Effects of Statistical Practices for Longitudinal Group Comparison of the Penetration-Aspiration Scale on Power and Effect Size Estimation: A Monte Carlo Simulation Study.

Multiple bolus trials are administered during clinical and research swallowing assessments to comprehensively capture an individual's swallowing function. Despite valuable information obtained from these boluses, it remains common practice to use a single bolus (e.g., the worst score) to describe the degree of dysfunction. Researchers also often collapse continuous or ordinal swallowing measures into categories, potentially exacerbating information loss. These practices may adversely affect statistical power to detect and estimate smaller, yet potentially meaningful, treatment effects. This study sought to examine the impact of aggregating and categorizing penetration-aspiration scale (PAS) scores on statistical power and effect size estimates. We used a Monte Carlo approach to simulate three hypothetical within-subject treatment studies in Parkinson's disease and head and neck cancer across a range of data characteristics (e.g., sample size, number of bolus trials, variability). Different statistical models (aggregated or multilevel) as well as various PAS reduction approaches (i.e., types of categorizations) were performed to examine their impact on power and the accuracy of effect size estimates. Across all scenarios, multilevel models demonstrated higher statistical power to detect group-level longitudinal change and more accurate estimates compared to aggregated (worst score) models. Categorizing PAS scores also reduced power and biased effect size estimates compared to an ordinal approach, though this depended on the type of categorization and baseline PAS distribution. Multilevel models should be considered as a more robust approach for the statistical analysis of multiple boluses administered in standardized swallowing protocols due to its high sensitivity and accuracy to compare group-level changes in swallowing function. Importantly, this finding appears to be consistent across patient populations with distinct pathophysiology (i.e., PD and HNC) and patterns of airway invasion. The decision to categorize a continuous or ordinal outcome should be grounded in the clinical or research question with recognition that scale reduction may negatively affect the quality of statistical inferences in certain scenarios.

Rehabilitating Cough Dysfunction in Parkinson's Disease: A Randomized Controlled Trial.

BACKGROUND: Disorders of airway protection (cough and swallowing) are pervasive in Parkinson's disease (PD) resulting in a high incidence of aspiration pneumonia and death. However, there are no randomized controlled trials comparing strength and skill-based approaches to improve airway protection in PD. OBJECTIVES: The aim of this study was to compare expiratory muscle strength training (EMST) and sensorimotor training for airway protection (smTAP) to improve cough-related outcomes in people with PD. METHODS: Participants with PD and dysphagia were recruited for this prospective phase II randomized-blinded controlled clinical trial. Participants completed baseline assessment, 5 weeks of EMST or smTAP, and a post-training assessment. Primary outcome measures included maximum expiratory pressure (MEP) and voluntary cough peak expiratory flow rate (PEFR). Mixed effects models were used to assess the effects of EMST and smTAP on outcomes. RESULTS: A total of 65 participants received either EMST (n = 34) or smTAP (n = 31). MEP improved from pre- to post-treatment for smTAP (P < 0.001, d = 0.19) and EMST (P < 0.001, d = 0.53). Voluntary PEFR increased from pre- to post-treatment for smTAP (P < 0.001, d = 0.19) and EMST (P < 0.001, d = 0.06). Moreover, reflex cough PEFR (P < 0.001, d = 0.64), reflex cough expired volume (P < 0.001, d = 0.74), and urge to cough (P = 0.018, OR = 2.70) improved for the smTAP group but not for the EMST group. CONCLUSIONS: This clinical trial confirmed the efficacy of smTAP to improve reflex and voluntary cough function, above and beyond EMST, the current gold standard. © 2022 International Parkinson and Movement Disorder Society.

Statistical Power and Swallowing Rehabilitation Research: Current Landscape and Next Steps.

Despite rapid growth in the number of treatments to rehabilitate dysphagia, studies often demonstrate mixed results with non-significant changes to functional outcomes. Given that power analyses are infrequently reported in dysphagia research, it remains unclear whether studies are adequately powered to detect a range of treatment effects. Therefore, this review sought to examine the current landscape of statistical power in swallowing rehabilitation research. Databases were searched for swallowing treatments using instrumental evaluations of swallowing and the penetration-aspiration scale as an outcome. Sensitivity power analyses based on each study's statistical test and sample size were performed to determine the minimum effect size detectable with 80% power. Eighty-nine studies with 94 treatment comparisons were included. Sixty-seven percent of treatment comparisons were unable to detect effects smaller than d = 0.80. The smallest detectable effect size was d = 0.29 for electrical stimulation, d = 0.49 for postural maneuvers, d = 0.52 for non-invasive brain stimulation, d = 0.61 for combined treatments, d = 0.63 for respiratory-based interventions, d = 0.70 for lingual strengthening, and d = 0.79 for oral sensory stimulation. Dysphagia treatments examining changes in penetration-aspiration scale scores were generally powered to reliably detect larger effect sizes and not smaller (but potentially clinically meaningful) effects. These findings suggest that non-significant results may be related to low statistical power, highlighting the need for collaborative, well-powered intervention studies that can detect smaller, clinically meaningful changes in swallowing function. To facilitate implementation, a tutorial on simulation-based power analyses for ordinal outcomes is provided ( https://osf.io/e6usd/ ).