Background

The primary goal for treating patients with obstructive hypertrophic cardiomyopathy (oHCM) is to improve their symptoms, function, and quality of life. Although the Kansas City Cardiomyopathy Questionnaire (KCCQ) is a valid, reliable, and sensitive measure for other etiologies of heart failure, its appropriateness for patients with oHCM is unknown.

Objectives

The purpose of this study was to establish the interpretability, validity, reliability, and responsiveness of the KCCQ in patients with oHCM.

Methods

Cognitive debriefing of the KCCQ was performed in 26 patients with oHCM. The validity, reliability, responsiveness, and interpretability of the KCCQ were tested in 196 participants from the EXPLORER-HCM trial by comparing each scale with relevant comparators, describing the internal reliability and the mean change in stable patients, and comparing the mean change in patients who reported different degrees of clinical change using a patient-reported global impression of change (PGIC).

Results

All KCCQ domains demonstrated strong correlations with external standards of symptoms, function, social limitation, and quality of life, including a recently designed instrument measuring symptoms not captured by the KCCQ (P < 0.0001 for all). Mean changes in stable patients were nonsignificant, ranging from 0.21 to 2.3 points (P > 0.30 for all), with high intraclass correlation coefficients. The mean changes in patients with small, moderate, and large clinical changes were consistent with the 5-, 10-, and 20-point mean differences observed in other etiologies of heart failure.

Conclusions

The KCCQ is well understood by patients with oHCM and has strong evidence of good psychometric performance. It can not only serve as a relevant endpoint in clinical trials of oHCM therapy, but may also prove useful in the clinical care of patients with oHCM. (Clinical Study to Evaluate Mavacamten [MYK-461] in Adults With Symptomatic Obstructive Hypertrophic Cardiomyopathy [EXPLORER-HCM]; NCT03470545)

中文翻译：

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堪萨斯城心肌病问卷在症状性梗阻性肥厚性心肌病中的验证

背景

治疗阻塞性肥厚型心肌病 (oHCM) 患者的主要目标是改善他们的症状、功能和生活质量。尽管堪萨斯城心肌病问卷 (KCCQ) 是一种有效、可靠和敏感的测量其他心力衰竭病因的方法，但它对 oHCM 患者的适用性尚不清楚。

目标

本研究的目的是确定 KCCQ 在 oHCM 患者中的可解释性、有效性、可靠性和反应性。

方法

对 26 名 oHCM 患者进行了 KCCQ 的认知汇报。KCCQ 的有效性、可靠性、反应性和可解释性在 EXPLORER-HCM 试验的 196 名参与者中进行了测试，通过将每个量表与相关比较器进行比较，描述稳定患者的内部可靠性和平均变化，并比较患者的平均变化他们使用患者报告的全球变化印象（PGIC）报告了不同程度的临床变化。

结果

所有 KCCQ 领域都表现出与症状、功能、社会限制和生活质量的外部标准密切相关，包括最近设计的测量 KCCQ 未捕获的症状的仪器（所有P < 0.0001）。稳定患者的平均变化不显着，范围从 0.21 到 2.3 分（所有P > 0.30），具有高组内相关系数。具有小、中和大临床变化的患者的平均变化与在其他心力衰竭病因中观察到的 5、10 和 20 点平均差异一致。

结论

KCCQ 为 oHCM 患者所熟知，并有良好的心理测量表现的有力证据。它不仅可以作为 oHCM 治疗临床试验的相关终点，还可以证明对 oHCM 患者的临床护理有用。（评估 Mavacamten [MYK-461] 在成人症状性阻塞性肥厚性心肌病 [EXPLORER-HCM] 中的临床研究；NCT03470545）