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Simulations For Designing And Interpreting Intervention Trials In Infectious Diseases., M Elizabeth Halloran, Kari Auranen, Sarah Baird, Nicole E Basta, Steven E Bellan, +several additional authors 2017 George Washington University

Simulations For Designing And Interpreting Intervention Trials In Infectious Diseases., M Elizabeth Halloran, Kari Auranen, Sarah Baird, Nicole E Basta, Steven E Bellan, +Several Additional Authors

Global Health Faculty Publications

BACKGROUND: Interventions in infectious diseases can have both direct effects on individuals who receive the intervention as well as indirect effects in the population. In addition, intervention combinations can have complex interactions at the population level, which are often difficult to adequately assess with standard study designs and analytical methods.

DISCUSSION: Herein, we urge the adoption of a new paradigm for the design and interpretation of intervention trials in infectious diseases, particularly with regard to emerging infectious diseases, one that more accurately reflects the dynamics of the transmission process. In an increasingly complex world, simulations can explicitly represent transmission dynamics ...


Novel Bayesian Adaptive Clinical Trial Designs In Early Phases, Haitao Pan 2017 The University of Texas MD Anderson Cancer Center UTHealth Graduate School of Biomedical Sciences

Novel Bayesian Adaptive Clinical Trial Designs In Early Phases, Haitao Pan

UT GSBS Dissertations and Theses (Open Access)

Early phase, or phase I and phase II, trials are the first step in testing new medicines that have been developed in the lab. The main goal of phase I clinical trials is to establish the recommended dose of new drugs for phase II trials. For the cytotoxic drugs, the goal is to find maximum tolerated dose (MTD). The guiding principle for dose escalation in phase I trials is to avoid exposing too many patients to subtherapeutic doses while preserving safety and maintaining rapid accrual. Therefore, dose escalation methods, especially Bayesian designs, are recommended to be used in phase I ...


Burden Of Atopic Dermatitis In The United States: Analysis Of Healthcare Claims Data In The Commercial, Medicare, And Medi-Cal Databases, Sulena Shrestha, Raymond Miao, Li Wang, Jingdong Chao, Huseyin Yuce, Wenhui Wei 2017 STATinMED Research/SIMR, Inc.

Burden Of Atopic Dermatitis In The United States: Analysis Of Healthcare Claims Data In The Commercial, Medicare, And Medi-Cal Databases, Sulena Shrestha, Raymond Miao, Li Wang, Jingdong Chao, Huseyin Yuce, Wenhui Wei

Publications and Research

Comparative data on the burden of atopic dermatitis (AD) in adults relative to the general population are limited. We performed a large-scale evaluation of the burden of disease among US adults with AD relative to matched non-AD controls, encompassing comorbidities, healthcare resource utilization (HCRU), and costs, using healthcare claims data. The impact of AD disease severity on these outcomes was also evaluated.


The Importance Of Inhaler Technique In Measuring And Calculating Inhaler Adherence, And Its Clinical Outcomes, Imran Sulaiman 2017 Royal College of Surgeons in Ireland

The Importance Of Inhaler Technique In Measuring And Calculating Inhaler Adherence, And Its Clinical Outcomes, Imran Sulaiman

PhD theses

Depending on the population studied, cross-sectional observational studies suggest that between 14%-90% of patients do not use their pressurized metered dose inhaler correctly, while 50-60% misuse a dry powder inhaler. This means that unless incorrect technique is acounted for a significant underestimation of how much medication the person actually obtained may be made.

The aim of this thesis was to objectively determine the frequency and importance of inhaler technique errors and to combine these with inhaler use to provide an acurate method of calculating adherence. I then investigated different patterns of inhaler use, determinants of inhaler use and the ...


Evaluation Of Multiple Interventions Using A Stepped Wedge Design, Vivian H. Lyons, Lingyu Li, James Hughes, Ali Rowhani-Rahbar 2017 University of Washington - Seattle Campus

Evaluation Of Multiple Interventions Using A Stepped Wedge Design, Vivian H. Lyons, Lingyu Li, James Hughes, Ali Rowhani-Rahbar

UW Biostatistics Working Paper Series

Background: Stepped wedge cluster randomized trials are a class of unidirectional crossover studies that have historically been limited to evaluating a single intervention. This design is especially suitable for pragmatic trials where the study feasibility can be improved with a phased introduction of the intervention. We examined variations of stepped wedge designs that would support evaluation of multiple interventions. Methods: We propose four different design variants for implementing a stepped wedge trial with two interventions: concurrent design, supplementation, replacement, and factorial designs. Analyses were conducted comparing the precision of the estimated intervention effects for the different designs. Results: Concurrent, supplementation ...


Randomized Clinical Trial To Evaluate The Pathogenicity Of Bibersteinia Trehalosi In Respiratory Disease Among Calves, Christy Jo Hanthorn, Reneé Dewell, Vickie L. Cooper, Timothy S. Frana, Paul J. Plummer, Chong Wang, Grant A. Dewell 2017 Iowa State University

Randomized Clinical Trial To Evaluate The Pathogenicity Of Bibersteinia Trehalosi In Respiratory Disease Among Calves, Christy Jo Hanthorn, Reneé Dewell, Vickie L. Cooper, Timothy S. Frana, Paul J. Plummer, Chong Wang, Grant A. Dewell

Grant A. Dewell

Bibersteinia trehalosi causes respiratory disease in ruminants particularly in wild and domestic sheep. Recently, there has been an increased number of B. trehalosi isolates obtained from diagnostic samples from bovine respiratory disease cases. This study evaluated the role of B. trehalosi in bovine respiratory disease using an intra-tracheal inoculation model in calves. Thirty six cross bred 2–3 month old dairy calves were inoculated intra-tracheally with either leukotoxin negative B. trehalosi, leukotoxin positive B. trehalosi isolate, Mannheimia haemolytica, a combination of leukotoxin negative B. trehalosi and M. haemolytica or negative control. Calves were euthanized and necropsy performed on day 10 ...


Denoising Tandem Mass Spectrometry Data, Felix Offei 2017 East Tennessee State Universtiy

Denoising Tandem Mass Spectrometry Data, Felix Offei

Electronic Theses and Dissertations

Protein identification using tandem mass spectrometry (MS/MS) has proven to be an effective way to identify proteins in a biological sample. An observed spectrum is constructed from the data produced by the tandem mass spectrometer. A protein can be identified if the observed spectrum aligns with the theoretical spectrum. However, data generated by the tandem mass spectrometer are affected by errors thus making protein identification challenging in the field of proteomics. Some of these errors include wrong calibration of the instrument, instrument distortion and noise. In this thesis, we present a pre-processing method, which focuses on the removal of ...


Polymer Micelle Formulation For The Proteasome Inhibitor Drug Carfilzomib: Anticancer Efficacy And Pharmacokinetic Studies In Mice, Ji Eun Park, Se-Eun Chun, Derek Alexander Reichel, Jee Sun Min, Su-Chan Lee, Songhee Han, Gongmi Ryoo, Yunseok Oh, Shin-Hyung Park, Heon-Min Ryu, Kyung Bo Kim, Ho-Young Lee, Soo Kyung Bae, Younsoo Bae, Wooin Lee 2017 Seoul National University, South Korea

Polymer Micelle Formulation For The Proteasome Inhibitor Drug Carfilzomib: Anticancer Efficacy And Pharmacokinetic Studies In Mice, Ji Eun Park, Se-Eun Chun, Derek Alexander Reichel, Jee Sun Min, Su-Chan Lee, Songhee Han, Gongmi Ryoo, Yunseok Oh, Shin-Hyung Park, Heon-Min Ryu, Kyung Bo Kim, Ho-Young Lee, Soo Kyung Bae, Younsoo Bae, Wooin Lee

Pharmaceutical Sciences Faculty Publications

Carfilzomib (CFZ) is a peptide epoxyketone proteasome inhibitor approved for the treatment of multiple myeloma (MM). Despite the remarkable efficacy of CFZ against MM, the clinical trials in patients with solid cancers yielded rather disappointing results with minimal clinical benefits. Rapid degradation of CFZ in vivo and its poor penetration to tumor sites are considered to be major factors limiting its efficacy against solid cancers. We previously reported that polymer micelles (PMs) composed of biodegradable block copolymers poly(ethylene glycol) (PEG) and poly(caprolactone) (PCL) can improve the metabolic stability of CFZ in vitro. Here, we prepared the CFZ-loaded PM ...


Effect Of Immediate Initiation Of Antiretroviral Therapy On Risk Of Severe Bacterial Infections In Hiv-Positive People With Cd4 Cell Counts Of More Than 500 Cells Per Μl: Secondary Outcome Results From A Randomised Controlled Trial., Jemma O'Connor, Michael J Vjecha, Andrew N Phillips, Brian Angus, David Cooper, Fred Gordin, +several additional authors 2017 George Washington University

Effect Of Immediate Initiation Of Antiretroviral Therapy On Risk Of Severe Bacterial Infections In Hiv-Positive People With Cd4 Cell Counts Of More Than 500 Cells Per Μl: Secondary Outcome Results From A Randomised Controlled Trial., Jemma O'Connor, Michael J Vjecha, Andrew N Phillips, Brian Angus, David Cooper, Fred Gordin, +Several Additional Authors

Medicine Faculty Publications

BACKGROUND: The effects of antiretroviral therapy on risk of severe bacterial infections in people with high CD4 cell counts have not been well described. In this study, we aimed to quantify the effects of immediate versus deferred ART on the risk of severe bacterial infection in people with high CD4 cell counts in a preplanned analysis of the START trial.

METHODS: The START trial was a randomised controlled trial in ART-naive HIV-positive patients with CD4 cell count of more than 500 cells per μL assigned to immediate ART or deferral until their CD4 cell counts were lower than 350 cells ...


Adaptive Non-Inferiority Margins Under Observable Non-Constancy, Brett S. Hanscom, Deborah J. Donnell, Brian D. Williamson, Jim Hughes 2017 Fred Hutchinson Cancer Research Center

Adaptive Non-Inferiority Margins Under Observable Non-Constancy, Brett S. Hanscom, Deborah J. Donnell, Brian D. Williamson, Jim Hughes

UW Biostatistics Working Paper Series

A central assumption in the design and conduct of non-inferiority trials is that the active-control therapy will have the same degree of effectiveness in the planned non-inferiority trial as it had in the prior placebo-controlled trials used to define the non-inferiority margin. This is referred to as the `constancy' assumption. If the constancy assumption fails, the chosen non-inferiority margin is not valid and the study runs the risk of approving an inferior product or failing to approve a beneficial product. The constancy assumption cannot be validated in a trial without a placebo arm, and it is unlikely ever to be ...


A Comparison Of The Quality Of Informed Consent For Clinical Trials Of An Experimental Hookworm Vaccine Conducted In Developed And Developing Countries., David J. Diemert, Lucas Lobato, Ashley Styczynski, Maria Zumer, Amanda Soares, Maria Flávia Gazzinelli 2017 George Washington University

A Comparison Of The Quality Of Informed Consent For Clinical Trials Of An Experimental Hookworm Vaccine Conducted In Developed And Developing Countries., David J. Diemert, Lucas Lobato, Ashley Styczynski, Maria Zumer, Amanda Soares, Maria Flávia Gazzinelli

Microbiology, Immunology, and Tropical Medicine Faculty Publications

Informed consent is one of the principal ethical requirements of conducting clinical research, regardless of the study setting. Breaches in the quality of the informed consent process are frequently described in reference to clinical trials conducted in developing countries, due to low levels of formal education, a lack of familiarity with biomedical research, and limited access to health services in these countries. However, few studies have directly compared the quality of the informed consent process in developed and developing countries using the same tool and in similar clinical trials. This study was conducted to compare the quality of the informed ...


A Predictive Probability Interim Design For Phase Ii Clinical Trials With Continuous Endpoints, Meng Liu 2017 University of Kentucky

A Predictive Probability Interim Design For Phase Ii Clinical Trials With Continuous Endpoints, Meng Liu

Theses and Dissertations--Epidemiology and Biostatistics

Phase II clinical trials aim to potentially screen out ineffective and identify effective therapies to move forward to randomized phase III trials. Single-arm studies remain the most utilized design in phase II oncology trials, especially in scenarios where a randomized design is simply not practical. Due to concerns regarding excessive toxicity or ineffective new treatment strategies, interim analyses are typically incorporated in the trial, and the choice of statistical methods mainly depends on the type of primary endpoints. For oncology trials, the most common primary objectives in phase II trials include tumor response rate (binary endpoint) and progression disease-free survival ...


Variance Prior Specification For A Basket Trial Design Using Bayesian Hierarchical Modeling, Kristen Cunanan, Alexia Iasonos, Ronglai Shen, Mithat Gonen 2017 Memorial Sloan Kettering Cancer Center

Variance Prior Specification For A Basket Trial Design Using Bayesian Hierarchical Modeling, Kristen Cunanan, Alexia Iasonos, Ronglai Shen, Mithat Gonen

Memorial Sloan-Kettering Cancer Center, Dept. of Epidemiology & Biostatistics Working Paper Series

Background: In the era of targeted therapies, clinical trials in oncology are rapidly evolving, wherein patients from multiple diseases are now enrolled and treated according to their genomic mutation(s). In such trials, known as basket trials, the different disease cohorts form the different baskets for inference. Several approaches have been proposed in the literature to efficiently use information from all baskets while simultaneously screening to find individual baskets where the drug works. Most proposed methods are developed in a Bayesian paradigm that requires specifying a prior distribution for a variance parameter, which controls the degree to which information is ...


Analysis Of Data From A Study To Identify Potential Biomarkers To Indicate Renal Injury, Mitchell D. Tahtinen 2017 Michigan Technological University

Analysis Of Data From A Study To Identify Potential Biomarkers To Indicate Renal Injury, Mitchell D. Tahtinen

Dissertations, Master's Theses and Master's Reports

Ureteropelvic junction obstruction is a disease in which flow from the kidney to the bladder is obstructed for extended periods of time causing irreversible damage to the kidney. Current tests to detect kidney damage caused by obstruction are not effective until significant damage occurs. The purpose of this report is to identify a panel of biomarkers in urine to detect kidney damage earlier by analyzing data collected from a two-part study. Currently, two established urinary biomarkers to indicate kidney damage are NGAL and KIM-1. Biomarkers of interest in this study are CD13, CD10, and CD26. Results from the linear mixed ...


When Is A Randomised Controlled Trial Health Equity Relevant? Development And Validation Of A Conceptual Framework, J. Jull, M. Whitehead, M. Petticrew, E. Kristjansson, D. Gough, Sarah Baird, +several additional authors 2017 George Washington University

When Is A Randomised Controlled Trial Health Equity Relevant? Development And Validation Of A Conceptual Framework, J. Jull, M. Whitehead, M. Petticrew, E. Kristjansson, D. Gough, Sarah Baird, +Several Additional Authors

Global Health Faculty Publications

Background Randomised controlled trials can provide evidence relevant to assessing the equity impact of an intervention, but such information is often poorly reported. We describe a conceptual framework to identify health equity-relevant randomised trials with the aim of improving the design and reporting of such trials.

Methods An interdisciplinary and international research team engaged in an iterative consensus building process to develop and refine the conceptual framework via face-to-face meetings, teleconferences and email correspondence, including findings from a validation exercise whereby two independent reviewers used the emerging framework to classify a sample of randomised trials.

Results A randomised trial can ...


Implementing An Mhealth System For Substance Use Disorders In Primary Care: A Mixed Methods Study Of Clinicians’ Initial Expectations And First Year Experiences, Marie-Louise Mares, David H. Gustafson, Joseph E. Glass, Andrew Quanbeck, Helene McDowell, Fiona McTavish, Amy Atwood, Lisa Marsch 2016 University of Wisconsin-Madison

Implementing An Mhealth System For Substance Use Disorders In Primary Care: A Mixed Methods Study Of Clinicians’ Initial Expectations And First Year Experiences, Marie-Louise Mares, David H. Gustafson, Joseph E. Glass, Andrew Quanbeck, Helene Mcdowell, Fiona Mctavish, Amy Atwood, Lisa Marsch

Open Dartmouth: Faculty Open Access Articles

Millions of Americans need but don’t receive treatment for substance use, and evidence suggests that addiction-focused interventions on smart phones could support their recovery. There is little research on implementation of addiction-related interventions in primary care, particularly in Federally Qualified Health Centers (FQHCs) that provide primary care to underserved populations. We used mixed methods to examine three FQHCs’ implementation of Seva, a smart-phone app that offers patients online support/discussion, health-tracking, and tools for coping with cravings, and offers clinicians information about patients’ health tracking and relapses. We examined (a) clinicians' initial perspectives about implementing Seva, and (b) the ...


Update On Schizophrenia And Bipolar Disorder: Focus On Cariprazine, Rona Jeannie Roberts, Lillian Jan Findlay, Peggy El-Mallakh, Rif S. El-Mallakh 2016 University of Louisville

Update On Schizophrenia And Bipolar Disorder: Focus On Cariprazine, Rona Jeannie Roberts, Lillian Jan Findlay, Peggy El-Mallakh, Rif S. El-Mallakh

Nursing Faculty Publications

Schizophrenia and bipolar disorder are severe psychiatric disorders that are frequently associated with persistent symptoms and significant dysfunction. While there are a multitude of psychopharmacologic agents are available for treatment of these illnesses, suboptimal response and significant adverse consequences limit their utility. Cariprazine is a new, novel antipsychotic medication with dopamine D2 and D3 partial agonist effects. Its safety and efficacy have been investigated in acute psychosis of schizophrenia, bipolar mania, bipolar depression, and unipolar depression. Efficacy has been demonstrated in schizophrenia and mania. It is unclear if cariprazine is effective in depression associated with unipolar or bipolar illness. Adverse ...


A Randomized, Double-Blind, Placebo-Controlled Phase Ii Trial Investigating The Safety And Immunogenicity Of Modified Vaccinia Ankara Smallpox Vaccine (Mva-Bn®) In 56-80-Year-Old Subjects, Richard N. Greenberg, Christine M. Hay, Jack T. Stapleton, Thomas C. Marbury, Eva Wagner, Eva Kreitmeir, Siegfried Röesch, Alfred von Krempelhuber, Philip Young, Richard Nichols, Thomas P. Meyer, Darja Schmidt, Josef Weigl, Garth Virgin, Nathaly Arndtz-Wiedemann, Paul Chaplin 2016 University of Kentucky

A Randomized, Double-Blind, Placebo-Controlled Phase Ii Trial Investigating The Safety And Immunogenicity Of Modified Vaccinia Ankara Smallpox Vaccine (Mva-Bn®) In 56-80-Year-Old Subjects, Richard N. Greenberg, Christine M. Hay, Jack T. Stapleton, Thomas C. Marbury, Eva Wagner, Eva Kreitmeir, Siegfried Röesch, Alfred Von Krempelhuber, Philip Young, Richard Nichols, Thomas P. Meyer, Darja Schmidt, Josef Weigl, Garth Virgin, Nathaly Arndtz-Wiedemann, Paul Chaplin

Internal Medicine Faculty Publications

Background Modified Vaccinia Ankara MVA-BN® is a live, highly attenuated, viral vaccine under advanced development as a non-replicating smallpox vaccine. In this Phase II trial, the safety and immunogenicity of Modified Vaccinia Ankara MVA-BN® (MVA) was assessed in a 56–80 years old population.

Methods MVA with a virus titer of 1 x 108 TCID50/dose was administered via subcutaneous injection to 56–80 year old vaccinia-experienced subjects (N = 120). Subjects received either two injections of MVA (MM group) or one injection of Placebo and one injection of MVA (PM group) four weeks apart. Safety was evaluated by ...


Is The Force Awakened? Publication Trends In Oncology Big Data As Phase Ii Cancerlinq Is Launched, Hind Rafei, Benjamin Viernes, Angelike P. Liappis, Dalia Abdelaziz Mobarek 2016 George Washington University

Is The Force Awakened? Publication Trends In Oncology Big Data As Phase Ii Cancerlinq Is Launched, Hind Rafei, Benjamin Viernes, Angelike P. Liappis, Dalia Abdelaziz Mobarek

GW Research Days 2016 - Present

Background: The American Society of Clinical Oncology launched CancerLinQ project in 2010 to provide real-time data collection, mining and visualization, clinical decision support, and quality feedback. Creation of a big data software platform is currently underway to power the CancerLinQ in the phase II of the project. This would allow for evidence driven practice and rapid learning for cancer care providers. Additionally, adequate knowledge about the utility of Big Data to encourage provider utilization in high Impact Factor (IF) journals is needed. We aimed to assess trends and quality of Big Data published in Oncology.

Methods: Peer-reviewed English papers published ...


Models For Hsv Shedding Must Account For Two Levels Of Overdispersion, Amalia Magaret 2016 University of Washington - Seattle Campus

Models For Hsv Shedding Must Account For Two Levels Of Overdispersion, Amalia Magaret

UW Biostatistics Working Paper Series

We have frequently implemented crossover studies to evaluate new therapeutic interventions for genital herpes simplex virus infection. The outcome measured to assess the efficacy of interventions on herpes disease severity is the viral shedding rate, defined as the frequency of detection of HSV on the genital skin and mucosa. We performed a simulation study to ascertain whether our standard model, which we have used previously, was appropriately considering all the necessary features of the shedding data to provide correct inference. We simulated shedding data under our standard, validated assumptions and assessed the ability of 5 different models to reproduce the ...


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