A Systematic Review of Botulinum Toxin Injection in Pediatric Dystonia

• Published in: Toxins Vol. 16; no. 7; p. 289
• Main Authors: Rasera, Andrea, Squintani, Giovanna Maddalena, Cerruto, Maria Angela
• Format: Journal Article
• Language: English
• Published: Switzerland MDPI AG 01.07.2024
• Subjects: Adolescent; Adults; Analgesics; Botulinum toxin; Botulinum Toxins - administration & dosage; Botulinum Toxins - adverse effects; Botulinum Toxins - therapeutic use; Care and treatment; Case reports; Cerebral palsy; Child; Child development; Child, Preschool; Children; Clinical trials; Drug approval; Dystonia; Dystonia - drug therapy; dystonia treatment; Dystonic Disorders - drug therapy; Effectiveness; Health aspects; Health services; Human motion; Humans; Kinases; Low level; Medical research; Medicine, Experimental; Movement disorders; Neuromuscular Agents - administration & dosage; Neuromuscular Agents - adverse effects; Neuromuscular Agents - therapeutic use; Patients; pediatric dystonia; Pediatrics; Quality of Life; Saliva; Side effects; Spasticity; Systematic review; Toxins; Treatment Outcome; botulinum toxin; pediatric dystonia; dystonia treatment
• ISSN: 2072-6651; 2072-6651
• DOI: 10.3390/toxins16070289

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.