Medical treatment of autism spectrum disorder in children: Current evidence, controversies, and clinical challenges

Table 1 Risperidone for pediatric autism spectrum disorder

Feature	Key findings
Indication	FDA-approved for irritability (aggression, self-injury, tantrums) associated with autistic disorder in ages 5-16 years
Efficacy	Strong evidence for reducing irritability, aggression, self-injury, and tantrums. Moderate evidence for reducing hyperactivity and stereotypy. Limited/inconsistent evidence for improving core social-communication symptoms
Key adverse effects	Weight gain: Significant, often rapid, linked to increased appetite; risk increases with duration and possibly younger age. Metabolic changes: Increased risk of insulin resistance, metabolic syndrome; changes in glucose, lipids, leptin, and adiponectin observed. Sedation/somnolence: Common, especially initially, often mild-moderate and transient. Hyperprolactinemia: Frequent, dose-dependent. Enuresis: Increased risk observed in long-term use. Tremor: Increased risk, though EPS is generally low
Cognitive effects	Appears to have no detrimental effect; some studies suggest potential minor improvements in attention/recognition memory in testable children
Pharmacokinetics/TDM	Sum trough concentration (risperidone + 9-hydroxyrisperidone-risperidone) correlates with efficacy and side effects (weight gain, sedation, prolactin). Proposed TDM target range: 3.5-7.0 μg/L to balance efficacy and weight gain. Simulation studies suggest TDM can improve achievement of the target range and potentially reduce side effects

FDA: Food and Drug Administration; EPS: Extrapyramidal symptoms; TDM: Therapeutic drug monitoring.