An Update on Pharmacotherapy for Autism Spectrum Disorder
Despite the high rates of medication usage in children with ASD, current evidence-based pharmacotherapy options are extremely limited. Antipsychotics treatment, particularly risperidone and aripiprazole, are effective in reducing irritability, stereotypy and hyperactivity, and MPH is effective in improving ADHD symptoms. Atomoxetine and alpha-2 agonists appear effective in reducing ADHD symptoms. SSRIs are not effective in improving RRB in children with ASD, and may lead to activating adverse events. Efficacy of AED is inconclusive. Even with the medications with evidence-based efficacy, their response rates and tolerability tend to be less favorable than data seen in typically developing children with similar symptoms. Therefore, clinicians should carefully weigh the risk/benefit ratio, closely monitor adverse events and periodically re-assess needs for continued pharmacotherapy for the target symptoms.
ASD is a lifelong neurodevelopmental disorder that often requires a comprehensive and multidisciplinary treatment. Nonpharmacologic options such as modifications in the setting and behavioral interventions may improve target symptoms without medications. Therefore, pharmacotherapy should be a part of comprehensive treatment.
Nevertheless, new pharmacotherapy options for severely impairing co-existing and core symptoms are in urgent need. However, developing new drugs for ASD faces a number of challenges.
First of all, largely unknown cause and wide genotypic and phenotypic heterogeneity bring an inherent challenge to treatment development for the population. Large-scale clinical trials that include highly heterogeneous subjects often fail to capture effects of treatment that may be indeed effective in a more homogeneous subgroup. Investigating ASD associated with monogenic disorders (e.g. FXS and tuberous sclerosis) may mitigate this challenge. In fact, targeted agents such as acamprosate for FXS and rapamycin for tuberous sclerosis are currently in clinical trials. Future research investigating genotypic and/or phenotypic characteristics influencing medication response and tolerability will be valuable in further individualizing pharmacotherapy.
Another substantial challenge in drug development for ASD is lack of gold standard outcome measures, particularly for the core symptoms. Future research utilizing biomarkers such as eye-tracking, electrophysiological measures and/or functional neuroimaging may aid in capturing treatment benefits more accurately.
Despite substantive challenges, autism treatment research has made considerable progress in recent years. With genetic testing and animal models rapidly increasing our understanding of ASD, the field is now at an exciting juncture, and researchers are eagerly anticipating important discoveries for targeted pharmacotherapy, which may one day be available in clinical settings in the near future.
Conclusion and Future Directions
Despite the high rates of medication usage in children with ASD, current evidence-based pharmacotherapy options are extremely limited. Antipsychotics treatment, particularly risperidone and aripiprazole, are effective in reducing irritability, stereotypy and hyperactivity, and MPH is effective in improving ADHD symptoms. Atomoxetine and alpha-2 agonists appear effective in reducing ADHD symptoms. SSRIs are not effective in improving RRB in children with ASD, and may lead to activating adverse events. Efficacy of AED is inconclusive. Even with the medications with evidence-based efficacy, their response rates and tolerability tend to be less favorable than data seen in typically developing children with similar symptoms. Therefore, clinicians should carefully weigh the risk/benefit ratio, closely monitor adverse events and periodically re-assess needs for continued pharmacotherapy for the target symptoms.
ASD is a lifelong neurodevelopmental disorder that often requires a comprehensive and multidisciplinary treatment. Nonpharmacologic options such as modifications in the setting and behavioral interventions may improve target symptoms without medications. Therefore, pharmacotherapy should be a part of comprehensive treatment.
Nevertheless, new pharmacotherapy options for severely impairing co-existing and core symptoms are in urgent need. However, developing new drugs for ASD faces a number of challenges.
First of all, largely unknown cause and wide genotypic and phenotypic heterogeneity bring an inherent challenge to treatment development for the population. Large-scale clinical trials that include highly heterogeneous subjects often fail to capture effects of treatment that may be indeed effective in a more homogeneous subgroup. Investigating ASD associated with monogenic disorders (e.g. FXS and tuberous sclerosis) may mitigate this challenge. In fact, targeted agents such as acamprosate for FXS and rapamycin for tuberous sclerosis are currently in clinical trials. Future research investigating genotypic and/or phenotypic characteristics influencing medication response and tolerability will be valuable in further individualizing pharmacotherapy.
Another substantial challenge in drug development for ASD is lack of gold standard outcome measures, particularly for the core symptoms. Future research utilizing biomarkers such as eye-tracking, electrophysiological measures and/or functional neuroimaging may aid in capturing treatment benefits more accurately.
Despite substantive challenges, autism treatment research has made considerable progress in recent years. With genetic testing and animal models rapidly increasing our understanding of ASD, the field is now at an exciting juncture, and researchers are eagerly anticipating important discoveries for targeted pharmacotherapy, which may one day be available in clinical settings in the near future.
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