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Safety and Efficacy of Paliperidone Palmitate in Pediatric Patients with Autism and Intellectual Disability.

Journal of autism and developmental disorders2026

Simpson Seth, Dominick Kelli C, Erickson Craig A, Lamy Martine

What this study means for families

Researchers studied a long-acting injectable medication (paliperidone palmitate) for managing irritability in 26 young people with autism and intellectual disability. The medication was given as monthly injections over 3 years. Most children tolerated it well, with only 3 stopping due to side effects. Parents and doctors reported improvements in behaviour and fewer hospital visits. The injection format helped families who struggled with daily medication compliance.

Summary by AutismInsights from published abstract. This is not a substitute for reading the original paper.

Research summary

This retrospective chart review examined long-acting injectable paliperidone palmitate (P-LAI) in 26 youth and transition-aged individuals with autism spectrum disorder and/or intellectual disability over 3 years. The study targeted irritability symptoms and evaluated outcomes using Clinical Global Impressions scales and hospital presentation rates. Results showed good tolerability with only 3 patients discontinuing due to side effects, average treatment duration of 21.1 months, and statistically significant improvements in clinical severity, global improvement scores, and reduced hospital visits. No BMI changes were observed.

Medication compliance difficulties were the primary reason for initiating this long-acting formulation.

Summary by AutismInsights from published abstract. This is not a substitute for reading the original paper.

Key findings

  • 1

    Statistically significant improvements in Clinical Global Impressions Severity and Improvement scores

    Confidence: moderateRelevance: Suggests meaningful clinical improvement in irritability symptoms
  • 2

    Statistically significant reduction in hospital presentations

    Confidence: moderateRelevance: Indicates potential reduction in crisis situations requiring emergency care
  • 3

    Good tolerability with only 11.5% discontinuation rate due to side effects

    Confidence: moderateRelevance: Suggests acceptable safety profile for this vulnerable population
  • 4

    No significant change in BMI during treatment

    Confidence: moderateRelevance: Important safety finding as weight gain is a common concern with antipsychotics

Summary by AutismInsights from published abstract. This is not a substitute for reading the original paper.

Clinical implications

P-LAI may be a viable option for managing irritability in youth with ASD/ID, particularly when medication compliance is challenging. The long-acting formulation addresses adherence issues common in this population. However, careful monitoring and individualised risk-benefit assessment remain essential given the vulnerable population and preliminary nature of evidence.

Summary by AutismInsights from published abstract. This is not a substitute for reading the original paper.

Limitations

Retrospective design limits causal inferences. Small sample size (n=26) reduces generalisability. No control group for comparison. Lack of standardised behavioural outcome measures beyond CGI scales. Unknown study type classification suggests incomplete methodology reporting.

Summary by AutismInsights from published abstract. This is not a substitute for reading the original paper.

Original abstract

This retrospective chart review examines the safety, tolerability and effectiveness of long acting injectable paliperidone palmitate (P-LAI) targeting irritability in twenty-six youth and transition-aged individuals with autism spectrum disorder (ASD) and/or intellectual disability (ID) over a 3-year window. Clinical response was evaluated via prospectively assigned Clinical Global Impressions Severity (CGI-S) and Improvement (CGI-I) scales as well as number of hospital presentations. P-LAI was well tolerated with only 3 patients stopping P-LAI due to side effects. The average duration of P-LAI treatment was 21.1 months.

Difficulty with medication compliance was the most common reason for initiating P-LAI. There was a statistically significant improvement in CGI-I, CGI-S and hospital visits and no change in BMI noted. Given the potential difficulty of medication administration in this population, this evidence of safety, tolerability as well as preliminary data supporting effectiveness is an important addition to the literature regarding psychopharmacologic management of irritability in youth with ASD and ID.

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Evidence Grade

Emerging

limited

Grade assigned by AutismInsights based on study type and published abstract.

Study Details

Journal
Journal of autism and developmental disorders
Year
2026
PMID
38740696
DOI
10.1007/s10803-024-06372-9

MeSH Terms

HumansPaliperidone PalmitateMaleFemaleIntellectual DisabilityChildAdolescentAntipsychotic AgentsIrritable MoodRetrospective StudiesTreatment OutcomeAutism Spectrum DisorderChild, Preschool