Very little high-quality evidence to support most medications for children with autism spectrum disorders

Article Outline

• Question
• Design
• Data Sources
• Study Selection and Assessment
• Outcomes
• Main Results
• Conclusions
• Commentary
• Copyright

McPheeters ML, Warren Z, Sathe N, Bruzek JL, Krishnaswami S, Jerome RN, et al. A Systematic Review of Medical Treatments for Children With Autism Spectrum Disorders. Pediatrics 2011;127:e1312-21.

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Question 

Among children with autism spectrum disorder (ASD), what medications are most effective at treating co-morbid symptoms?

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Design 

Systematic review.

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Data Sources 

Medline, PsycInfo, and ERIC (Education Resources Information Center) databases from 2000 to May 2010, regulatory data for approved medications, and reference lists of included articles.

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Study Selection and Assessment 

Two reviewers independently assessed each study against predetermined inclusion/exclusion criteria. Studies of secretin were not included in this review. They extracted data regarding participant and intervention characteristics, assessment techniques, and outcomes, and assigned overall quality and strength-of-evidence ratings on the basis of predetermined criteria.

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Outcomes 

Effects on core symptoms of ASDs or common comorbid symptoms including sleep, anxiety, hyperactivity, and challenging behavior (eg, irritability/agitation).

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Main Results 

Evidence supports the benefit of risperidone and aripiprazole for challenging and repetitive behaviors in children with ASDs. Evidence also supports significant adverse effects of these medications. Insufficient strength of evidence is present to evaluate the benefits or adverse effects for any other medical treatments for ASDs, including serotonin-reuptake inhibitors and stimulant medications.

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Conclusions 

Although many children with ASDs are currently treated with medical interventions, strikingly little evidence exists to support benefit for most treatments. Risperidone and aripiprazole have shown benefit for challenging and repetitive behaviors, but associated adverse effects limit their use to patients with severe impairment or risk of injury.

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Commentary 

This review adds to our knowledge of effective medical treatment for children with ASD primarily for what it does not provide – much new evidence. Their evaluations in this study are rigorous; setting the bar high provides some limitations to what many clinicians would consider effective and evidence-based treatments. For example, there are only four randomized clinical trials of risperidone that meet the group's review criteria out of dozens of reports in the literature, the rest falling short due to small study population, lack of randomization or blinding, etc. This review also does not endeavor to conduct meta-analysis of the studies, but rather accepts the data presented and highlights the specifics of each (eg, the target symptoms for treatment and the measurements used). Finally, the authors bring to light the implications of using some treatments that are shown to be efficacious but, at the same time, carry significant risks. The result is that there was only sufficient evidence to support the use of two medications – risperidone and aripiprazole – among the many agents that are commonly used in this population. Analyses of this type are important in sorting out the best evidence from the more common case reports that many clinicians read and sometimes too quickly implement in their clinical practice.