Language
English
Publication Date
9-10-2025
Journal
Toxins
DOI
10.3390/toxins17090455
PMID
41003519
PMCID
PMC12474189
PubMedCentral® Posted Date
9-10-2025
PubMedCentral® Full Text Version
Post-print
Abstract
Blepharospasm (BSP) is characterized by excessive orbicularis oculi muscle activity leading to abnormal blinking and involuntary eyelid closure. Botulinum neurotoxin (BoNT) injections are the main treatment for BSP, but they only partially and transiently relieve symptoms, leading to a waxing and waning therapeutic response. A patient-centered outcome (PCO) tool that measures BSP symptoms in a simple and efficient way could inform the development of better treatments. Using a stepwise modified Delphi approach, potential PCO items were first identified using the Dystonia Coalition Database with data from over 200 individuals with BSP who had provided responses to existing clinical assessment scales. These items were then analyzed for contribution to overall severity using a Random Forests approach, and redundant items were merged and revised in a series of iterative meetings with a specialist panel along with input from patient advocacy group representatives and focus groups. An online survey was conducted with 330 individuals with BSP to validate and verify the items' relevance. Finally, the specialist panel provided content validity ratio, which was repeated until it showed good agreement for relevance and clarity of all items. In the end, an easy-to-use PCO tool designed for smartphones and tablets containing 17 items covering three symptom domains (motor, disability, and psychosocial/quality of life) was created. This novel PCO tool for BSP may be used to characterize the cyclical response that an individual patient experiences from BoNT treatments and provide a vital tool for future investigations of longer-acting BoNT preparations or adjunctive therapies.
Keywords
Humans, Blepharospasm, Delphi Technique, Female, Male, Middle Aged, Aged, Patient Reported Outcome Measures, Botulinum Toxins, Adult, Patient-Centered Care, Treatment Outcome, Neuromuscular Agents, blepharospasm, patient-centered outcomes, botulinum neurotoxin, self-assessments, symptom monitoring, treatment tracking
Published Open-Access
yes
Recommended Citation
Berman, Brian D; Qeadan, Fares; Henderson, Amanda D; et al., "Development of a Patient-Centered Outcome Tool for Blepharospasm: A Stepwise Modified Delphi Study" (2025). Faculty and Staff Publications. 5245.
https://digitalcommons.library.tmc.edu/baylor_docs/5245