Few Psychotropic Drugs Have Established Evidence in Autism

Megan Brooks

November 28, 2011

November 28, 2011 — Only a few psychotropic medications have an "established" evidence base for the treatment of symptoms associated with autism spectrum disorders (ASD); however, there is "preliminary" or "promising" evidence for a larger group of agents, new research suggests.

The evidence for most psychotropic medications for ASD is "thin, though increasing," Matthew Siegel, MD, medical director of the developmental disorders program at Spring Harbor Hospital, Maine Medical Center, in Westbrook, told Medscape Medical News.

"Part of our goal was to grade the research that is out there and give clinicians a tool to figure out how to use the evidence for existing agents to make better treatment decisions," he said.

Autism affects more than 1 in 110 American children, and it is estimated that up to three quarters of them suffer from a cooccurring mental illness, such as anxiety or attention deficit hyperactivity disorder. It is also estimated that 45% of children with ASD are prescribed psychotropic medication.

The research is published online November 9 in the Journal of Autism and Developmental Disorders.

Raising the Prescribing Bar

Dr. Siegel and his colleague, Amy A. Beaulieu, MSSW, systematically reviewed, synthesized, and rated the evidence from 33 randomized controlled trials, published in peer-reviewed journals, of psychotropic medications commonly prescribed to children with ASD.

The researchers found established evidence for only 3 medications — all of them antipsychotics.

Specifically, risperidone and aripiprazole have established evidence for the treatment of irritability and hyperactivity, haloperidol has established evidence for the treatment of negative behavioral symptoms, and aripiprazole also has established evidence for the treatment of stereotypy, the researchers note.

Six agents have "at least promising or preliminary evidence of benefit," Dr. Siegel said, including methylphenidate, which has a promising level of evidence for the treatment of hyperactivity associated with ASD.

Medications with preliminary evidence include naltrexone and atomoxetine for hyperactivity, risperidone for repetitive behavior and stereotypy, and pentoxifylline in combination with risperidone for irritability and social withdrawal.

"It is my hope that this article helps raise the bar for prescribers and directs them toward the evidence," Dr. Siegel said.

The paper includes a table that lists primary target symptoms, such as aggression and inattention, and the level of evidence for each drug.

"It's a good starting point for treatment decisions," Dr. Siegel said.

"Sobering" News

"The humbling or sobering news," he told Medscape Medical News, "is that we still have no medicines that treat the core features of autism — social/interaction and language impairments and repetitive behaviors. We really do not have anything at the moment that substantially affects any of those. The good news, however, is that the pace of research is really accelerating. Certainly one day we'd like to have a drug that treats some of the core symptoms."

Many of the studies, Dr. Siegel noted, are hampered by the fact that there currently is no validated tool to diagnose mental illness in children with autism.

"In other words, if you want to decide whether a child with autism has depression...there is no means to do so. That's certainly different than for adults or for children without autism; there is a knowledge gap there," he said.

The study was supported in part by a grant from the Pond Family Foundation and through a cooperative agreement between the Maine Department of Health and Human Services and the University of Southern Maine. The authors have disclosed no relevant financial relationships.

J Autism Dev Disord. Published online November 9, 2011. Abstract


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