Economic and Quality-of-Life Outcomes of Natriuretic Peptide-Guided Therapy for Heart Failure

Abstract

Background: The GUIDE-IT (GUIDing Evidence Based Therapy Using Biomarker Intensified Treatment in Heart Failure) trial prospectively compared the efficacy of an N-terminal pro-B-type natriuretic peptide (NT-proBNP)-guided heart failure treatment strategy (target NT-proBNP level <1,000 pg/ml) with optimal medical therapy alone in high-risk patients with heart failure and reduced ejection fraction. When the study was stopped for futility, 894 patients had been enrolled.

Objectives: The purpose of this study was to assess treatment-related quality-of-life (QOL) and economic outcomes in the GUIDE-IT trial.

Methods: The authors prospectively collected a battery of QOL instruments at baseline and 3, 6, 12, and 24 months post-randomization (collection rates 90% to 99% of those eligible). The principal pre-specified QOL measures were the Kansas City Cardiomyopathy Questionnaire (KCCQ) Overall Summary Score and the Duke Activity Status Index (DASI). Cost data were collected for 735 (97%) U.S.

Results: Baseline variables were well balanced in the 446 patients randomized to the NT-proBNP-guided therapy and 448 to usual care. Both the KCCQ and the DASI improved over the first 6 months, but no evidence was found for a strategy-related difference (mean difference [biomarker-guided - usual care] at 24 months of follow-up 2.0 for DASI [95% confidence interval (CI): -1.3 to 5.3] and 1.1 for KCCQ [95% CI: -3.7 to 5.9]). Total winsorized costs averaged $5,919 higher in the biomarker-guided strategy (95% CI: -$1,795, +$13,602) over 15-month median follow-up.

Conclusions: A strategy of NT-proBNP-guided HF therapy had higher total costs and was not more effective than usual care in improving QOL outcomes in patients with heart failure and a reduced ejection fraction. (Guiding Evidence Based Therapy Using Biomarker Intensified Treatment [GUIDE-IT]; NCT01685840).

Keywords: biomarkers; economics; heart failure; left ventricular dysfunction; quality of life.

Copyright © 2018 American College of Cardiology Foundation. All rights reserved.

Figures

Appendix Figure 1:. KCCQ Treatment Differences in Subgroups at 6 Months in the GUIDE-IT Trial

This figure shows the difference in KCCQ Overall Summary scores between the biomarker-guided strategy and the usual care strategy in pre-specified subgroups. CI=Confidence interval.

Appendix Figure 2:. DASI Treatment Differences in Subgroups at 6 Months in the GUIDE-IT Trial

This figure shows the difference in DASI scores between the biomarker-guided strategy and the usual care strategy in pre-specified subgroups. CI=Confidence interval.

Appendix Figure 3:. Mean Inpatient Costs, by Quintile of Total Cost*

*Quintiles were based on total observed costs in the overall sample. BG=biomarker group; UC=usual care.

Appendix Figure 4:. Mean Inpatient Admission Count, by Quintile of Total Cost*

*Quintiles were based on total observed costs in the overall sample. BG=biomarker group; UC=usual care.

Appendix Figure 5:. Mean Inpatient Costs for Heart Failure Admissions, by Quintile of Total Cost*

*Quintiles were based on total observed costs in the overall sample. BG=biomarker group; UC=usual care.

Appendix Figure 6:. Mean Inpatient Costs for Non-Heart Failure Admissions, by Quintile of Total Cost*

*Quintiles were based on total observed costs in the overall sample. BG=biomarker group; UC=usual care.

Appendix Figure 7:. Mean Inpatient Heart Failure Admission Count, by Quintile of Total Cost*

*Quintiles were based on total observed costs in the overall sample. BG=biomarker group; UC=usual care.

Appendix Figure 8:. Mean Inpatient Non-Heart Failure Admission Count, by Quintile of Total Cost*

*Quintiles were based on total observed costs in the overall sample. BG=biomarker group; UC=usual care.

Appendix Figure 9:. Mean Inpatient Winsorized Cost, by Quintile of Total Cost*

*Quintiles were based on total observed costs in the overall sample. **Costs and counts exceeding the 95th percentile value in the overall sample were set to the 95th percentile. BG=biomarker group; UC=usual care.

Appendix Figure 10:. Mean Inpatient Winsorized Admission Count, by Quintile of Total Cost*

*Quintiles were based on total observed costs in the overall sample. **Costs and counts exceeding the 95th percentile value in the overall sample were set to the 95th percentile. BG=biomarker group; UC=usual care.

Central Illustration: Economic and Quality-of-Life Outcomes with Natriuretic Peptide Guided

Therapy in Heart Failure. The GUIDE-IT trial prospectively compared the efficacy of a NT-proBNP-guided heart failure treatment strategy (target NT-proBNP level <11000 pg/mL) with optimal medical therapy alone in high-risk patients with heart failure and reduced ejection fraction. Treatment-related quality of life and economic outcomes were assessed. The patient population and randomization allocation are shown in the top left corner. Winsorized costs and total costs in USD are presented in the lower left. The scores for the Kansas City Cardiomyopathy Questionnaires and the Duke Activity Status Index are presented by treatment group at baseline and follow-up intervals for both the biomarker-guided treatment group and the usual care group in the charts on the right.

Figure 1:. KCCQ Treatment Differences in the GUIDE-IT Trial.

This figure is a visual representation of the data in Table 1 and Online Table 1. The circles show the mean difference in scores, measured in KCCQ score units, while the bars indicate the 95% confidence interval around that estimate. KCCQ=Kansas City Cardiomyopathy Questionnaire; QOL=quality of life.