What is the efficacy of medications for the treatment of autism spectrum disorder (ASD)?

Updated: Sep 30, 2019
  • Author: James Robert Brasic, MD, MPH; Chief Editor: Caroly Pataki, MD  more...
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Although 70% of children with autism spectrum disorder receive medications, only limited evidence exists that the beneficial effects outweigh the adverse effects. [159] No pharmacologic agent is effective in the treatment of the core behavioral manifestations of autistic disorder, but drugs may be effective in treating associated behavioral problems and comorbid disorders. [160, 161]

The second-generation antipsychotic agents risperidone and aripiprazole provide beneficial effects on challenging and repetitive behaviors in children with autism spectrum disorder, although these patients may experience significant adverse effects. [162] Risperidone and aripiprazole have been approved by the US Food and Drug Administration (FDA) for irritability associated with autistic disorder. The second-generation antipsychotic agent ziprasidone may help to control aggression, irritability, and agitation. [163]

Serotonergic drugs are reportedly beneficial for improving behavior in autism. Hyperactivity often improves with methylphenidate therapy.

Additionally, treatments may be indicated for an underlying condition. For example, children with biotin-responsive infantile encephalopathy improve with the addition of biotin.

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