2025 American Academy of Neurology Abstract Website

Amy Lin¹, Raphael Pamintuan², Minnie Luu³, Yukang Han⁴, Richard Barbano⁵, Brian Berman⁶, Jeanne Feuerstein⁷, H. Jinnah⁸, Davide Martino⁹, Joel Perlmutter¹⁰, Aparna Wagle-Shukla¹¹, Cynthia Comella¹², Glenn Stebbins¹², Sarah Pirio Richardson¹³, David Peterson¹⁴
¹UC San Diego Parkinson & Other Movement Disorders Center, ²University of California, San Diego, ³University of California, Los Angeles, ⁴Johns Hopkins University, ⁵University of Rochester, ⁶Virginia Commonwealth University, ⁷University of Colorado School of Medicine, ⁸Emory University, ⁹Department of Clinical Neurosciences, University of Calgary, ¹⁰Washington University in St. Louis, ¹¹UF Fixel, ¹²Rush University Medical Center, ¹³University of New Mexico, ¹⁴Salk Institute for Biological Sciences

Objective:

Compare a novel patient-reported outcome with a clinician-reported assessment of head tremor severity in isolated cervical dystonia (CD).

Background:

Over 50% of people with CD have head tremor, often treated with botulinum toxin injections (BoNT). However, efficacy trials of BoNT in CD did not assess changes in tremor. Additionally, patient-reported outcomes (PRO) of head tremor in CD have not been studied, but their importance is increasingly recognized by the FDA.

Design/Methods:

We assessed changes in both patient-reported and clinician-reported head tremor severity, by comparing peak effectiveness and trough periods just prior to BoNT. The patient-reported metric was rated on a Likert-scale of 0 to 10 in response to the question, “How much shaking or tremor do you have in your neck or head?” The clinician-reported metric assessed the patients as they attempted to maintain a midline head posture and at the null point, using a global severity scale (0 to 10).

Results:

We assessed data from 16 cycles (32 timepoints) of BoNT. For a majority of cases, patients and clinicians disagreed on the presence of tremor (n=18, 56.3%). Of those 18 instances, there were 16 instances where patients perceived tremor that was not noted in the video segments. Among instances where patients reported having tremor at time of trough (n=12), 6 patients (50%) reported improvement in tremor after BoNT. The change from trough did not reach statistical significance based on 1-sided Wilcoxon signed rank test for either the patient (S= -19.5, p=0.066) or clinician (S=5.5, p=0.59) reports. The patient and clinician reports of change in tremor before and after BoNT were not concordant (Kendall t = -0.22, p = 0.38).

Conclusions:

There was a non-significant trend towards improvement in head tremor based on patient report. Patient and clinician reports are not interchangeable when assessing response to BoNT for head tremor in CD.