Article
Health Care Sciences & Services
Yeonhee Park
Summary: This paper proposes a personalized risk-based screening design using Bayesian covariate-adjusted response-adaptive randomization for improved clinical trial outcomes under personalized medicine. The design effectively controls error rates and allocates more patients to better interventions.
JOURNAL OF PERSONALIZED MEDICINE
(2022)
Article
Medicine, Research & Experimental
Jin Huang, David L. Roth
Summary: This article introduces an overall imbalance index (I) based on standardized differences in means on site characteristics. The theoretical distribution of I is derived and simulation studies show its robust properties under varying covariate circumstances. It is recommended that values of I below the 10th percentile indicate sufficient overall site balance in Covariate-constrained randomization (CCR) trials.
Article
Medicine, General & Internal
Bruno Giraudeau, Agnes Caille, Sandra M. Eldridge, Charles Weijer, Merrick Zwarenstein, Monica Taljaard
Summary: Pragmatic trials aim to generate evidence to inform decisions, but heterogeneity in patient characteristics and other factors can affect outcomes. Different sources of heterogeneity in trials should be managed, and some heterogeneity can be acceptable. Heterogeneity in centers and patients should be welcomed, control interventions should reflect usual care, and flexibility in interventions should be allowed. Objective outcomes and intention-to-treat analysis should be favored. Process analyses can provide further understanding of trial results.
Article
Pediatrics
Sunita Vohra, Salima Punja, Hsing Jou, Michael Schlegelmilch, Beverly Wilson, Maria Spavor, Paul Grundy, Andrew S. Mackie, Jennifer Conway, Dawn Hartfield
Summary: Research has shown that using a hospital-based PIM service is safe and effective for alleviating PNVA symptoms. Future studies should be conducted in other pediatric inpatient divisions and locations to determine the reproducibility of the findings.
Article
Psychology, Mathematical
John M. Ferron, Diep Nguyen, Robert F. Dedrick, Shannon M. Suldo, Elizabeth Shaunessy-Dedrick
Summary: The researchers developed an adaptive, low-assumption, and statistically valid method to test for intervention effects. By applying masked visual analysis techniques to cluster RCTs, they demonstrated the benefits of testing effects in small-scale cluster RCTs without a priori specification of a statistical model or test statistic.
BEHAVIOR RESEARCH METHODS
(2022)
Article
Mathematical & Computational Biology
Dustin J. Rabideau, Rui Wang
Summary: This article proposes a framework for calculating randomization-based p-values and CIs for marginal treatment effects in SWT, using test statistics derived from individual-level generalized linear models. It also explores how study design features impact various SWT analysis methods. Data from the XpertMTB/RIF tuberculosis trial are reanalyzed to illustrate the method and compare it to alternatives.
STATISTICS IN MEDICINE
(2021)
Article
Biology
Xin Lu, Tianle Liu, Hanzhong Liu, Peng Ding
Summary: Complete randomization may not achieve covariate balance in finite samples, while rerandomization can discard undesired treatment assignments to ensure covariate balance in realized experiments. Cluster rerandomization combines cluster-randomized experiments with rerandomization to balance covariates at individual or cluster levels, for which there is currently no existing asymptotic theory. This study provides a design-based theory for cluster rerandomization and compares two schemes that utilize prior information on covariate importance.
Article
Mathematical & Computational Biology
Patty Chondros, Obioha C. Ukoumunne, Jane M. Gunn, John B. Carlin
Summary: Simulation was used to compare the efficiency of matched-pair design, stratified design, and simple design in cluster randomized trials. Results showed that matched-pair design was generally the most efficient when the matching correlation was moderate to strong, while stratified design and simple design were more efficient for weak matching correlations.
STATISTICS IN MEDICINE
(2021)
Article
Mathematical & Computational Biology
Dustin J. Rabideau, Rui Wang
Summary: Cluster randomized trials (CRTs) randomize groups of people to different interventions, with existing methods relying on distributional assumptions or a large number of clusters. Randomization-based inference offers a distribution-free alternative that does not require a large number of clusters.
Article
Medicine, General & Internal
Lauralyn Ann McIntyre, Dean A. Fergusson, Tracy McArdle, Alison Fox-Robichaud, Shane W. English, Claudio Martin, John Marshall, Deborah J. Cook, Ian D. Graham, Steven Hawken, Colin McCartney, Kusum Menon, Raphael Saginur, Andrew Seely, Ian Stiell, Kednapa Thavorn, Charles Weijer, Akshai Iyengar, John Muscedere, Alan J. Forster, Monica Taljaard, Canadian Crit Care Trials Grp
Summary: This pilot trial aimed to evaluate the effect of normal saline and Ringer's lactate on clinically important outcomes. The study found a higher adherence rate with normal saline, but also observed more adverse events associated with it. Overall, conducting a large-scale trial in the future is feasible, but addressing logistical challenges during the planning stages will be crucial.
Review
Critical Care Medicine
David J. Cook, William B. Rutherford, Damon C. Scales, Neill K. J. Adhikari, Brian H. Cuthbertson
Summary: Cluster randomized controlled trials in critical care often involve a small and fixed number of relatively large clusters. Reporting on key methodological aspects of these trials is often inadequate.
CRITICAL CARE MEDICINE
(2021)
Editorial Material
Health Care Sciences & Services
Jose A. Sacristan, J. Andre Knottnerus
Summary: Although Evidence-based medicine (EBM) and Patient-centered medicine (PCM) are often seen as conflicting paradigms, they both aim to improve the care of individual patients. A new research method oriented towards individual patients has been proposed, incorporating patients' preferences into personalized research protocols within clinical trials.
JOURNAL OF CLINICAL EPIDEMIOLOGY
(2021)
Review
Medicine, General & Internal
Min Gao, Yangxi Huang, Qianyi Wang, Zejuan Gu, Guozhen Sun
Summary: This study aims to conduct a network meta-analysis to compare the effectiveness of different exercise training in patients with HF. The research will synthesize evidence on the impact of exercise rehabilitation on patients with HF and assess the strength of evidence using a grading system.
Article
Statistics & Probability
Anqi Zhao, Peng Ding
Summary: The split-plot design originated from agricultural science and provides a convenient way to accommodate hard-to-change factors by assigning different interventions at the whole-plot and subplot levels. Design-based inference assumes no outcome models and relies solely on the controllable randomization mechanism determined by the physical experiment.
ANNALS OF STATISTICS
(2022)
Review
Urology & Nephrology
Tuomas P. Kilpelainen, Kari A. O. Tikkinen, Gordon H. Guyatt, Robin W. M. Vernooij
Summary: In randomized controlled trials, investigators should carefully assess the credibility of subgroup effects to avoid inappropriately claiming them. Subgroup analyses should be based on criteria such as a priori hypotheses, support from prior evidence, low likelihood of chance explanations, and testing only a small number of subgroup hypotheses.
EUROPEAN UROLOGY FOCUS
(2021)
Article
Surgery
Kellie L. Mathis, Erin M. Green, Daniel J. Sargent, Conor Delaney, Clifford L. Simmang, Heidi Nelson
Article
Oncology
Harry H. Yoon, David Tougeron, Qian Shi, Steven R. Alberts, Michelle R. Mahoney, Garth D. Nelson, Suresh G. Nair, Stephen N. Thibodeau, Richard M. Goldberg, Daniel J. Sargent, Frank A. Sinicrope
CLINICAL CANCER RESEARCH
(2014)
Article
Oncology
Dan Sha, Adam M. Lee, Qian Shi, Steven R. Alberts, Daniel J. Sargent, Frank A. Sinicrope, Robert B. Diasio
CLINICAL CANCER RESEARCH
(2014)
Article
Oncology
Jocelin Huang, Suresh G. Nair, Michelle R. Mahoney, Garth D. Nelson, Anthony F. Shields, Emily Chan, Richard M. Goldberg, Sharlene Gill, Morton S. Kahlenberg, James T. Quesenberry, Stephen N. Thibodeau, Thomas C. Smyrk, Axel Grothey, Frank A. Sinicrope, Thomas A. Webb, Gist H. Farr, Barbara A. Pockaj, Jeffrey L. Berenberg, Margaret Mooney, Daniel J. Sargent, Steven R. Alberts
CLINICAL COLORECTAL CANCER
(2014)
Article
Medicine, Research & Experimental
Ming-Wen An, Sumithra J. Mandrekar, Martin J. Edelman, Daniel J. Sargent
CONTEMPORARY CLINICAL TRIALS
(2014)
Article
Oncology
Yan Liu, Saskia Litiere, Elisabeth G. E. de Vries, Daniel Sargent, Lalitha Shankar, Jan Bogaerts, Lesley Seymour
EUROPEAN JOURNAL OF CANCER
(2014)
Article
Oncology
Saskia Litiere, Elisabeth G. E. de Vries, Lesley Seymour, Dan Sargent, Lalitha Shankar, Jan Bogaerts
EUROPEAN JOURNAL OF CANCER
(2014)
Article
Pharmacology & Pharmacy
Sin-Ho Jung, Daniel J. Sargent
JOURNAL OF BIOPHARMACEUTICAL STATISTICS
(2014)
Article
Oncology
Amanda I. Phipps, Qian Shi, Polly A. Newcomb, Garth D. Nelson, Daniel J. Sargent, Steven R. Alberts, Paul J. Limburg
JOURNAL OF CLINICAL ONCOLOGY
(2013)
Article
Oncology
Sumithra J. Mandrekar, Ming-Wen An, Jeffrey Meyers, Axel Grothey, Jan Bogaerts, Daniel J. Sargent
JOURNAL OF CLINICAL ONCOLOGY
(2014)
Article
Oncology
Dan Rosmarin, Claire Palles, David Church, Enric Domingo, Angela Jones, Elaine Johnstone, Haitao Wang, Sharon Love, Patrick Julier, Claire Scudder, George Nicholson, Anna Gonzalez-Neira, Miguel Martin, Daniel Sargent, Erin Green, Howard McLeod, Ulrich M. Zanger, Matthias Schwab, Michael Braun, Matthew Seymour, Lindsay Thompson, Benjamin Lacas, Valerie Boige, Nuria Ribelles, Shoaib Afzal, Henrik Enghusen, Soren Astrup Jensen, Marie-Christine Etienne-Grimaldi, Gerard Milano, Mia Wadelius, Bengt Glimelius, Hans Garmo, Milena Gusella, Thierry Lecomte, Pierre Laurent-Puig, Eva Martinez-Balibrea, Rohini Sharma, Jesus Garcia-Foncillas, Zdenek Kleibl, Alain Morel, Jean-Pierre Pignon, Rachel Midgley, David Kerr, Ian Tomlinson
JOURNAL OF CLINICAL ONCOLOGY
(2014)
Article
Health Care Sciences & Services
Jeff A. Sloan, Daniel J. Sargent, Paul J. Novotny, Paul A. Decker, Randolph S. Marks, Heidi Nelson
JOURNAL OF PAIN AND SYMPTOM MANAGEMENT
(2014)
Article
Multidisciplinary Sciences
Hanna K. Sanoff, Lindsay A. Renfro, Pradeep Poonnen, Pratibha Ambadwar, Daniel J. Sargent, Richard M. Goldberg, Howard McLeod
Article
Mathematical & Computational Biology
A. S. C. Conlon, J. M. G. Taylor, D. J. Sargent
STATISTICS IN MEDICINE
(2014)
Article
Computer Science, Interdisciplinary Applications
L. A. Renfro, Q. Shi, Y. Xue, J. Li, H. Shang, D. J. Sargent
COMPUTATIONAL STATISTICS & DATA ANALYSIS
(2014)