Saturday, September 7, 2024
9/7/2024
The dystonias overall are a rare neurologic disorder. Blepharospasm (BSP), is one of the most common forms of dystonia. BSP is characterized by involuntary abnormal facial expressions and eye closures. In addition to these overt motor abnormalities, the disorder is also associated with non-motor symptoms including anxiety and depression. Treatment options for BSP are suboptimal. Many oral medications have been tried but their efficacy is minimal and limited by dose-dependent adverse side effects. Botulinum neurotoxin (BoNT) injections repeated every 3-4 months are the primary treatment of choice. Although BoNT is highly efficacious for many patients, for a variety of reasons about 1/3 of patients discontinue BoNT treatment, and of those who continue treatment about 1/3 are unsatisfied with the response. Because treatment options are suboptimal, there is an active effort to find better strategies for treating BSP, as evidenced by dozens of trials listed on ClinicalTrials.gov. However, the most common clinical outcome assessments used to measure motor abnormalities are intrinsically subjective assessments and therefore suffer from inter-rater variability. This reduces our power to detect treatment effects in clinical trials. Technology-based objective measures have the potential to circumvent this variability. Advances in computer vision technology have enabled the measurement of facial expressions and eye closure from 2-D images of the face in conventional video recordings. One of the long-term objectives of our group is to leverage these advances to develop software that can capture and quantify motor abnormalities across multiple types of focal dystonia. We are calling this system the Computational Motor Objective Rater (CMOR). In this project specifically targeting BSP, our aims are 1) to evaluate CMOR’s convergent validity with patient reports of severity of facial spasms and excessive blinking and 2) to determine CMOR’s sensitivity to changes in severity associated with interventions. To accomplish these aims, we will conduct CMOR analyses of motor symptoms from video recordings of 100 BSP patients enrolled in a separate Dystonia Coalition project to evaluate the variability of efficacy of BoNT. That project will also acquire patient reports in the form of a patient centered outcome with specific questions about the prominent motor features of BSP and the patient’s global impression of change (PGIC) in response to each BoNT treatment. Collectively the results will provide important information about CMOR’s validity and a quantitative basis for sample size estimates for future clinical trials in BSP.
HHS’ Tracking Accountability in Government Grants System (TAGGS) website provides access to detailed descriptions of grants, loans, aggregated direct payments and other types of financial assistance awarded by the HHS Operating Divisions (OPDIVs) and the Office of the Secretary Staff Divisions (STAFFDIVs).
