Abstract 12936: Quality of Life Assessments as Inclusion Criteria in Heart Failure Clinical Trials

Abstract

Background: Patient-reported quality of life (QoL) assessments, such as the Kansas City Cardiomyopathy Questionnaire (KCCQ) or Minnesota Living with Heart Failure Questionnaire (MLHFQ), reflect an individual’s health status and symptom severity at a given timepoint, and may have utility as inclusion criteria in heart failure (HF) clinical trials. Method: The Heart Failure Collaboratory-Academic Research Consortium, a group of stakeholders including patients, investigators, and representatives from government and industry, convened to review data from multiple HF clinical trials discuss the use of patient reported QoL-based inclusion criteria in clinical trials of HF. Result: Patient-reported QoL assessments, such as the KCCQ, have high test-retest reliability and, in contrast with the physician-assigned New York Heart Association (NYHA) class, are not subject to clinician bias or influenced by knowledge of prognostic variables such as Left Ventricular Ejection Fraction (LVEF) or N-terminal pro B-type Natriuretic Peptide (NT-proBNP). The U.S. FDA has qualified KCCQ as an effective clinical outcomes assessment for HF, and recent clinical trials that demonstrated substantial reductions in morbidity and mortality also showed improvements in KCCQ scores, provided patients had a decrement at baseline. However, KCCQ use is limited by both complicated clinical interpretability, with total symptom score consisting of aggregate scores from different physical and symptomatic domains and uncertainty around the threshold of clinically important difference, and by its length, with average completion time of 5 to 8 minutes. Conclusion: Patient-reported functional assessments have potential as inclusion criteria in clinical trials of HF by ensuring the enrollment of patients with a diminished QoL who have the potential to improve in response to therapeutic intervention, especially in clinical trials with QoL-based endpoints.