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Published on: June 10, 2025
Minimally Clinically Important Difference in Health Status Scores in Patients With HFrEF vs HFpEF
Javed Butler1, Muhammad Shahzeb Khan2, JoAnn Lindenfeld3
1Department of Medicine, University of Mississippi, Jackson, Mississippi, USA.
Insights
Clinically meaningful improvement in the Kansas City Cardiomyopathy Questionnaire (KCCQ) is at least 9 points for heart failure with reduced ejection fraction (HFrEF) and 7 points for heart failure with preserved ejection fraction (HFpEF). These thresholds aid in evaluating treatment effectiveness in heart failure patients.
Area of Science:
- Cardiology
- Clinical Trials
- Patient-Reported Outcomes
Background:
- Establishing clinically significant thresholds for patient-reported outcome measures like the Kansas City Cardiomyopathy Questionnaire (KCCQ) is crucial for differentiating between heart failure with preserved ejection fraction (HFpEF) and heart failure with reduced ejection fraction (HFrEF).
- Existing data on these thresholds are less defined for HFpEF compared to HFrEF.
Purpose of the Study:
- To estimate meaningful thresholds for improvement or deterioration in the KCCQ-Total Symptom Score (TSS).
- To compare these thresholds between patients with HFrEF and HFpEF.
Main Methods:
- A secondary analysis of the EMPERIAL program data was conducted.
- Anchor- and distribution-based approaches were utilized, with Patient Global Impression of Severity (PGIS) as the primary anchor.
- Mean changes in KCCQ-TSS from baseline to week 12 were calculated based on PGIS categories.
Main Results:
- The study included 312 HFrEF and 315 HFpEF patients.
- A meaningful threshold for KCCQ-TSS improvement was identified as ≥9 points for HFrEF and ≥7 points for HFpEF.
- These thresholds demonstrated acceptable sensitivity and specificity in distinguishing meaningful change.
Conclusions:
- A change of ≥9 points in KCCQ-TSS signifies the minimal clinically important difference for improvement in HFrEF patients.
- A change of ≥7 points in KCCQ-TSS signifies the minimal clinically important difference for improvement in HFpEF patients.
- These findings confirm a broad range of 5-10 points as meaningful thresholds for KCCQ-TSS change in heart failure management.
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 preserved 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 corresponding 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.
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