Long-Term Improvement and Safety of Aripiprazole for Irritability and Adaptive Function in Asian Children and Adolescents with Autistic Disorder: A 52-Week, Multinational, Multicenter, Open-Label Study.

This year-long study followed 58 Asian children and teens (ages 6-17) with autism who took aripiprazole medication. The results showed continued improvements in challenging behaviors, daily living skills, and reduced family stress. Most children tolerated the medication well, though about 6 in 10 experienced some side effects. The most common side effects were common cold symptoms and weight gain. Three serious events happened but weren't caused by the medication.

This 52-week open-label study evaluated long-term aripiprazole treatment (2-15 mg/day) in 58 Asian children and adolescents (6-17 years) with autism spectrum disorder who had completed a prior 12-week study. Results showed sustained improvements across multiple domains including irritability (ABC), global functioning (CGI), repetitive behaviors (CY-BOCS), adaptive functioning (VABS), and parenting stress (PSI-SF). The medication was generally well-tolerated with 58.62% experiencing treatment-emergent adverse events, most commonly nasopharyngitis (20.69%) and weight gain (18.97%). Weight increase was the most frequent adverse drug reaction (15.52%).

Three serious adverse events occurred but were not considered drug-related. No clinically significant extrapyramidal symptoms were observed.

Results support aripiprazole as a viable long-term treatment option for irritability in autism, but require careful weight monitoring. The sustained benefits across multiple domains suggest comprehensive therapeutic effects. However, open-label design necessitates cautious interpretation and individualized risk-benefit assessment in clinical practice.

Open-label design without placebo control limits interpretation of efficacy. Small sample size (58 participants) and focus on Asian populations may limit generalizability. Lack of comparison group makes it difficult to distinguish treatment effects from natural development or regression to mean.

Evaluate the long-term improvement and safety of aripiprazole in treating irritability in Asian children and adolescents (6-17 years) with autistic disorder.A 52-week, open-label, flexibly dosed (2-15 mg/day) study on the improvement and safety of aripiprazole in patients with autistic disorder who had completed an antecedent 12-week open-label study. The evaluation of efficacy was conducted using the Aberrant Behavior Checklist (ABC), Clinical Global Impression (CGI) scale, Child Yale-Brown Obsessive-Compulsive Scale (CY-BOCS), Vineland Adaptive Behavior Scale (VABS), and the Parenting Stress Index-Short Form (PSI-SF). Safety and tolerability measurements included adverse events, vital signs, electrocardiography, laboratory tests, body weight, and extrapyramidal symptoms (EPSs).During the 52-week treatment, all effectiveness variables, including ABC, CGI, CY-BOCS, VABS, and PSI-SF scores, showed improvement. Regarding safety, the proportion of patients who experienced any treatment-emergent adverse events (TEAEs) was 58.62% (34/58 subjects, 75 cases).

The most common TEAE was nasopharyngitis reported in 20.69% (15/58 subjects, 15 cases) and the other TEAE with an incidence of ≥10% was weight increases in 18.97% (11/58 subjects, 11 cases). Of them, 27.59% (16/58 subjects, 28 cases) experienced adverse drug reactions (ADRs). The most common ADR was weight increase reported in 15.52% (9/58 subjects, nine cases). The incidence of serious adverse events (SAEs) was 5.17% (3/58 subjects, three cases), which were epiphysiolysis, seizure, and a suicide attempt, but these were not ADRs.

There were no clinically significant changes found in the evaluation of EPSs.Aripiprazole showed improvement for behavioral problems and adaptive functioning and was well tolerated in patients with autistic disorder until nearly a year after drug use. The Clinical Trial Registration number: NCT02069977.