Neuropediatrics 2025; 56(S 01): S1-S24
DOI: 10.1055/s-0045-1812149
Movement Disorders

Therapeutic Potential of Cannabinoids in Pediatric Dystonia: Insights from a Systematic Review and Clinical Cases

Authors

  • Y. Schipper

    1   University Clinic for Paediatrics, Inselspital, Bern, Switzerland

  • R. Reitman

    2   Division of Neurology, Department of Pediatrics, The Hospital for Sick Children, Toronto, Canada

  • C. Gorodetsky

    2   Division of Neurology, Department of Pediatrics, The Hospital for Sick Children, Toronto, Canada

  • A. Ménétrey

    2   Division of Neurology, Department of Pediatrics, The Hospital for Sick Children, Toronto, Canada

  • S. Grunt

    3   Development & Rehabilitation, University Clinic for Paediatrics Inselspital, Bern, Switzerland
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Background/Purpose: Dystonia is a movement disorder characterized by sustained or intermittent involuntary muscle contractions, causing contorted movements or abnormal posturing. Symptoms often significantly impair quality of life, yet current pharmacological treatments are limited and frequently insufficient. Interestingly, cannabinoids have emerged as promising therapeutics; however, clinical evidence supporting their use, particularly in pediatric populations, remains sparse. This study presents a comprehensive systematic review of existing literature, complemented by an analysis of the largest cohort of pediatric patients treated with cannabinoids for dystonia.

Methods: A systematic review was conducted following PRISMA guidelines, searching PubMed, EMBASE, and Cochrane for peer-reviewed studies published up to December 2024 on medical cannabinoid use in pediatric dystonia. In parallel, data from The Hospital for Sick Children were retrospectively analyzed, including 36 pediatric dystonia patients treated with cannabinoids. The patients were then categorized according to demographic information, clinical presentations, and genetic diagnoses and evaluated for functional improvement and adverse effects.

Results: The systematic review identified six primary studies, which generally reported positive effects. In contrast, the cohort (mean age: 7.1 years, range: 2–14; equal male/female ratio) included a variety of genetic etiologies (e.g., MECP2, ATP1A3, KCNQ2, MCT8, and GABRA1) and nongenetic causes, including hypoxic-ischemic brain injuries. Preliminary findings show that while some patients experienced symptomatic relief with cannabinoid treatment, many outcomes remain inconclusive.

Conclusion: While our systematic review suggests that cannabinoids consistently improve dystonia symptoms, our cohort study highlights the variability in response and a substantial proportion of unknown outcomes. Larger, prospective trials are needed to validate these early findings.



Publication History

Article published online:
26 September 2025

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