ArticleThe Pediatric infectious disease journal2018
Defining Study Outcomes That Better Reflect Individual Response to Treatment.
Article in The Pediatric infectious disease journal, 2018. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Not yet cited in PubMed.
What it found
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The abstract states no effect estimate the extractor could read, or names no intervention and outcome on the map, so this paper lights no cell and moves no belief. It is still indexed, cited and linked below.
The trial behind it
Trials whose registry record cites this paper, or whose number appears in the abstract. A trial that started after this paper was published is citing it as background, not reporting it.
Neither the registry nor the abstract names a trial number. If this is a trial report, that itself is worth knowing.
Who cites it
0 citing papers in PubMed, 4 citations in OpenAlex.
No citing paper in PubMed yet.
Corrections and comments
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Authors and funding
10 authors at 5 institutions in 3 countries.
Funding
Abstract
backgroundMost clinical trials comparing treatments evaluate the separate effects on each of several efficacy and toxicity outcomes. However, population-averaged summary measures of treatment differences may not accurately reflect individual responses to treatment, and drawing conclusions about which treatment is "best" is straightforward if one treatment is superior across all outcomes, but challenging when this is not the case.
methodsWe created a study outcome based on expert opinion, which captures the risk/benefit profile of response to a treatment. Treatments were compared using this ordered outcome with standard statistical techniques. To illustrate the approach, we used as an example a study designed to evaluate initial antiretroviral therapy (ART) in human immunodeficiency virus-1-infected infants, in which results were contradictory across the study's primary and secondary efficacy and toxicity outcomes. The proposed risk/benefit outcome was evaluated retrospectively in each participant.
resultsIn the International Maternal Pediatric Adolescent AIDS Clinical Trials P1060 study, one treatment regimen (lopinavir/ritonavir-based ART) was superior to the other (nevirapine-based ART) in reducing viral load (primary outcome) but inferior for immunologic and growth outcomes (important secondary outcomes in resource-limited settings). Treatment comparisons using the risk/benefit outcome indicated that the lopinavir/ritonavir-based ART regimen had a higher proportion of participants with the best overall response to treatment. Comparisons focusing on individual-level responses for the secondary outcomes also favored lopinavir/ritonavir-based ART, results that differed from the original population-averaged analyses ones.
conclusionsDesigning studies prospectively using risk/benefit outcomes focusing on an individual's responses to treatment more closely matches the needs of clinicians making decisions about how best to treat patients in clinical settings.
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Registered trials
Read under generation 80e0d062 · epoch 390. Bibliography from PubMed, PubMed Central and OpenAlex; grants from NIH RePORTER; trial links from ClinicalTrials.gov; estimates, votes and beliefs from the OpenQuestion graph.