Health Policy Research Institute, Department of Medicine, University of California, Irvine, CA 92697, USA.
Med Care. 2010 Jun;48(6 Suppl):S9-16. doi: 10.1097/MLR.0b013e3181d99161.
Interest in comparative effectiveness research and the rising number of negative or "small effect" trials have stimulated research into differential response to treatment among subgroups of patients.
To develop and test the Potential for Benefit Scale (PBS), a composite measure to identify subgroups of patients with differential potential for response to treatment, using diabetes as a model.
Cross-sectional and longitudinal cohort study.
Type 2 diabetes patients (n = 1361) were identified from 7 outpatient clinics serving a diverse population. Of these, 611 completed a 1-year follow-up.
To represent patients' health status, we used the Total Illness Burden Index, the Physical Function Index of the SF-36, the Center for Epidemiologic Studies Depression Scale, and the Diabetes Burden Scale. To represent personality characteristics related to health, we used the Provider-Dependent Health Care Orientation scale. We assessed the contribution of these measures to a composite scale of patients' potential for treatment response in terms of self-reported medication adherence and glycemic control.
Principal components analysis confirmed associations among these measures. The internal consistency reliability of the PBS was adequate (Cronbach alpha = 0.65). Patients in the lowest versus highest quartile of the PBS reported poorer adherence (18% vs. 55%, P < 0.001) and poorer glycemic control at baseline (mean hemoglobin A1c values: 7.75 vs. 7.39, P < 0.001). Those in the highest quartile of the PBS also were more likely to reach target values for glycemic control (HbA1c <7%) at 1-year follow-up, (adjusted OR = 1.61, P < 0.05).
The PBS, a composite scale, may be helpful in identifying patients with differential potential for response to treatment.
对比较疗效研究的兴趣以及越来越多的负面或“小效应”试验刺激了对患者亚组之间治疗反应差异的研究。
以糖尿病为模型，开发并测试潜在获益量表（PBS），这是一种识别对治疗反应有不同潜在获益的患者亚组的综合衡量标准。
横断面和纵向队列研究。
从为不同人群服务的 7 家门诊诊所中确定了 2 型糖尿病患者（n=1361）。其中 611 人完成了 1 年的随访。
为了代表患者的健康状况，我们使用了总疾病负担指数、SF-36 的身体功能指数、流行病学研究中心抑郁量表和糖尿病负担量表。为了代表与健康相关的人格特征，我们使用了依赖提供者的医疗保健取向量表。我们根据自我报告的药物依从性和血糖控制情况，评估这些措施对患者治疗反应潜在能力的综合量表的贡献。
主成分分析证实了这些措施之间的关联。PBS 的内部一致性信度良好（Cronbach alpha=0.65）。PBS 最低四分位与最高四分位的患者报告的依从性较差（18%比 55%，P<0.001），基线时血糖控制较差（平均糖化血红蛋白值：7.75 比 7.39，P<0.001）。在 PBS 最高四分位的患者在 1 年随访时也更有可能达到血糖控制的目标值（HbA1c<7%），（调整后的 OR=1.61，P<0.05）。
PBS 是一种综合量表，可能有助于识别对治疗反应有不同潜在获益的患者。
