Abstract 12321: Patient-Reported Outcomes in Clinical Studies of Patients With Hypertrophic Cardiomyopathy

Introduction: Therapy for hypertrophic cardiomyopathy (HCM) is primarily targeted toward alleviation of symptoms and improvement in function and quality of life. Yet, the contemporary landscape of patient-reported outcomes (PROs) in HCM clinical research has not been investigated. Methods: ClinicalTrials.gov was queried to identify clinical studies of HCM that reported PROs as outcome measures. All studies of HCM as the disease condition were included, and PROs were identified using specific search terms in the Outcome Measures field. Study characteristics were collected and compared between those that did versus did not report PROs. Results: From November 1987 to February 2022, 181 studies including participants with HCM were registered on ClinicalTrials.gov. Of these, 35 (19%) included PROs as primary or secondary outcome measures. Studies reporting PROs were more likely to be interventional (85.7% vs. 46.6%; p<0.001) and to involve randomization (65.7% vs. 24.7%; p=0.003) compared with those that did not report PROs. Prior to 2007, no clinical studies that reported PROs were registered in ClinicalTrials.gov; however, PRO reporting has increased over the last 15 years. Ninety-four percent of studies that reported PROs as outcomes enrolled fewer than 500 participants. In total, 66 PRO tools or domains were listed as outcome measures. Of these, the Kansas City Cardiomyopathy Questionnaire (KCCQ) was the most often used. Conclusion: Only approximately one in five registered clinical studies of participants with HCM report PROs. Only one PRO tool, the KCCQ, has been validated in studies of patients with HCM. HCM-specific symptoms such as palpitations, dizziness, angina, syncope, and psychiatric impacts are not adequately assessed by existing PRO tools. Future research is needed to develop and validate PRO tools that adequately capture HCM-specific symptoms and longitudinal health status trajectories in patients with varying HCM subphenotypes.