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Multicenter Study
. 2010 Jun;48(6 Suppl):S9-16.
doi: 10.1097/MLR.0b013e3181d99161.

Who can respond to treatment? Identifying patient characteristics related to heterogeneity of treatment effects

Sherrie H Kaplan  1 , John Billimek, Dara H Sorkin, Quyen Ngo-Metzger, Sheldon Greenfield
Affiliations
Multicenter Study

Who can respond to treatment? Identifying patient characteristics related to heterogeneity of treatment effects

Sherrie H Kaplan et al. Med Care. 2010 Jun.

Abstract

Background: 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.

Objective: 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.

Design: Cross-sectional and longitudinal cohort study.

Subjects and setting: 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.

Measures: 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.

Results: 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).

Conclusions: The PBS, a composite scale, may be helpful in identifying patients with differential potential for response to treatment.

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Figures

FIGURE 1
FIGURE 1
Conceptual model for the development of the Potential for Benefit Scale (PBS).
See this image and copyright information in PMC

References

    1. Greenfield S, Billimek J, Pellegrini F, et al. Comorbidity affects the relationship between glycemic control and cardiovascular outcomes in diabetes: a cohort study. Ann Intern Med. 2009;151:854–860. - PubMed
    1. Greenfield S, Kravitz RL, Duan N, et al. Heterogeneity of treatment effects: implications for guidelines, payment, and quality assessment. Am J Med. 2007;120:S3–S9. - PubMed
    1. Greenfield S, Kravitz RL. Engaging the implications of subgroup heterogeneity: the increasing inadequacy of randomized trials for guidelines, payment and quality. Washington, DC: Institute of Medicine Report on the Learning Healthcare Environment: National Academy of Sciences; 2007.
    1. Kravitz RL, Duan N, Braslow J. Evidence-based medicine, heterogeneity of treatment effects, and the trouble with averages. Milbank Q. 2004;82:661–687. - PMC - PubMed
    1. Kent D, Hayward R. When Averages hide individual differences in clinical trials: analyzing the results of clinical trials to expose individual patients’ risks might help doctors make better treatment decisions. Am Sci. 2007;95:60–68.

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