Flynn Kathryn E, Lin Li, Ellis Stephen J, Russell Stuart D, Spertus John A, Whellan David J, Piña Ileana L, Fine Lawrence J, Schulman Kevin A, Weinfurt Kevin P
Center for Clinical and Genetic Economics, Duke Clinical Research Institute, Duke University School of Medicine, Durham, NC 27715, USA.
Am Heart J. 2009 Oct;158(4 Suppl):S64-71. doi: 10.1016/j.ahj.2009.07.010.
Patient-reported outcomes are increasingly used to assess the efficacy of new treatments. Understanding relationships between these and clinical measures can facilitate their interpretation. We examined associations between patient-reported measures of health-related quality of life and clinical indicators of disease severity in a large, heterogeneous sample of patients with heart failure.
Patient-reported measures, including the Kansas City Cardiomyopathy Questionnaire (KCCQ) and the EuroQol Visual Analog Scale (VAS), and clinical measures, including peak VO(2), 6-minute walk distance, and New York Heart Association (NYHA) class, were assessed at baseline in 2331 patients with heart failure. We used general linear models to regress patient-reported measures on each clinical measure. Final models included for significant sociodemographic variables and 2-way interactions.
The KCCQ was correlated with peak VO(2) (r = .21) and 6-minute walk distance (r = .27). The VAS was correlated with peak VO(2) (r = .09) and 6-minute walk distance (r = .11). Using the KCCQ as the response variable, a 1-SD difference in peak Vo(2) (4.7 mL/kg/min) was associated with a 2.86-point difference in the VAS (95% CI, 1.98-3.74) and a 4.75-point difference in the KCCQ (95% CI, 3.78-5.72). A 1-SD difference in 6-minute walk distance (105 m) was associated with a 2.78-point difference in the VAS (95% CI, 1.92-3.64) and a 5.92-point difference in the KCCQ (95% CI, 4.98-6.87); NYHA class III was associated with an 8.26-point lower VAS (95% CI, 6.59-9.93) and a 12.73-point lower KCCQ (95% CI, 10.92-14.53) than NYHA class II.
These data may inform deliberations about how to best measure benefits of heart failure interventions, and they generally support the practice of considering a 5-point difference on the KCCQ and a 3-point difference on the VAS to be clinically meaningful.
患者报告的结果越来越多地用于评估新治疗方法的疗效。了解这些结果与临床指标之间的关系有助于对其进行解读。我们在一个大型、异质性心力衰竭患者样本中，研究了患者报告的健康相关生活质量测量指标与疾病严重程度临床指标之间的关联。
在2331例心力衰竭患者基线时，评估患者报告的测量指标，包括堪萨斯城心肌病问卷（KCCQ）和欧洲五维度健康量表视觉模拟量表（VAS），以及临床测量指标，包括峰值摄氧量（VO₂）、6分钟步行距离和纽约心脏协会（NYHA）心功能分级。我们使用一般线性模型，将患者报告的测量指标对每个临床测量指标进行回归分析。最终模型纳入了显著的社会人口统计学变量和双向交互作用。
KCCQ与峰值VO₂（r = 0.21）和6分钟步行距离（r = 0.27）相关。VAS与峰值VO₂（r = 0.09）和6分钟步行距离（r = 0.11）相关。以KCCQ作为反应变量，峰值VO₂每相差1个标准差（4.7 mL/kg/min），VAS相差2.86分（95%CI，1.98 - 3.74），KCCQ相差4.75分（95%CI，3.78 - 5.72）。6分钟步行距离每相差1个标准差（105 m），VAS相差2.78分（95%CI，1.92 - 3.64），KCCQ相差5.92分（95%CI，4.98 - 6.87）；与NYHA心功能Ⅱ级相比，NYHA心功能Ⅲ级的VAS低8.26分（95%CI，6.59 - 9.93），KCCQ低12.73分（95%CI，10.92 - 14.53）。
这些数据可能为如何最佳衡量心力衰竭干预措施的益处提供参考，并且总体上支持将KCCQ相差5分和VAS相差3分视为具有临床意义的做法。
