CoPE II: Individualizing patient-reported outcomes in patient care for vocal fold paralysis in the clinic and in research

PROJECT SUMMARY Developing an individualized approach to interpreting patient-reported outcome measures (PROM) scores is essential to providing quality care. In this proposal, we plan to create an individualized minimally clinically important difference (MCID) that is specific to each patient in order to improve the ability of PROMs to assess individual patient preferences and measure responsiveness to treatment in heterogeneous patient populations. Specifically, we will evaluate how clinically relevant information (e.g., baseline severity, sociodemographic characteristics) affects the interpretation of PROMs and how this information should be incorporated when making clinical decisions for individual patients. Unilateral vocal fold paralysis (paralysis) is a life-changing condition caused by injury to one recurrent laryngeal nerve and results in temporary or permanent glottal incompetence. Effects on health and quality of life can include loss of voice, swallowing dysfunction, and psychosocial distress. Paralysis is suited for developing the individualized MCID because (1) treatments exist, but effectiveness is poorly characterized; (2) decision-making is based on individual patient preferences; and (3) patient symptoms are heterogeneous, affected by injury severity and unique patient characteristics. Our team has pioneered statistical theory to develop the individualized MCID and simultaneously developed and validated the Cord Paralysis Experience (CoPE) – a novel PROM designed to assess disability specific to paralysis. The proposed study will develop an individualized MCID that incorporates each patient's injury severity, vocal demands, and sociodemographic characteristics, which can be used to improve the interpretability of PROMs at the point-of-care and align patient-provider expectations. To do this, we will perform an observational cohort study that measures pre- and post-treatment CoPE score changes among adults treated for acute paralysis (<3 months from injury, when symptoms are most severe) with standard-of- care interventions across a 37-site national collaborative of high-volume voice centers. The specific aims for this project are: (1) create individualized minimal clinically important differences (iMCID) that account for each patient's baseline injury severity, vocal demands, and sociodemographic characteristics, (2) test the heterogeneity of treatment effect among patients with acute paralysis to identify factors associated with treatment effectiveness of speech therapy and/or injection augmentation, and (3) develop a method to contextualize patient preferences using the iMCID with expected treatment effects in order to align patient- provider expectations. This work will revolutionize how the MCID is applied in heterogeneous patient populations while enabling clinical trials to improve the care of patients with paralysis, and making CoPE a useful, standardized way to assess disability in patients with paralysis at the point-of-care.