Dataset Information

The Use of the Restricted Mean Survival Time as a Treatment Measure in HIV/AIDS Clinical Trial: Reanalysis of the ACTG A5257 Trial.

ABSTRACT:

Background

The restricted mean survival time (RMST) measures have not been used as primary measure of efficacy in HIV/AIDS clinical trials. In this work, we aim to compare analysis based on the difference in RMST (Δ-RMST) measure and 2 other treatment-effect measures in a recent HIV equivalence trial, and to investigate the performance and characteristics of Δ-RMST-based analysis in a simulation study.

Setting and methods

We reanalyzed a recent HIV equivalence trial (ACTG A5257 trial) with hazard ratio and Δ-RMST, and then compared the results with the original analysis based on risk difference estimated by Kaplan-Meier curves (RDKM). In a simulation study, we investigated the performance and operating characteristics of Δ-RMST-based analysis in the setting of non-proportional hazards (PH) ratio.

Results

In the ACTG A5257 trial, analyses based on Δ-RMST globally led to similar conclusions as the published finding based on RDKM. By contrast, analyses based on hazard ratio provided some discordant equivalence conclusions compared both with the initial analyses based on RDKM and the Δ-RMST. Results of simulation study indicate that the violation of the PH assumption has an impact on Δ-RMST-based analysis regarding the probability of declaring equivalence.

Conclusions

Although the RMST-based analysis is an alternative measure of efficacy in HIV/AIDS, clinical trials such an analysis can be strongly impacted by departures from the PH assumption.

SUBMITTER: Abulizi X

PROVIDER: S-EPMC6642730 | biostudies-literature | 2019 May

REPOSITORIES: biostudies-literature

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Publications

The Use of the Restricted Mean Survival Time as a Treatment Measure in HIV/AIDS Clinical Trial: Reanalysis of the ACTG A5257 Trial.

Abulizi Xianmixikemaier X Ribaudo Heather J HJ Flandre Philippe P

Journal of acquired immune deficiency syndromes (1999) 20190501 1

<h4>Background</h4>The restricted mean survival time (RMST) measures have not been used as primary measure of efficacy in HIV/AIDS clinical trials. In this work, we aim to compare analysis based on the difference in RMST (Δ-RMST) measure and 2 other treatment-effect measures in a recent HIV equivalence trial, and to investigate the performance and characteristics of Δ-RMST-based analysis in a simulation study.<h4>Setting and methods</h4>We reanalyzed a recent HIV equivalence trial (ACTG A5257 tr ...[more]

PMID: 30789450

Similar Datasets

Project description:Background/objectivesRestricted mean survival time (RMST) summarizes treatment effect in terms of a gain or loss in the event-free days. It remains uncertain whether communicating treatment benefit and harm using RMST-based summary is more effective than conventional summary based on absolute and relative risk reduction. We compared the effect of RMST-based approach and conventional approach on decisional conflict using an example of intensive versus standard blood pressure-lowering strategies.DesignOn-line survey.SettingA convenience sample of patients in the United States.ParticipantsTwo hundred adults aged 65 and older with hypertension requiring anti-hypertensive treatment (response rate 85.5%).InterventionsParticipants were randomly assigned to either RMST-based summary or conventional summary about the benefit and harm of blood pressure-lowering strategies.MeasurementsDecisional Conflict Scale (DCS), ranging from 0 (no conflict) to 100 (high conflict), and preference for intensive blood pressure-lowering strategy.ResultsParticipants assigned to RMST-based approach (n = 100) and conventional approach (n = 100) had similar age (mean [standard deviation, SD]: 72.3 [5.6] vs 72.8 [5.5] years) and proportions of female (50 [50.0%] vs 61 [61.0%]) and white race (92 [92.0%] vs 92 [92.0%]). The mean (SD) DCS score was 25.2 (15.0) for RMST-based approach and 25.6 (14.1) for conventional approach (p = 0.84). The number (%) of participants who preferred intensive strategy was 10 (10.0%) for RMST-based approach and 14 (14.0%) for conventional approach (p = 0.52). The results were consistent in subgroups defined by age, sex, education level, cardiovascular disease status, and predicted mortality risk categories.ConclusionIn a sample of relatively healthy older adults with hypertension, RMST-based approach was as effective as conventional approach on decisional conflict about choosing a blood pressure-lowering strategy. This study provides proof-of-concept evidence that RMST-based approach can be used in conjunction with absolute and relative risk reduction for communicating treatment benefit and harm in a decision aid.

Dataset Information

The Use of the Restricted Mean Survival Time as a Treatment Measure in HIV/AIDS Clinical Trial: Reanalysis of the ACTG A5257 Trial.

Background

Setting and methods

Results

Conclusions

Publications

The Use of the Restricted Mean Survival Time as a Treatment Measure in HIV/AIDS Clinical Trial: Reanalysis of the ACTG A5257 Trial.

Similar Datasets

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