A Systematic Review of Botulinum Toxin Injection in Pediatric Dystonia

Author:

Rasera Andrea1,Squintani Giovanna Maddalena2,Cerruto Maria Angela3

Affiliation:

1. Neurology Unit, “Azienda Provinciale per i Servizi Sanitari”, Trento “Santa Chiara” Hospital, Largo Medaglie d’oro 9, 38122 Trento, Italy

2. Section of Neurophysiology, Neurology Unit, “Azienda Ospedaliera Universitaria Integrata Verona”, Verona “Borgo Trento” Hospital, Piazzale Aristide Stefani 1, 37126 Verona, Italy

3. Urology Clinic, Department of Surgery, Dentistry, Paediatrics and Gynaecology, University of Verona, Piazzale Aristide Stefani 1, 37126 Verona, Italy

Abstract

Botulinum toxin (BT), a first-line treatment for focal dystonias in adults, has gained USA Food and Drug Administration approval for pediatric upper and lower extremity spasticity and sialorrhea, though its use in children younger than 2 years old is still considered off-label treatment for all pathologies. Dosing, treatment strategies and outcome measures lack international consensus, and they are often extrapolated from adult or spasticity guidelines. This review aims to evaluate the best available evidence on the efficacy and safety of BT therapy in pediatric dystonia (age under 21 years old), isolated or associated with other medical conditions. A comprehensive search in PubMed, Scopus and Web of Science was conducted, including only articles in English. Although no randomized controlled trials are still present, 12 articles were included with an overall of 57 patients. All the papers demonstrate that BT can improve motor function, decrease pain and ameliorate quality of life, with minimal adverse effects in pediatric patients affected by pure or mixed dystonic motor disorders. Despite the low level of evidence, our review shows that BT could be an efficacious treatment for these pediatric patients. The frequent generalized involvement, together with the heterogeneous nature of childhood dystonic forms, sometimes intermingled with spasticity, prompts further multicenter clinical trials or prospective studies with a higher level of evidence to shed light on the efficacy and safety profile of BT in pediatric dystonia.

Publisher

MDPI AG

Reference40 articles.

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3. Botulinum Toxin Therapy: A Comprehensive Review on Clinical and Pharmacological Insights;Journal of Clinical Medicine;2025-03-16

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