Minimally Clinically Important Difference in Health Status Scores in Patients With HFrEF vs HFpEF

BACKGROUND Differences in clinically important thresholds in patient-reported outcomes measures such as the Kansas City Cardiomyopathy Questionnaire (KCCQ) remain less well-established in patients with heart failure with pre-served ejection fraction (HFpEF) versus heart failure with reduced ejection fraction (HFrEF). OBJECTIVES The purpose of this study was to estimate meaningful thresholds for improvement or deterioration in the KCCQ-Total Symptom Score (TSS) in patients with HFrEF versus HFpEF. METHODS This secondary analysis of EMPERIAL program used anchor-and distribution-based approaches to estimate thresholds for improvement or deterioration in the KCCQ-TSS using Patient Global Impression of Severity (PGIS) as the primary anchor. Mean change in KCCQ-TSS from baseline to week 12 was calculated for each PGIS.RESULTS A total of 312 HFrEF and 315 HFpEF patients were enrolled. At week 12, mean changes in KCCQ-TSS corre-sponding to PGIS changes of any improvement, 1-category improvement, and 1-category deterioration were 13 +/- 17, 12 +/- 17,-3 +/- 16 points in HFrEF, and 15 +/- 18, 13 +/- 17,-7 +/- 18 points in HFpEF. Threshold for meaningful within -patient change in KCCQ-TSS was >= 9 points in HFrEF and >= 7 points in HFpEF patients. Sensitivity and specificity of >= 9 points/>= 7 points change was 0.65 and 0.70 for HFrEF and 0.64 and 0.66 for HFpEF. Cumulative distribution function curves of KCCQ-TSS change from baseline to week 12 showed a shift to higher scores in both HFrEF and HFpEF patients.CONCLUSIONS In the EMPERIAL program, a change in KCCQ-TSS of >= 9 points in HFrEF and >= 7 points in HFpEF represents the minimal clinically important difference for improvement, confirming the broad range of 5-10 points as meaningful thresholds. (J Am Coll Cardiol HF 2022;10:651-661)(c) 2022 The Authors. Published by Elsevier on behalf of the American College of Cardiology Foundation. This is an open access article under the CC BY license (http://creativecommons.org/licenses/by/4.0/).

Automated summary

This study establishes meaningful thresholds for patient-reported outcomes measures in heart failure patients and identifies the minimal clinically important difference in KCCQ-TSS for both HFrEF and HFpEF patients.