Article
Oncology
Jennifer K. Litton, Meredith M. Regan, Lajos Pusztai, Hope S. Rugo, Sara M. Tolaney, Elizabeth Garrett-Mayer, Laleh Amiri-Kordestani, Reva K. Basho, Ana F. Best, Jean-Francois Boileau, Carsten Denkert, Jared C. Foster, Nadia Harbeck, Heather A. Jacene, Tari A. King, Ginny Mason, Ciara C. O'Sullivan, Tatiana M. Prowell, Andrea L. Richardson, Karla A. Sepulveda, Mary Lou Smith, Judy A. Tjoe, Gulisa Turashvili, Wendy A. Woodward, Lynn Pearson Butler, Elena I. Schwartz, Larissa A. Korde
Summary: The STEEP criteria, updated as STEEP 2.0 in 2021, provide standardized definitions for adjuvant breast cancer end points. A working group called NeoSTEEP was established to evaluate and align neoadjuvant breast cancer trial end points.
JOURNAL OF CLINICAL ONCOLOGY
(2023)
Article
Clinical Neurology
Jennifer A. Janusz, Bonita P. Klein-Tasman, Jonathan M. Payne, Pamela L. Wolters, Heather L. Thompson, Staci Martin, Peter de Blank, Nicole Ullrich, Allison del Castillo, Maureen Hussey, Kristina K. Hardy, Kristina Haebich, Tena Rosser, Mary Anne Toledo-Tamula, Karin S. Walsh
Summary: The study reviewed parent-report social skills measures to recommend outcomes for use in clinical trials of social deficit in children and adolescents with neurofibromatosis type 1. After evaluation, the committee recommended two measures: the Social Responsiveness Scale-2 (SRS-2) and the Social Skills Improvement System-Rating Scale (SSIS-RS). Each measure assesses different aspects of social functioning, with SSIS-RS suitable for broader social functioning studies and SRS-2 best for targeting problematic social behaviors associated with ASD.
Article
Clinical Neurology
Jennifer A. Janusz, Bonita P. Klein-Tasman, Jonathan M. Payne, Pamela L. Wolters, Heather L. Thompson, Staci Martin, Peter de Blank, Nicole Ullrich, Allison del Castillo, Maureen Hussey, Kristina K. Hardy, Kristina Haebich, Tena Rosser, Mary Anne Toledo-Tamula, Karin S. Walsh, REiNS Int Collaboration
Summary: Two parent-report social skills measures, the SRS-2 and SSIS-RS, were recommended for use in clinical trials of social deficit in children and adolescents with NF1. Each measure assesses different aspects of social functioning, with the SSIS-RS suitable for broader social functioning studies and the SRS-2 best for targeting problematic social behaviors associated with ASD. Researchers should consider their study goals when selecting a measure, with specific recommendations provided for their use.
Review
Ophthalmology
Michalis Georgiou, Kaoru Fujinami, Michel Michaelides
Summary: Inherited retinal diseases are a group of disorders characterized by photoreceptor degeneration or dysfunction, leading to severe vision loss. The advancements in genetics, retinal imaging, and molecular biology have paved the way for the development of treatments such as gene therapy and ongoing clinical trials targeting various retinal dystrophies. The current research aims to explore novel therapies for these diseases and improve patient outcomes.
CLINICAL AND EXPERIMENTAL OPHTHALMOLOGY
(2021)
Article
Mathematical & Computational Biology
Peijin Wang, Shein-Chung Chow
Summary: This article discusses methods for sample size re-estimation in clinical trials, including the adjusted effect size (AES) approach and the iterated expectation/variance (IEV) approach, which take into account the variability of observed responses. Results show that the IEV approach generally performs best in controlling type I error inflation, but may lead to a larger increase in sample size when detecting smaller effect sizes.
STATISTICS IN MEDICINE
(2021)
Editorial Material
Oncology
Boris Freidlin, Larissa A. Korde, Edward L. Korn
Summary: Recent therapeutic advances have improved patient survival in various cancer settings. To accelerate the development of new therapies, many cancer trials now designate OS as a secondary end point. This review assesses current practices and provides recommendations for designing and reporting trials with OS as a secondary end point to ensure data integrity.
JOURNAL OF CLINICAL ONCOLOGY
(2023)
Article
Oncology
Sara M. Tolaney, Elizabeth Garrett-Mayer, Julia White, Victoria S. Blinder, Jared C. Foster, Laleh Amiri-Kordestani, E. Shelley Hwang, Judith M. Bliss, Eileen Rakovitch, Jane Perlmutter, Patricia A. Spears, Elizabeth Frank, Nadine M. Tung, Anthony D. Elias, David Cameron, Neelima Denduluri, Ana F. Best, Angelo DiLeo, Lawrence Baizer, Lynn Pearson Butler, Elena Schwartz, Eric P. Winer, Larissa A. Korde
Summary: This study assessed the adherence of primary efficacy end points in adjuvant breast cancer trials to standardized definitions. Simulation modeling revealed the impact of excluding second nonbreast primary cancers on outcomes, leading to the recommendation of adding invasive breast cancer-free survival as an additional end point.
JOURNAL OF CLINICAL ONCOLOGY
(2021)
Review
Geriatrics & Gerontology
Graziella D'Arrigo, Stefanos Roumeliotis, Claudia Torino, Giovanni Tripepi
Summary: A crucial step in planning a randomized clinical trial (RCT) is the calculation of sample size, which determines the optimal number of patients needed to ensure the study has enough power to detect differences in specific endpoints between study arms. This calculation involves inputting variables such as the expected effect size, alpha error (α), beta error (β), and the allocation ratio in order to determine the number of participants allocated to each arm of the RCT.
AGING CLINICAL AND EXPERIMENTAL RESEARCH
(2021)
Review
Immunology
Patrick Santos, Fausto Almeida
Summary: Extracellular vesicles play crucial roles in processes such as inflammation, cell proliferation, and immune response, while also being implicated in the pathogenesis of diseases like cancer and infectious diseases. Research suggests that EV-based vaccines have promising therapeutic potential for cancer and infectious diseases, showing exciting results in inducing immune responses.
FRONTIERS IN IMMUNOLOGY
(2021)
Article
Rheumatology
Yura Kim, Gregory Levin, Nikolay P. Nikolov, Robert Abugov, Rebecca Rothwell
Summary: Defining appropriate clinical end points for clinical trials targeting osteoarthritis pathophysiology is challenging. This study proposed concept clinical end points that directly capture clinical benefit and evaluated their feasibility. The findings suggest that using composite end points can substantially reduce the required sample sizes compared to using total knee replacement alone.
ARTHRITIS CARE & RESEARCH
(2022)
Review
Biochemistry & Molecular Biology
Eugenia D. Namiot, Aleksandr Sokolov, Vladimir N. Chubarev, Vadim V. Tarasov, Helgi B. Schioth
Summary: Nanoparticles, particularly lipid-based ones, have played a crucial role in the development and manufacturing of the Pfizer and Moderna COVID-19 vaccines. They offer advantages such as reduced toxicity and increased bioavailability, and can be modified for specific tissues or cells. The number of clinical trials using nanoparticles for drug delivery is growing rapidly due to advancements in nanotechnology, biotechnology, and medicine.
INTERNATIONAL JOURNAL OF MOLECULAR SCIENCES
(2023)
Article
Health Care Sciences & Services
Benjamin R. Baer, Mario Gaudino, Stephen E. Fremes, Mary Charlson, Martin T. Wells
Summary: The fragility index, a clinically interpretable metric, is proposed as a supplement to traditional sample size calculations in clinical trial design. By using this approach, higher power and lower error rates can be achieved compared to standard tests.
JOURNAL OF CLINICAL EPIDEMIOLOGY
(2021)
Article
Oncology
Val Gebski, Andreas Obermair, Monika Janda
Summary: In addition to morbidity and mortality, quality of life (QoL) is an important outcome of cancer treatments. This study proposes a method to incorporate QoL as coprimary endpoints in clinical trial designs and provides metrics to evaluate QoL outcomes. The analysis of a clinical trial comparing laparoscopic and abdominal surgery for endometrial cancer showed that laparoscopic surgery had a shorter time to achieving a clinically important difference in physical and functional well-being QoL domains compared to abdominal surgery. The methods introduced in this study can help healthcare professionals counsel patients about both QoL and clinical outcomes simultaneously.
JOURNAL OF CLINICAL ONCOLOGY
(2022)
Article
Immunology
Jessica Howard-Anderson, Toshimitsu Hamasaki, Weixiao Dai, Deborah Collyar, Daniel Rubin, Sumathi Nambiar, Tori Kinamon, Carol Hill, Steven P. Gelone, David Mariano, Takamichi Baba, Thomas L. Holland, Sarah B. Doernberg, Henry F. Chambers, Vance G. Fowler, Scott R. Evans, Helen W. Boucher
Summary: The study applied the DOOR method in three registrational drug trials for complicated urinary tract infections and found no significant difference between the treatment and control groups. DOOR can improve understanding of the trade-offs between clinical efficacy and safety.
CLINICAL INFECTIOUS DISEASES
(2023)
Article
Mathematical & Computational Biology
Fengqing Zhang, Jiangtao Gou
Summary: Graphical approach provides a useful framework for multiplicity adjustment in clinical trials, but there is a lack of theoretical guidance on how to specify initial weight and transition probability to meet clinical preference and minimize sample size. We propose statistical methods to optimize sample size over initial weight and transition probability in a graphical approach, and prove that commonly used graphical approaches are suboptimal. Our methods are flexible and applicable to different types of endpoints. Two hypothetical clinical trial designs are presented to illustrate the application of our methods.
STATISTICS IN MEDICINE
(2023)
