Department of Medicine, University of Verona, 37124 Verona, Italy.
Department of Diagnostics and Public Health, University of Verona, 37124 Verona, Italy.
Toxins (Basel). 2024 Jul 4;16(7):306. doi: 10.3390/toxins16070306.
Since its first approval by the Food and Drug Administration in 1989 for strabismus, botulinum toxin indications of use have been widely expanded. Due to its anticholinergic properties, this toxin is currently approved in adult patients for the treatment of a wide range of neuromuscular, otolaryngologic, orthopedic, gastrointestinal, and urologic disorders. Approved pediatric indications of use include the treatment of blepharospasm associated with dystonia, strabismus, lower-limb spasticity, focal spasticity in patients with cerebral palsy, and neurogenic detrusor overactivity. Alongside these approved indications, botulinum toxin is extensively used off-label. Although several clinical studies have shown that botulinum toxin is effective and well-tolerated in children, uncertainties persist regarding its long-term effects on growth and appropriate dosing in this population. As such, further research is needed to better define the botulinum toxin risk-benefit profile and expand approved uses in pediatrics. This narrative review aimed to provide a broad overview of the evidence concerning the clinical effectiveness and safety of BoNT with respect to its principal authorized and non-authorized pediatric therapeutic indications, as well as to describe perspectives on its future use in children.
自 1989 年美国食品和药物管理局首次批准用于斜视以来，肉毒毒素的用途已广泛扩展。由于其抗胆碱能特性，目前已批准该毒素用于治疗成人的各种神经肌肉、耳鼻喉科、骨科、胃肠道和泌尿科疾病。已批准的儿科用途包括治疗与肌张力障碍相关的眼睑痉挛、斜视、下肢痉挛、脑瘫患者的局部痉挛以及神经性逼尿肌过度活动。除了这些批准的适应症外，肉毒毒素还被广泛地超适应证使用。尽管多项临床研究表明，肉毒毒素在儿童中有效且耐受良好，但对于其在该人群中的长期影响和适当剂量仍存在不确定性。因此，需要进一步的研究来更好地确定肉毒毒素的风险效益概况，并扩大儿科的批准用途。本综述性叙述旨在广泛概述有关 BoNT 在其主要授权和非授权儿科治疗适应症方面的临床有效性和安全性的证据，并描述其在儿童中的未来应用前景。
