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Updated: April 4th, 2024 22:22

2022 article

Group sequential methods for interim monitoring of randomized clinical trials with time-lagged outcome

Tsiatis, A. A., & Davidian, M. (2022, September 18). *STATISTICS IN MEDICINE*, Vol. 9.

author keywords: augmented inverse probability weighting; early stopping; influence function; proportion of information

MeSH headings : Humans; Randomized Controlled Trials as Topic; Odds Ratio; Research Design

TL;DR:
Treatment effect estimators that take account of censoring and incorporate covariate information at an interim analysis are derived using semiparametric theory and are demonstrated to lead to stronger evidence for early stopping than standard approaches.
(via Semantic Scholar)

UN Sustainable Development Goal Categories

3. Good Health and Well-being
(Web of Science)

Sources: Web Of Science, NC State University Libraries

Added: October 3, 2022

2021 review

Data and Safety Monitoring of COVID-19 Vaccine Clinical Trials

[Review of ]. *JOURNAL OF INFECTIOUS DISEASES*, *224*(12), 1995–2000.

author keywords: SARS-CoV-2; COVID19; clinical trials; vaccines; data and safety monitoring

MeSH headings : Aged; COVID-19 / prevention & control; COVID-19 Vaccines / administration & dosage; COVID-19 Vaccines / adverse effects; COVID-19 Vaccines / immunology; Humans; SARS-CoV-2; United States; Vaccines

TL;DR:
A single 11-member data and safety monitoring board monitors all government-funded trials to ensure coordinated oversight, promote harmonized designs, and allow shared insights related to safety across trials.
(via Semantic Scholar)

UN Sustainable Development Goal Categories

3. Good Health and Well-being
(Web of Science; OpenAlex)

Source: Web Of Science

Added: February 28, 2022

2021 article

Estimating vaccine efficacy over time after a randomized study is unblinded

Tsiatis, A. A., & Davidian, M. (2021, August 13). *BIOMETRICS*, Vol. 8.

author keywords: crossover; inverse probability weighting; potential outcomes; randomized phase 3 vaccine trial; waning vaccine efficacy

MeSH headings : COVID-19 / prevention & control; COVID-19 Vaccines; Humans; Pandemics / prevention & control; Randomized Controlled Trials as Topic; Research Design; SARS-CoV-2; Vaccine Efficacy

TL;DR:
This work focuses on the particular features of the Moderna trial and proposes a statistical framework based on a potential outcomes formulation within which methods for inference on potential waning of VE over time and estimation ofVE at any postvaccination time are developed.
(via Semantic Scholar)

UN Sustainable Development Goal Categories

3. Good Health and Well-being
(Web of Science; OpenAlex)

Sources: Web Of Science, NC State University Libraries

Added: August 23, 2021

2021 article

Estimation of the odds ratio in a proportional odds model with censored time-lagged outcome in a randomized clinical trial

Tsiatis, A. A., Davidian, M., & Holloway, S. T. (2021, December 17). *BIOMETRICS*, Vol. 12.

author keywords: augmented inverse probability weighting; censored ordinal categorical outcome; covariate adjustment; COVID-19 treatment; estimating function; marked point process

TL;DR:
An estimator for the OR in a proportional odds model with censored, time‐lagged categorical outcome that incorporates additional baseline and time‐dependent covariate information and demonstrates that it can result in considerable gains in efficiency relative to simpler approaches is proposed.
(via Semantic Scholar)

UN Sustainable Development Goal Categories

3. Good Health and Well-being
(Web of Science)

Sources: Web Of Science, ORCID, NC State University Libraries

Added: December 17, 2021

2021 article

Rejoinder: Estimating vaccine efficacy over time after a randomized study is unblinded

Tsiatis, A. A., & Davidian, M. (2021, August 22). *BIOMETRICS*, Vol. 8.

MeSH headings : Randomized Controlled Trials as Topic; Vaccine Efficacy

TL;DR:
Responding to the points they raise has enhanced the understanding of the area and the role of the work within it, and all note challenges posed by viral variants.
(via Semantic Scholar)

UN Sustainable Development Goal Categories

3. Good Health and Well-being
(OpenAlex)

Sources: Web Of Science, NC State University Libraries

Added: August 30, 2021

2021 article

Risks associated with discontinuation of oral anticoagulation in newly diagnosed patients with atrial fibrillation: Results from the GARFIELD-AF Registry

Cools, F., Johnson, D., Camm, A. J., Bassand, J.-P., Verheugt, F. W. A., Yang, S., … Kakkar, A. K. (2021, July 23). *JOURNAL OF THROMBOSIS AND HAEMOSTASIS*, Vol. 7.

author keywords: anticoagulation; antiplatelet; atrial fibrillation; discontinuation; marginal structure models; outcomes

MeSH headings : Administration, Oral; Anticoagulants / adverse effects; Atrial Fibrillation / complications; Atrial Fibrillation / diagnosis; Atrial Fibrillation / drug therapy; Humans; Registries; Risk Factors; Stroke / diagnosis; Stroke / etiology; Stroke / prevention & control

TL;DR:
Discontinuation for ≥7 consecutive days was associated with significantly higher all-cause mortality, stroke/SE and MI risk, and caution should be exerted when considering any OAC discontinuation beyond 7 days.
(via Semantic Scholar)

UN Sustainable Development Goal Categories

3. Good Health and Well-being
(Web of Science; OpenAlex)

Sources: Web Of Science, NC State University Libraries

Added: August 2, 2021

2020 review

Independent increments in group sequential tests: a review

[Review of ]. *SORT-STATISTICS AND OPERATIONS RESEARCH TRANSACTIONS*, *44*(2), 223–264.

UN Sustainable Development Goal Categories

3. Good Health and Well-being
(Web of Science)

Source: Web Of Science

Added: January 25, 2021

2019 review

Application of a sequential multiple assignment randomized trial (SMART) design in older patients with chronic lymphocytic leukemia

[Review of ]. *ANNALS OF ONCOLOGY*, *30*(4), 542–550.

author keywords: SMART; CLL; design strategies; adaptive interventions; randomized clinical trials

MeSH headings : Age Factors; Aged; Antineoplastic Combined Chemotherapy Protocols / therapeutic use; Data Analysis; Disease Progression; Feasibility Studies; Humans; Leukemia, Lymphocytic, Chronic, B-Cell / drug therapy; Leukemia, Lymphocytic, Chronic, B-Cell / mortality; Progression-Free Survival; Randomized Controlled Trials as Topic; Research Design; Sample Size

TL;DR:
SMART design strategies can be used in cancer clinical trials with adaptive interventions to identify optimal treatment strategies and are appropriate when comparing adaptive interventions, which are defined by an individual's sequence of treatment decisions and guided by intermediate outcomes.
(via Semantic Scholar)

UN Sustainable Development Goal Categories

3. Good Health and Well-being
(Web of Science)

Sources: Web Of Science, NC State University Libraries

Added: June 17, 2019

2019 book

Dynamic Treatment Regimes

Source: Crossref

Added: January 22, 2022

2019 journal article

Identical and Nonidentical Twins: Risk and Factors Involved in Development of Islet Autoimmunity and Type 1 Diabetes

*DIABETES CARE*, *42*(2), 192–199.

MeSH headings : Adolescent; Adult; Autoantibodies / analysis; Autoantibodies / blood; Autoimmunity / genetics; Autoimmunity / physiology; Child; Child, Preschool; Diabetes Mellitus, Type 1 / diagnosis; Diabetes Mellitus, Type 1 / epidemiology; Diabetes Mellitus, Type 1 / genetics; Diabetes Mellitus, Type 1 / prevention & control; Disease Progression; Diseases in Twins / diagnosis; Diseases in Twins / epidemiology; Diseases in Twins / genetics; Diseases in Twins / immunology; Environment; Female; Genetic Predisposition to Disease; Glutamate Decarboxylase / immunology; Humans; Insulin / metabolism; Islets of Langerhans / immunology; Male; Mass Screening / methods; Risk Factors; Seroepidemiologic Studies; Siblings; Twins / genetics; Twins, Dizygotic / genetics; Twins, Dizygotic / statistics & numerical data; Twins, Monozygotic / genetics; Twins, Monozygotic / statistics & numerical data; Young Adult

TL;DR:
Risk of type 1 diabetes at 3 years is high for initially multiple and single autoantibody–positive identical twins and multiple autoantibia–positive nonidentical twins.
(via Semantic Scholar)

UN Sustainable Development Goal Categories

3. Good Health and Well-being
(Web of Science; OpenAlex)

Source: Web Of Science

Added: April 20, 2020

2018 journal article

Interpretable Dynamic Treatment Regimes

*JOURNAL OF THE AMERICAN STATISTICAL ASSOCIATION*, *113*(524), 1541–1549.

author keywords: Decision lists; Interpretability; Precision medicine; Research-practice gap; Treatment regimes; Tree-based methods

TL;DR:
An estimator of an optimal treatment regime composed of a sequence of decision rules, each expressible as a list of “if-then” statements that can be presented as either a paragraph or as a simple flowchart that is immediately interpretable to domain experts is proposed.
(via Semantic Scholar)

UN Sustainable Development Goal Categories

3. Good Health and Well-being
(Web of Science)

Sources: Web Of Science, NC State University Libraries

Added: February 18, 2019

2018 journal article

Modeling survival distribution as a function of time to treatment discontinuation: A dynamic treatment regime approach

*BIOMETRICS*, *74*(3), 900–909.

author keywords: Dynamic treatment; Inverse weighting; Observational study; Survival distribution; Time-varying confounding; Treatment discontinuation

MeSH headings : Cardiovascular Diseases / therapy; Computer Simulation; Humans; Kaplan-Meier Estimate; Models, Statistical; Survival Analysis; Time-to-Treatment; Withholding Treatment / statistics & numerical data

TL;DR:
The intervention is cast in the form of a dynamic regime “terminate treatment optionally at time v unless a mandatory treatment‐terminating event occurs prior to v” and the distribution of time to event as a function of treatment regime v is considered.
(via Semantic Scholar)

UN Sustainable Development Goal Categories

3. Good Health and Well-being
(Web of Science)

Sources: Web Of Science, NC State University Libraries

Added: October 16, 2018

2018 journal article

Optimal two-stage dynamic treatment regimes from a classification perspective with censored survival data

*BIOMETRICS*, *74*(4), 1180–1192.

author keywords: Doubly robust; Inverse probability weighting; Optimal treatment regime; Sequential multiple assignment randomized trial; Support vector machine; Value search

MeSH headings : Acute Disease; Algorithms; Biometry / methods; Computer Simulation; Decision Support Techniques; Humans; Leukemia; Outcome Assessment, Health Care / methods; Outcome Assessment, Health Care / standards; Randomized Controlled Trials as Topic; Support Vector Machine; Survival Analysis

TL;DR:
A method for estimation of an optimal regime involving two decision points when the outcome of interest is a censored survival time is proposed, based on maximizing a locally efficient, doubly robust, augmented inverse probability weighted estimator for average outcome over a class of regimes.
(via Semantic Scholar)

UN Sustainable Development Goal Categories

3. Good Health and Well-being
(Web of Science)

16. Peace, Justice and Strong Institutions
(OpenAlex)

Sources: Web Of Science, NC State University Libraries

Added: February 25, 2019

2017 journal article

Effect of oral insulin on prevention of diabetes in relatives of patients with type 1 diabetes a randomized clinical trial

*Journal of the American Medical Association*, *318*(19), 1891–1902.

Source: NC State University Libraries

Added: August 6, 2018

2017 journal article

Optimal treatment regimes for survival endpoints using locally-efficient doubly-robust estimator from a classification perspective

*Lifetime Data Analysis*, *23*(4), 585–604.

author keywords: Classification; Doubly-robust; Observational survival study; Optimal treatment regime; Value search

MeSH headings : Computer Simulation; Coronary Artery Bypass; Coronary Artery Disease / mortality; Coronary Artery Disease / therapy; Decision Making; Humans; Life Tables; Models, Statistical; Monte Carlo Method; Percutaneous Coronary Intervention; Survival Analysis; Treatment Outcome

TL;DR:
This work derives a locally efficient, doubly robust, augmented inverse probability weighted complete case estimator for the value function with censored survival data and study the large sample properties of this estimator.
(via Semantic Scholar)

UN Sustainable Development Goal Categories

3. Good Health and Well-being
(Web of Science)

Sources: Web Of Science, NC State University Libraries

Added: August 6, 2018

2017 journal article

Survival benefit of lung transplantation in the modern era of lung allocation

*Annals of the American Thoracic Society*, *14*(2), 172–181.

Source: NC State University Libraries

Added: August 6, 2018

2016 journal article

A log rank type test in observational survival studies with stratified sampling

*LIFETIME DATA ANALYSIS*, *22*(2), 280–298.

author keywords: Cox proportional hazards model; Log rank test; Observational study; Stratified sampling; Survival analysis

MeSH headings : Computer Simulation; Coronary Artery Disease / mortality; Coronary Artery Disease / therapy; Humans; Models, Statistical; Observational Studies as Topic / statistics & numerical data; Probability; Proportional Hazards Models; Sampling Studies; Survival Analysis

TL;DR:
This paper generalizes the augmented inverse probability weighted complete case estimators for treatment-specific survival distribution proposed in Bai et al. (Biometrics 69:830–839, 2013) and develops the log rank type test in both cases.
(via Semantic Scholar)

UN Sustainable Development Goal Categories

3. Good Health and Well-being
(Web of Science)

Source: Web Of Science

Added: August 6, 2018

2016 article

Estimating optimal treatment regimes from a classification perspective (vol 1, pg 103, 2012)

*STAT*, Vol. 5, pp. 278–278.

UN Sustainable Development Goal Categories

3. Good Health and Well-being
(Web of Science)

Sources: Web Of Science, NC State University Libraries

Added: August 6, 2018

2015 journal article

Estimation After a Group Sequential Trial

*STATISTICS IN BIOSCIENCES*, *7*(2), 187–205.

author keywords: Exponential family; Frequentist inference; Generalized sample average; Joint modeling; Likelihood inference; Missing at random; Sample average

TL;DR:
It is shown that the ordinary sample average is a viable option for estimation following a group sequential trial, for a wide class of stopping rules and for random outcomes with a distribution in the exponential family, and it is consistent and asymptotically unbiased.
(via Semantic Scholar)

UN Sustainable Development Goal Categories

3. Good Health and Well-being
(Web of Science)

10. Reduced Inequalities
(OpenAlex)

Sources: Web Of Science, NC State University Libraries

Added: August 6, 2018

2015 journal article

Using Decision Lists to Construct Interpretable and Parsimonious Treatment Regimes

*BIOMETRICS*, *71*(4), 895–904.

author keywords: Decision lists; Exploratory analyses; Interpretability; Personalized medicine; Treatment regimes

MeSH headings : Biometry / methods; Breast Neoplasms / drug therapy; Clinical Protocols; Clinical Trials as Topic / statistics & numerical data; Computer Simulation; Decision Trees; Depression / therapy; Evidence-Based Medicine / statistics & numerical data; Female; Humans; Models, Statistical; Precision Medicine / statistics & numerical data

TL;DR:
A simple, yet flexible class of treatment regimes whose members are representable as a short list of if–then statements are proposed, which are immediately interpretable and are therefore an appealing choice for broad application in practice.
(via Semantic Scholar)

UN Sustainable Development Goal Categories

3. Good Health and Well-being
(Web of Science)

Sources: Web Of Science, NC State University Libraries

Added: August 6, 2018

2015 journal article

Using pilot data to size a two-arm randomized trial to find a nearly optimal personalized treatment strategy

*Statistics in Medicine*, *35*(8), 1245–1256.

author keywords: personalized medicine; sample size calculation; treatment regimes; nonregular asymptotics; projection confidence region

MeSH headings : Biostatistics; Computer Simulation; Confidence Intervals; Data Interpretation, Statistical; Evidence-Based Practice / statistics & numerical data; Female; Fertility; Humans; Male; Models, Statistical; Pilot Projects; Precision Medicine / statistics & numerical data; Pregnancy; Randomized Controlled Trials as Topic / statistics & numerical data; Regression Analysis; Sample Size

TL;DR:
This work offers a simple and robust method for powering a single stage, two‐armed randomized clinical trial when the primary aim is estimating the optimal single stage personalized treatment strategy.
(via Semantic Scholar)

UN Sustainable Development Goal Categories

3. Good Health and Well-being
(Web of Science; OpenAlex)

Sources: Crossref, Web Of Science, NC State University Libraries

Added: August 6, 2018

2014 article

Combining Biomarkers to Optimize Patient Treatment Recommendations Discussions

Laber, E. B., Tsiatis, A. A., Davidian, M., & Holloway, S. T. (2014, September). *BIOMETRICS*, Vol. 70, pp. 707–710.

MeSH headings : Biomarkers, Tumor / blood; Biometry / methods; Breast Neoplasms / blood; Breast Neoplasms / therapy; Data Interpretation, Statistical; Female; Humans; Male; Outcome Assessment, Health Care / methods

TL;DR:
It is shown how the method of KJH can be used with existing policy-search methods to construct a treatment rule that is interpretable, logistically feasible, parsimonious, or otherwise appealing.
(via Semantic Scholar)

UN Sustainable Development Goal Categories

3. Good Health and Well-being
(Web of Science)

4. Quality Education
(OpenAlex)

Sources: Web Of Science, NC State University Libraries

Added: August 6, 2018

2014 journal article

On random sample size, ignorability, ancillarity, completeness, separability, and degeneracy: Sequential trials, random sample sizes, and missing data

*STATISTICAL METHODS IN MEDICAL RESEARCH*, *23*(1), 11–41.

author keywords: Frequentist inference; generalized sample average; informative cluster size; joint modeling; likelihood inference; missing at random; random cluster size

MeSH headings : Likelihood Functions; Models, Statistical; Probability; Sample Size

TL;DR:
The aim is to place various related examples into a single framework derived from the joint modeling of the outcomes and sampling process and so derive generic results that in turn provide insight, and in some cases practical consequences, for different settings.
(via Semantic Scholar)

UN Sustainable Development Goal Categories

3. Good Health and Well-being
(Web of Science)

Sources: Web Of Science, NC State University Libraries

Added: August 6, 2018

2014 journal article

Q- and A-Learning Methods for Estimating Optimal Dynamic Treatment Regimes

*STATISTICAL SCIENCE*, *29*(4), 640–661.

author keywords: Advantage learning; bias-variance trade-off; model misspecification; personalized medicine; potential outcomes; sequential decision-making

TL;DR:
This work provides a detailed account of Q- and A-learning methods, study their performance, and illustrate them using data from a depression study.
(via Semantic Scholar)

UN Sustainable Development Goal Categories

3. Good Health and Well-being
(Web of Science)

Sources: Web Of Science, NC State University Libraries

Added: August 6, 2018

2013 journal article

Assessing the Causal Effect of Organ Transplantation on the Distribution of Residual Lifetime

*BIOMETRICS*, *69*(4), 820–829.

author keywords: Causal inference; G-estimation; Lung transplantation; Martingale theory; Structural nested failure time models

MeSH headings : Adolescent; Adult; Age Distribution; Aged; Aged, 80 and over; Causality; Data Interpretation, Statistical; Humans; Internationality; Life Expectancy; Lung Diseases / mortality; Lung Diseases / surgery; Lung Transplantation / mortality; Middle Aged; Outcome Assessment, Health Care / methods; Survival Rate; Young Adult

TL;DR:
A version of G‐estimation is developed that accounts for the fact that treatment assignment is not independent across individuals to estimate the parameters of a structural nested failure time model and leads to valid inference of the effect of transplantation on the distribution of residual lifetime.
(via Semantic Scholar)

UN Sustainable Development Goal Categories

3. Good Health and Well-being
(Web of Science)

Sources: Web Of Science, NC State University Libraries

Added: August 6, 2018

2013 journal article

Doubly-Robust Estimators of Treatment-Specific Survival Distributions in Observational Studies with Stratified Sampling

*BIOMETRICS*, *69*(4), 830–839.

author keywords: Cox proportional hazard model; Double robustness; Observational study; Stratified sampling; Survival analysis

MeSH headings : Coronary Artery Disease / mortality; Coronary Artery Disease / surgery; Data Interpretation, Statistical; Humans; Observational Studies as Topic / methods; Outcome Assessment, Health Care / methods; Prevalence; Reproducibility of Results; Sample Size; Sensitivity and Specificity; Statistical Distributions; Survival Analysis; Treatment Outcome; United States / epidemiology

TL;DR:
Semiparametric theory is used to derive a doubly robust estimator of the treatment-specific survival distribution in cases where it is believed that all the potential confounders are captured.
(via Semantic Scholar)

UN Sustainable Development Goal Categories

3. Good Health and Well-being
(Web of Science)

Source: Web Of Science

Added: August 6, 2018

2013 journal article

Efficient estimation of the distribution of time to composite endpoint when some endpoints are only partially observed

*LIFETIME DATA ANALYSIS*, *19*(4), 513–546.

author keywords: Augmented inverse probability weighted estimator; Composite endpoint; Missing data; Nelson-Aalen estimator; Semi-parametric efficiency; Withdrawal

MeSH headings : Biostatistics; Clinical Trials as Topic / statistics & numerical data; Computer Simulation; Endpoint Determination / statistics & numerical data; Humans; Linear Models; Longitudinal Studies; Models, Statistical; Proportional Hazards Models; Registries / statistics & numerical data; Statistics, Nonparametric; Time Factors

TL;DR:
The theory of augmented inverse probability weighted estimating equations is used to show how partial information on the composite endpoint for subjects who withdraw from the study can be incorporated in a principled way into the estimation of the distribution of time to composite endpoint, typically leading to increased efficiency without relying on additional assumptions above those that would be made by standard approaches.
(via Semantic Scholar)

UN Sustainable Development Goal Categories

3. Good Health and Well-being
(Web of Science)

Source: Web Of Science

Added: August 6, 2018

2013 journal article

Robust estimation of optimal dynamic treatment regimes for sequential treatment decisions

*BIOMETRIKA*, *100*(3), 681–694.

author keywords: A-learning; Double robustness; Outcome regression; Propensity score; Q-learning

TL;DR:
This work proposes an alternative to Q- and A-learning that maximizes a doubly robust augmented inverse probability weighted estimator for population mean outcome over a restricted class of regimes.
(via Semantic Scholar)

UN Sustainable Development Goal Categories

3. Good Health and Well-being
(Web of Science)

Sources: Web Of Science, NC State University Libraries, ORCID

Added: August 6, 2018

2012 journal article

A Robust Method for Estimating Optimal Treatment Regimes

*BIOMETRICS*, *68*(4), 1010–1018.

author keywords: Doubly robust estimator; Inverse probability weighting; Outcome regression; Personalized medicine; Potential outcomes; Propensity score

MeSH headings : Breast Neoplasms / epidemiology; Breast Neoplasms / therapy; Clinical Trials as Topic / methods; Computer Simulation; Decision Support Systems, Clinical; Female; Humans; Models, Statistical; Outcome Assessment, Health Care / methods; Prevalence; Regression Analysis; Treatment Outcome

TL;DR:
A doubly robust augmented inverse probability weighted estimator is used for finding the optimal regime within a class of regimes by finding the regime that optimizes an estimator of overall population mean outcome.
(via Semantic Scholar)

UN Sustainable Development Goal Categories

3. Good Health and Well-being
(Web of Science; OpenAlex)

Sources: Web Of Science, NC State University Libraries

Added: August 6, 2018

2012 journal article

Estimating optimal treatment regimes from a classification perspective

*Stat*, *1*(1), 103–114.

author keywords: classification; doubly robust estimator; inverse probability weighting; personalized medicine; potential outcomes; propensity score

TL;DR:
This work proposes a novel and general framework that transforms the problem of estimating an optimal treatment regime into a classification problem wherein the optimal classifier corresponds to the optimalreatment regime.
(via Semantic Scholar)

UN Sustainable Development Goal Categories

3. Good Health and Well-being
(Web of Science; OpenAlex)

Sources: Crossref, ORCID, NC State University Libraries

Added: January 23, 2020

2012 journal article

Mixed model analysis of censored longitudinal data with flexible random-effects density

*BIOSTATISTICS*, *13*(1), 61–73.

author keywords: Censoring; HCV; HIV; Limit of quantification; Longitudinal data; Random effects

MeSH headings : Antiviral Agents / therapeutic use; Bias; Biostatistics; Data Interpretation, Statistical; Hepatitis C / drug therapy; Hepatitis C / virology; Humans; Interferon-alpha / therapeutic use; Linear Models; Longitudinal Studies; Models, Statistical; Statistics, Nonparametric; Viral Load / drug effects

TL;DR:
A mixed model framework for censored longitudinal data in which the random effects are represented by the flexible seminonparametric density is developed andSimulations show that this approach can lead to reduction in bias and increase in efficiency relative to assuming Gaussian random effects.
(via Semantic Scholar)

UN Sustainable Development Goal Categories

3. Good Health and Well-being
(Web of Science)

Sources: Web Of Science, NC State University Libraries

Added: August 6, 2018

2011 journal article

Connections between survey calibration estimators and semiparametric models for incomplete data discussion

*International Statistical Review*, *79*(2), 221–223.

Source: NC State University Libraries

Added: August 6, 2018

2011 journal article

Improved Doubly Robust Estimation When Data Are Monotonely Coarsened, with Application to Longitudinal Studies with Dropout

*BIOMETRICS*, *67*(2), 536–545.

author keywords: Coarsening at random; Discrete hazard; Dropout; Longitudinal data; Missing at random

MeSH headings : Acquired Immunodeficiency Syndrome; Biometry / methods; Computer Simulation; Humans; Longitudinal Studies; Models, Statistical; Statistical Distributions

TL;DR:
This work proposes a DR estimator applicable in general monotone coarsening problems that achieves comparable or improved performance relative to existing DR methods, which is demonstrated via simulation studies and by application to data from an AIDS clinical trial.
(via Semantic Scholar)

UN Sustainable Development Goal Categories

3. Good Health and Well-being
(Web of Science)

Sources: Web Of Science, NC State University Libraries

Added: August 6, 2018

2011 journal article

Inference on treatment effects from a randomized clinical trial in the presence of premature treatment discontinuation: the SYNERGY trial

*BIOSTATISTICS*, *12*(2), 258–269.

author keywords: Dynamic treatment regime; Inverse probability weighting; Potential outcomes; Proportional hazards model

MeSH headings : Acute Coronary Syndrome / drug therapy; Algorithms; Computer Simulation; Endpoint Determination; Enoxaparin / administration & dosage; Enoxaparin / adverse effects; Enoxaparin / therapeutic use; Hemorrhage / etiology; Heparin / administration & dosage; Heparin / adverse effects; Heparin / therapeutic use; Humans; Intention to Treat Analysis; Models, Statistical; Monte Carlo Method; Mortality; Multicenter Studies as Topic; Probability; Proportional Hazards Models; Randomized Controlled Trials as Topic / methods; Survival Analysis; Treatment Outcome; Withholding Treatment / statistics & numerical data

TL;DR:
The Superior Yield of the New Strategy of Enoxaparin, Revascularization, and GlYcoprotein IIb/IIIa inhibitors (SYNERGY) was a randomized, open-label, multicenter clinical trial comparing 2 anticoagulant drugs on the basis of time-to-event endpoints.
(via Semantic Scholar)

UN Sustainable Development Goal Categories

3. Good Health and Well-being
(Web of Science; OpenAlex)

Sources: Web Of Science, NC State University Libraries

Added: August 6, 2018

2011 journal article

Semiparametric estimation of treatment effect with time-lagged response in the presence of informative censoring

*LIFETIME DATA ANALYSIS*, *17*(4), 566–593.

author keywords: Informative censoring; Influence function; Logrank test; Nuisance tangent space; Proportional hazards model; Regular and asymptotically linear estimators

MeSH headings : Anti-HIV Agents / administration & dosage; Anti-HIV Agents / therapeutic use; CD4 Lymphocyte Count; Data Interpretation, Statistical; HIV / growth & development; HIV Infections / drug therapy; Humans; Models, Statistical; Randomized Controlled Trials as Topic / methods; Treatment Outcome

TL;DR:
Semiparametric theory is used to derive a class of consistent and asymptotically normal estimators for the treatment effect parameter which are applicable when the response variable is right censored and are more efficient then the estimators which do not consider the auxiliary covariates.
(via Semantic Scholar)

UN Sustainable Development Goal Categories

3. Good Health and Well-being
(Web of Science)

Source: Web Of Science

Added: August 6, 2018

2010 journal article

A Generalized Estimator of the Attributable Benefit of an Optimal Treatment Regime

*BIOMETRICS*, *66*(2), 512–522.

author keywords: Attributable risk (AR); Causal inference; Influence function; Optimal treatment regime

MeSH headings : Algorithms; Cardiac Catheterization; Combined Modality Therapy; Coronary Artery Bypass; Humans; Methods; Observation; Precision Medicine / methods; Regression Analysis; Risk Assessment

TL;DR:
Using a measure that is a generalization of attributable risk (AR) and notions of potential outcomes, an estimator is derived for the proportion of events that could have been prevented had the optimal treatment regime been implemented.
(via Semantic Scholar)

UN Sustainable Development Goal Categories

3. Good Health and Well-being
(Web of Science; OpenAlex)

Source: Web Of Science

Added: August 6, 2018

2010 journal article

Multiple Imputation Approaches for the Analysis of Dichotomized Responses in Longitudinal Studies with Missing Data

*BIOMETRICS*, *66*(4), 1202–1208.

author keywords: Logistic regression; Missing data; Multiple imputation; Repeated measures

MeSH headings : Bayes Theorem; Biometry / methods; Humans; Likelihood Functions; Logistic Models; Longitudinal Studies / statistics & numerical data; Time Factors

TL;DR:
It is proposed to apply degrees of freedom to account for the uncertainty associated with variance-covariance parameter estimates for the repeated measures model to be more efficient.
(via Semantic Scholar)

UN Sustainable Development Goal Categories

3. Good Health and Well-being
(Web of Science)

16. Peace, Justice and Strong Institutions
(OpenAlex)

Source: Web Of Science

Added: August 6, 2018

2009 journal article

Improving efficiency and robustness of the doubly robust estimator for a population mean with incomplete data

*BIOMETRIKA*, *96*(3), 723–734.

author keywords: Causal inference; Enhanced propensity score model; Missing at random; No unmeasured confounders; Outcome regression

TL;DR:
This work proposes alternative doubly robust estimators that achieve comparable or improved performance relative to existing methods, even with some estimated propensity scores close to zero.
(via Semantic Scholar)

UN Sustainable Development Goal Categories

3. Good Health and Well-being
(Web of Science)

Sources: Web Of Science, NC State University Libraries

Added: August 6, 2018

2008 article

Covariate adjustment for two-sample treatment comparisons in randomized clinical trials: A principled yet flexible approach

Tsiatis, A. A., Davidian, M., Zhang, M., & Lu, X. (2008, October 15). *STATISTICS IN MEDICINE*, Vol. 27, pp. 4658–4677.

author keywords: baseline variables; clinical trials; covariate adjustment; efficiency; semiparametric theory; variable selection

MeSH headings : Algorithms; Data Interpretation, Statistical; Humans; Randomized Controlled Trials as Topic / statistics & numerical data; Sampling Studies; Statistics, Nonparametric; Treatment Outcome

TL;DR:
App appealing to the theory of semiparametrics is led naturally to a characterization of all treatment effect estimators and to principled, practically feasible methods for covariate adjustment that yield the desired gains in efficiency and that allow covariate relationships to be identified and exploited while circumventing the usual concerns.
(via Semantic Scholar)

UN Sustainable Development Goal Categories

3. Good Health and Well-being
(Web of Science)

Sources: Web Of Science, NC State University Libraries

Added: August 6, 2018

2008 journal article

Empirical estimation of life expectancy from large clinical trials: Use of left-truncated, right-censored survival analysis methodology

*STATISTICS IN MEDICINE*, *27*(26), 5525–5555.

author keywords: life expectancy; cost-effectiveness; methods; clinical trials; survival

MeSH headings : Adult; Aged; Aged, 80 and over; Cardiovascular Diseases / economics; Cardiovascular Diseases / epidemiology; Cerebrovascular Disorders / economics; Cerebrovascular Disorders / epidemiology; Cohort Studies; Cost-Benefit Analysis; Female; Health Care Costs; Humans; Life Expectancy; Male; Markov Chains; Middle Aged; Models, Statistical; Observation; Proportional Hazards Models; Randomized Controlled Trials as Topic / methods; Survival Analysis

TL;DR:
Methods for using empirical patient‐level data to extrapolate survival in large clinical trials and cohorts beyond a limited follow‐up period in which most patients remain alive are described and a novel combination of models that estimate the hazard rate not only as a function of time but also as afunction of patient age are accomplished.
(via Semantic Scholar)

UN Sustainable Development Goal Categories

3. Good Health and Well-being
(Web of Science)

Source: Web Of Science

Added: August 6, 2018

2008 journal article

Improving efficiency of inferences in randomized clinical trials using auxiliary covariates

*BIOMETRICS*, *64*(3), 707–715.

author keywords: covariate adjustment; hypothesis test; k-arm trial; Kruskal-Wallis test; log odds ratio; longitudinal data; semiparametric theory

MeSH headings : Acquired Immunodeficiency Syndrome / drug therapy; Analysis of Variance; Angina, Unstable / blood; Angina, Unstable / drug therapy; Anti-HIV Agents / therapeutic use; Anticoagulants / therapeutic use; Biometry / methods; Humans; Linear Models; Logistic Models; Models, Statistical; Monte Carlo Method; Odds Ratio; Platelet Glycoprotein GPIIb-IIIa Complex / metabolism; Randomized Controlled Trials as Topic / statistics & numerical data; Treatment Outcome

TL;DR:
Taking a semiparametric theory perspective, this work proposes a broadly applicable approach to adjustment for auxiliary covariates to achieve more efficient estimators and tests for treatment parameters in the analysis of randomized clinical trials.
(via Semantic Scholar)

UN Sustainable Development Goal Categories

3. Good Health and Well-being
(Web of Science)

Sources: Web Of Science, NC State University Libraries

Added: August 6, 2018

2008 journal article

Improving the efficiency of the log-rank test using auxiliary covariates

*BIOMETRIKA*, *95*(3), 679–694.

UN Sustainable Development Goal Categories

3. Good Health and Well-being
(Web of Science)

Source: Web Of Science

Added: August 6, 2018

2008 journal article

Optimal two-stage group-sequential designs

*JOURNAL OF STATISTICAL PLANNING AND INFERENCE*, *138*(2), 489–499.

author keywords: adaptive; backward induction; group-sequential; optimal design; two-stage

UN Sustainable Development Goal Categories

3. Good Health and Well-being
(Web of Science)

16. Peace, Justice and Strong Institutions
(OpenAlex)

Source: Web Of Science

Added: August 6, 2018

2008 journal article

Quality of life with defibrillator therapy or amiodarone in heart failure

*NEW ENGLAND JOURNAL OF MEDICINE*, *359*(10), 999–1008.

MeSH headings : Activities of Daily Living; Adult; Amiodarone / therapeutic use; Anti-Arrhythmia Agents / therapeutic use; Defibrillators, Implantable / adverse effects; Drug Therapy, Combination; Female; Follow-Up Studies; Heart Failure / drug therapy; Heart Failure / mortality; Heart Failure / psychology; Heart Failure / therapy; Humans; Male; Quality of Life; Survival Analysis

TL;DR:
In a large primary-prevention population with moderately symptomatic heart failure, single-lead ICD therapy was not associated with any detectable adverse quality-of-life effects during 30 months of follow-up.
(via Semantic Scholar)

UN Sustainable Development Goal Categories

3. Good Health and Well-being
(Web of Science)

Source: Web Of Science

Added: August 6, 2018

2006 journal article

Adaptive two-stage designs in phase II clinical trials

*STATISTICS IN MEDICINE*, *25*(19), 3382–3395.

author keywords: two-stage adaptive design; backward induction; Bayesian decision theory; simulated annealing

MeSH headings : Clinical Trials, Phase II as Topic / methods; Humans; Research Design; Sample Size

TL;DR:
This paper proposes an adaptive two‐stage design which allows the sample size at the second stage to depend on the results at the first stage, the optimality criterion being minimum expected sample size under the null hypothesis.
(via Semantic Scholar)

UN Sustainable Development Goal Categories

3. Good Health and Well-being
(Web of Science)

16. Peace, Justice and Strong Institutions
(OpenAlex)

Source: Web Of Science

Added: August 6, 2018

2006 journal article

Cost-effectiveness of defibrillator therapy or amiodarone in chronic stable heart failure - Results from the Sudden Cardiac Death in Heart Failure Trial (SCD-HeFT)

*CIRCULATION*, *114*(2), 135–142.

author keywords: amiodarone; defibrillators, implantable; heart failure, congestive; quality-adjusted life years; cost-benefit analysis

MeSH headings : Adult; Cost-Benefit Analysis; Death, Sudden, Cardiac / epidemiology; Defibrillators, Implantable / economics; Electroshock; Equipment Design; Heart Failure / surgery; Humans; Medical Records; Randomized Controlled Trials as Topic / economics; Retrospective Studies; Treatment Outcome; United States / epidemiology

TL;DR:
Prophylactic use of single-lead, shock-only I CD therapy is economically attractive in patients with stable, moderately symptomatic heart failure with an ejection fraction ≤35%, particularly those in NYHA class II, as long as the benefits of ICD therapy observed in the SCD-HeFT persist for at least 8 years.
(via Semantic Scholar)

UN Sustainable Development Goal Categories

3. Good Health and Well-being
(Web of Science; OpenAlex)

Source: Web Of Science

Added: August 6, 2018

2006 article

Information-based monitoring of clinical trials

Tsiatis, A. A. (2006, October 15). *STATISTICS IN MEDICINE*, Vol. 25, pp. 3236–3244.

author keywords: group-sequential test; statistical information

MeSH headings : Clinical Trials Data Monitoring Committees; Clinical Trials as Topic / methods; Clinical Trials as Topic / standards; Clinical Trials as Topic / statistics & numerical data; Humans; Sample Size

TL;DR:
It is argued in this paper that statistical precision is directly related to Statistical Information and that the study should continue until the requisite statistical information is obtained and that this type of methodology is best suited with group‐sequential trials which monitor the data periodically and allow for estimation of the statistical information as the study progresses.
(via Semantic Scholar)

UN Sustainable Development Goal Categories

3. Good Health and Well-being
(Web of Science)

Source: Web Of Science

Added: August 6, 2018

2006 journal article

On closed form semiparametric estimators for measurement error models

*Statistica Sinica*, *16*(1), 183–193.

Source: NC State University Libraries

Added: August 6, 2018

2006 journal article

Semiparametric efficient estimation of survival distributions in two-stage randomisation designs in clinical trials with censored data

*BIOMETRIKA*, *93*(1), 163–177.

author keywords: censoring; influence function; inverse probability weighted estimator; potential outcome; regular asymptotically linear estimator; two-stage design

TL;DR:
This paper derives the best regular asymptotically linear estimator for the survival distribution and related quantities of treatment regimes and proposes an estimator which is easily computable and is more efficient than existing estimators.
(via Semantic Scholar)

UN Sustainable Development Goal Categories

3. Good Health and Well-being
(Web of Science)

Source: Web Of Science

Added: August 6, 2018

2006 book

Semiparametric theory and missing data

New York: Springer.

Source: NC State University Libraries

Added: August 6, 2018

2006 journal article

Semiparametric transformation models for the case-cohort study

*BIOMETRIKA*, *93*(1), 207–214.

author keywords: case-cohort design; martingale; transformation model; weighted estimating equation

TL;DR:
Weighted estimating equations are proposed for simultaneous estimation of the regression parameters and the transformation function and it is shown that the resulting regression estimators are asymptotically normal, with variance-covariance matrix that has a closed form and can be consistently estimated by the usual plug-in method.
(via Semantic Scholar)

UN Sustainable Development Goal Categories

3. Good Health and Well-being
(Web of Science)

Source: Web Of Science

Added: August 6, 2018

2005 journal article

Comparison between two partial likelihood approaches for the competing risks model with missing cause of failure

*LIFETIME DATA ANALYSIS*, *11*(1), 29–40.

author keywords: cause-specific hazard; martingale; missing at random; semiparametric efficiency

MeSH headings : Cause of Death; Humans; Life Tables; Likelihood Functions; Models, Statistical; Proportional Hazards Models; Risk; Risk Assessment; Sensitivity and Specificity; Survival Analysis; United States

TL;DR:
It is shown that the estimator for the regression coefficients based on the Dewanji partial likelihood is not only consistent and asymptotically normal, but also semiparametric efficient.
(via Semantic Scholar)

UN Sustainable Development Goal Categories

3. Good Health and Well-being
(Web of Science)

Source: Web Of Science

Added: August 6, 2018

2005 journal article

Conducting economic evaluations alongside multinational clinical trials: Toward a research consensus

*AMERICAN HEART JOURNAL*, *149*(3), 434–443.

MeSH headings : Clinical Trials as Topic / economics; Clinical Trials as Topic / methods; Clinical Trials as Topic / trends; Costs and Cost Analysis / classification; Forecasting; Health Care Costs / classification; Health Resources / economics; Humans; Internationality; Multicenter Studies as Topic / economics; Multicenter Studies as Topic / methods; Multicenter Studies as Topic / trends; Research Design; Resource Allocation / methods; Terminology as Topic; Treatment Outcome; United States

TL;DR:
A framework for classifying multinational economic evaluations according to (a) the sources of an analyst's estimates of resource use and clinical effectiveness and (b) the analyst's method of estimating costs is proposed.
(via Semantic Scholar)

UN Sustainable Development Goal Categories

3. Good Health and Well-being
(Web of Science)

Source: Web Of Science

Added: August 6, 2018

2005 journal article

Locally efficient semiparametric estimators for generalized skew-elliptical distributions

*JOURNAL OF THE AMERICAN STATISTICAL ASSOCIATION*, *100*(471), 980–989.

UN Sustainable Development Goal Categories

3. Good Health and Well-being
(Web of Science)

Source: Web Of Science

Added: August 6, 2018

2005 journal article

Locally-efficient robust estimation of haplotype-disease association in family-based studies

*BIOMETRIKA*, *92*(3), 559–571.

author keywords: family-based association study; haplotype; nuisance parameter

TL;DR:
This work develops locally-efficient estimators of the effect of haplotypes on disease that are robust to incorrect estimates of haplotype frequencies and is demonstrated with a simulation study of a case-parent design.
(via Semantic Scholar)

UN Sustainable Development Goal Categories

3. Good Health and Well-being
(Web of Science)

Source: Web Of Science

Added: August 6, 2018

2005 journal article

Semiparametric estimation of treatment effect in a pretest-posttest study with missing data

*Statistical Science*, *20*(3), 261–282.

Source: NC State University Libraries

Added: August 6, 2018

2005 journal article

Semiparametric estimators for the regression coefficients in the linear transformation competing risks model with missing cause of failure

*BIOMETRIKA*, *92*(4), 875–891.

UN Sustainable Development Goal Categories

3. Good Health and Well-being
(Web of Science)

Source: Web Of Science

Added: August 6, 2018

2005 journal article

Semiparametric inference in observational duration-response studies, with duration possibly right-censored

*BIOMETRIKA*, *92*(3), 605–618.

author keywords: confounding; infusion trial; missing data; survival analysis

TL;DR:
Estimating a functional relationship between the response and a treatment duration policy, continuously in time, is the focus of this paper.
(via Semantic Scholar)

UN Sustainable Development Goal Categories

3. Good Health and Well-being
(Web of Science; OpenAlex)

Source: Web Of Science

Added: August 6, 2018

2005 article

Statistical issues arising in the Women's Health Initiative - Discussion

Tsiatis, A. A., & Davidian, M. (2005, December). *BIOMETRICS*, Vol. 61, pp. 933–935.

UN Sustainable Development Goal Categories

3. Good Health and Well-being
(Web of Science)

5. Gender Equality
(OpenAlex)

Sources: Web Of Science, NC State University Libraries

Added: August 6, 2018

2004 journal article

Differential Treatment Benefit of Platelet Glycoprotein IIb/IIIa Inhibition With Percutaneous Coronary Intervention Versus Medical Therapy for Acute Coronary Syndromes

*Circulation*, *109*(5), 641–646.

author keywords: trials; glycoproteins; coronary disease

MeSH headings : Acute Disease; Aged; Combined Modality Therapy; Coronary Disease / drug therapy; Coronary Disease / surgery; Female; Humans; Male; Middle Aged; Myocardial Revascularization; Patient Selection; Platelet Glycoprotein GPIIb-IIIa Complex / antagonists & inhibitors; Randomized Controlled Trials as Topic; Research Design; Syndrome; Treatment Outcome

TL;DR:
Clinical-practice decisions and practice guidelines should be based on overall trial results and not analyses of post-randomization subgroups, based primarily on inappropriate subgroup analyses performed across the glycoprotein IIb/IIIa inhibitor trials.
(via Semantic Scholar)

UN Sustainable Development Goal Categories

3. Good Health and Well-being
(Web of Science; OpenAlex)

Sources: Web Of Science, Crossref, NC State University Libraries

Added: August 6, 2018

2004 journal article

Estimating mean response as a function of treatment duration in an observational study, where duration may be informatively censored

*BIOMETRICS*, *60*(2), 315–323.

author keywords: censored covariates; censored treatment; confounding; infusion length; inverse weighting; observational study; propensity score; survival analysis

MeSH headings : Biometry; Clinical Protocols; Coronary Artery Disease / drug therapy; Data Interpretation, Statistical; Eptifibatide; Humans; Peptides / therapeutic use; Platelet Aggregation Inhibitors / therapeutic use; Randomized Controlled Trials as Topic / statistics & numerical data; Time Factors; Treatment Outcome

TL;DR:
An estimator for mean response as a function of treatment‐duration policy under conditions where treatment must be terminated prematurely due to an adverse event or until a treatment‐censoring event occurs, whichever comes first is proposed.
(via Semantic Scholar)

UN Sustainable Development Goal Categories

3. Good Health and Well-being
(Web of Science)

Source: Web Of Science

Added: August 6, 2018

2004 journal article

Joint modeling of longitudinal and time-to-event data: An overview

*Statistica Sinica*, *14*(3), 809–834.

Source: NC State University Libraries

Added: August 6, 2018

2004 journal article

Locally efficient semiparametric estimators for functional measurement error models

*Biometrika*, *91*(4), 835–848.

Source: NC State University Libraries

Added: August 6, 2018

2004 journal article

Marginal structural models for analyzing causal effects of time-dependent treatments: An application in perinatal epidemiology

*AMERICAN JOURNAL OF EPIDEMIOLOGY*, *159*(10), 926–934.

author keywords: causality; confounding factors (epidemiology); epidemiologic methods; longitudinal studies; models, structural

MeSH headings : Adult; Anemia / diet therapy; Anemia / epidemiology; Anemia / prevention & control; Causality; Confounding Factors, Epidemiologic; Dietary Supplements; Epidemiologic Research Design; Female; Humans; Iron / administration & dosage; Logistic Models; Models, Statistical; North Carolina / epidemiology; Pregnancy; Pregnancy Complications, Hematologic / diet therapy; Pregnancy Complications, Hematologic / epidemiology; Pregnancy Complications, Hematologic / prevention & control; Random Allocation; Time Factors

TL;DR:
The objective of this paper is to illustrate the fitting of an MSM for the causal effect of iron supplement use during pregnancy (time-varying treatment) on odds of anemia at delivery in the presence of time-dependent confounding.
(via Semantic Scholar)

UN Sustainable Development Goal Categories

3. Good Health and Well-being
(Web of Science; OpenAlex)

Sources: Web Of Science, NC State University Libraries

Added: August 6, 2018

2004 journal article

Optimal duration of eptifibatide infusion in percutaneous coronary intervention (an ESPRIT Substudy)

*AMERICAN JOURNAL OF CARDIOLOGY*, *94*(7), 926–929.

MeSH headings : Angioplasty, Balloon, Coronary; Biomarkers / blood; Coronary Disease / therapy; Creatine Kinase / blood; Creatine Kinase, MB Form; Double-Blind Method; Eptifibatide; Humans; Infusions, Intra-Arterial; Isoenzymes / blood; Myocardial Infarction / blood; Myocardial Infarction / etiology; Myocardial Infarction / mortality; North America / epidemiology; Peptides / therapeutic use; Platelet Aggregation Inhibitors / therapeutic use; Platelet Glycoprotein GPIIb-IIIa Complex / antagonists & inhibitors; Platelet Glycoprotein GPIIb-IIIa Complex / therapeutic use; Postoperative Complications / blood; Postoperative Complications / etiology; Postoperative Complications / mortality; Survival Analysis; Time Factors; Treatment Outcome

TL;DR:
The investigators sought to determine the optimal length of the infusion of eptifibatide after PCI by analyzing the outcomes of patients enrolled in the Enhanced Suppression of the Platelet IIb/IIIa Receptor With Integrilin Therapy trial who were randomized to treatment with ePTifib atide.
(via Semantic Scholar)

UN Sustainable Development Goal Categories

3. Good Health and Well-being
(Web of Science; OpenAlex)

Source: Web Of Science

Added: August 6, 2018

2004 journal article

Optimal estimator for the survival distribution and related quantities for treatment policies in two-stage randomization designs in clinical trials

*BIOMETRICS*, *60*(1), 124–133.

author keywords: induction therapy; influence functions; intent-to-treat; inverse probability weighted estimator; maintenance therapy; missing data; potential outcomes; survival analysis

MeSH headings : Antineoplastic Agents / therapeutic use; Biometry; Granulocyte-Macrophage Colony-Stimulating Factor / therapeutic use; Humans; Leukemia, Myeloid, Acute / drug therapy; Leukemia, Myeloid, Acute / mortality; Linear Models; Models, Statistical; Nonlinear Dynamics; Randomized Controlled Trials as Topic / statistics & numerical data; Recombinant Proteins; Statistics, Nonparametric; Survival Analysis

TL;DR:
This article derives estimators that are easy to compute and are more efficient than previous estimators for the survival distribution and shows how to improve efficiency further by taking into account additional information from auxiliary variables.
(via Semantic Scholar)

UN Sustainable Development Goal Categories

3. Good Health and Well-being
(Web of Science)

Source: Web Of Science

Added: August 6, 2018

2004 journal article

The prognostic importance of comorbidity for mortality in patients with stable coronary artery disease

*ACC Current Journal Review*, *13*(4), 11–12.

UN Sustainable Development Goal Categories

3. Good Health and Well-being
(OpenAlex)

Source: Crossref

Added: August 28, 2020

2004 journal article

The prognostic importance of comorbidity for mortality in patients with stable coronary artery disease

*Journal of the American College of Cardiology*, *43*(4), 576–582.

MeSH headings : Black People / statistics & numerical data; Cardiac Catheterization; Cohort Studies; Comorbidity; Coronary Artery Disease / epidemiology; Coronary Artery Disease / ethnology; Coronary Artery Disease / mortality; Databases, Factual / statistics & numerical data; Female; Follow-Up Studies; Humans; Male; Middle Aged; Prognosis; Proportional Hazards Models; Stroke Volume / physiology; Survival Analysis; Time Factors; Ventricular Function, Left / physiology; White People / statistics & numerical data

TL;DR:
Comorbid disease is strongly associated with long-term survival in patients with CAD and co-existing illnesses should be measured and considered in clinical trials, disease registries, quality comparisons, and counseling of individual patients.
(via Semantic Scholar)

UN Sustainable Development Goal Categories

3. Good Health and Well-being
(OpenAlex)

Source: Crossref

Added: August 28, 2020

2003 journal article

On the inefficiency of the adaptive design for monitoring clinical trials

*BIOMETRIKA*, *90*(2), 367–378.

author keywords: adaptive design; group-sequential test; likelihood ratio test

TL;DR:
It is shown that adaptive designs are inefficient and that one can improve uniformly on such adaptive designs using standard group-sequential tests based on the sequentially computed likelihood ratio test statistic.
(via Semantic Scholar)

UN Sustainable Development Goal Categories

3. Good Health and Well-being
(Web of Science)

Source: Web Of Science

Added: August 6, 2018

2003 journal article

Semiparametric estimation of treatment effect in a pretest-posttest study

*BIOMETRICS*, *59*(4), 1046–1055.

author keywords: analysis of covariance; counterfactuals; influence function; inverse probability weighting; semiparametric model; t-test

MeSH headings : Antiviral Agents / therapeutic use; Biometry / methods; CD4 Lymphocyte Count; Clinical Trials as Topic; Computer Simulation; HIV Infections / drug therapy; Humans; Models, Statistical; Randomized Controlled Trials as Topic; Statistics, Nonparametric

TL;DR:
This work exploits recent developments in the literature on missing data and causal inference to derive the class of all consistent treatment effect estimators, identify the most efficient such estimator, and outline strategies for implementation of estimators that may improve on popular methods.
(via Semantic Scholar)

UN Sustainable Development Goal Categories

3. Good Health and Well-being
(Web of Science; OpenAlex)

Sources: Web Of Science, NC State University Libraries

Added: August 6, 2018

2002 journal article

A semiparametric likelihood approach to joint modeling of longitudinal and time-to-event data

*BIOMETRICS*, *58*(4), 742–753.

author keywords: informative censoring; mixed model; proportional hazards; SNP density; survival

MeSH headings : Anti-HIV Agents / therapeutic use; Biometry / methods; Computer Simulation; Disease Progression; HIV Infections / drug therapy; Humans; Likelihood Functions; Longitudinal Studies; Monte Carlo Method; Proportional Hazards Models; Randomized Controlled Trials as Topic / methods

TL;DR:
This work proposes a likelihood-based approach that requires only the assumption that the random effects have a smooth density, and implementation via the EM algorithm is described, and performance and the benefits for uncovering noteworthy features are illustrated.
(via Semantic Scholar)

UN Sustainable Development Goal Categories

3. Good Health and Well-being
(Web of Science; OpenAlex)

Sources: Web Of Science, NC State University Libraries

Added: August 6, 2018

2002 journal article

An estimator for the proportional hazards model with multiple longitudinal covariates measured with error

*Biostatistics (Oxford, England)*, *3*(4), 511–528.

Source: NC State University Libraries

Added: August 6, 2018

2002 journal article

Effects of insulin in relatives of patients with type 1 diabetes mellitus

*New England Journal of Medicine*, *346*(22), 1685–1691.

Source: NC State University Libraries

Added: August 6, 2018

2002 journal article

Estimation of survival distributions of treatment policies in two-stage randomization designs in clinical trials

*BIOMETRICS*, *58*(1), 48–57.

author keywords: induction therapy; intent to treat; inverse weighting; maintenance therapy; potential outcomes; survival analysis

MeSH headings : Aged; Computer Simulation; Granulocyte-Macrophage Colony-Stimulating Factor / therapeutic use; Humans; Leukemia, Myeloid, Acute / drug therapy; Models, Statistical; Randomized Controlled Trials as Topic / methods; Survival Analysis; Treatment Outcome

TL;DR:
It is proposed consistent estimators for the survival distribution and mean restricted survival time for each treatment policy in such two‐stage studies and derive large‐sample properties.
(via Semantic Scholar)

UN Sustainable Development Goal Categories

3. Good Health and Well-being
(Web of Science; OpenAlex)

Sources: Web Of Science, NC State University Libraries

Added: August 6, 2018

2002 journal article

Median regression with censored cost data

*BIOMETRICS*, *58*(3), 643–649.

author keywords: cost analysis; estimating equation; median regression; simulated annealing; survival analysis

MeSH headings : Biometry; Colorectal Neoplasms / economics; Colorectal Neoplasms / therapy; Costs and Cost Analysis / statistics & numerical data; Health Care Costs / statistics & numerical data; Humans; Models, Statistical; Regression Analysis

TL;DR:
This article proposes semiparametric procedures for estimating the parameters in median regression models based on weighted estimating equations when censoring is present and the methods are applied to a dataset for medical costs of patients with colorectal cancer.
(via Semantic Scholar)

UN Sustainable Development Goal Categories

3. Good Health and Well-being
(Web of Science)

Source: Web Of Science

Added: August 6, 2018

2002 journal article

Multiple imputation methods for testing treatment differences in survival distributions with missing cause of failure

*Biometrika*, *89*(1), 238–244.

Source: NC State University Libraries

Added: August 6, 2018

2001 journal article

A semiparametric estimator for the proportional hazards model with longitudinal covariates measured with error

*BIOMETRIKA*, *88*(2), 447–458.

UN Sustainable Development Goal Categories

3. Good Health and Well-being
(Web of Science)

Source: Web Of Science

Added: August 6, 2018

2001 journal article

Causal inference on the difference of the restricted mean lifetime between two groups

*BIOMETRICS*, *57*(4), 1030–1038.

author keywords: causal inference; Cox's proportional hazard model; martingale process; observational study; restricted lifetime; stochastic integral; survival analysis

MeSH headings : Biometry; Coronary Disease / mortality; Coronary Disease / therapy; Humans; Life Tables; Models, Statistical; Proportional Hazards Models; Stochastic Processes; Survival Analysis

TL;DR:
Estimators for the difference of the restricted mean lifetime between two groups that account for treatment imbalances in prognostic factors assuming a proportional hazards relationship are proposed and large‐sample properties of these estimators based on martingale theory for counting processes are derived.
(via Semantic Scholar)

UN Sustainable Development Goal Categories

3. Good Health and Well-being
(Web of Science)

Source: Web Of Science

Added: August 6, 2018

2001 journal article

Computationally simple accelerated failure time regression for interval censored data

*BIOMETRIKA*, *88*(3), 703–711.

author keywords: AIDS; current status data; nonparametric maximum likelihood; survival analysis

UN Sustainable Development Goal Categories

3. Good Health and Well-being
(Web of Science)

Source: Web Of Science

Added: August 6, 2018

2001 journal article

Efficiency study of estimators for a treatment effect in a pretest-posttest trial

*AMERICAN STATISTICIAN*, *55*(4), 314–321.

UN Sustainable Development Goal Categories

3. Good Health and Well-being
(Web of Science; OpenAlex)

Source: Web Of Science

Added: August 6, 2018

2001 journal article

Flexible sample size considerations using information-based interim monitoring

*DRUG INFORMATION JOURNAL*, *35*(4), 1095–1112.

author keywords: sample size reestimation; information-based design and monitoring; adaptive design

TL;DR:
It is argued that the precision with which a treatment effect is estimated is directly related to the statistical information in the data and is well suited to being adopted in conjunction with a group sequential clinical trial where the data are monitored routinely anyway.
(via Semantic Scholar)

UN Sustainable Development Goal Categories

3. Good Health and Well-being
(Web of Science)

Source: Web Of Science

Added: August 6, 2018

2001 journal article

Interim monitoring of group sequential trials using spending functions for the type I and type II error probabilities

*DRUG INFORMATION JOURNAL*, *35*(4), 1113–1121.

author keywords: group sequential trials; information time; error spending function

UN Sustainable Development Goal Categories

3. Good Health and Well-being
(Web of Science)

Source: Web Of Science

Added: August 6, 2018

2001 journal article

Multiple imputation methods for estimating regression coefficients in the competing risks model with missing cause of failure

*BIOMETRICS*, *57*(4), 1191–1197.

author keywords: cause-specific hazard; missing at random; partial likelihood

MeSH headings : Breast Neoplasms / metabolism; Breast Neoplasms / mortality; Breast Neoplasms / therapy; Cause of Death; Clinical Trials as Topic / statistics & numerical data; Female; Humans; Likelihood Functions; Models, Statistical; Neoplasms, Hormone-Dependent / metabolism; Neoplasms, Hormone-Dependent / mortality; Neoplasms, Hormone-Dependent / therapy; Proportional Hazards Models; Receptors, Estrogen / metabolism; Regression Analysis; Risk; Treatment Failure

UN Sustainable Development Goal Categories

3. Good Health and Well-being
(Web of Science; OpenAlex)

Source: Web Of Science

Added: August 6, 2018

2001 journal article

Testing equality of survival functions of quality-adjusted lifetime

*BIOMETRICS*, *57*(3), 861–867.

author keywords: counting processes; hypothesis test; martingale processes; quality of life; survival analysis

MeSH headings : Biometry; Breast Neoplasms / mortality; Breast Neoplasms / therapy; Clinical Trials as Topic / statistics & numerical data; Female; Humans; Models, Statistical; Quality-Adjusted Life Years; Survival Analysis

TL;DR:
A test statistic is presented that becomes the ordinary log‐rank test when quality‐adjusted lifetime is the same as the survival time and is applied to a breast cancer trial for comparing the distribution of quality-adjusted lifetime between two treatment regimes.
(via Semantic Scholar)

UN Sustainable Development Goal Categories

3. Good Health and Well-being
(Web of Science; OpenAlex)

Source: Web Of Science

Added: August 6, 2018

2001 journal article

Using auxiliary time-dependent covariates to recover information in nonparametric testing with censored data

*LIFETIME DATA ANALYSIS*, *7*(2), 125–141.

author keywords: Kaplan-Meier estimate; longitudinal; missing data; survival; two-sample test

MeSH headings : Data Interpretation, Statistical; Humans; Longitudinal Studies; Probability; Prognosis; Survival Analysis; United States

TL;DR:
This work proposes a test statistic based on the statistic of Pepe and Fleming (1989, 1991) that incorporates prognostic time-dependent covariate information and it is shown to be effective at detecting survival differences in crossing hazards settings where the logrank test performs poorly.
(via Semantic Scholar)

UN Sustainable Development Goal Categories

3. Good Health and Well-being
(Web of Science)

Source: Web Of Science

Added: August 6, 2018

2001 journal article

Utilizing propensity scores to estimate causal treatment effects with censored time-lagged data

*BIOMETRICS*, *57*(4), 1207–1218.

author keywords: censoring; confounding; inverse weighting; Kaplan-Meier; observational study; propensity score; survival analysis

MeSH headings : Analysis of Variance; Biometry; Clinical Trials as Topic / statistics & numerical data; Coronary Disease / economics; Coronary Disease / therapy; Data Interpretation, Statistical; Health Care Costs / statistics & numerical data; Humans; Models, Statistical

TL;DR:
A method for estimating treatment effects in nonrandomized studies with right‐censored responses is presented and an estimator for comparing two treatments by applying inverse weights to the complete cases is derived.
(via Semantic Scholar)

UN Sustainable Development Goal Categories

3. Good Health and Well-being
(Web of Science)

Source: Web Of Science

Added: August 6, 2018

2000 journal article

Cost-effectiveness of platelet glycoprotein IIb/IIIa inhibition with eptifibatide in patients with non-ST-elevation acute coronary syndromes

*CIRCULATION*, *101*(4), 366–371.

author keywords: coronary disease; cost-benefit analysis; glycoproteins; eptifibatide

MeSH headings : Angina, Unstable / drug therapy; Angina, Unstable / economics; Cost-Benefit Analysis; Eptifibatide; Female; Follow-Up Studies; Humans; Length of Stay; Male; Middle Aged; Myocardial Infarction / drug therapy; Myocardial Infarction / economics; Peptides / economics; Peptides / therapeutic use; Platelet Aggregation Inhibitors / economics; Platelet Aggregation Inhibitors / therapeutic use; Platelet Glycoprotein GPIIb-IIIa Complex / antagonists & inhibitors; Prospective Studies; Risk Factors; United States

TL;DR:
Based on the results observed in the US PURSUIT patients, the routine addition of eptifibatide to standard care for non-ST-elevation acute coronary syndrome patients is economically attractive by conventional standards.
(via Semantic Scholar)

UN Sustainable Development Goal Categories

3. Good Health and Well-being
(Web of Science)

Source: Web Of Science

Added: August 6, 2018

2000 journal article

Estimating medical costs with censored data

*BIOMETRIKA*, *87*(2), 329–343.

author keywords: cost analysis; counting process; efficiency; martingale; missing data; semiparametrics; survival analysis

TL;DR:
A class of weighted estimators which account appropriately for censoring are introduced and are shown to be consistent and asymptotically normal with easily estimated variances in a sample of individuals whose medical costs may be right censored.
(via Semantic Scholar)

UN Sustainable Development Goal Categories

3. Good Health and Well-being
(Web of Science)

16. Peace, Justice and Strong Institutions
(OpenAlex)

Source: Web Of Science

Added: August 6, 2018

2000 article

Estimating the distribution of quality-adjusted life with censored data

Tsiatis, A. A. (2000, April). *AMERICAN HEART JOURNAL*, Vol. 139, pp. S177–S181.

UN Sustainable Development Goal Categories

3. Good Health and Well-being
(Web of Science)

Source: Web Of Science

Added: August 6, 2018

2000 journal article

Human immunodeficiency virus type 1 RNA level and CD4 count as prognostic markers and surrogate end points: A meta-analysis

*AIDS Research and Human Retroviruses*, *16*(12), 1123–1133.

Source: NC State University Libraries

Added: August 6, 2018

2000 journal article

Testing for differences in survival with delayed ascertainment

*BIOMETRICS*, *56*(1), 145–153.

author keywords: ascertainment bias; counting process; logrank statistic; Martingale; U statistic

MeSH headings : Acquired Immunodeficiency Syndrome / drug therapy; Acquired Immunodeficiency Syndrome / immunology; Acquired Immunodeficiency Syndrome / mortality; Anti-HIV Agents / therapeutic use; Biometry; CD4 Lymphocyte Count; Clinical Trials as Topic / statistics & numerical data; Humans; Survival Analysis

TL;DR:
A class of test statistics that extends the usual class of U statistics is proposed that can be more powerful than the current practice when differential ascertainment is present and to illustrate the potential loss of information when lagging follow‐up to control for ascertainment delays, an AIDS clinical trial is re analyzed.
(via Semantic Scholar)

UN Sustainable Development Goal Categories

3. Good Health and Well-being
(Web of Science)

Source: Web Of Science

Added: August 6, 2018

2000 journal article

Using conditional logistic regression to fit proportional odds models to interval censored data

*BIOMETRICS*, *56*(2), 511–518.

author keywords: AIDS; current status data; nonparametric maximum likelihood; survival analysis

MeSH headings : Acquired Immunodeficiency Syndrome / drug therapy; Acquired Immunodeficiency Syndrome / immunology; Acquired Immunodeficiency Syndrome / mortality; Anti-HIV Agents / therapeutic use; Biometry / methods; CD4 Lymphocyte Count; Drug Therapy, Combination; Humans; Likelihood Functions; Models, Statistical; Regression Analysis; Statistics, Nonparametric; Survival Analysis; Zalcitabine / therapeutic use; Zidovudine / therapeutic use

TL;DR:
An easily implemented approach to fitting the proportional odds regression model to interval‐censored data is presented based on using conditional logistic regression routines in standard statistical packages to sidestep complications that attend estimation of the baseline odds ratio function.
(via Semantic Scholar)

UN Sustainable Development Goal Categories

3. Good Health and Well-being
(Web of Science)

Source: Web Of Science

Added: August 6, 2018

1999 journal article

Efficient estimation of the distribution of quality-adjusted survival time

*BIOMETRICS*, *55*(4), 1101–1107.

author keywords: counting process; Martingale process; quality of life; semiparametric efficiency; survival analysis

MeSH headings : Biometry; Breast Neoplasms / mortality; Breast Neoplasms / therapy; Clinical Trials as Topic / statistics & numerical data; Computer Simulation; Female; Humans; Models, Statistical; Quality-Adjusted Life Years; Survival Analysis

TL;DR:
Using the general representation theorem for missing data processes, this paper is able to derive an estimator that is more efficient than the one proposed in Zhao and Tsiatis (1997, Biometrika 84, 339-348).
(via Semantic Scholar)

UN Sustainable Development Goal Categories

3. Good Health and Well-being
(Web of Science; OpenAlex)

Source: Web Of Science

Added: August 6, 2018

1998 journal article

Estimating the parameters in the Cox model when covariate variables are measured with error

*BIOMETRICS*, *54*(4), 1407–1419.

author keywords: cumulative hazards; measurement error; nonparametric maximum likelihood; proportional hazards model; regression calibration; semiparametric method

TL;DR:
A likelihood-based approach is introduced, which is referred to as the semiparametric method, and it is shown that this method is an appealing alternative to the Cox proportional hazards model to analyze the relationship between survival and CD4 count in patients with AIDS.
(via Semantic Scholar)

UN Sustainable Development Goal Categories

3. Good Health and Well-being
(Web of Science)

Sources: Web Of Science, NC State University Libraries

Added: August 6, 2018

1998 journal article

Semiparametric efficient estimation in the generalized odds-rate class of regression models for right-censored time-to-event data

*Lifetime Data Analysis*, *4*(4), 355–391.

MeSH headings : Biometry / methods; Clinical Trials as Topic; Data Interpretation, Statistical; Humans; Lung Neoplasms / radiotherapy; Models, Statistical; Odds Ratio; Randomized Controlled Trials as Topic; Regression Analysis; Time Factors; United States; United States Department of Veterans Affairs

TL;DR:
The generalized odds-rate class of regression models for time to event data is indexed by a non-negative constant ρ and estimators for β and exp(α(t)) are constructed and it is shown that the estimator for β is semiparametric efficient in the sense that it attains the semiparmetric variance bound.
(via Semantic Scholar)

UN Sustainable Development Goal Categories

3. Good Health and Well-being
(Web of Science)

16. Peace, Justice and Strong Institutions
(OpenAlex)

Source: NC State University Libraries

Added: August 6, 2018

1998 journal article

Use of simulation and bootstrap in information-based group sequential studies

*Statistics in Medicine*, *17*(1), 75–87.

Source: NC State University Libraries

Added: August 6, 2018

1997 journal article

A consistent estimator for the distribution of quality adjusted survival time

*BIOMETRIKA*, *84*(2), 339–348.

author keywords: counting process; martingale process; quality of life; survival analysis

TL;DR:
A method is presented that enables the survival distribution of quality adjusted lifetime to be calculated using martingale theory for counting processes, and it is shown that the estimator is asymptotically consistent, normally distributed, and its asymPTotic variance estimate can be obtained analytically.
(via Semantic Scholar)

UN Sustainable Development Goal Categories

3. Good Health and Well-being
(Web of Science)

Source: Web Of Science

Added: August 6, 2018

1997 journal article

A joint model for survival and longitudinal data measured with error

*BIOMETRICS*, *53*(1), 330–339.

author keywords: EM algorithm; longitudinal data; repeated measures; survival

TL;DR:
This work argues that the Cox proportional hazards regression model method is superior to naive methods where one maximizes the partial likelihood of the Cox model using the observed covariate values and improves on two-stage methods where empirical Bayes estimates of the covariate process are computed and then used as time-dependent covariates.
(via Semantic Scholar)

UN Sustainable Development Goal Categories

3. Good Health and Well-being
(Web of Science)

Source: Web Of Science

Added: August 6, 2018

1997 journal article

Semiparametric efficiency and its implication on the design and analysis of group-sequential studies

*JOURNAL OF THE AMERICAN STATISTICAL ASSOCIATION*, *92*(440), 1342–1350.

UN Sustainable Development Goal Categories

3. Good Health and Well-being
(Web of Science)

Source: Web Of Science

Added: August 6, 2018

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