Review
Clinical Neurology
Jon B. Toledo, Carla Abdelnour, Rimona S. Weil, Daniel Ferreira, Federico Rodriguez-Porcel, Andrea Pilotto, Kathryn A. Wyman-Chick, Michel J. Grothe, Joseph P. M. Kane, Angela Taylor, Arvid Rongve, Sonja Scholz, James B. Leverenz, Bradley F. Boeve, Dag Aarsland, Ian G. McKeith, Simon Lewis, Iracema Leroi, John P. Taylor
Summary: Dementia with Le bodies (DLB) is clinically characterized by visual hallucinations, fluctuations, rapid eye movement (REM) sleep behavioral disorder, and parkinsonism. However, neuropathological studies have shown the coexistence of Alzheimer's disease, TAR DNA-binding protein 43 (TDP-43), and cerebrovascular pathologies in DLB cases. These co-pathologies should be taken into consideration in clinical trials for DLB individuals.
ALZHEIMERS & DEMENTIA
(2023)
Editorial Material
Medicine, General & Internal
Michael A. Steinman, Cynthia M. Boyd, Kenneth E. Schmader
Summary: Complementary approaches are necessary to enhance the evidence for informing care for older adults.
JAMA-JOURNAL OF THE AMERICAN MEDICAL ASSOCIATION
(2021)
Article
Multidisciplinary Sciences
Jie Xu, Hao Zhang, Hansi Zhang, Jiang Bian, Fei Wang
Summary: The restrictive eligibility criteria of clinical trials can limit the generalizability of trial results. To address this issue, a novel machine learning approach was developed using real-world data extracted from electronic health records (EHRs) to inform the design of clinical trial eligibility criteria. The approach identified patient subgroups with similar clinical characteristics and similar risk levels of severe adverse events (SAEs). Testing on previous clinical trials, the algorithm successfully identified patient subgroups that were more likely or less likely to experience SAEs, providing data-driven recommendations for refining exclusion criteria.
SCIENTIFIC REPORTS
(2023)
Article
Medicine, General & Internal
Emma Svennberg, Leif Friberg, Viveka Frykman, Faris Al-Khalili, Johan Engdahl, Marten Rosenqvist
Summary: A randomized controlled trial in Sweden showed that systematic screening for atrial fibrillation in older populations can reduce mortality and morbidity compared to no screening.
Article
Neurosciences
Kelly J. Atkins, David A. Scott, Brendan Silbert, Kerryn E. Pike, Lis Evered
Summary: This study aims to conduct a prospective, single-blind, pragmatic, randomized-controlled trial to compare the effects of a novel multidisciplinary perioperative intervention with current standard of care practices. Targeting delirium risk reduction strategies, the intervention emphasizes early mobilization, nutrition, hydration, cognitive orientation, etc., in an effort to reduce the risk of postoperative delirium and associated cognitive decline.
JOURNAL OF ALZHEIMERS DISEASE
(2021)
Article
Neurosciences
Shadi Zarei, Sarah Colman, Aviva Rostas, Amer M. Burhan, Li Chu, Simon J. C. Davies, Peter Derkach, Sarah Elmi, Maria Hussain, Philip Gerretsen, Ariel Graff-Guerrero, Zahinoor Ismail, Donna Kim, Linda Krisman, Rola Moghabghab, Benoit H. Mulsant, Vasavan Nair, Bruce G. Pollock, Soham Rej, Jyll Simmons, Lisa Van Bussel, Tarek K. Rajji, Sanjeev Kumar
Summary: The StaN study aims to compare the effectiveness of an Integrated Care Pathway with treatment-as-usual in treating agitation in dementia patients. Structured behavioral interventions in the study are designed and implemented based on considerations including personalization, evidence base, dose, duration, measurement, and environmental factors.
JOURNAL OF ALZHEIMERS DISEASE
(2022)
Review
Urology & Nephrology
Allison Tong, Nicole Scholes-Robertson, Carmel Hawley, Andrea K. Viecelli, Simon A. Carter, Adeera Levin, Brenda R. Hemmelgarn, Tess Harris, Jonathan C. Craig
Summary: Patient involvement in clinical trial design is crucial for improving participant recruitment and retention, increasing the uptake of interventions, and enhancing the impact of findings on patient outcomes. However, in nephrology, patients have limited involvement in trial design, which can compromise the relevance and reliability of trial evidence.
NATURE REVIEWS NEPHROLOGY
(2022)
Article
Medicine, General & Internal
Abir Ghorayeb, Julie L. Darbyshire, Marta W. Wronikowska, Peter J. Watkinson
Summary: The objective of this study was to develop and validate a questionnaire for assessing the usability of clinical decision support systems (CDSS) and identifying usability issues that may impact patient safety and quality of care. Mixed research methods were used, and the Healthcare Systems Usability Scale (HSUS) was developed, which consists of four subscales related to patient safety, workflow integration, work effectiveness, and user control. The HSUS is a reliable tool for testing CDSS usability and identifying potential usability issues.
Review
Oncology
Anna Spreafico, Aaron R. Hansen, Albiruni R. Abdul Razak, Philippe L. Bedard, Lillian L. Siu
Summary: Clinical trials have evolved from traditional cytotoxic chemotherapy studies to biomarker-driven evaluations, aiming to provide safer and more effective treatments for patients. The future of cancer clinical trials will require a framework that efficiently transforms scientific discoveries into clinical applications, offering personalized treatment strategies and contributing to global knowledge and collective learning through collaboration among all stakeholders.
Article
Medicine, General & Internal
Elisa Neuvonen, Jenni Lehtisalo, Tiia Ngandu, Esko Levaelahti, Riitta Antikainen, Tuomo Hanninen, Tiina Laatikainen, Jaana Lindstrom, Teemu Paajanen, Hilkka Soininen, Timo Strandberg, Jaakko Tuomilehto, Miia Kivipelto, Alina Solomon
Summary: Depressive symptoms may affect improvement in cognitive abilities, with clinically significant symptoms potentially impacting the effectiveness of interventions, warranting further attention in dementia prevention strategies.
JOURNAL OF CLINICAL MEDICINE
(2022)
Review
Oncology
Stephane Champiat, Lambros Tselikas, Siham Farhane, Thibault Raoult, Matthieu Texier, Emilie Lanoy, Christophe Massard, Caroline Robert, Samy Ammari, Thierry De Baere, Aurelien Marabelle
Summary: Intratumoral immunotherapies have shown promising effects by stimulating the immune system through the injection of immune stimulatory products in certain solid tumor types. This approach can transform cold tumors into hot tumors, enhance the response rates to cancer immunotherapies, and reduce side effects, leading to improved long-term survival rates for patients.
CLINICAL CANCER RESEARCH
(2021)
Article
Clinical Neurology
Amy K. Liu, Katherine L. Possin, Kristen M. Cook, Shalini Lynch, Sarah Dulaney, Jennifer J. Merrilees, Tamara Braley, Rachel E. Kiekhofer, Stephen J. Bonasera, Isabel E. Allen, Winston Chiong, Amy M. Clark, Julie Feuer, Joan Ewalt, Elan L. Guterman, Rosalie Gearhart, Bruce L. Miller, Kirby P. Lee
Summary: This study evaluated the effect of the Care Ecosystem (CE) collaborative dementia care program on medication use among community-dwelling persons living with dementia (PLWD). The results showed that the CE program significantly reduced the use of potentially inappropriate medications (PIMs), as well as the total number of medications and specific medication classes.
ALZHEIMERS & DEMENTIA
(2023)
Article
Medical Informatics
Christian Gulden, Romina Blasini, Azadeh Nassirian, Alexandra Stein, Fatma Betul Altun, Melanie Kirchner, Hans-Ulrich Prokosch, Martin Boeker
Summary: This study investigates the use of HL7 FHIR as a standardized format for exchanging and storing clinical trial records, presenting an open-source central trial registry as a prototype. The results demonstrate that FHIR resources facilitate automated data exchange between trial centers and central study registries, establishing a harmonized view of study information from heterogeneous sources.
JMIR MEDICAL INFORMATICS
(2021)
Article
Clinical Neurology
Armando Miranda, Eve Rubovits, Raksha Mudar, Vania Leung, Minakshi Raj
Summary: This study evaluates the responsibilities of family caregivers in ADRD clinical trials and examines how these responsibilities are communicated on clinicaltrials.gov. The results show that less than half of the trial information pages include caregiver responsibilities, such as giving consent, caregiver training, monitoring patient's response, and communicating with the study team. The study suggests that consistently including caregiver responsibilities can better prepare them and facilitate recruitment and retention of diverse participants.
ALZHEIMERS & DEMENTIA
(2023)
Article
Multidisciplinary Sciences
Haoxiang Yang, Ozge Suerer, Daniel Duque, David P. Morton, Bismark Singh, Spencer J. Fox, Remy Pasco, Kelly Pierce, Paul Rathouz, Victoria Valencia, Zhanwei Du, Michael Pignone, Mark E. Escott, Stephen I. Adler, S. Claiborne Johnston, Lauren Ancel Meyers
Summary: Community mitigation strategies to combat COVID-19 can have substantial socioeconomic costs, but judicious implementation and relaxation of restrictions can enhance public health benefits while reducing costs. Establishing triggers for increasing mitigation measures based on daily COVID-19 hospital admissions can ensure sufficient ICU capacity and minimize the duration of strict mitigation measures. The study suggests that tracking COVID-19 hospital admissions and enacting staged measures can help cities worldwide to navigate future pandemic waves effectively.
NATURE COMMUNICATIONS
(2021)
Article
Biology
Kendra Davis-Plourde, Monica Taljaard, Fan Li
Summary: This article presents a method for calculating sample size in SW-CRTs with subclusters, which can properly differentiate between-period and within-period correlation coefficients. It uses an extended block exchangeable correlation matrix to characterize the dependencies of outcomes within clusters and derives a closed-form sample size expression for Gaussian outcomes. For non-Gaussian outcomes, it proposes a generic sample size algorithm based on linearization.
Article
Health Care Sciences & Services
Oluwaseun J. Adeyemi, Jean-Baptiste Bouillon-Minois, Nina Siman, Allison M. Cuthel, Keith S. Goldfeld, Corita R. Grudzen
Summary: This study re-validated an assessment tool for evaluating the knowledge and attitudes of emergency providers towards hospice and palliative care. Three underlying constructs were identified, including self-rated knowledge, support for practice, and views on provider-patient communication.
AMERICAN JOURNAL OF HOSPICE & PALLIATIVE MEDICINE
(2023)
Article
Biology
Siyun Yang, Mirjam Moerbeek, Monica Taljaard, Fan Li
Summary: Pragmatic trials in healthcare interventions often use cluster randomization, but methods for determining sample size and power for continuous coprimary endpoints are lacking. We propose a method based on multivariate linear mixed models to address this gap and demonstrate its effectiveness through simulation studies.
Article
Mathematical & Computational Biology
Fan Li, Xinyuan Chen, Zizhong Tian, Denise Esserman, Patrick J. Heagerty, Rui Wang
Summary: This article explores the heterogeneity of treatment effects in different patient subpopulations in cluster randomized trials and presents a novel analytical design formula that can be widely applied to evaluate effect modifiers at different levels. The effectiveness of this new method is validated through simulation studies and real-world trial examples.
Review
Health Care Sciences & Services
Can Meng, Denise Esserman, Fan Li, Yize Zhao, Ondrej Blaha, Wenhan Lu, Yuxuan Wang, Peter Peduzzi, Erich J. Greene
Summary: Simulation studies are crucial for evaluating the performance of statistical models in analyzing complex survival data. This article provides researchers with a fundamental understanding of generating competing risks data, inducing cluster-level correlation, and combining them in simulation studies.
STATISTICAL METHODS IN MEDICAL RESEARCH
(2023)
Article
Mathematical & Computational Biology
Kendra Davis-Plourde, Monica Taljaard, Fan Li
Summary: In this article, the authors propose computationally efficient power and sample size procedures for stepped wedge cluster randomized trials (SW-CRTs) with multivariate outcomes. They derive the joint distribution of the intervention test statistics under a multivariate linear mixed model and provide an example using the intersection-union test for co-primary outcomes. The authors also prove that the multivariate linear mixed model leads to a more efficient treatment effect estimator compared to the univariate linear mixed model under certain assumptions.
STATISTICS IN MEDICINE
(2023)
Article
Public, Environmental & Occupational Health
Guangyu Tong, Fan Li, Xinyuan Chen, Shashivadan P. Hirani, Stanton P. Newman, Wei Wang, Michael O. Harhay
Summary: Many studies face challenges with clustering and nonmortality outcomes that are truncated due to death. Traditional missing-data methods and causal estimands are not ideal for statistical inference in the presence of these issues. We developed a Bayesian estimator using principal stratification to address these problems and provide a causal interpretation of the treatment effect. Our simulation and reanalysis of a clinical trial showed promising results. Assessing the SACE estimand in studies with clustering and informative truncation can offer valuable insights into a subset of study participants.
AMERICAN JOURNAL OF EPIDEMIOLOGY
(2023)
Article
Public, Environmental & Occupational Health
AnnaMarie S. O'Neill, Jason T. Newsom, Em F. Trubits, Miriam R. Elman, Anda Botoseneanu, Heather G. Allore, Corey L. Nagel, David A. Dorr, Ana R. Quinones
Summary: This study aimed to clarify the number of distinct trajectories of morbidity accumulation and the potential associations between race/ethnicity and socioeconomic status. Three trajectories of morbidity accumulation were identified, with NH Black adults experiencing disadvantages and Hispanic adults having advantages compared to NH White adults. These findings have implications for addressing health disparities through more precise targeting of public health interventions.
SSM-POPULATION HEALTH
(2023)
Article
Mathematical & Computational Biology
Guangyu Tong, Jiaqi Tong, Fan Li
Summary: This paper investigates the design planning of multicenter individually randomized group treatment trials, provides analytical sample size formulas, and finds that considering center-level correlation can reduce sample size.
BIOMETRICAL JOURNAL
(2023)
Article
Medicine, Research & Experimental
Brennan C. Kahan, Fan Li, Bryan Blette, Vipul Jairath, Andrew Copas, Michael Harhay
Summary: This study re-analyzed a cluster-randomized trial and found that estimates from different methods can differ, which may be due to the presence of informative cluster size. Careful consideration and evaluation of the estimand and assumptions underlying each estimator can help ensure the appropriate choice of analysis method.
Article
Computer Science, Interdisciplinary Applications
Can Meng, Mary Ryan, Paul J. Rathouz, Elizabeth L. Turner, John S. Preisser, Fan Li
Summary: This article introduces a newly developed R package ORTH.Ord for analyzing correlated ordinal outcomes using GEE models with finite-sample bias corrections. The package implements a modified version of alternating logistic regressions with estimation based on orthogonalized residuals. The simulation study shows that the ORTH method with bias-correction provides less biased estimates and closer coverage to the nominal level.
COMPUTER METHODS AND PROGRAMS IN BIOMEDICINE
(2023)
Article
Mathematical & Computational Biology
Lara Maleyeff, Rui Wang, Sebastien Haneuse, Fan Li
Summary: This study proposes a method for testing treatment effect heterogeneity in cluster randomized trials. Through a generalized linear mixed model, we derive sample size expressions for binary effect modifiers and develop a computationally efficient Monte Carlo approach for continuous effect modifiers. Our findings contribute to filling the methodological gap in existing research.
STATISTICS IN MEDICINE
(2023)
Article
Rheumatology
Hailey Baker, Rebecca Fine, Fenn Suter, Heather Allore, Betty Hsiao, Vaidehi Chowdhary, Elizabeth Lavelle, Ping Chen, Richard Hintz, Lisa G. Suter, Abhijeet Danve
Summary: This study evaluated the effectiveness of a computerized decision support system in improving infectious disease screening for patients with autoimmune rheumatic disease (ARD). The implementation of the system significantly improved screening rates for tuberculosis, hepatitis B, and hepatitis C, suggesting it can enhance patient safety.
ARTHRITIS CARE & RESEARCH
(2023)
Article
Cardiac & Cardiovascular Systems
Cenjing Zhu, Rachel P. Dreyer, Fan Li, Erica S. Spatz, Cesar Caraballo-Cordovez, Shiwani Mahajan, Valeria Raparelli, Erica C. Leifheit, Yuan Lu, Harlan M. Krumholz, John A. Spertus, Gail D'Onofrio, Louise Pilote, Judith H. Lichtman
Summary: Marital stress is significantly associated with worse health outcomes in young patients with acute myocardial infarction, highlighting the need for routine screening and interventions.
JOURNAL OF THE AMERICAN HEART ASSOCIATION
(2023)
Article
Public, Environmental & Occupational Health
Xueqi Wang, Keith S. Goldfeld, Monica Taljaard, Fan Li
Summary: This article develops formal methods for sample size and power analyses for testing subgroup-specific treatment effects in parallel-arm cluster-randomized trials (CRTs) with a continuous outcome and a binary subgroup variable. The authors point out that the variances of the subgroup-specific treatment effect estimators and their covariance are given by weighted averages of the variance of the overall average treatment effect estimator and the variance of the heterogeneous treatment effect estimator. The proposed sample size methods are validated through a simulation study.
PREVENTION SCIENCE
(2023)
