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Efficacy and safety of incobotulinumtoxinA (NT 201, Xeomin) in the treatment of blepharospasm—A randomized trial

Authors


  • Relevant conflicts of interest/financial disclosures: The study was sponsored by Merz Pharmaceuticals GmbH. Susanne Grafe and Angelika Hanschmann are employees of the sponsor. Joseph Jankovic was coordinating investigator, and Cynthia Comella investigator of this study. Full financial disclosures and author roles may be found in the online version of this article.

Abstract

IncobotulinumtoxinA (NT 201, Xeomin) is a highly purified botulinum neurotoxin type A formulation, free from complexing proteins. A randomized, placebo-controlled, double-blind trial of efficacy and safety compared incobotulinumtoxinA (up to 50 U per eye) to placebo administered in a single treatment session to patients with blepharospasm. All patients had documented satisfactory response to 2 previous treatments with botulinum neurotoxin type A other than incobotulinumtoxinA and had Jankovic Rating Scale severity subscores ≥ 2. Patients (n = 109) were randomized in a 2:1 ratio to incobotulinumtoxinA or placebo and followed up to 20 weeks; 94% completed the study. A significant difference was observed in the primary efficacy variable (change in Jankovic Rating Scale severity subscore rated by an independent rater 6 weeks following treatment), favoring incobotulinumtoxinA by 1.0 point (95% CI [0.5–1.4]; P < .001). Functional impairment, as measured by the Blepharospasm Disability Index, improved by 0.5 points (95% CI [0.2–0.7]; P = .002) compared with placebo. There was a strong correlation between the 2 scale scores. In addition, all secondary outcome measures favored incobotulinumtoxinA. Patients rated the mean therapeutic effect of incobotulinumtoxinA significantly better than placebo (P < .001). Adverse events were reported in 70.3% of incobotulinumtoxinA patients and 58.8% of placebo patients. Eyelid ptosis (18.9% vs 5.9%), dry eye (18.9% vs 11.8%), and dry mouth (14.9% vs 2.9%) occurred most frequently. Tolerability was rated good/very good by 91.9% of incobotulinumtoxinA versus in 85.2% of placebo patients. In conclusion, incobotulinumtoxinA was well tolerated and was associated with statistically significant and clinically relevant reductions in blepharospasm severity and functional impairment. © 2011 Movement Disorder Society

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