Medications are often used to treat behavioral problems--such as aggression, self-injurious behavior, and severe tantrums--that keep the person with autism spectrum disorders (ASD) from functioning more effectively at home or school. The medications used are those that have been developed to treat similar symptoms in other disorders. Many of these medications are prescribed "off-label." This means they have not been officially approved by the U.S. Food and Drug Administration (FDA) for use in children, but the doctor prescribes the medications if he or she feels they are appropriate for your child. Further research needs to be done to ensure not only the efficacy but the safety of psychotropic agents used in the treatment of children and adolescents.

A child with ASD may not respond in the same way to medications as typically developing children. It is important that parents work with a doctor who has experience with children with autism. A child should be monitored closely while taking a medication. The doctor will prescribe the lowest dose possible to be effective. Ask the doctor about any side effects the medication may have and keep a record of how your child responds to the medication. It will be helpful to read the "patient insert" that comes with your child's medication. Some people keep the patient inserts in a small notebook to be used as a reference. This is most useful when several medications are prescribed.

Anxiety and Depression

The selective serotonin reuptake inhibitors (SSRI's) are the medications most often prescribed for symptoms of anxiety, depression, and/or obsessive-compulsive disorder (OCD). The SSRIs fluoxetine and sertraline are approved by the FDA for children age 7 and older with obsessive-compulsive disorder. Fluoxetine is also approved for children age 8 and older for the treatment of depression.

Two others that have been approved for OCD are fluvoxamine, age 8 and older; and clomipramine, age 10 and older.¹ Treatment with these medications can be associated with decreased frequency of repetitive, ritualistic behavior and improvements in eye contact and social contacts.

Despite the relative safety and popularity of SSRIs and other antidepressants, some studies have suggested that they may have unintentional effects on some people, especially adolescents and young adults. In 2004, after a thorough review of data, the FDA adopted a "black box" warning label on all antidepressant medications to alert the public about the potential increased risk of suicidal thinking or attempts in children and adolescents taking antidepressants. In 2007, the agency extended the warning to include young adults up to age 25. A black box warning is the most serious type of warning on prescription drug labeling. The warning emphasizes that children, adolescents, and young adults taking antidepressants should be closely monitored, especially during the initial weeks of treatment, for any worsening depression, suicidal thinking or behavior, or any unusual changes in behavior such as sleeplessness, agitation, or withdrawal from normal social situations.

Behavioral Problems

Antipsychotic medications have been used to treat severe behavioral problems. These medications work by reducing the activity in the brain of the neurotransmitter dopamine. Among the older, typical antipsychotics, such as haloperidol, thioridazine, fluphenazine, and chlorpromazine, haloperidol was found in more than one study to be more effective than a placebo in treating serious behavioral problems.² However, haloperidol, while helpful for reducing symptoms of aggression, can also have adverse side effects, such as sedation, muscle stiffness, and abnormal movements.

Placebo-controlled studies of newer "atypical" antipsychotics were being conducted on children with autism in recent years. The first such study, conducted by the National Institute of Mental Health (NIMH)-supported Research Units on Pediatric Psychopharmacology (RUPP) Autism Network, was on risperidone.³ Results of the 8-week study were reported in 2002 and showed that risperidone was effective and well tolerated for the treatment of severe behavioral problems in children with autism. The most common side effects were increased appetite, weight gain, and sedation.

On October 6, 2006, the FDA approved risperidone for the symptomatic treatment of irritability in autistic children and adolescents ages 5 to 16. The approval is the first for the use of a drug to treat behaviors associated with autism in children. These behaviors are included under the general heading of irritability, and include aggression, deliberate self-injury, and temper tantrums.

Other atypical antipsychotics that were studied recently with encouraging results are olanzapine and ziprasidone. Ziprasidone has not been associated with significant weight gain. Olanzapine (as well as other antipsychotic medications) is used off-label for the treatment of aggression and other serious behavioral disturbances in children, including children with autism.

Seizures

Seizures are found in some people with ASD, most often in those who have low intelligence quotient (IQ) or are mute. They are treated with one or more of the anticonvulsants. These include such medications as carbamazepine, lamotrigine, topiramate, and valproic acid. The level of the medication in the blood should be monitored carefully and adjusted so that the least amount possible is used to be effective. Although medication usually reduces the number of seizures, it cannot always eliminate them.

Inattention and Hyperactivity

Stimulant medications such as methylphenidate, used safely and effectively in persons with attention deficit hyperactivity disorder, have also been prescribed for children with autism. These medications may decrease impulsivity and hyperactivity in some children, especially those higher functioning children.

Several other medications have been used to treat ASD symptoms; among them are other antidepressants, naltrexone, lithium, and some of the benzodiazepines such as diazepam and lorazepam. The safety and efficacy of these medications in children with autism have not been proven. Since people may respond differently to different medications, your child's unique history and behavior will help your doctor decide which medication might be most beneficial.

References

1. Newschaffer, C.J., Johns Hopkins Bloomberg School of Public Health. (2003, June 20). Autism among us: Rising concerns and the public health response [Internet video]. Public Health Training Network.
2. McDougle, C.J., Stigler, K.A., & Posey, D.J. (2003). Treatment of aggression in children and adolescents with autism and conduct disorder. Journal of Clinical Psychiatry, 64(supplement 4), 16-25.
3. Research Units on Pediatric Psychopharmacology Network. (2002). Risperidone in children with autism and serious behavioral problems. New England Journal of Medicine, 347(5), 314-321.