Risperidone and aripiprazole are approved by the USA Food and Drug Administration for the treatment of irritability and aggression in children from the ages of 5 and 6 years, respectively. However, there are no approved medications for the treatment of autism spectrum disorder (ASD) core signs and symptoms. Nevertheless, early intervention is recognized as key to improving long-term outcomes. This retrospective case study included 10 children (mean age, 2 years 10 months) with ASD who presented with persistent irritability and aggression before 4 years of age that was unresponsive to behavioral interventions and sufficiently severe to consider pharmacological intervention with risperidone or aripiprazole combined with standard supportive therapies. Besides ameliorating comorbid behaviors, improvement was observed in ASD core signs and symptoms for all patients, with minimal-to-no symptoms observed in 60% of patients according to the Childhood Autism Rating Scale 2-Standard Test and Clinical Global Impression scales. Excessive weight gain in two patients was the only adverse effect observed that required intervention. This is the first study to suggest that ASD can potentially be treated in very young children (<4 years). Clinical trials are urgently required to validate these findings among this pediatric population.

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Background: There are a number of medications prescribed to address comorbid challenging behaviors in children with autism spectrum disorder (ASD), including risperidone and aripiprazole. This retrospective case series reports the use of these drugs in children aged 2 to 13 years. Methodology: A total of 82 children (mean age, 5 years; 79% male) with ASD treated at the Kids Neuro Clinic and Rehab Center in Dubai between January 2020 and September 2021 were included in this retrospective case series. All patients had comorbid challenging behaviors that were resistant to standard supportive therapies alone and warranted pharmacological intervention. The Childhood Autism Rating Scale—2nd Edition Standard form (CARS2-ST) and the Clinical Global Impression (CGI)—Severity (CGI-S) and CGI—Improvement (CGI-I) scales were used to assess the severity of ASD at baseline and to monitor response to treatment with risperidone or aripiprazole. Results: Besides the expected improvement in comorbid challenging behaviors, 79/82 patients (96%) attained a CGI-I score of 2 or 1 following treatment, and 35/82 patients (43%) achieved both a CGI-I score of 1 and minimal-to-no symptoms as per the CARS2-ST test, with complete resolution of their ASD signs and symptoms. The differences in the overall mean CARS2-ST and CGI-S scores pre- and post-treatment were statistically significant (Z = −7.86, p < 0.0001 for both), with pre- and post-treatment mean values of 42 and 23 for CARS2-ST, respectively, and 6 and 2 for CGI-S, respectively. The main side effects were asymptomatic elevated prolactin (n = 12) and excessive weight gain (n = 2). Conclusions: When combined with standard supportive therapies, chronic treatment with antipsychotic medications, either with or without medications for attention deficit hyperactivity disorder, was well tolerated and effective in the treatment of ASD core symptoms and comorbid behaviors in young children. Double-blind, placebo-controlled clinical trials are needed to verify these findings.