Review
Endocrinology & Metabolism
Jan W. Eriksson, Bjorn Eliasson, Louise Bennet, Johan Sundstrom
Summary: This narrative review introduces a new approach called registry-based randomised clinical trials (RRCTs) for navigation in the challenging landscape of clinical drug development in diabetes. The decline in the number of clinical trials in diabetes is attributed to various environmental factors, and RRCTs offer a cost-effective and scientifically sound solution for conducting large-scale diabetes trials in real-world settings.
Review
Medicine, General & Internal
Miriam Ortiz, Anna Katharina Koch, Holger Cramer, Klaus Linde, Gabriele Rotter, Michael Teut, Benno Brinkhaus, Heidemarie Haller
Summary: This study aimed to systematically review the clinical effects of hydrotherapy according to Kneipp. The results suggested that hydrotherapy showed positive effects in some conditions and outcomes, but the high risk of bias and heterogeneity of the included studies made it difficult to ascertain the treatment effects. Higher-quality studies on Kneipp hydrotherapy are urgently needed.
Review
Cardiac & Cardiovascular Systems
Kevin C. Harris, Andrew S. Mackie, Frederic Dallaire, Michael Khoury, Joel Singer, William T. Mahle, Terry P. Klassen, Brian W. McCrindle
Summary: Pediatric cardiology has evolved with time, with an increasing recognition of the importance of randomised controlled trials. While the number of impactful trials has increased, there remains a relative scarcity of such trials in the field.
CANADIAN JOURNAL OF CARDIOLOGY
(2021)
Article
Immunology
Nobuhiro Asai, Daisuke Sakanashi, Hiroyuki Suematsu, Hideo Kato, Mao Hagihara, Arufumi Shiota, Yusuke Koizumi, Yuka Yamagishi, Hiroshige Mikamo
Summary: This study reviewed all candidemia patients in the institute from 2014 to 2018 and found that only 24% of the patients were eligible for clinical trials. Compared to those who were not eligible, the eligible patients had better performance status, fewer comorbidities and longer overall survival times. This suggests that clinical trials may not accurately reflect the real world among candidemia patients.
JOURNAL OF MICROBIOLOGY IMMUNOLOGY AND INFECTION
(2022)
Article
Medicine, General & Internal
Stuart McLennan, Barbara Nussbaumer-Streit, Lars G. Hemkens, Matthias Briel
Summary: In this study, there is generally a consensus among Swiss stakeholders and international funders that systematic evidence assessments are important in justifying new clinical trials. However, barriers at individual, organizational, and political levels hinder the implementation of this practice. More explicit requirements from funders are needed to clarify the level of comprehensiveness required in summarizing existing evidence for different types of clinical trials.
Article
Gastroenterology & Hepatology
Jordi Bruix
Summary: Clinical trials rely on strict definitions and criteria, but real-world clinical practice may not follow the same guidelines, leading to discrepancies in patient selection, disease assessment, and treatment decisions. Understanding these differences is crucial in refining criteria for drug activity, toxicity, and treatment failure in order to achieve evidence-based clinical practice and precision oncology.
JOURNAL OF HEPATOLOGY
(2021)
Review
Oncology
Malia Alexandra Foo, Mingliang You, Shing Leng Chan, Gautam Sethi, Glenn K. Bonney, Wei-Peng Yong, Edward Kai-Hua Chow, Eliza Li Shan Fong, Lingzhi Wang, Boon-Cher Goh
Summary: Multiple three-dimensional tumour organoid models assisted by multi-omics and AI have contributed to preclinical drug development and precision medicine. However, there are challenges in translating these models to clinical applications. This review identifies potential strategies to overcome these limitations.
BIOMARKER RESEARCH
(2022)
Article
Medicine, General & Internal
Mathias Luhr Hansen, Caroline Kamp Jorgensen, Lehana Thabane, Eliana Rulli, Elena Biagioli, Maria Chiaruttini, Lawrence Mbuagbaw, Ole Mathiesen, Christian Gluud, Janus Christian Jakobsen
Summary: This study aims to estimate the range of observed intervention effects for all-cause mortality in trials included in Cochrane Reviews. Cochrane Reviews will be clustered according to the registered Cochrane Review Group, and statistical analyses will be conducted for each group and overall. The results will report the median relative risk, IQR, and the proportion of trials within different ranges of relative risk.
Review
Obstetrics & Gynecology
Giulia Borghese, Antonio Raffone, Diego Raimondo, Gabriele Saccone, Antonio Travaglino, Eugenia Degli Esposti, Manuela Mastronardi, Paolo Salucci, Fulvio Zullo, Renato Seracchioli
Summary: This study evaluated the effectiveness of different adhesion barriers in preventing de novo adhesion development after laparoscopic myomectomy, and found that oxidized regenerated cellulose, auto-crosslinked hyaluronic acid gel, and polyethylene glycol amine plus dextran aldehyde polymers were the most promising methods for adhesion prevention.
INTERNATIONAL JOURNAL OF GYNECOLOGY & OBSTETRICS
(2021)
Review
Oncology
Alexia Iasonos, John O'Quigley
Summary: Phase 1 trials in oncology have expanded their objectives from assessing tolerability to addressing multiple objectives under the heading of early-phase trials. The aim is to obtain reliable evidence of clinical activity for drug approvals. Designing a Phase 1 trial that can address multiple objectives is challenging and requires adherence to ethical and scientific principles.
BRITISH JOURNAL OF CANCER
(2021)
Editorial Material
Biochemistry & Molecular Biology
Calum A. MacRae, Rahul C. Deo, Stanley Y. Shaw
Summary: Despite the increasing ability to understand and correct molecular derangements in disease, genomics and novel phenotypic assays are not uniformly deployed in clinical practice, hindering translational research and identification of clinically actionable sub-types of disease. Historic examples demonstrate how stakeholders' perspectives in the healthcare ecosystem can influence adoption of innovations in healthcare. Considering these factors from discovery to implementation can accelerate adoption of new molecular and digital phenotypes in a 'learning' healthcare ecosystem.
TRENDS IN MOLECULAR MEDICINE
(2021)
Editorial Material
Medicine, General & Internal
Prathit A. Kulkarni, Hardeep Singh
Summary: This article examines the use of generative artificial intelligence in healthcare, specifically its role in assisting clinical diagnoses, and the challenges associated with its use, including ensuring the accuracy of clinical data.
JAMA-JOURNAL OF THE AMERICAN MEDICAL ASSOCIATION
(2023)
Review
Cardiac & Cardiovascular Systems
Bo Liang, Ning Gu
Summary: Traditional Chinese medicine has a long history and is widely used in clinical practice, but its role in the prevention and treatment of coronary artery disease remains unclear due to lack of scientific evidence. Randomized controlled trials have shown that traditional Chinese medicine can be used as a complementary method in managing coronary artery disease, with similar safety profile to conventional medications. Further research with long-term endpoints is needed to fully assess its safety and efficacy.
FRONTIERS IN CARDIOVASCULAR MEDICINE
(2021)
Article
Computer Science, Interdisciplinary Applications
Kabir Soeny, Barbara Bogacka, Byron Jones
Summary: Personalized medicine is crucial in designing therapies for specific patients or patient groups. Therapeutic drug monitoring is used in clinical settings for certain drugs, but may not be applicable for drugs in development due to insufficient pharmacokinetic information. A new methodology presented in this paper optimizes dose regimens to maintain desired blood concentration levels, demonstrating high efficiency and minimal exposure variations for subjects in clinical trials.
COMPUTER METHODS AND PROGRAMS IN BIOMEDICINE
(2021)
Review
Pediatrics
Rian Hayes, Jack Hartnett, Gergana Semova, Cian Murray, Katherine Murphy, Leah Carroll, Helena Plapp, Louise Hession, Jonathan O'Toole, Danielle McCollum, Edna Roche, Elinor Jenkins, David Mockler, Tim Hurley, Matthew McGovern, John Allen, Judith Meehan, Frans B. Plotz, Tobias Strunk, Willem P. de Boode, Richard Polin, James L. Wynn, Marina Degtyareva, Helmut Kuester, Jan Janota, Eric Giannoni, Luregn J. Schlapbach, Fleur M. Keij, Irwin K. M. Reiss, Joseph Bliss, Joyce M. Koenig, Mark A. Turner, Christopher Gale, Eleanor J. Molloy
Summary: Neonatal sepsis, a leading cause of infant mortality, has diverse definitions based on microbiological culture, laboratory tests, and clinical signs. An international consensus-based definition could improve research comparability and clinical outcomes.
PEDIATRIC RESEARCH
(2023)
Article
Infectious Diseases
Paul Loubet, Linda Wittkop, Laetitia Ninove, Mathieu Chalouni, Benoit Barrou, Jean-Yves Blay, Maryvonne Hourmant, Eric Thouvenot, Martine Laville, Bruno Laviolle, Jean-Daniel Lelievre, Jacques Morel, Stephanie Nguyen Quoc, Jean-Philippe Spano, Benjamin Terrier, Anne Thiebaut, Jean-Francois Viallard, Francois Vrtovsnik, Sophie Circosta, Laure Esterle, Axel Levier, Philippe Vanhems, Eric Tartour, Beatrice Parfait, Xavier de Lamballerie, Odile Launay
Summary: This study aimed to investigate the 1-month humoral response to two or three doses of mRNA COVID-19 vaccine in specific populations compared with healthy adults. The results showed a lower humoral response in the specific populations. However, the third dose of the vaccine had a positive effect on the immune response.
CLINICAL MICROBIOLOGY AND INFECTION
(2023)
Review
Critical Care Medicine
Justyna A. Karolak, Carrie L. Welch, Christian Mosimann, Katarzyna Bzdega, James D. West, David Montani, Melanie Eyries, Mary P. Mullen, Steven H. Abman, Matina Prapa, Stefan Graf, Nicholas W. Morrell, Anna R. Hemnes, Frederic Perros, Rizwan Hamid, Malcolm P. O. Logan, Jeffrey Whitsett, Csaba Galambos, Pawel Stankiewicz, Wendy K. Chung, Eric D. Austin
Summary: Over the past decade, the significance of the TBX4 gene in respiratory diseases has been recognized. TBX4 is associated with congenital disorders involving respiratory and skeletal structures, but its exact role in human development is not fully understood. Through human and animal studies, we discuss the developmental, tissue-specific, and pathological functions of TBX4, as well as review the published TBX4 variants that result in different disease phenotypes. We also outline future research directions to enhance our understanding of TBX4 function and the impact of TBX4 disruption on development.
AMERICAN JOURNAL OF RESPIRATORY AND CRITICAL CARE MEDICINE
(2023)
Review
Nutrition & Dietetics
Giorgina Barbara Piccoli, Tommy Cederholm, Carla Maria Avesani, Stephan J. L. Bakker, Vincenzo Bellizzi, Cristina Cuerda, Adamasco Cupisti, Alice Sabatino, Stephane Schneider, Massimo Torreggiani, Denis Fouque, Juan Jesus Carrero, Rocco Barazzoni
Summary: Increased life expectancy presents significant challenges to healthcare systems globally, including the rise in chronic kidney disease (CKD) and malnutrition-protein-energy wasting (PEW) in older adults, leading to poor clinical outcomes. A critical review paper by ESPEN and ERN-ERA aims to summarize key concepts in preventing and treating CKD progression and impaired nutritional status, and provide guidance on defining optimal protein and energy intake in varying CKD severity. Careful assessment and individualized approach are necessary, considering malnutrition-PEW status, kidney function, and patient preferences. Greater awareness and research are needed to improve nutritional care for this growing patient subgroup.
CLINICAL NUTRITION
(2023)
Article
Endocrinology & Metabolism
Benedicte Gaborit, Sara Fernandes, Paul Loubet, Laetitia Ninove, Anne Dutour, Bertrand Cariou, Muriel Coupaye, Karine Clement, Sebastien Czernichow, Claire Carette, Noemie Resseguier, Laure Esterle, Sabrina Kali, Marie Houssays, Xavier de Lamballerie, Linda Wittkop, Odile Launay, Martine Laville
Summary: Patients with diabetes and obesity have weaker immune responses to COVID-19 vaccines, especially one month after the second dose. Post-vaccination serological checks are necessary for these high-risk populations.
METABOLISM-CLINICAL AND EXPERIMENTAL
(2023)
Article
Urology & Nephrology
Maxime Espi, Xavier Charmetant, Cyrille Mathieu, Alexandre Lalande, Didier Decimo, Laetitia Koppe, Caroline Pelletier, Anne Ovize, Alexia Barbry, Emmanuel Morelon, Emilie Kalbacher, Denis Fouque, Olivier Thaunat
KIDNEY INTERNATIONAL REPORTS
(2023)
Article
Cardiac & Cardiovascular Systems
Nathan Mewton, Erwan Donal, Francois Picard, Francois Derimay, Daniel Grinberg, Delphine Maucort Boulch, Thomas Bochaton, Nicolas Piriou, Amelie De Lorgeril, Geraldine Samson, Frederic Rouleau, Benjamin Riche, Jean Noel Trochu
Summary: The study aimed to assess the distribution of primary and secondary heart failure events in a severe heart failure population and their association with 2-year all-cause mortality. The results showed that primary heart failure decompensations were three times more frequent than secondary decompensations, but both types had a similar impact on mortality risk. Patients with only primary or only secondary decompensation had a significantly increased risk of death.
AMERICAN HEART JOURNAL
(2023)
Article
Medicine, Research & Experimental
Viviana Giannuzzi, Lucia Ruggieri, Rosa Conte, Cristina Manfredi, Mariagrazia Felisi, Christine Kubiak, Mihaela Matei, Salma Malik, Jacques Demotes, Adriana Ceci, Donato Bonifazi
Summary: In pediatric clinical research, implementing ethical and regulatory requirements, training, and facilities are crucial for proper management of specimens. The Pediatric Clinical Research Infrastructure Network (PedCRIN) has developed a tool to guide investigators and sponsors in managing specimens and data in compliance with European rules. This tool addresses the specific need for guidance in managing biosamples in pediatric trials.
CTS-CLINICAL AND TRANSLATIONAL SCIENCE
(2023)
Article
Public, Environmental & Occupational Health
Kossi D. Abalo, Sophie Malekzadeh-Milani, Sebastien Hascoet, Serge Dreuil, Tiphaine Feuillet, Cecilia Damon, Helene Bouvaist, Ivan Bouzguenda, Sarah Cohen, Claire Dauphin, Sylvie Di Filippo, Stephanie Douchin, Francois Godart, Patrice Guerin, Pauline Helms, Clement Karsenty, Bruno Lefort, Pierre Mauran, Caroline Ovaert, Jean-Francois Piechaud, Jean-Benoit Thambo, Choonsik Lee, Mark P. Little, Damien Bonnet, Marie-Odile Bernier, Estelle Rage
Summary: Pediatric patients with congenital heart disease who undergo cardiac catheterization (CC) may be exposed to low dose ionizing radiation (LDIR). However, this study found no increased risk of lympho-hematopoietic malignancies and lymphoma associated with LDIR in these patients.
EUROPEAN JOURNAL OF EPIDEMIOLOGY
(2023)
Article
Medicine, Research & Experimental
Isak Mazanti Cold, Joshua Buron Feinberg, Axel Brandes, Ulla Davidsen, Ulrik Dixen, Helena Dominguez, Uffe Jakob Ortved Gang, Christian Gluud, Rakin Hadad, Kit Engedal Kristensen, Doan Tuyet van Le, Emil Eik Nielsen, Michael Hecht Olsen, Ole Dyg Pedersen, Ilan Esra Raymond, Ahmad Sajadieh, Anne Merete Boas Soja, Janus Christian Jakobsen
Summary: This study aims to compare strict rate control with lenient rate control in patients with atrial fibrillation. The primary outcome is the physical component score of the SF-36 questionnaire. All secondary outcomes will be hypothesis-generating. The analysis will be based on the intention-to-treat principle and use linear regression for adjustment.
Article
Gastroenterology & Hepatology
Emma Wischlen, Noemie Laverdure, Domitille Erard, Barbara Rohmer, Olivier Boillot, Remi Dubois, Alain Lachaux, Sophie Collardeau-Frachon, Valerie Hervieu, Jerome Dumortier
Summary: Post-transplantation evolution of progressive familial intrahepatic cholestasis type 2 patients can be complicated by antibody-induced bile salt export pump deficiency (AIBD). There is no consensus on its management. We describe a patient who presented two episodes, 9 years apart. The first episode was refractory to plasmapheresis and intravenous immunoglobulin (IVIG) started 2 months after AIBD onset, leading to graft loss. The second episode responded to plasmapheresis, IVIG and rituximab initiated less than 2 weeks after the beginning of symptoms, allowing for long-term recovery. This case suggests that intensive treatment with minimum delay after symptoms onset could sponsor a better evolution.
CLINICS AND RESEARCH IN HEPATOLOGY AND GASTROENTEROLOGY
(2023)
Editorial Material
Cardiac & Cardiovascular Systems
Thomas Bochaton, Nathan Mewton
EUROPEAN HEART JOURNAL-ACUTE CARDIOVASCULAR CARE
(2023)
Article
Medicine, Research & Experimental
Marie Isabel Rasmussen, Mathias Luhr Hansen, Adelina Pellicer, Christian Gluud, Eugene Dempsey, Jonathan Mintzer, Simon Hyttel-Sorensen, Anne Marie Heuchan, Cornelia Hagmann, Ebru Ergenekon, Gabriel Dimitriou, Gerhard Pichler, Gunnar Naulaers, Guoqiang Cheng, Jakub Tkaczyk, Hans Fuchs, Monica Fumagalli, Saudamini Nesargi, Siv Fredly, Tomasz Szczapa, Anne Mette Plomgaard, Bo Molholm Hansen, Janus Christian Jakobsen, Gorm Greisen
Summary: The SafeBoosC-III follow-up study aims to assess mortality, neurodevelopmental disability, or any harm in trial participants at 2 years of corrected age. The lack of funding for trial-specific assessment local costs is a challenge that needs to be addressed.
Article
Health Care Sciences & Services
Caroline Kamp Jorgensen, Markus Harboe Olsen, Niklas Nielsen, Theis Lange, Lawrence Mbuagbaw, Lehana Thabane, Laurent Billot, Nadine Binder, Silvio Garattini, Rita Banzi, Jacques Demotes, Elena Biagioli, Eliana Rulli, Guido Bertolini, Giovanni Nattino, Ole Mathiesen, Valter Torri, Christian Gluud, Janus Christian Jakobsen
Summary: The Centre for Statistical and Methodological Excellence (CESAME) aims to improve the accuracy and reliability of clinical trial results by developing standardized methods and guidelines. CESAME's work will benefit patients worldwide in various medical specialties by enhancing the internal and external validity of randomized clinical trials.
HEALTH SERVICES INSIGHTS
(2023)
Article
Critical Care Medicine
Pierre-Edouard Bollaert, Alexandra Monnier, Francis Schneider, Laurent Argaud, Julio Badie, Claire Charpentier, Ferhat Meziani, Michel Bemer, Jean-Pierre Quenot, Marie Buzzi, Herve Outin, Cedric Bruel, Laurent Ziegler, Sebastien Gibot, Jean-Marc Virion, Camille Alleyrat, Guillaume Louis, Nelly Agrinier
Summary: The POINCARE-2 trial aimed to assess the effectiveness of a fluid balance control strategy on mortality in critically ill patients. However, the conservative strategy did not reduce mortality in these patients.
