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A few medications show benefit for repetitive behaviors or associated symptoms. The clearest evidence favors the use of atypical antipsychotics to address challenging behaviors. The antipsychotics risperidone and aripiprazole each have at least two randomized trials demonstrating improvement in parent-reported challenging behavior. Parent-reported hyperactivity and noncompliance also showed significant improvement. In addition, repetitive behavior showed improvement with both risperidone and aripiprazole. However, both medications may cause significant side effects, including marked weight gain, sedation, and risk of extrapyramidal symptoms (muscle stiffness or tremor). These side effects should limit use of these drugs to patients with severe impairment or risk of injury.
Injection of a substance called secretin has been studied as a treatment for ASDs in eight controlled trials. None showed significantly greater improvements in measures of language, cognition or any other autistic symptoms when compared with placebo. We do not recommend that secretin be used in children with ASDs.
An analysis of five clinical trials of melatonin in children with ASDs showed this supplement was associated with significant improvement in sleep onset and duration. Melatonin has been proven safe and is relatively inexpensive to purchase in many health food stores.
Evidence-Based research on medications:
Oxytocin and autism: a systematic review of randomized controlled trials 1 (subscription needed)This article systematically reviews all clinical trials of the medication oxytocin in persons with Autism Spectrum Disorders (ASD) as of March, 2013. Oxytocin is a naturally occurring substance synthesized in the human hypothalamus and released into the bloodstream by the pituitary gland. In synthetic form, it is often used for inducing labor or managing postpartum hemorrhage. Because of some positive results in non-autistic individuals, oxytocin was hypothesized to improve social symptoms of ASD. Neither this drug nor any others are FDA approved for this use.Six randomized controlled trials were identified. A total of 101 persons with ASD were enrolled; only six females were included. All trials lasted six weeks or less. Some studies utilized intranasal administration while others administered oxytocin intravenously; doses varied by study. Studies used different outcomes to measure effectiveness. The studies’ risk of bias, based on the randomization process, blinding, and attrition rate, ranged from medium to high. Although some studies reported potentially promising findings, results were inconsistent. No adverse events requiring medical attention were reported. Additional research with larger population samples and more females must be conducted before this treatment can be recommended.
Mitigation of sociocommunicational deficits of autism through oxytocin-induced recovery of medial prefrontal activity: A randomized trial. 2 Researchers in Japan conducted a randomized clinical trial (RCT) of oxytocin to assess its efficacy in mitigating social and communication deficits in high functioning men with autism spectrum disorders (ASD). They used a within-subject crossover design, where 40 men were randomized to receive a single dose of intra-nasal oxytocin or placebo before a psychological task.The researchers created a psychological task which required participants to make judgments regarding the intentions of others based on communicational content in which verbal and nonverbal information conflicted. The men viewed short videos in which professional actors spoke an emotional word (verbal information) with an emotional facial expression and expressive voice (nonverbal information). The videos consisted of two types of emotionally congruent videos with negative (NV−V−) or positive (NV+V+) nonverbal and verbal information and two types of incongruent videos with negative nonverbal and positive verbal (NV−V+) or positive nonverbal and negative verbal (NV+V−) information. On the basis of the video content participants were asked to make a “friend or foe” judgment of the actor. Persons with ASD tend to respond less to nonverbal communication; the hypothesis was that frequency of nonverbal information–based judgments (NVJs), the response time for NVJs, and brain activity of the medial prefrontal cortex (measured by Functional Magnetic Resonance Imaging) during NVJs would be higher with oxytocin than placebo. Compared to placebo, intranasal oxytocin led to significantly more frequent NVJs, with significantly shorter response time. The magnitude of neural effects was predictive of the behavioral effects. The authors concluded that intranasal administration of oxytocin enables highly functioning individuals with ASD to exhibit more typical and smoother behavioral responses to social communication when verbal and nonverbal information conflicts.
1 Preti A, Melis M, Siddi S, Vellante M, Doneddu G, Fadda R. Oxytocin and autism: a systematic review of randomized controlled trials. Journal of child and adolescent psychopharmacology. Mar 2014;24(2):54-68. Journal of autism and developmental disorders. Jan 2013;43(1):57-67.