Article
Multidisciplinary Sciences
Jean-Pierre Boissel, David Perol, Herve Decousus, Ingrid Klingmann, Marc Hommel
Summary: This research aims to reduce ethical losses in randomized controlled clinical trials by introducing the concept of unnecessary participants and increasing the proportion of responders, which leads to a decrease in unnecessary participants and a more ethical trial.
Article
Biology
Nora Hutchinson, Hannah Moyer, Deborah A. Zarin, Jonathan Kimmelman
Summary: This article describes the importance of informativeness in guiding clinical, policy, or research decisions. The study found that the majority of randomized interventional trials designed to guide clinical practice possess features that may compromise their ability to do so.
News Item
Biochemistry & Molecular Biology
Sofia Moutinho
Summary: Risk-based screening approaches can decrease excessive detection, while self-testing and new diagnostics can detect diseases at an early stage.
Article
Obstetrics & Gynecology
Justin W. J. Lim, Kar Y. Lor, Rohan D'Souza
Summary: This study aimed to develop a tool for assessing the breadth and completeness of outcomes measured in pregnancy trials. Results showed a lack of comprehensiveness in reported outcomes in studies on obesity in pregnant patients. The COR Tool exhibited only a weak correlation with Cochrane's RoB 2.0 Tool.
ACTA OBSTETRICIA ET GYNECOLOGICA SCANDINAVICA
(2021)
Article
Health Care Sciences & Services
Mira G. P. Zuidgeest, Iris Goetz, Anna-Katharina Meinecke, Daniel Boateng, Elaine A. Irving, Ghislaine J. M. van Thiel, Paco M. J. Welsing, Katrien Oude-Rengerink, Diederick E. Grobbee
Summary: Methodologies incorporating Real World Elements into clinical trial design, also known as pragmatic trials, provide an attractive opportunity to assess treatment strategies in routine care and guide decision making. However, the adoption of these methods has been slow due to uncertainties about the acceptability of trial results, lack of experience with the methodology, and operational challenges. To address these issues, we developed the GetReal Trial Tool, an online interface that allows users to assess the impact of design choices on generalizability to routine clinical practice while considering factors such as bias, precision, acceptability, and feasibility. The tool is based on scientific literature and input from experts in various fields, aiming to help researchers optimize trial design and facilitate the translation of evidence from pragmatic trials to clinical practice.
JOURNAL OF CLINICAL EPIDEMIOLOGY
(2022)
Article
Oncology
Talha Qaiser, Ching-Yi Lee, Michel Vandenberghe, Joe Yeh, Marios A. Gavrielides, Jason Hipp, Marietta Scott, Joachim Reischl
Summary: Understanding factors that impact prognosis is important for cancer treatment decisions. This study investigates the use of AI and digital pathology to predict overall survival in lung and bladder urothelial carcinoma, showing promising results.
NPJ PRECISION ONCOLOGY
(2022)
Article
Gastroenterology & Hepatology
Elisa Pose, Abiguei Torrents, Enric Reverter, Valeria Perez-Campuzano, Isabel Campos-Varela, Emma Avitabile, Jordi Gratacos-Gines, Jose Castellote, Lluis Castells, Jordi Colmenero, Jaume Tort, Pere Gines, Gonzalo Crespo
Summary: This study found that approximately 9% of patients with alcohol-related decompensated cirrhosis can be delisted due to improvement. Predictors for delisting after improvement include low MELD score, high platelet levels, and higher probabilities for women. Long-term outcomes after delisting are generally favorable.
JOURNAL OF HEPATOLOGY
(2021)
Article
Mathematical & Computational Biology
Victor B. Talisa, Chung-Chou H. Chang
Summary: The study suggests that in clinical trials, identifying a subpopulation with estimated treatment effects that are beneficial and enrolling future study subjects from this subpopulation can improve the chances of success in follow-up trials.
STATISTICS IN MEDICINE
(2021)
Article
Clinical Neurology
Jeffrey T. Guptill, Michael Benatar, Volkan Granit, Ali A. Habib, James F. Howard, Carolina Barnett-Tapia, Richard J. Nowak, Ikjae Lee, Katherine Ruzhansky, Mazen M. Dimachkie, Gary R. Cutter, Henry J. Kaminski
Summary: The lack of standardization in outcome measures in myasthenia gravis clinical trials has led to confusion and variability in data. A group of experts summarized key outcome measures and made recommendations for improvements. Changes to outcome measure instructions and specific instruments were proposed, with a focus on the Quantitative MG score requiring the most attention. Training materials and revised documents will be made available to study teams.
Article
Cell Biology
Mehdi Ghasemi, Charles P. Emerson, Lawrence J. Hayward
Summary: Facioscapulohumeral muscular dystrophy (FSHD) is a debilitating disease with variable age of onset, severity, and progression. Research progress in understanding the underlying mechanisms and developing novel therapies targeting DUX4 expression has accelerated in recent years, providing hope for future clinical trials to improve patient outcomes.
News Item
Multidisciplinary Sciences
Heidi Ledford
Summary: Base editing is being tested in CAR-T-cell treatments for leukaemia, allowing specific modifications to a cell's genome.
Article
Health Care Sciences & Services
Alexander G. Mathioudakis, Markus Fally, Jan Hansel, Rebecca C. Robey, Faiuna Haseeb, Thomas Williams, Ahmed Kouta, Tobias Welte, Dan G. Wootton, Mike Clarke, Grant Waterer, Paul Dark, Paula R. Williamson, Jorgen Vestbo, Timothy W. Felton
Summary: This review identifies significant variations in outcomes and measurement instruments reported in clinical trials of pneumonia management. Important outcomes are often omitted. A rigorous core outcome set is urgently needed to address this issue.
JOURNAL OF CLINICAL EPIDEMIOLOGY
(2023)
Article
Psychology, Multidisciplinary
Kevin J. Munro, William M. Whitmer, Antje Heinrich
Summary: Clinical trials are crucial for evaluating interventions in preventing, diagnosing, or treating health conditions, with a focus on improving practice in healthcare. The lack of high-quality evidence in hearing health practice highlights the importance of understanding key elements defining trial quality, such as design, blinding, and outcome measures. The complexity of outcome measures within hearing health emphasizes the need to consider how measurement instruments impact interpretation, accuracy estimation, and the meaningfulness of differences to individuals with hearing loss.
FRONTIERS IN PSYCHOLOGY
(2021)
Article
Oncology
Jerold Loh, Jiaxuan Wu, Jenny Chieng, Aurora Chan, Wei-Peng Yong, Raghav Sundar, Soo-Chin Lee, Andrea Wong, Joline S. J. Lim, David S. P. Tan, Ross Soo, Boon-Cher Goh, Bee-Choo Tai, Cheng E. Chee
Summary: Patient selection is crucial in Phase I studies, particularly in heavily pre-treated patients where prognosis estimation is challenging. Existing prognostic models like the Royal Marsden Hospital (RMH) score or the neutrophil-lymphocyte ratio (NLR) have not been validated in current novel therapies or in the Asian Phase I population.
BRITISH JOURNAL OF CANCER
(2023)
Article
Medicine, General & Internal
Hui Zhou, Mi Yao, Xiaodan Gu, Mingrui Liu, Ruifeng Zeng, Qin Li, Tingjia Chen, Wen He, Xiao Chen, Gang Yuan
Summary: This study evaluated the application and characteristics of patient-reported outcome (PRO) instruments as primary and secondary outcomes in randomized clinical trials in China. The results showed an increase in the use of PROs in clinical trials conducted in China. However, patient opinion is still rarely measured. The use of PROs is geographically unevenly distributed. The development of PRO instruments, especially those suitable for the Chinese population, may be beneficial. Further expansion of PROs with respect to the scope of diseases is needed to avoid missing important data.
Article
Biochemistry & Molecular Biology
Henry R. Kranzler, Richard Feinn, Timothy Pond, Emily Hartwell, Joel Gelernter, Richard C. Crist, Katie Witkiewitz
Summary: Topiramate reduces drinking and alcohol-related problems during treatment, but the effects diminish significantly after discontinuation. The rs2832407, a single nucleotide polymorphism (SNP) in the GRIK1 gene, does not significantly moderate the treatment outcomes.
Article
Biochemistry & Molecular Biology
Emily E. Hartwell, Alison K. Merikangas, Shefali S. Verma, Marylyn D. Ritchie, Henry R. Kranzler, Rachel L. Kember
Summary: This study investigated the relationship between SUD PRS and primary phenotypes, finding associations but not diagnostic predictability. PheWAS analysis revealed cross-trait associations in psychiatric disorders and medical conditions.
Article
Substance Abuse
Chamindi Seneviratne, David A. Gorelick, Kevin G. Lynch, Clayton Brown, Danielle Romer, Timothy Pond, Kyle Kampman, Henry R. Kranzler
Summary: This study aimed to test the efficacy of ondansetron in treating alcohol use disorder (AUD) and explore whether genotype influences treatment response. The results showed that ondansetron did not significantly reduce drinking intensity, and genotype did not have a significant impact on treatment response.
ALCOHOLISM-CLINICAL AND EXPERIMENTAL RESEARCH
(2022)
Article
Substance Abuse
Victoria R. R. Votaw, Katie Witkiewitz, M. Lee Van Horn, Richard C. C. Crist, Timothy Pond, Henry R. R. Kranzler
Summary: This study aimed to investigate whether a single-nucleotide polymorphism (rs2832407) in GRIK1 moderates the effects of topiramate treatment for drinking reduction. The results showed that topiramate treatment was effective in reducing daily heavy drinking and desire to drink, and this effect was not influenced by the rs2832407*C-allele homozygotes.
Editorial Material
Substance Abuse
Henry R. R. Kranzler
ALCOHOLISM-CLINICAL AND EXPERIMENTAL RESEARCH
(2023)
Article
Substance Abuse
Henry R. R. Kranzler, Richard Feinn, Heng Xu, Brendan L. L. Ho, Divya Saini, Olivia R. R. Nicastro, Anya Jacoby, Sylvanus Toikumo, Joel Gelernter, Emily E. E. Hartwell, Rachel L. L. Kember
Summary: This study investigated the impact of genetic risk for substance use disorders (SUDs) on the onset and progression of substance use. The results showed that European-ancestry individuals and males had an earlier onset and quicker progression of substance use, and the polygenic risk scores (PRSs) had predictive value for substance use milestones and symptom progression. In African-ancestry individuals, PRSs also predicted some substance use milestones, albeit to a lesser extent due to a smaller discovery sample.
Article
Substance Abuse
Heng Xu, Sylvanus Toikumo, Richard C. Crist, Klaudia Glogowska, Zeal Jinwala, Joseph D. Deak, Amy C. Justice, Joel Gelernter, Emma C. Johnson, Henry R. Kranzler, Rachel L. Kember
Summary: This study used multi-trait analysis to identify novel loci associated with substance use disorders in European and African populations. The results showed that multi-trait analysis can improve the genetic modeling of substance use traits and uncover genes that were not previously linked to substance use. Additionally, it enhanced the effectiveness of polygenic risk scores.
Article
Substance Abuse
Michelle S. S. Naps, Shirley H. H. Leong, Emily E. E. Hartwell, Christopher T. T. Rentsch, Henry R. R. Kranzler
Summary: This study investigated the effects of topiramate, a medication used to treat alcohol use disorder, on serum bicarbonate concentrations. The study found that the effects of the medication on bicarbonate concentrations were similar regardless of the presence of alcohol use disorder or the dosage of topiramate. Therefore, it is recommended to monitor baseline and periodic serum bicarbonate concentrations during topiramate therapy.
ALCOHOL-CLINICAL AND EXPERIMENTAL RESEARCH
(2023)
Review
Substance Abuse
Henry R. Kranzler, Emily E. Hartwell
Summary: Chronic heavy alcohol use has negative impacts on neurotransmitter systems and causes various medical, psychiatric, and social problems. Evidence-based medications for treating alcohol use disorder (AUD) are not widely used in clinical practice. Pharmacogenetic approaches have gained interest but have not yet yielded strong enough results for routine clinical care.
ALCOHOL-CLINICAL AND EXPERIMENTAL RESEARCH
(2023)
Article
Psychiatry
Youshu Cheng, Cecilia Dao, Hang Zhou, Boyang Li, Rachel L. Kember, Sylvanus Toikumo, Hongyu Zhao, Joel Gelernter, Henry R. Kranzler, Amy C. Justice, Ke Xu
Summary: Smoking behaviors and alcohol use disorder commonly co-occur and are moderately heritable. Previous single-trait GWAS studies have identified multiple loci for smoking and AUD. However, limited by small samples, GWASs aiming to identify loci contributing to co-occurring smoking and AUD have been less informative. Using multi-trait analysis of GWASs (MTAG) with data from the Million Veteran Program, this study identified novel loci associated with smoking initiation and cessation. Functional annotation highlighted biologically important regions contributing to smoking behaviors. MTAG results did not enhance discovery for smoking behaviors and alcohol consumption compared to single-trait GWAS. This study provides new insights into the pleiotropic effects of genetic variants on smoking behavior and AUD through the application of MTAG.
TRANSLATIONAL PSYCHIATRY
(2023)
Article
Medicine, General & Internal
Thomas. J. J. Schnitzer, Rebecca. L. L. Robinson, Lars Viktrup, Joseph. C. C. Cappelleri, Andrew. G. G. Bushmakin, Leslie Tive, Mia Berry, Chloe Walker, James Jackson
Summary: Patients taking opioids for osteoarthritis pain had more prior treatments, higher pain intensity, and worse quality of life compared to those taking nonopioid medications. Adjusting for demographics and pain intensity, patients prescribed opioids were less satisfied with their overall regimen, had lower belief in medication effectiveness, and had more concerns about treatment effectiveness and addiction compared to patients prescribed nonopioid regimens. These findings were consistent even when analyzing patients with a medication regimen duration of at least 30 days.
JOURNAL OF CLINICAL MEDICINE
(2023)
Review
Substance Abuse
Juliana I. Byanyima, Xinyi Li, Sianneh A. Vesslee, Henry R. Kranzler, Zhenhao Shi, Corinde E. Wiers
Summary: Opioid use disorder (OUD) is a chronic condition with serious adverse consequences, including high incarceration rates and overdose-related fatalities. Medications used for OUD treatment have been shown to cause metabolic impairments, but the effects vary depending on the individual's metabolic condition. Further research is needed to determine the best approach for reducing the risk of metabolic disorders in OUD treatment.
CURRENT ADDICTION REPORTS
(2023)
Article
Economics
Stuart Silverman, Elizabeth Packnett, Anthony Zagar, Sheena Thakkar, Patricia Schepman, Warachal Faison, Cory Hultman, Nicole M. Zimmerman, Rebecca L. Robinson
Summary: Higher healthcare costs were observed in Black Medicaid patients with knee/hip osteoarthritis despite lower rates of inpatient admission. These increased costs cannot be attributed to either longer or more frequent hospitalizations; no significant difference in either the length of stay or the number of hospitalizations was observed when comparing Black patients to White patients.
JOURNAL OF MEDICAL ECONOMICS
(2023)
Article
Psychology, Clinical
Zachary L. Mannes, Ofir Livne, Justin Knox, Deborah S. Hasin, Henry R. Kranzler
Summary: This study aims to examine the prevalence and clinical correlates of opioid withdrawal syndrome (OWS) among adults in the U.S. who engage in non-medical use of prescription opioids (NMOU). Results revealed that OWS is prevalent among individuals with NMOU and is associated with psychiatric disorders and opioid use disorder (OUD).
AMERICAN JOURNAL OF DRUG AND ALCOHOL ABUSE
(2023)
Article
Substance Abuse
Carrie M. Mintz, Justin Knox, Sarah M. Hartz, Deborah S. Hasin, Silvia S. Martins, Henry R. Kranzler, Emily Greene, Elvin H. Geng, Richard A. Grucza, Laura J. Bierut
Summary: This study analyzed demographic disparities in the implementation of SBIRT for unhealthy alcohol use. The results showed that gender, age, race, and insurance status were factors that influenced the likelihood of screening and intervention. Addressing these disparities and reducing attrition from treatment could improve outcomes for individuals with unhealthy alcohol use.
ALCOHOL-CLINICAL AND EXPERIMENTAL RESEARCH
(2023)