Concomitant medication use in children with autism spectrum disorder: Data from the Autism Biomarkers Consortium for Clinical Trials.

This study looked at what medications children with autism are taking. Of 280 children studied, about 4 out of 10 were on medications that affect the brain and behavior. Half of these children were taking more than one medication. Common medications included melatonin for sleep, stimulants for attention, antidepressants, and antipsychotics.

Children on antipsychotics seemed to have more challenges. The researchers suggest that excluding children on medications from studies might miss important insights.

This study analyzed medication use patterns among 280 school-age children with autism spectrum disorder in the Autism Biomarkers Consortium for Clinical Trials. Researchers found that 42.5% were taking psychotropic medications, with half of those children on multiple medications (polypharmacy). The most common medications were melatonin, stimulants, SSRIs, alpha agonists, and antipsychotics. Children taking antipsychotics showed trends toward greater overall impairment.

The study highlights that excluding children on medications from clinical trials may reduce the representativeness of research samples and potentially exclude those who might benefit most from new treatments.

High medication use rates suggest need for coordinated care between autism specialists and prescribers. Polypharmacy patterns indicate importance of medication reviews. Including medicated children in research trials may improve clinical relevance and identify those most likely to benefit from new interventions.

The study is descriptive and cross-sectional, limiting causal inferences about medication effects. Sample may not be representative of all children with autism. Severity of impairment relationships with antipsychotics described only as trends without statistical significance testing reported.

Children with autism spectrum disorder are prescribed a variety of medications that affect the central nervous system (psychotropic medications) to address behavior and mood. In clinical trials, individuals taking concomitant psychotropic medications often are excluded to maintain homogeneity of the sample and prevent contamination of biomarkers or clinical endpoints. However, this choice may significantly diminish the clinical representativeness of the sample. In a recent multisite study designed to identify biomarkers and behavioral endpoints for clinical trials (the Autism Biomarkers Consortium for Clinical Trials), school-age children with autism spectrum disorder were enrolled without excluding for medications, thus providing a unique opportunity to examine characteristics of psychotropic medication use in a research cohort and to guide future decisions on medication-related inclusion criteria.

The aims of the current analysis were (1) to quantify the frequency and type of psychotropic medications reported in school-age children enrolled in the ABC-CT and (2) to examine behavioral features of children with autism spectrum disorder based on medication classes. Of the 280 children with autism spectrum disorder in the cohort, 42.5% were taking psychotropic medications, with polypharmacy in half of these children. The most commonly reported psychotropic medications included melatonin, stimulants, selective serotonin reuptake inhibitors, alpha agonists, and antipsychotics. Descriptive analysis showed that children taking antipsychotics displayed a trend toward greater overall impairment.

Our findings suggest that exclusion of children taking concomitant psychotropic medications in trials could limit the clinical representativeness of the study population, perhaps even excluding children who may most benefit from new treatment options.