Comparison of Outcome Measures (Subjective, Objective, and Patient-Based) in Laryngeal Dystonia Treatment With Botulinum Toxin A Injection

Abstract

Outcome assessment in laryngeal dystonia is hindered by lack of consensus on a core set of outcome measures to quantify treatment effect and disease severity on quality of life. Potential outcome measure domains include objective voice, clinician reported, and patient reported outcome measures (PROMs) for determining treatment success and longitudinal disease tracking. We aim to determine correlations between a selections of outcome measure tools following Botulinum toxin injection. A selection of instruments were administered to assess adductor laryngeal dystonia patient outcomes before and after Botulinum toxin injection. Voice samples recorded using a cellular telephone application were used for objective acoustic measures (CPPS, acoustic voice quality index) and speech language pathologist perceptual analysis (CAPE-V). Additionally, patients completed a PROMs battery consisting of the Voice Handicap Index-10, Communicative Participation Item Bank-10, OMNI-Vocal Effort Scale, 3 visual analog scale (VAS) questions. Changes in these outcome measures pre-post treatment were compared between each other and with a global rating of change questionnaire (GRCQ) using Spearman's rank correlation coefficients. Twenty six patients (20 female, mean age 57.7 years) participated. Using an anchor based GRCQ, patients reported Botox efficacy was the only outcome measure found to have significant correlation (r=0.54, P=0.022); all other outcome measures did not meet statistically significant correlation. Amongst the selected outcome tools, several moderate-strong correlations were identified, largely for outcome measures within the same domain. Most notable were correlations between the patient reported OMNI-VES and VAS questions (r > 0.68, P < 0.05), clinician CAPE-V strain and overall severity (r=0.900, P < 0.001), and acoustic voice quality index with sustained vowel CPPs (r=-0.797, P=0.002). Correlation between outcome measures instruments used for patients with adductor laryngeal dystonia requires further attention. Weak correlations with an anchor based GRCQ were found for this study's selected outcome instruments. A select number of correlations were found between outcome instruments within each of the individual outcome measure domains (patient perception, clinical perception, objective acoustics), but there was largely a lack of correlation found for instruments between these three separate domains.