Interpreting the Kansas City Cardiomyopathy Questionnaire in Clinical Trials and Clinical Care

Spertus, John A.; Jones, Philip G.; Sandhu, Alexander T.; Arnold, Suzanne V.

doi:10.1016/j.jacc.2020.09.542

Cited by 260 publications

(282 citation statements)

References 34 publications

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“…Pooled results of the three large-scale RCTs reporting the KCCQ score showed significant improvement by SGLT2i compared with placebo in T2DM patients with stage C HF. As for the magnitude of the effect, analysis of the T2DM subgroup in DAPA-HF trial [10] showed that more patients reported an increase of at least 5 points in the SGLT2i group compared with the placebo group (58.9% vs 49.9%), yielding a number needed to treat of 14 patients with dapagliflozin for one to be clinically better in eight months, which showed a considerable benefit [65]. The MD in the change of the KCCQ score was 4.1 points (95% CI 1.3 to 7.0) in the SOLOIST-WHF trial and 2.41 (95% CI 0.64 to 4.17) in the T2DM subgroup in EMPEROR-Reduced trial, but the numbers needed to treat were not calculable.…”

Section: Discussionmentioning

confidence: 99%

Effect of sodium–glucose cotransporter 2 inhibitors on cardiac structure and function in type 2 diabetes mellitus patients with or without chronic heart failure: a meta-analysis

Zhao

Wang

et al. 2021

Cardiovasc Diabetol

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Background Although the benefits of sodium–glucose cotransporter 2 inhibitors (SGLT2i) on cardiovascular events have been reported in patients with type 2 diabetes mellitus (T2DM) with or without heart failure (HF), the impact of SGLT2i on cardiac remodelling remains to be established. Methods We searched the PubMed, Embase, Cochrane Library and Web of Science databases up to November 16th, 2020, for randomized controlled trials reporting the effects of SGLT2i on parameters of cardiac structure, cardiac function, plasma N-terminal pro-brain natriuretic peptide (NT-proBNP) level or the Kansas City Cardiomyopathy Questionnaire (KCCQ) score in T2DM patients with or without chronic HF. The effect size was expressed as the mean difference (MD) or standardized mean difference (SMD) and its 95% confidence interval (CI). Subgroup analyses were performed based on the stage A–B or stage C HF population and HF types. Results Compared to placebo or other antidiabetic drugs, SGLT2i showed no significant effects on left ventricular mass index, left ventricular end diastolic volume index, left ventricular end systolic volume index, or left atrial volume index. SGLT2i improved left ventricular ejection fraction only in the subgroup of HF patients with reduced ejection fraction (MD 3.16%, 95% CI 0.11 to 6.22, p = 0.04; I2 = 0%), and did not affect the global longitudinal strain in the overall analysis including stage A–B HF patients. SGLT2i showed benefits in the E/e’ ratio (MD − 0.45, 95% CI − 0.88 to − 0.03, p = 0.04; I2 = 0%), plasma NT-proBNP level (SMD − 0.09, 95% CI − 0.16 to − 0.03, p = 0.004; I2 = 0%), and the KCCQ score (SMD 3.12, 95% CI 0.76 to 5.47, p = 0.01; I2 = 0%) in the overall population. Conclusion The use of SGLT2i was associated with significant improvements in cardiac diastolic function, plasma NT-proBNP level, and the KCCQ score in T2DM patients with or without chronic HF, but did not significantly affect cardiac structural parameters indexed by body surface area. The LVEF level was improved only in HF patients with reduced ejection fraction.

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Section: Discussionmentioning

confidence: 99%

Effect of sodium–glucose cotransporter 2 inhibitors on cardiac structure and function in type 2 diabetes mellitus patients with or without chronic heart failure: a meta-analysis

Zhao

Wang

et al. 2021

Cardiovasc Diabetol

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“…It is notable that the developer of the KCCQ and colleagues recently published a framework for interpreting the KCCQ as a trial endpoint. Their state‐of‐the‐art review 12 advocates using the original KCCQ guidelines where small, moderate‐to‐large, and large‐to‐very large clinical changes are defined by changes of 5, 10, and 20 points, respectively. Before this proposed framework can be used with confidence by clinicians and patients, it may be necessary to address how change scores from numerous trials (as noted above) should be clinically interpreted where different quantiles of KCCQ change were required to demonstrate a prognostic association with clinical outcomes.…”

Section: Change Relative To Baselinementioning

confidence: 99%

Current challenges for using the Kansas City Cardiomyopathy Questionnaire to obtain a standardized patient‐reported health status outcome

Stogios

Fezza

Wong

et al. 2021

European J of Heart Fail

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“…More often, some patients improve a lot, some a little, some do not change, and others get worse. Without reporting the proportions of patients with different magnitudes of clinical change, it is difficult to interpret the benefits of therapy on patients' health status 3 . Of course, the same applies to dichotomous outcomes such as death.…”

Section: Figurementioning

confidence: 99%

“…5 Despite this attempt to offer a standardized approach for interpreting thresholds of change, there has been wide variation in the reporting of the KCCQ in clinical trials and observational studies. 2 To support a more standardized approach, we recently published a state-of-the-art review specifically on this topic, 3 where we outlined how to interpret scores cross-sectionally in both a clinical and prognostic context; how to report and interpret changes in scores; how to best report the KCCQ as outcomes in trials; and how to account for missing data due to death. Despite the guidance offered over the past two decades, clinical trialists often develop their own approaches to reporting health status data.…”

mentioning

confidence: 99%

Standardizing the standard: reporting health status in clinical trials

Arnold

Butler

Spertus

2021

European J of Heart Fail

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In ∼20 BCE, Marcus Vipsanius Agrippa, a powerful Roman general and Augustus Caesar's right-hand man, made a decree that the Roman 'foot' would be precisely 11.65 in., or the length of Agrippa's foot. 1 Prior to this, the length of a foot was determined based on each individual's own body part, which can vary by several inches depending on their height. The importance of standardizing the length of a foot cannot be understated, as this paved the way for the Romans' achievements in engineering and construction, including aqueducts, roadways, and monuments. This system of measurement brought order to randomness. It did not really matter what the actual length of a foot was; just that it was precise, reproducible, and accepted as the standard. Although the Kansas City Cardiomyopathy Questionnaire (KCCQ) can reliably and validly assess health status in patients with heart failure, the current reporting of health status measures in clinical trials is similar to the era before Agrippa. Stogios et al., 2 in their perspective piece, call for the need to standardize reporting of these measures. We could not agree more. The KCCQ is a continuous measure with scores ranging from 0 to 100. As with any continuous measure, the interpretation of outcomes, particularly average outcomes, can be more challenging than with discrete events such as hospitalizations or deaths. When trials compare means or mean changes in scores, the clinical significance of these differences may not be clear, particularly in large studies with higher statistical power. For example, few individual patients, if any, actually change by the same amount as the mean of the population. More often, some patients improve a lot, some a little, some do not change, and others get worse. Without reporting the proportions of patients with different magnitudes of clinical change, it is difficult to interpret the benefits of therapy on patients' health status. 3 Of course, the same applies to dichotomous outcomes such as death. If a trial shows a 2% difference in deaths between the groups, obviously this does not mean that every patient in the worse group is 2% dead or 2% more dead. Moreover, it may also by difficult to judge whether a certain percentage reduction in death is meaningful, especially if there are risks from treatment, as this also requires interpretation and judgement.

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Interpreting the Kansas City Cardiomyopathy Questionnaire in Clinical Trials and Clinical Care

Cited by 260 publications

References 34 publications

Effect of sodium–glucose cotransporter 2 inhibitors on cardiac structure and function in type 2 diabetes mellitus patients with or without chronic heart failure: a meta-analysis

Effect of sodium–glucose cotransporter 2 inhibitors on cardiac structure and function in type 2 diabetes mellitus patients with or without chronic heart failure: a meta-analysis

Current challenges for using the Kansas City Cardiomyopathy Questionnaire to obtain a standardized patient‐reported health status outcome

Standardizing the standard: reporting health status in clinical trials

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