Medication effective in treating children with autism

July 31, 2002

COLUMBUS, Ohio - A team of researchers has found that the drug risperidone, considered effective in treating adults with schizophrenia, is safe for children and reduces severe behavioral symptoms in youths with autism, according to a study published in this week's (8/1) New England Journal of Medicine.

The trial, the largest study to date examining medication options for children with autistic disorder, was conducted at five U.S. institutions, including The Ohio State University College of Medicine and Public Health.

The eight-week trial showed that 69 percent of participants showed a positive response to risperidone, an antipsychotic agent, compared to 12 percent of those taking placebo. Though increasingly sophisticated educational programs and behavior therapy are considered important elements of treatment for children with autism, the safety and effectiveness of medication is being analyzed for children with behaviors that can place particular strain on families, said Michael Aman, an Ohio State psychologist and lead OSU author of the study.

The finding holds promise in treating children with autism who have some of the most severe related behavioral disturbances. Most children with autism exhibit such characteristics as highly compulsive behavior, delayed language, learning challenges and a preference for isolation. Irritability, tantrums and aggression are not universal characteristics, but are "reasonably common," Aman said.

"Many of these kids present families with very serious challenges," he said. "Parents have to educate themselves about the disorder, and then access health care, occupational and speech therapy, special education and possibly applied behavioral analysis to help the children develop skills. Even then, the children may still present behavioral problems. That's a lot on your plate.

"Risperidone is popular in clinical circles, and this is one of those times when a drug's popularity is for a good reason," he said. "It was gratifying to see that the children taking the medication showed marked improvement in irritability, and even to show signs of clinically meaningful but smaller changes in other behaviors, such as overactivity and repetitive behaviors."

Children selected for the study were those exhibiting highly irritable behavior, including tantrums, sudden mood swings, self-injury and aggression. A total of 101 children between ages 5 and 17 participated in the trial across the five study sites; of those, 49 received risperidone and 52 received a placebo in the double-blind study.

Researchers used two widely employed measures to determine outcomes after eight weeks of treatment: the irritability subscale of the Aberrant Behavior Checklist and the rating on the Clinical Global Impressions - Improvement (CGI-I) scale. Clinicians assessing trial participants in weekly visits did not know which children were receiving the medication.

Treatment with risperidone resulted in a 57 percent reduction in the irritability score, compared with a 14 percent decrease in the placebo group. The overall positive response was 69 percent in the risperidone group compared with 12 percent in the placebo group. A positive response was defined as at least a 25 percent decrease in the irritability score combined with a rating of "much improved" or "very much improved" on the CGI-I scale.

In two-thirds of the children with a positive response, the benefit was maintained for six months.

Risperidone is one of a new generation of antipsychotic agents, and carries fewer side effects than previously used antipsychotic drugs, such as haloperidol, the only other antipsychotic drug shown in more than one study to effectively treat serious behavioral problems in children with autism. However, many clinicians avoid using haloperidol in children because of concerns about short- and long-term side effects.

Risperidone caused no serious side effects in children taking the drug, and most adverse effects were mild and short-term. Children most commonly experienced fatigue and increased appetite accompanied by weight gain. Some caretakers also reported mild tremor, rigidity,

difficulty swallowing, restlessness and spasms in children taking risperidone.

The trial was conducted by the Autism Network of the Research Units on Pediatric Psychopharmacology (RUPP). Study sites, in addition to Ohio State, were the University of California, Los Angeles, Indiana University, Yale University and the Kennedy Krieger Institute at Johns Hopkins University.

"This study network enabled five universities to pool their resources and locate a large number of children with the same conditions and carry out a protocol in a uniform way," said Aman, a professor of psychology and psychiatry in Ohio State's Nisonger Center for Mental Retardation and Developmental Disabilities. "The size of this trial gave us adequate power to detect even modest effects. It turned out that the effect was much more than modest, and was substantial for these irritable types of behaviors."

"The size of the sample also supports subgroup analyses to find out which children are most likely to respond," said L. Eugene Arnold, an Ohio State child psychiatrist who chaired the steering committee for the study and an OSU co-investigators in the multidisciplinary project. "Those analyses are currently being done and they should yield additional information valuable to clinicians who have to decide on a treatment plan," Arnold said.

The study was supported by contracts from the National Institute of Mental Health and the National Institutes of Health, with medication donated by Janssen Pharmaceutica.

Though the RUPP study findings apply only to children with autism who have severe behavioral disorders, Aman also is lead author of a study published in the American Journal of Psychiatry Aug. 1 showing that risperidone is effective in treating children with severe conduct problems. The 118 children participating in this trial were those with "disruptive behavior disorders" characterized by a chronic pattern of anti-social, maladaptive behavior that often involves impulsive aggression against property, people or animals; deceitfulness; and, sometimes, violation of laws. Participants who took risperidone showed substantial reductions in symptoms of aggression, tantrums and property destruction.

Aman and his Nisonger Center colleagues - Arnold, Ronald Lindsay and Yaser Ramadan - now are seeking participants for new research on hyperactivity in children with autism, and will participate in an upcoming multisite study of combined risperidone and behavior therapies for treatment of autism.

An estimated 20 children in 10,000 are affected by autism. Aman said individuals with autism are described as an "orphan" population because their relatively small number has resulted in little research being conducted in the past on the disorder. However, significant government support for autism research is helping to end those years of neglect, he said.

"The National Institute of Mental Health has identified autism as a significant child and adolescent condition that needs attention and has put significant support behind it," Aman said. "This is particularly meaningful for families living with children who have this condition."
The Ohio State University's academic medical center includes the College of Medicine and Public Health, University Hospitals, The James Cancer Hospital and Solove Research Institute, University Hospitals East, OSU & Harding Behavioral Healthcare and Medicine, the Dorothy M. Davis Heart and Lung Research Institute, a network of community care sites and the Richard M. Ross Heart Hospital, to be completed in 2004.

The OSU College of Medicine has highly regarded programs in research and physician training. It includes more than 1,000 faculty members, more than 800 students pursuing medical degrees and some 570 students enrolled in undergraduate and graduate programs.

Contact: Emily Caldwell, Medical Center Communications, 614-293-3737,

Ohio State University Wexner Medical Center

Related Autism Articles from Brightsurf:

Autism-cholesterol link
Study identifies genetic link between cholesterol alterations and autism.

National Autism Indicators Report: the connection between autism and financial hardship
A.J. Drexel Autism Institute released the 2020 National Autism Indicators Report highlighting the financial challenges facing households of children with autism spectrum disorder (ASD), including higher levels of poverty, material hardship and medical expenses.

Autism risk estimated at 3 to 5% for children whose parents have a sibling with autism
Roughly 3 to 5% of children with an aunt or uncle with autism spectrum disorder (ASD) can also be expected to have ASD, compared to about 1.5% of children in the general population, according to a study funded by the National Institutes of Health.

Adulthood with autism
The independence that comes with growing up can be scary for any teenager, but for young adults with autism spectrum disorder and their caregivers, the transition from adolescence to adulthood can seem particularly daunting.

Brain protein mutation from child with autism causes autism-like behavioral change in mice
A de novo gene mutation that encodes a brain protein in a child with autism has been placed into the brains of mice.

Autism and theory of mind
Theory of mind, or the ability to represent other people's minds as distinct from one's own, can be difficult for people with autism.

Potential biomarker for autism
A study of young children with autism spectrum disorder published in JNeurosci reveals altered brain waves compared to typically developing children during a motor control task.

Autism often associated with multiple new mutations
Most autism cases are in families with no previous history of the disorder.

State laws requiring autism coverage by private insurers led to increases in autism care
A new study led by researchers at the Johns Hopkins Bloomberg School of Public Health has found that the enactment of state laws mandating coverage of autism spectrum disorder (ASD) was followed by sizable increases in insurer-covered ASD care and associated spending.

Autism's gender patterns
Having one child with autism is a well-known risk factor for having another one with the same disorder, but whether and how a sibling's gender influences this risk has remained largely unknown.

Read More: Autism News and Autism Current Events is a participant in the Amazon Services LLC Associates Program, an affiliate advertising program designed to provide a means for sites to earn advertising fees by advertising and linking to