Development of a Patient-Centered Outcome Tool for Blepharospasm: A Stepwise Modified Delphi Study.

Area of Science:

• Neurology
• Ophthalmology
• Patient-Reported Outcomes

Background:

• Blepharospasm (BSP) involves involuntary eyelid closure due to excessive orbicularis oculi muscle activity.
• Current treatments like Botulinum neurotoxin (BoNT) offer only partial, temporary relief, resulting in fluctuating patient responses.
• A need exists for a simple, efficient tool to measure BSP symptoms from the patient's perspective.

Purpose of the Study:

• To develop and validate a novel patient-centered outcome (PCO) tool for assessing blepharospasm (BSP) symptoms.
• To create a tool that can capture the cyclical nature of BSP symptoms and treatment responses.
• To provide a resource for future research on improved BSP treatments.

Main Methods:

• A stepwise modified Delphi approach was employed, starting with data from over 200 individuals with BSP.
• Item analysis utilized Random Forests, followed by iterative refinement with specialist panels, patient advocacy groups, and focus groups.
• Online surveys with 330 individuals with BSP validated item relevance, with final content validity ratio ensuring clarity and agreement.

Main Results:

• A 17-item patient-centered outcome (PCO) tool for blepharospasm (BSP) was successfully developed.
• The tool covers three key domains: motor symptoms, disability, and psychosocial impact/quality of life.
• The PCO tool is designed for easy use on smartphones and tablets.

Conclusions:

• The developed PCO tool offers a user-friendly method for evaluating BSP symptoms and their impact.
• This tool can characterize the waxing and waning response to Botulinum neurotoxin (BoNT) treatments in individual patients.
• It serves as a vital instrument for future investigations into novel therapies, including longer-acting BoNT preparations or adjunctive treatments for blepharospasm.