Objective: To perform an evidence-based review of the safety and efficacy of botulinum neurotoxin (BoNT) in the treatment of movement disorders. Methods: A literature search was performed including MEDLINE and Current Contents for therapeutic articles relevant to BoNT and selected movement disorders. Authors reviewed, abstracted, and classified articles based on American Academy of Neurology criteria (Class I–IV). Results: The highest quality literature available for the respective indications was as follows: blepharospasm (two Class II studies); hemifacial spasm (one Class II and one Class III study); cervical dystonia (seven Class I studies); focal upper extremity dystonia (one Class I and three Class II studies); focal lower extremity dystonia (one Class II study); laryngeal dystonia (one Class I study); motor tics (one Class II study); and upper extremity essential tremor (two Class II studies). Recommendations: Botulinum neurotoxin should be offered as a treatment option for the treatment of cervical dystonia (Level A), may be offered for blepharospasm, focal upper extremity dystonia, adductor laryngeal dystonia, and upper extremity essential tremor (Level B), and may be considered for hemifacial spasm, focal lower limb dystonia, and motor tics (Level C). While clinicians’ practice may suggest stronger recommendations in some of these indications, evidencebased conclusions are limited by the availability of data. Neurology ® 2008;70:1699–1706

https://n.neurology.org/content/neurology/70/19/1691.full.pdf

Assessment: Botulinum neurotoxin for the treatment of movement disorders (an evidence-based review) Report of the Therapeutics and Technology Assessment Subcommittee of the American Academy of Neurology

The updated European Consensus 2009 on the use of Botulinum toxin for children with cerebral palsy

Objective: To evaluate published evidence of efficacy and safety of pharmacologic treatments for childhood spasticity due to cerebral palsy. Methods: A multidisciplinary panel systematically reviewed relevant literature from 1966 to July 2008. Results: For localized/segmental spasticity, botulinum toxin type A is established as an effective treatment to reduce spasticity in the upper and lower extremities. There is conflicting evidence regarding functional improvement. Botulinum toxin type A was found to be generally safe in children with cerebral palsy; however, the Food and Drug Administration is presently investigating isolated cases of generalized weakness resulting in poor outcomes. No studies that met criteria are available on the use of phenol, alcohol, or botulinum toxin type B injections. For generalized spasticity, diazepam is probably effective in reducing spasticity, but there are insufficient data on its effect on motor function and its side-effect profile. Tizanidine is possibly effective, but there are insufficient data on its effect on function and its side-effect profile. There were insufficient data on the use of dantrolene, oral baclofen, and intrathecal baclofen, and toxicity was frequently reported. Recommendations: For localized/segmental spasticity that warrants treatment, botulinum toxin type A should be offered as an effective and generally safe treatment (Level A). There are insufficient data to support or refute the use of phenol, alcohol, or botulinum toxin type B (Level U). For generalized spasticity that warrants treatment, diazepam should be considered for short-term treatment (Level B), and tizanidine may be considered (Level C). There are insufficient data to support or refute use of dantrolene, oral baclofen, or continuous intrathecal baclofen (Level U). Neurology ® 2010;74:336 –343

https://n.neurology.org/content/neurology/74/4/336.full.pdf

Practice Parameter: Pharmacologic treatment of spasticity in children and adolescents with cerebral palsy (an evidence-based review) Report of the Quality Standards Subcommittee of the American Academy of Neurology and the Practice Committee of the Child Neurology Society

The aim of the study was to review the psychometric properties and use of goal attainment scaling (GAS) in paediatric rehabilitation research. We performed a critical literature review searching: (1) all studies whose main focus was to assess the psychometric properties of GAS in paediatric rehabilitation; and (2) all effect studies in paediatric rehabilitation that used GAS as one of the outcome measures. Three articles in the first group and six in the second group met the inclusion criteria. None of the studies had investigated the content reliability of the scales. Interrater reliability had been investigated in one study and had been found to be good. Only one trial had assessed the content validity of the developed scales, which was found to be acceptable. Comparisons showed that GAS, because of its idiosyncratic nature, measures different constructs from those measured by some related instruments. Low concurrent validity was found. All included studies reported good sensitivity to change. We conclude that the literature supports promising qualities of GAS in paediatric rehabilitation. GAS is a responsive method for individual goal setting and for treatment evaluation. However, current knowledge about its reliability when used with children is insufficient. There is a need for further development of GAS and its application for children of different ages and disabilities, across therapists of different disciplines.

https://onlinelibrary.wiley.com/doi/pdf/10.1111/j.1469-8749.2007.00550.x

Goal attainment scaling in paediatric rehabilitation: a critical review of the literature

Botulinum neurotoxin type-A (BoNT-A) has been used in association with other interventions in the management of spasticity in children with cerebral palsy (CP) for almost two decades. This consensus statement is based on an extensive review of the literature by an invited international committee. The use of BoNT-A in the lower limbs of children with spasticity caused by CP is reported using the American Academy of Neurology Classification of Evidence for therapeutic intervention. Randomized clinical trials have been grouped into five areas of management, and the outcomes are presented as treatment recommendations. The assessment of children with CP and evaluation of outcomes following injection of BoNT-A are complex, and therefore, a range of measures and the involvement of a multidisciplinary team is recommended. The committee concludes that injection of BoNT-A in children with CP is generally safe although systemic adverse events may occur, especially in children with more physical limitations (GMFCS V). The recommended dose levels are intermediate between previous consensus statements. The committee further concludes that injection of BoNT-A is effective in the management of lower limb spasticity in children with CP, and when combined with physiotherapy and the use of orthoses, these interventions may improve gait and goal attainment.

/pdf/botulinum-toxin-assessment-intervention-and-after-care-for-w20jnrxohe.pdf

Botulinum toxin assessment, intervention and after‐care for lower limb spasticity in children with cerebral palsy: international consensus statement

Adductor spasticity in children with cerebral palsy (CP) impairs motor function and development. In a placebo-controlled, double-blind, randomized multicentre study, we evaluated the effects of botulinum toxin A(BTX-A) in 61 children (37 males, 24 females; mean age 6 years 1 month [SD 3y 1mo]) with CP (leg-dominated tetraparesis, n=39; tetraparesis, n=22; GMFCS level I, n=3; II, n=6; III, n=17; IV, n=29; V, n=6). Four weeks after treatment, a significant superiority of BTX-A was observed in the primary outcome measure (knee–knee distance ‘fast catch’, p=0.002), the Ashworth scale (p=0.001), and the Goal Attainment Scale (p=0.037).

https://onlinelibrary.wiley.com/doi/pdf/10.1017/S0012162206000041

Nico Breitbach-Faller

Papers

Treatment of adductor spasticity with BTX-A in children with CP: a randomized, double-blind, placebo-controlled study.