exhibit this behavior. Behavior therapy and medications are the two main forms of treatment.

"At Kennedy Krieger, the behavioral psychologists, neurologists, developmental pediatricians, speech and language therapists, social workers, occupational therapists, psychiatrists and other specialists work together to learn about the various aspects and situations in which this behavior occurs. We then find ways to treat those behaviors," Dr. Tierney says.

Researchers randomly assigned 101 subjects, 82 males and 19 females, ages 5 to 17, to receive either risperidone or placebo. 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 was 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 of a medication ever observed in a study of children with autism.

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

Several medications previously have been used 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 former types of 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 who have serious behavioral disturbances.

The study was conducted at eight sites of the Research Units of Pediatric Psychopharmacology (RUPP) network, which is funded by the National Institute of Mental Health (NIMH), with patients seen at five of the sites. 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:

  • Kennedy Krieger Institute, Principal Investigators Elaine Tierney, M.D., Co-Investigators Jaswinder Ghuman, M.D., Nilda M. Gonzalez, M.D., Marco Grados, M.D.;
  • 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.;
  • 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.;
  • 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.;
  • Nathan Kline Institute, Data Management James Robinson, M.E.D., Don McMahon, M.S.

More information on the trial is available on the NIMH Clinical Trial's web site: http://www.clinicaltrials.gov/ct/gui/c/a1r/show/NCT00005014?order=2&JSer...

Kennedy Krieger Institute is dedicated to helping children and adolescents with disabilities resulting from disorders of the brain achieve their potential and participate as fully as possible in family, community and school life. For more information, visit us on the web at www.kennedykrieger.org.

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