MONDAY, April 4 (HealthDay News) -- Little evidence supports the
use of most medications for treating autism in children, with the
exception of the anti-psychotic drugs risperidone and aripiprazole
that have shown success in controlling severely disruptive,
hyperactive and repetitive behaviors, a new study shows.
However, anti-psychotics can have significant side effects,
including substantial weight gain, sedation and tremors or
rigidity, and should only be used when absolutely necessary, the
"The medications are efficacious, but they have significant side effects that should limit their use to patients with severe impairment or at risk of injury," said study author Melissa McPheeters, co-director of the Vanderbilt Evidence-Based Practice Center in Nashville.
Other medications sometimes prescribed for children with autism
-- including selective serotonin reuptake inhibitors (SSRIs) to
treat repetitive behaviors and stimulants used to treat
hyperactivity and impulsive behavior -- have scant evidence
supporting their use, according to the study. It was published
online April 4 in the journal
A randomized, controlled trial on SSRIs showed no benefit in
controlling repetitive behaviors, according to the study. And while
one study did show that stimulants may help with hyperactivity, far
more research is needed.
A second study in the same issue of the journal analyzed seven
randomized controlled trials on secretin, which is used to treat
peptic ulcers, and the study found no benefit in treating autism
symptoms and that it wasn't even worth further study. (Animals
studies have showed secretin affects the central nervous system,
which sparked the interest in using the drug for children with
autism, but those hopes have not been borne out, the researchers
A third study in the journal looked at 34 studies on behavioral
interventions in preschoolers. The research generally shows such
interventions help children improve their language and social
skills, but most studies were poorly designed, and more research is
needed, the authors of the new study said.
"We see a real variability in response, with some subgroups having dramatically positive effects and some children remaining very impaired," McPheeters said.
An estimated one in 110 U.S. children have an autism spectrum
disorder, which is often marked by problems with language, social
and communication skills, and repetitive behaviors.
But the condition can vary widely among individuals, with some
people never developing the ability to speak and others going on to
"This trio of review articles is important in that they remind parents and professionals how difficult it is to effectively treat children with autism, how little research there is to support some of our medical and developmental interventions and how daunting it is to conduct well-designed clinical research effectively," said Dr. Andrew Adesman, chief of developmental and behavioral Pediatrics at Steven and Alexandra Cohen Children's Medical Center of New York.
Children with autism also often have other conditions, such as
aggressive or disruptive behaviors, seizure disorders and
hyperactivity. No medications target the communication issues or
problems with social functioning that define autism itself, experts
said. Instead, physicians prescribe drugs to try to ease those
"The drugs are used to treat co-morbid symptoms, not the core autism symptoms," McPheeters explained.
In the review, the researchers looked at studies conducted
between 2000 and 2010 on the use of various drugs in children aged
12 and under with an autism spectrum disorder. The study on
medications included nine studies on anti-psychotics, five on SSRIs
and four on stimulants.
While the evidence for anti-psychotics is strong, far more
research is needed on newer anti-psychotic drugs that may have
fewer side effects, McPheeters said.
As for the early behavioral interventions, "we have a small
number of studies that suggest positive outcomes, but we are in
critical need of replication, extension and controlled studies,"
She added: "Even though the paper says there is low and
insufficient strength of evidence, that should not be interpreted
as the interventions are not effective, but as encouragement for
The three studies were part of an 18-month long project done in
conjunction with the Agency for Healthcare Research and Policy to
review all of the evidence on treatments for autism spectrum
disorders for children 12 and under published after 2000, said
McPheeters, the senior author of the report that will be published
soon on the AHRQ Web site.
That report started with more than 4,000 abstracts and was
narrowed down to 183 studies in all
For the medication study, studies had to include at least 30
people, while for the behavioral study, studies had to include at
least 10 people.
"The report includes studies that are very rigorous and also studies that we rated to have poor quality," McPheeters said. "We felt like it was important to make sure we covered the waterfront so that people could see what all was out there."
Another challenge for physicians is determining which children
will respond best to which treatment, said Dr. Eric Hollander,
director of the Compulsive, Impulsive and Autism Spectrum Disorders
Program at Montefiore Medical Center in New York City.
"There is no single treatment that works for all patients," Hollander said.
To learn more, visit