GLP-1 Agonist Therapy for Advanced Heart Failure With Reduced Ejection Fraction

Margulies, Kenneth B.; Anstrom, Kevin J.; Hernandez, Adrian F.; Redfield, Margaret M.; Shah, Monica; Braunwald, Eugene; Cappola, Thomas P.

doi:10.1161/circheartfailure.114.000346

Cited by 41 publications

(22 citation statements)

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“…A clinical composite may also be considered (Massie et al, 2010), although like the Finkelstein and Schoenfeld endpoint it is too ad hoc to make a general program useful, and it is easily coded. A macro was included for the unmatched win-ratio composite (derive_WR) at the web link above; see the supplementary material to Pocock et al (2012) although note the small error in the variance equation which should sum U 2 from 1 to N. The code was validated in a number of ways including reproducing power estimates for current trials such as FIGHT which uses a global rank of three outcomes (Margulies et al, 2014) and BLAST using an average Z-score for five outcomes (Felker et al, 2015), both of which used data simulations for sample size estimation. The macros have also been used to evaluate these composites (Brown, Anstrom, Felker, & Ezekowitz, 2016).…”

Section: Resultsmentioning

confidence: 99%

“…They were used, for example, in the Functional Impact of GLP-1 for Heart Failure Treatment (FIGHT) study which compared Liraglutide and placebo groups using a global rank composite comprising mortality, hospital readmission and time-averaged proportional change in N-terminal pro-B-type natriuretic peptide (NTproBNP) level (Margulies et al, 2014). They were also used in the biased ligand of the angiotensin receptor study in acute heart failure (BLAST-AHF) study which used an average Z-score to BROWN & EZEKOWITZ 217 compare three dose groups and a placebo in acute patients with heart failure .…”

Section: Methodsmentioning

confidence: 99%

“…The global rank composite is becoming increasingly popular in phase II research; see the FIGHT study where the global rank was comprised of three outcomes (Margulies et al, 2014). Its appeal is the simplicity of construction and openness to input from researchers regarding prioritizing outcomes.…”

Section: Power Estimation For Composite Endpoints 220mentioning

confidence: 99%

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Vol. 16, No. 2 (Full Issue)

2017

J. Mod. App. Stat. Meth.

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EDITORIAL ASSISTANCEJMASM (ISSN 1538−9472, http://digitalcommons.wayne.edu/jmasm) is an independent, open access electronic journal, published biannually in May and November by JMASM Inc. (PO Box 48023, Oak Park, MI, 48237) in collaboration with the Wayne State University Library System. JMASM seeks to publish (1) new statistical tests or procedures, or the comparison of existing statistical tests or procedures, using computer-intensive Monte Carlo, bootstrap, jackknife, or resampling methods, (2) the study of nonparametric, robust, permutation, exact, and approximate randomization methods, and (3) applications of computer programming, preferably in Fortran (all other programming environments are welcome), related to statistical algorithms, pseudo-random number generators, simulation techniques, and self-contained executable code to carry out new or interesting statistical methods.Journal correspondence (other than manuscript submissions) and requests for advertising may be forwarded to ea@jmasm.com. See back matter for instructions for authors. IntroductionComputer simulation studies represent an important tool for investigating statistical procedures difficult or impossible to study using mathematical theory or real data. Descriptors of these studies vary (e.g., statistical experiment, Monte Carlo simulation, computer experiment), but the examples of Hoaglin and Andrews (1975) and Hauck and Anderson (1984) are followed here with use of the term simulation studies. Extensive descriptions of simulation studies can be found in Lewis and Orav (1989) and Santner, Williams, and Notz (2003). In the behavioral sciences simulation studies have been used to study a wide array of statistical methods (e.g., Cribbie, Fiksenbaum, & Wilcox, 2012; Depaoli, 2012; Enders, Baraldi, & Cham, 2014; Hu & Bentler, 1999; Tomarken & Serlin, 1986). The general goal of these studies is to provide evidence of the behavior of statistical methods under a variety of data conditions that improves statistical practice and informs future statistical research. The goal here is to encourage methodological researchers to treat these studies as statistical sampling EXPERIMENTAL DESIGN AND DATA ANALYSIS IN SIMULATION 4 experiments subject to established principles of experimental design and data analysis.An underappreciated facet of simulation studies in statistics is their role in enhancing the reproducibility of scientific findings. The importance of reproducibility has gained momentum in numerous scientific arenas because of growing evidence that many findings cannot be replicated (Stodden, 2015). Concerns over reproducibility and the role of statistics were captured in Statistics and science: A report of the London workshop on the future of the statistical sciences (2014) which noted: "The reproducibility problem goes far beyond statistics, of course, because it involves the entire reward structure of the scientific enterprise. Nevertheless, statistics is a very important ingredient in both the problem and the remedy." (p. 27) Simulati...

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Section: Resultsmentioning

confidence: 99%

Section: Methodsmentioning

confidence: 99%

See 1 more Smart Citation

Vol. 16, No. 2 (Full Issue)

2017

J. Mod. App. Stat. Meth.

View full text Add to dashboard Cite

show abstract

“…They were used, for example, in the Functional Impact of GLP-1 for Heart Failure Treatment (FIGHT) study which compared Liraglutide and placebo groups using a global rank composite comprising mortality, hospital readmission and time-averaged proportional change in N-terminal pro-B-type natriuretic peptide (NTproBNP) level (Margulies et al, 2014). They were also used in the biased ligand of the angiotensin receptor study in acute heart failure (BLAST-AHF) study which used an average Z-score to compare three dose groups and a placebo in acute patients with heart failure .…”

Section: Methodsmentioning

confidence: 99%

“…Patients proceed down the hierarchy until they fail on an outcome according to some criterion. A decision rule employing criteria for failure is not necessary for a global rank composite, but we follow the approach of Felker and Maisel (2010) here; 'global rank' is a generic term and various specifications could fall under this label (Califf, Harrelson-Woodlief, & Topol, 1990;Finkelstein & Schoenfeld, 1999;Lachin, 1999;Margulies et al, 2014;Pocock, Ariti, Collier, & Wang, 2012;Temkin et al, 2007). The intention is to assign every patient a rank which reflects the severity of response.…”

Section: Global Rank (%Derive_gr)mentioning

confidence: 99%