Difficulties with differentiating botulinum toxin treatment effects in essential blepharospasm

• Published in: Journal of Neural Transmission Vol. 118; no. 6; pp. 925 - 943
• Main Authors: Wabbels, Bettina, Jost, Wolfgang H., Roggenkämper, Peter
• Format: Journal Article
• Language: English
• Published: Vienna Springer Vienna 01.06.2011; Springer Nature B.V
• Subjects: Blepharospasm - diagnosis; Blepharospasm - drug therapy; Blepharospasm - physiopathology; Botulinum Toxins, Type A - pharmacology; Botulinum Toxins, Type A - therapeutic use; Clinical Trials as Topic - methods; Diagnosis, Differential; Endpoint Determination - methods; Humans; Medicine; Medicine & Public Health; Movement Disorders - Review; Movement Disorders - Review Article; Neurology; Neurosciences; Outcome Assessment, Health Care - methods; Psychiatry; Severity of Illness Index; Blepharospasm Disability Index; Dystonia; Jankovic Rating Scale; Botulinum toxin; Blepharospasm
• ISSN: 0300-9564; 1435-1463
• DOI: 10.1007/s00702-010-0546-9

Blepharospasm is a focal dystonia in which the extraocular muscles contract repetitively, leading to excessive blinking and forced eyelid closure. Botulinum toxin type A (BoNTA) is the primary symptomatic treatment for blepharospasm and its effects have been evaluated using numerous rating scales. The main scales in use today were initially used to determine whether BoNTA treatment was superior to placebo, and most controlled trials have confirmed this. More recently, these scales have been used to determine whether there are efficacy differences between different BoNTs in blepharospasm. However, although the scales used in these trials are able to differentiate the effects of BoNT from placebo, they may not be sensitive enough to differentiate between BoNTs. Most of the scales include only four possible points for each item, which would necessitate a 25% greater improvement in one group than the other to detect any differences. Current scales are also relatively insensitive to patients with mild disability who may experience mainly psychosocial problems related to their blepharospasm. Clinical trials comparing BoNTs that include substantial numbers of mildly affected patients may be unlikely to find differences because the scales do not adequately measure mild symptoms. Additional challenges with evaluating blepharospasm include the lack of precision and objectivity of current measures, symptom variability, the need to evaluate aspects of the disorder that are most important to patients, and the different types of blepharospasm. Although no single scale may be able to capture all relevant aspects of blepharospasm, more sensitive and patient-centered scales are needed.