Journal ArticleJ Pediatr · August 2025
OBJECTIVE: To develop and pilot an intervention to support communication and decision-making for critically ill infants. STUDY DESIGN: In this single-arm, mixed-methods, prospective, feasibility study, we enrolled infants, parents, and clinicians at a sing ...
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Journal ArticleJ Biopharm Stat · April 20, 2025
Biomarker-guided designs are increasingly used to evaluate personalized treatments based on patients' biomarker status in Phase II and III clinical trials. With adaptive enrichment, these designs can improve the efficiency of evaluating the treatment effec ...
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Journal ArticleStat Med · February 28, 2025
The accessibility of individual participant-level data (IPD) enhances the evaluation of moderation effects of patient covariates. It facilitates the provision of accurate estimation of intervention effects and confidence intervals by incorporating covariat ...
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Journal ArticlemedRxiv · February 12, 2025
BACKGROUND: Prophylactic steroids are often used to reduce the systemic inflammatory response to cardiopulmonary bypass in infants undergoing heart surgery. The STRESS trial found that the likelihood of a worse outcome did not differ between infants random ...
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Journal ArticlePediatrics · February 10, 2025
OBJECTIVE: Intensive health behavior and lifestyle treatment (IHBLT) is recommended for children aged 6-18 years with obesity. The objective was to evaluate the effectiveness of Fit Together, a health care and parks and recreation partnership to deliver IH ...
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Journal ArticleStat Methods Med Res · February 2025
Meta-analysis of randomized controlled trials is commonly used to evaluate treatments and inform policy decisions because it provides comprehensive summaries of all available evidence. However, meta-analyses are limited to draw population inference of trea ...
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Journal ArticleBiom J · February 2025
Network meta-analysis (NMA) extends pairwise meta-analysis to compare multiple treatments simultaneously by combining "direct" and "indirect" comparisons of treatments. The availability of individual participant data (IPD) makes it possible to evaluate tre ...
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Journal ArticleAm J Epidemiol · January 31, 2025
One of the key limitations of electronic health records (EHR) data is that not all health care encounters are observed. The degree to which patient information is captured is referred to as observability. Poor observability, and in particular differential ...
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Journal ArticleHealth Services and Outcomes Research Methodology · January 1, 2025
Combining data from diverse sources including randomized controlled trials (RCTs) and observational datasets holds the potential to increase sample size, improve external validity, and gain a well-rounded view of the question under study. However, the prac ...
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ConferenceCirculation · November 12, 2024
Background:
Prophylactic steroids are often used to reduce the systemic inflammatory response to cardiopulmonary bypass in infants undergoing heart surgery. The STRESS trial found that the likelihood ...
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Journal ArticleObstet Gynecol · May 1, 2024
OBJECTIVE: To evaluate the influence of the ARRIVE (A Randomized Trial of Induction Versus Expectant Management) trial and the coronavirus disease 2019 (COVID-19) pandemic on racial and ethnic differences in labor induction, pregnancy-associated hypertensi ...
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Journal ArticleStat Med · March 30, 2024
Individualized treatment decisions can improve health outcomes, but using data to make these decisions in a reliable, precise, and generalizable way is challenging with a single dataset. Leveraging multiple randomized controlled trials allows for the combi ...
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Journal ArticleSci Rep · February 24, 2024
While novel oral anticoagulants are increasingly used to reduce risk of stroke in patients with atrial fibrillation, vitamin K antagonists such as warfarin continue to be used extensively for stroke prevention across the world. While effective in reducing ...
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Journal ArticleJ Clin Epidemiol · December 2023
OBJECTIVES: We sought to assess and report harms that were observed but not disclosed previously in clinical trials of gabapentin. STUDY DESIGN AND SETTING: We reanalyzed individual participant data from six randomized parallel trials of gabapentin for neu ...
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Journal ArticleStat Sci · November 2023
Estimating treatment effects conditional on observed covariates can improve the ability to tailor treatments to particular individuals. Doing so effectively requires dealing with potential confounding, and also enough data to adequately estimate effect mod ...
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Journal ArticleJ Child Neurol · October 2023
Here, we describe the process of development of the methodology for an international multicenter natural history study of alternating hemiplegia of childhood as a prototype disease for rare neurodevelopmental disorders. We describe a systematic multistep a ...
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Journal ArticleJACC CardioOncol · October 2023
BACKGROUND: Androgen deprivation therapy is the cornerstone of treatment for patients with advanced prostate cancer. Meta-analysis of small, oncology-focused trials suggest gonadotropin-releasing hormone (GnRH) antagonists may be associated with fewer adve ...
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Journal ArticleAm J Respir Crit Care Med · September 1, 2023
Rationale: Idiopathic pulmonary fibrosis (IPF) is a progressive lung disease for which novel therapies are needed. External controls (ECs) could enhance IPF trial efficiency, but the direct comparability of ECs versus concurrent controls is unknown. Object ...
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Journal ArticleBMC Med Res Methodol · June 26, 2023
BACKGROUNDS: Meta-analyses can be a powerful tool but need to calibrate potential unrepresentativeness of the included trials to a target population. Estimating target population average treatment effects (TATE) in meta-analyses is important to understand ...
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Journal ArticleBMJ Open · June 16, 2023
OBJECTIVE: We studied the safety and efficacy of hydroxychloroquine (HCQ) as pre-exposure prophylaxis for COVID-19 in healthcare workers (HCWs), using a meta-analysis of randomised controlled trials (RCTs). DATA SOURCES: PubMed and EMBASE databases were se ...
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Journal ArticleCirculation · June 6, 2023
BACKGROUND: There is uncertainty surrounding the use of direct oral anticoagulants (DOACs) in patients with kidney dysfunction. METHODS: Using the COMBINE AF (A Collaboration Between Multiple Institutions to Better Investigate Non-Vitamin K Antagonist Oral ...
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Journal ArticlePediatr Infect Dis J · May 1, 2023
BACKGROUND: Racial inequities influence health outcomes in the United States, but their impact on sepsis outcomes among children is understudied. We aimed to evaluate for racial inequities in sepsis mortality using a nationally representative sample of ped ...
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Journal ArticleBMC Med · March 29, 2023
BACKGROUND: Studies included in a meta-analysis are often heterogeneous. The traditional random-effects models assume their true effects to follow a normal distribution, while it is unclear if this critical assumption is practical. Violations of this betwe ...
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Journal ArticleJ Allergy Clin Immunol · March 2023
BACKGROUND: It is unclear how the efficacy of tezepelumab, approved for the treatment of type 2 high and low asthma, compares to the efficacy of other biologics for type 2-high asthma. OBJECTIVES: We sought to conduct an indirect comparison of tezepelumab ...
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Journal ArticleOtol Neurotol · March 1, 2023
OBJECTIVE: Comprehensively analyze tumor control and treatment complications for jugular paraganglioma patients undergoing surgery versus stereotactic radiosurgery (SRS). DATABASES REVIEWED: EMBASE, Medline, and Scopus. METHODS: The databases were searched ...
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Journal ArticleCommun Stat Theory Methods · 2023
When effect modifiers influence the decision to participate in randomized trials, generalizing causal effect estimates to an external target population requires the knowledge of two scores - the propensity score for receiving treatment and the sampling sco ...
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Journal ArticleEpidemiol Rev · December 21, 2022
In clinical trials, harms (i.e., adverse events) are often reported by simply counting the number of people who experienced each event. Reporting only frequencies ignores other dimensions of the data that are important for stakeholders, including severity, ...
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Journal ArticleJ Allergy Clin Immunol · November 2022
BACKGROUND: The comparative safety and efficacy of the biologics currently approved for asthma are unclear. OBJECTIVE: We compared the safety and efficacy of mepolizumab, benralizumab, and dupilumab in individuals with severe eosinophilic asthma. METHODS: ...
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Journal ArticleClin Trials · October 2022
BACKGROUND/AIM: The number of coronavirus disease 2019 deaths and cases continues to increase globally. Novel therapies are urgently needed to treat patients with coronavirus disease 2019. We sought to provide a critical review of trials designed during th ...
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ConferenceValue Health · May 2022
OBJECTIVES: To assess the cost-effectiveness of systemic treatments for metastatic castration-sensitive prostate cancer from the US healthcare sector perspective with a lifetime horizon. METHODS: We built a partitioned survival model based on a network met ...
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Journal ArticleJ Natl Cancer Inst · February 7, 2022
BACKGROUND: For nonmetastatic castration-resistant prostate cancer (nmCRPC), 3 drugs under patent protection-apalutamide, enzalutamide, and darolutamide-were approved based on randomized, placebo-controlled trials; 1 drug with generic availability, abirate ...
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Journal ArticleCirculation · January 25, 2022
BACKGROUND: Direct oral anticoagulants (DOACs) are preferred over warfarin for stroke prevention in atrial fibrillation. Meta-analyses using individual patient data offer substantial advantages over study-level data. METHODS: We used individual patient dat ...
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Journal ArticleCommun Stat Simul Comput · 2022
Policymakers use results from randomized controlled trials to inform decisions about whether to implement treatments in target populations. Various methods - including inverse probability weighting, outcome modeling, and Targeted Maximum Likelihood Estimat ...
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Journal ArticleClin Infect Dis · July 15, 2021
BACKGROUND: Globally, pneumonia is the leading cause of death among children. Few data exist regarding the effect of Haemophilus influenzae type b (Hib) vaccine and 13-valent pneumococcal conjugate vaccine (PCV-13) on the burden of childhood pneumonia in A ...
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Journal ArticleBMJ Open · June 30, 2021
INTRODUCTION: Gabapentin (Neurontin) is prescribed widely for conditions for which it has not been approved by regulators, including certain neuropathic pain conditions. There is limited evidence that gabapentin is safe and effective for the treatment of n ...
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Journal ArticleEpidemiology · May 1, 2021
Generalizability methods are increasingly used to make inferences about the effect of interventions in target populations using a study sample. Most existing methods to generalize effects from sample to population rely on the assumption that subgroup-speci ...
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Journal ArticleAm Heart J · March 2021
BACKGROUND: Non-vitamin K antagonist oral anticoagulants (NOACs) are the preferred class of medications for prevention of stroke and systemic embolism in patients with atrial fibrillation unless contraindications exist. Five large, international, randomize ...
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Journal ArticleJAMA Oncol · March 1, 2021
IMPORTANCE: Multiple systemic treatments are available for metastatic castration-sensitive prostate cancer (mCSPC), with unclear comparative effectiveness and safety and widely varied costs. OBJECTIVE: To compare the effectiveness and safety determined in ...
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Journal ArticleClin Trials · February 2021
BACKGROUND/AIMS: Regulatory approval of a drug or device involves an assessment of not only the benefits but also the risks of adverse events associated with the therapeutic agent. Although randomized controlled trials (RCTs) are the gold standard for eval ...
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Journal ArticleIndian Heart J · 2021
BACKGROUND: The initial enthusiasm for thrombectomy during percutaneous coronary intervention (PCI) of ST-elevation myocardial infarction (STEMI) patients has given way to restraint. There has been some limited interest whether it is beneficial in a few se ...
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Journal ArticleOpen Forum Infect Dis · November 2020
BACKGROUND: Bloodstream infections (BSIs) occur frequently after hematopoietic stem cell transplantation (HSCT). We examined the microbiology of BSI in pediatric HSCT recipients over a 2-decade period at our institution to inform empirical antimicrobial pr ...
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Journal ArticleJAMA Cardiol · May 1, 2020
IMPORTANCE: Antithrombotic treatment in patients with atrial fibrillation (AF) and percutaneous coronary intervention (PCI) presents a balancing act with regard to bleeding and ischemic risks. OBJECTIVES: To evaluate the safety and efficacy of 4 antithromb ...
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Journal ArticleTrials · September 5, 2019
BACKGROUND: Adverse events (AEs) in clinical trials may be reported in multiple sources. Different methods for reporting adverse events across trials or across sources for a single trial may produce inconsistent information about the adverse events associa ...
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Journal ArticleJ Clin Epidemiol · September 2019
OBJECTIVE: We examined nonsystematic adverse events (AEs) in Part 2 (of 2) of a study describing the assessment and reporting AEs in clinical trials. STUDY DESIGN AND SETTING: We examined 21 trials of gabapentin for neuropathic pain (52 sources) and seven ...
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Journal ArticleJ Clin Epidemiol · September 2019
OBJECTIVES: We examined systematic adverse events (AEs) in Part 1 (of 2) of a study describing the assessment and reporting of AEs in clinical trials. STUDY DESIGN AND SETTING: We examined 52 public and nonpublic data sources about trials of quetiapine for ...
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Journal ArticleJAMA Cardiol · August 1, 2019
IMPORTANCE: The antithrombotic treatment of patients with atrial fibrillation (AF) and coronary artery disease, in particular with acute coronary syndrome (ACS) and/or percutaneous coronary intervention (PCI), poses a significant treatment dilemma in clini ...
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Journal ArticleAnn Plast Surg · August 2019
BACKGROUND: Keloid disease treatment continues to be unsatisfactory with high recurrence rates. We evaluated the literature regarding the effectiveness of keloid excision with various adjuvant treatments following surgery and assessed recurrence rates. MET ...
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Journal ArticleAm J Epidemiol · January 1, 2019
Propensity score methods are an important tool to help reduce confounding in nonexperimental studies. Most propensity score methods assume that covariates are measured without error. However, covariates are often measured with error, which leads to biased ...
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Journal ArticleInt J Methods Psychiatr Res · December 2018
OBJECTIVES: Few head-to-head comparisons of cocaine dependence medications exist, and combining data from different randomized controlled trials (RCTs) is fraught with methodological challenges including limited generalizability of the RCT findings. This s ...
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Journal ArticleTrials · September 17, 2018
Clinical trials and systematic reviews of clinical trials inform healthcare decisions. There is growing concern, however, about results from clinical trials that cannot be reproduced. Reasons for nonreproducibility include that outcomes are defined in mult ...
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Journal ArticleJournal of the Royal Statistical Society Series C Applied Statistics · August 1, 2018
In network meta-analysis, it is often desirable to synthesize different types of studies, featuring aggregated data and individual patient level data. However, existing methods do not sufficiently consider the quality of studies across different types of d ...
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Journal ArticleSyst Rev · March 19, 2018
The correct title of the article [1] should be "Integrating multiple data sources (MUDS) for meta-analysis to improve patient-centered outcomes research: a protocol". ...
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Journal ArticleAlzheimers Res Ther · February 16, 2018
The correct title of the article [1] should be "Integrating multiple data sources (MUDS) for meta-analysis to improve patient-centered outcomes research: a protocol". The article is a protocol for a methodological study, not a systematic review. ...
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Journal ArticlePsychometrika · December 2017
Propensity score methods are an important tool to help reduce confounding in non-experimental studies and produce more accurate causal effect estimates. Most propensity score methods assume that covariates are measured without error. However, covariates ar ...
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Journal ArticleJ Clin Epidemiol · November 2017
OBJECTIVES: The objective of this study was to determine whether disagreements among multiple data sources affect systematic reviews of randomized clinical trials (RCTs). STUDY DESIGN AND SETTING: Eligible RCTs examined gabapentin for neuropathic pain and ...
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Journal ArticleStat Methods Med Res · October 2017
Network meta-analysis expands the scope of a conventional pairwise meta-analysis to simultaneously compare multiple treatments, synthesizing both direct and indirect information and thus strengthening inference. Since most of trials only compare two treatm ...
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Journal ArticleJ Clin Epidemiol · June 2017
OBJECTIVE: To identify variations in outcomes and results across reports of randomized clinical trials (RCTs). STUDY DESIGN AND SETTING: Eligible RCTs examined gabapentin for neuropathic pain and quetiapine for bipolar depression, reported in public (e.g., ...
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Chapter · January 1, 2017
Network meta-analysis (NMA) is a statistical technique to assess various treatment effects and compare their benefits or harms simultaneously in a systematic review. In comparative effectiveness research, NMA offers useful information to help patients and ...
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Journal ArticleRes Synth Methods · March 2016
Bayesian statistical approaches to mixed treatment comparisons (MTCs) are becoming more popular because of their flexibility and interpretability. Many randomized clinical trials report multiple outcomes with possible inherent correlations. Moreover, MTC d ...
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Journal ArticleOphthalmology · January 2016
TOPIC: Primary open-angle glaucoma (POAG) is a highly prevalent condition worldwide and the most common cause of irreversible sight loss. The objective is to assess the comparative effectiveness of first-line medical treatments in patients with POAG or ocu ...
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Journal ArticleSyst Rev · November 2, 2015
BACKGROUND: Systematic reviews should provide trustworthy guidance to decision-makers, but their credibility is challenged by the selective reporting of trial results and outcomes. Some trials are not published, and even among clinical trials that are publ ...
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Journal ArticleStat Med · September 10, 2015
Availability of individual patient-level data (IPD) broadens the scope of network meta-analysis (NMA) and enables us to incorporate patient-level information. Although IPD is a potential gold mine in biomedical areas, methodological development has been sl ...
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Journal ArticleRes Aging · August 2015
Improving quality of nursing homes (NHs) is a major social priority, yet few studies examine the role of facility characteristics for residents' quality of life (QOL). This study goes beyond cross-sectional analyses by examining the predictors of NH reside ...
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Journal ArticlePharm Stat · 2014
The Drug Information Association Bayesian Scientific Working Group (BSWG) was formed in 2011 with a vision to ensure that Bayesian methods are well understood and broadly utilized for design and analysis and throughout the medical product development proce ...
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Chapter · January 1, 2014
Meta-analysis is a statistical technique that compares the effectiveness or safety of two treatments by incorporating the findings from several independent studies (DerSimonian and Laird, 1986). Network meta-analysis (NMA), also sometimes referred to as mu ...
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Journal ArticleMed Decis Making · July 2013
OBJECTIVES: Bayesian statistical methods are increasingly popular as a tool for meta-analysis of clinical trial data involving both direct and indirect treatment comparisons. However, appropriate selection of prior distributions for unknown model parameter ...
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Journal ArticleAIDS · July 31, 2011
OBJECTIVE: To compare incidence and distribution of non-AIDS-defining events (NADEs) among HIV-1-infected adults receiving combination antiretroviral therapy (cART) in urban sub-Saharan African versus United States settings. DESIGN: Retrospective cohort an ...
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