Medicine and Health Sciences Commons^™

The Influence Of Flavor On The Abuse Liability Of A Heated Tobacco Product And Its Feasibility As A Menthol Cigarette Substitute, Augustus White

Theses and Dissertations

Heated Tobacco Products (HTPs) purport to expose people that use cigarettes to fewer of the harmful or potentially harmful constituents of tobacco while still delivering reinforcing amounts of nicotine (Auer, Concha-Lozano et al., 2017). An exemplar of the HTP class, IQOS, and its three varieties of “HeatSticks” have been authorized by the Food and Drug Administration (FDA) as “Modified Risk Tobacco Products” (MRTP). However, as the FDA is planning to ban menthol as a characterizing flavor in cigarettes, questions remain regarding whether characterizing flavors should be permitted in HTPs (FDA, 2022e). New evidence regarding HTP abuse liability (i.e., the likelihood …

Determining Call-To-Entry Rate And Recruitment Barriers In Clinical Studies For Community Clinics Serving Low-Income Populations: A Cohort Study, Elizabeth M Vaughan, Salim Virani, Mahmoud Al Rifai, Victor J Cardenas, Craig A Johnston, Laura Porterfield, Zuleica Santiago Delgado, Susan L Samson, Vanessa Schick, Aanand D Naik

Journal Articles

BACKGROUND: Recruitment for clinical studies is challenging. to overcome barriers, investigators have previously established call-to-entry rates to assist in planning. However, rates specific to low-income minority populations are needed to account for additional barriers to enrolment these individuals face.

OBJECTIVE: to obtain a call-to-entry rate in a low-income uninsured Hispanic population with chronic disease.

METHODS: We used data from four of our randomised clinical studies to determine the call-to-entry rate for individuals (n=1075) with or at risk for type 2 diabetes: participants needed/potential participants contacted=recruitment rate (yield). Research staff contacted potential participants to enrol in a study that evaluated 6 …

A Phase I Trial Of Sirolimus With "7&3" Induction Chemotherapy In Patients With Newly Diagnosed Acute Myeloid Leukemia, Neil Palmisiano, Grace Jeschke, Lindsay Wilde, Onder Alpdogan, Matthew Carabasi, Joanne Filicko-O'Hara, Dolores Grosso, Thomas Klumpp, Ubaldo Martinez, John L Wagner, Martin Carroll, Alexander Perl, Margaret Kasner

Department of Medicine Faculty Papers

Chemotherapy remains a primary treatment for younger AML patients, though many relapse. Data from our group have shown that highly phosphorylated S6 in blasts may predict response to sirolimus given with chemotherapy. We report the results of a phase I study of this combination in newly diagnosed AML and the pharmacodynamic analysis of pS6 before and after treatment. Subjects received sirolimus (12 mg on day 1, 4 mg daily, days 2-10), then idarubicin and cytarabine (days 4-10). Response was assessed at hematologic recovery or by day 42 using a modified IWG criteria. Fifty-five patients received sirolimus. Toxicity was similar to …

Prospective Assessment Of Inter-Rater Reliability Of A Neonatal Adverse Event Severity Scale., Thomas Salaets, Thierry Lacaze-Masmonteil, Isamu Hokuto, Cheri Gauldin, Amjad Taha, Anne Smits, Liesbeth Thewissen, Ilse Van Horebeek, Armuchou Shoraisham, Khorshid Mohammad, Manami Suzuki, Shiori Komachi, Kurt Michels, Mark A. Turner, Karel Allegaert, Tamorah Lewis

Manuscripts, Articles, Book Chapters and Other Papers

Introduction: To ensure the quality of clinical trial safety data, universal data standards are required. In 2019 the International Neonatal Consortium (INC) published a neonatal adverse event severity scale (NAESS) to standardize the reporting of adverse event (AE) severity. In this study the reliability of AE severity grading with INC NAESS was prospectively assessed in a real-world setting. Methods: Severity of AEs was assessed by two independent observers at each of four centers across the world. In each center two series of 30 neonatal adverse events were assessed by both observers: in a first phase with a generic (Common Terminology …

Interactions Between Mediterranean Diet Supplemented With Dairy Foods And The Gut Microbiota Influence Cardiovascular Health In An Australian Population, Jocelyn M. Choo, Karen J. Murphy, Alexandra T. Wade, Yanan Wang, Ella L. Bracci, Courtney R. Davis, Kathryn A. Dyer, Richard J. Woodman, Jonathan M. Hodgson, Geraint B. Rogers

Research outputs 2022 to 2026

The impact of a Mediterranean diet on the intestinal microbiome has been linked to its health benefits. We aim to evaluate the effects of a Mediterranean diet supplemented with dairy foods on the gut microbiome in Australians at risk of cardiovascular disease. In a randomised controlled cross-over study, 34 adults with a systolic blood pressure ≥120 mmHg and with risk factors for cardiovascular disease were randomly allocated to a Mediterranean diet with 3–4 daily serves of dairy foods (Australian recommended daily intake (RDI) of 1000–1300 mg per day (MedDairy)) or a low-fat (LFD) control diet. Between each 8-week diet, participants …

An Artificial Intelligence (Ai)-Based Approach To Clinical Trial Recruitment: The Impact Of Viz Recruit On Enrollment In The Embolise Trial, Ameer E. Hassan, Saisree Ravi, Sohum Desai, Hamzah M. Saei, Ermias Mckennon, Wondwossen G. Tekle

School of Medicine Publications and Presentations

Background

Materials and methods

Precision Medicine Approach To Alzheimer’S Disease: Rationale And Implications, Dale E. Bredesen, Kat Toups, Ann Hathaway, Deborha Gordon, Henrianna Chung, Cyrus Raji, Alan Boyd, Benjamin D. Hill, Sharon Hausman-Cohen, Mouna Attarha, Won Jong Chwa, Alexei Kurakin, Michael Jarrett

University Faculty and Staff Publications

The neurodegenerative disease field has enjoyed extremely limited success in the development of effective therapeutics. One potential reason is the lack of disease models that yield accurate predictions and optimal therapeutic targets. Standard clinical trials have pre-determined a single treatment modality, which may be unrelated to the primary drivers of neurodegeneration. Recent proof-of-concept clinical trials using a precision medicine approach suggest a new model of Alzheimer’s disease (AD) as a chronic innate encephalitis that creates a network insufficiency. Identifying and addressing the multiple potential contributors to cognitive decline for each patient may represent a more effective strategy. Here we review …

Efficacy And Safety Of Acupuncture For Depression: A Systematic Review And Meta-Analysis, Binglei Chen, Carol C. Wang, Khui Hung Lee, Jianhong C. Xia, Zongting Luo

Research outputs 2022 to 2026

Acupuncture is widely accepted as a therapeutic option for managing depression. However, evidence from clinical trials remains controversial. This review aims to synthesize the best available evidence on the efficacy and safety of acupuncture in managing depression. The review was performed according to PRISMA (Preferred Reporting Items for Systematic Reviews and Meta-Analysis) guidelines. Five databases and the relevant trial registries were searched from the inception to October 2021. Randomized clinical trials of acupuncture for managing depression, published in English, were selected for inclusion. The quality of included studies was assessed using the Cochrane risk of bias tool. Netmeta and dmetar …

The Impact Of The 2022 Ukraine/Russian Conflict On Cancer Clinical Trials, Alice Talbot, Sophia G. Connor, Kate Austin, Tara Hannon, Eli Gabbay, Timothy D. Clay

Research outputs 2022 to 2026

Since the invasion of Ukraine in February 2022, clinical trial conduct has become extremely challenging due to damage to the healthcare infrastructure and patient displacement. This current study aimed to estimate the number of cancer clinical trials at risk of impact from the conflict. A descriptive analysis and narrative review were completed using data from cancer clinical trials with sites in Russia or Ukraine using the ‘clinical trials.gov’ online database between February 2022 and May 2022. There were 508 clinical trials involving sites in Ukraine or Russia. Most were multinational studies (470 of 508; 93 %). The majority of studies …

Visualization Literacy And Decision-Making In Healthcare, Stacy Weil

Translational Projects (Open Access)

The ability of workers in the healthcare industry to analyze, interpret and communicate with health data is critical to decision-making and impacts both health and business outcomes. Optimal decision-making requires having real-time access to information that provides useful insights and that lends itself to collaborative decision-making. Data visualizations have the potential to facilitate decision-making in healthcare when presented as a dashboard. However, dashboards have shown varying results in both effectiveness and adoption. Data or graphical literacy challenges experienced by health team members could complicate strategic decision-making through an inability to correctly interpret or summarize the information presented in a dashboard. …

Creating And Implementing A Principal Investigator Tool Kit For Enhancing Accrual To Late Phase Clinical Trials: Development And Usability Study., Kristin A Higgins, Alexandra Thomas, Nancy Soto, Rebecca Paulus, Thomas J George, Thomas B Julian, Sharon Hartson Stine, Merry Jennifer Markham, Maria Werner-Wasik

Kimmel Cancer Center Faculty Papers

BACKGROUND: Accrual to oncology clinical trials remains a challenge, particularly during the COVID-19 pandemic. For late phase clinical trials funded by the National Cancer Institute, the development of these research protocols is a resource-intensive process; however, mechanisms to optimize patient accrual after trial activation are underdeveloped across the National Clinical Trial Network (NCTN). Low patient accrual can lead to the premature closure of clinical trials and can ultimately delay the availability of new, potentially life-saving therapies in oncology.

OBJECTIVE: The purpose of this study is to formally create an easily implemented tool kit of resources for investigators of oncology clinical …

Validation Of Remote Height And Weight Assessment In A Rural Randomized Clinical Trial., Bethany Forseth Hanson, Ann M. Davis, Dana Bakula, Megan Murray, Kelsey Dean, Rebecca E. Swinburne Romine, Kandace Fleming

Manuscripts, Articles, Book Chapters and Other Papers

BACKGROUND: The purpose of this study is to describe and assess a remote height and weight protocol that was developed for an ongoing trial conducted during the SARS COV-2 pandemic.

METHODS: Thirty-eight rural families (children 8.3 ± 0.7 years; 68% female; and caregivers 38.2 ± 6.1 years) were provided detailed instructions on how to measure height and weight. Families obtained measures via remote data collection (caregiver weight, child height and weight) and also by trained staff. Differences between data collection methods were examined.

RESULTS: Per absolute mean difference analyses, slightly larger differences were found for child weight (0.21 ± 0.21 …

Abobotulinumtoxina Efficacy And Safety In Children With Upper Limb Spasticity Previously Treated With Botulinum Toxin, Edward Dabrowski, Mauricio R. Delgado, Joyce Oleszek, Nigar Dursun, Jorge Carranza Del-Rio, Marcin Bonikowski, Benjamin Regnault, Simon Page, Ann Tilton

Conference Presentation Abstracts

Introduction: Primary endpoint analysis of this phase 3 study confirmed abobotulinumtoxinA (aboBoNT-A 8 U/kg and 16 U/kg) significantly reduced hypertonia versus the 2 U/kg low-dose control and was generally well tolerated (Delgado, et al, 2021). However, the impact of previous botulinum neurotoxin (BoNT) injections on treatment outcomes remains largely unexplored.

Methods: We present subgroup analyses of a phase 3 study conducted in children (aged 2-17) with cerebral palsy (Gross Motor Function Classification System [GMFCS] Levels I-IV) and spasticity in ≥1 upper limb. In the first treatment cycle, 210 children were randomized to treatment with aboBoNT-A 2 U/kg, 8 U/kg or …

Efficacy Of Cenobamate For Uncontrolled Focal Seizures In Patients With Previous Epilepsy-Related Surgery: Post Hoc Analysis Of A Phase 3, Multicenter, Open-Label Study, Bassel Abou-Khalil, Sami Aboumatar, Pavel Klein, Gregory L Krauss, Michael R Sperling, William E Rosenfeld

Department of Neurology Faculty Papers

Objective: This post hoc analysis of 10 US study sites from a long-term open-label phase 3 study of adjunctive cenobamate evaluated the efficacy of cenobamate in patients with prior epilepsy-related surgery.

Methods: Patients with uncontrolled focal seizures despite taking stable doses of 1-3 concomitant antiseizure medications (ASMs) received increasing doses of cenobamate (12.5, 25, 50, 100, 150, 200 mg/day) at 2-week intervals over 12 weeks (target dose, 200 mg/day). Further increases up to 400 mg/day using biweekly 50-mg/day increments were allowed during the maintenance phase. Dose adjustments of cenobamate and concomitant ASMs were allowed. Data were assessed until the last …

Akt Inhibition In The Central Nervous System Induces Signaling Defects Resulting In Psychiatric Symptomatology, Apostolia-Maria Tsimberidou, Antonis Skliris, Alan Valentine, Jamie Shaw, Ursula Hering, Henry Hiep Vo, Tung On Chan, Roger S Armen, Jeffrey R Cottrell, Jen Q Pan, Philip N Tsichlis

Center for Translational Medicine Faculty Papers

Background: Changes in the expression and activity of the AKT oncogene play an important role in psychiatric disease. We present translational data assessing the role of AKT in psychiatric symptoms.

Methods: (1) We assessed the protein activity of an AKT3 mutant harboring a PH domain mutation (Q60H) detected in a patient with schizophrenia, the corresponding AKT1 mutant (Q61H), and wild-type AKT1 and AKT3 transduced in AKT-null mouse fibroblasts and modeled the Q61H mutation onto the crystal structure of the Akt1 PH domain. (2) We analyzed the results of earlier genome-wide association studies to determine the distribution of schizophrenia-associated single-nucleotide polymorphisms …

The Texas Community-Engagement Research Alliance Against Covid-19 In Disproportionately Affected Communities (Tx Ceal) Consortium, Rebecca A. Seguin-Fowler, Chris Amos, Bettina M. Beech, Robert L. Ferrer, Lorna Mcneill, Jasmine J. Opusunju, Emily Spence, Erika L. Thompson, Luis R. Torres-Hostos, Jamboor K. Vishwanatha

School of Social Work Faculty Publications and Presentations

The coronavirus disease 2019 (COVID-19) pandemic requires urgent implementation of effective community-engaged strategies to enhance education, awareness, and inclusion of underserved communities in prevention, mitigation, and treatment efforts. The Texas Community-Engagement Alliance Consortium was established with support from the United States’ National Institutes of Health (NIH) to conduct community-engaged projects in selected geographic locations with a high proportion of medically underserved minority groups with a disproportionate burden of COVID-19 disease and hospitalizations. The purpose of this paper is to describe the development of the Consortium. The Consortium organized seven projects with focused activities to address COVID-19 clinical and vaccine trials …

Concurrent P53 Mutation In Egfr Mutant Non-Small Cell Lung Cancer Is Associated With Resistance To First And Second Generation Egfr Tyrosine Kinase Inhibitors, A Meta-Analysis, Marissa Viola, Adam Kolatorowicz, Jun Wang

Research Day

Epidermal growth factor receptor (EGFR) mutant lung cancers tend to respond well to EGFR tyrosine kinase inhibitors (TKIs). However, resistance has been described. Molecular studies have revealed that concurrent mutations of tumor driver genes are associated with TKI resistance. To delineate the role of concurrent mutation of tumor suppressor gene p53 in TKI resistance, a meta-analysis was performed using published observations of EGFR mutant lung cancer patients treated with first or second generation TKIs. 31 studies are included in the analysis following a search of PubMed. Probability of TKI resistance and progress free survival (PFS) were compared in patients with …

Homoeopathic Pathogenetic Trial Of Mentha Piperita L.: A Multi-Centric, Double-Blind, Randomised And Placebo-Controlled Trial, Pritha Mehra, Amulya Ratna Sahoo, B. S. J. Rajakumar, Jai P. Singh, B S. Arya, Anil Khurana, Raj Kumar Manchanda

Indian Journal of Research in Homoeopathy

Introduction: Mentha piperita L, a lesser-known and partly proved drug in homoeopathy, is used extensively as a herbal medicine.

Objective: The objective of this study is to elicit the pathogenetic response of Mentha piperita in comparison to placebo.

Materials and Methods: A multicentre, double-blind, placebo-controlled and randomised clinical trial was carried out at three centres with 46 relatively healthy provers. After randomisation, 32 provers were given verum in 6C, 12C, 30C and 200C potencies and in the placebo group,14 provers were administered identical, un-medicated globules. All the changes were recorded by the provers and elaborated by proving …

Emerging Evidence And Treatment Perspectives From Randomized Clinical Trials In Systemic Sclerosis: Focus On Interstitial Lung Disease, Caterina Oriana Aragona, Antonio Giovanni Versace, Carmelo Ioppolo, Daniela La Rosa, Rita Lauro, Maria Concetta Tringali, Simona Tomeo, Guido Ferlazzo, William Neal Roberts, Alessandra Bitto, Natasha Irrera, Gianluca Bagnato

Internal Medicine Faculty Publications

Systemic sclerosis (SSc) is a complex rare autoimmune disease with heterogeneous clinical manifestations. Currently, interstitial lung disease (ILD) and cardiac involvement (including pulmonary arterial hypertension) are recognized as the leading causes of SSc-associated mortality. New molecular targets have been discovered and phase II and phase III clinical trials published in the last 5 years on SSc-ILD will be discussed in this review. Details on the study design; the drug tested and its dose; the inclusion and exclusion criteria of the study; the concomitant immunosuppression; the outcomes and the duration of the study were reviewed. The two most common drugs used …

Designing A Clinical Study With Dietary Supplements: It's All In The Details, Z. Elizabeth Floyd, David M. Ribnicky, Ilya Raskin, Daniel S. Hsia

Faculty and Student Publications

A successful randomized clinical trial of the effect of dietary supplements on a chosen endpoint begins with developing supporting data in preclinical studies while paying attention to easily overlooked details when planning the related clinical trial. In this perspective, we draw on our experience studying the effect of an ethanolic extract from Artemisia dracunculus L. (termed PMI-5011) on glucose homeostasis as a potential therapeutic option in providing resilience to metabolic syndrome (MetS). Decisions on experimental design related to issues ranging from choice of mouse model to dosing levels and route of administration in the preclinical studies will be discussed in …

Cost-Effectiveness Analysis Of Combined Cognitive And Vocational Rehabilitation In Patients With Mild-To-Moderate Tbi: Results From A Randomized Controlled Trial, Emilie Isager Howe, Nada Andelic, Silje C R Fure, Cecilie Røe, Helene L. Søberg, Torgeir Hellstrøm, Øystein Spjelkavik, Heidi Enehaug, Juan Lu, Helene Ugelstad, Marianne Løvstad, Eline Aas

Family Medicine and Population Health Publications

Background

Traumatic brain injury (TBI) represents a financial burden to the healthcare system, patients, their families and society. Rehabilitation interventions with the potential for reducing costs associated with TBI are demanded. This study evaluated the cost-effectiveness of a randomized, controlled, parallel group trial that compared the effectiveness of a combined cognitive and vocational intervention to treatment as usual (TAU) on vocational outcomes.

Methods

One-hundred sixteen participants with mild-to-moderate TBI were recruited from an outpatient clinic at Oslo University Hospital, Norway. They were randomized to a cognitive rehabilitation intervention (Compensatory Cognitive Training, CCT) and Supported Employment (SE) or TAU in a …

Stakeholder Perspectives Of A Pilot Multicomponent Delirium Prevention Intervention For Adult Patients With Advanced Cancer In Palliative Care Units: A Behaviour Change Theory-Based Qualitative Study, Anna Green, Annmarie Hosie, Jane L. Phillips, Slavica Kochovska, Beverly Noble, Meg Brassil, Anne Cumming, Peter G. Lawlor, Shirley H. Bush, Jan M. Davis, Layla Edwards, Jane Hunt, Julie Wilcock, Carl Phillipson, Eugene Wesley Ely, Cynthia Parr, Melanie Lovell, Meera Agar

Nursing Papers and Journal Articles

Background: Theory-based and qualitative evaluations in pilot trials of complex clinical interventions help to understand quantitative results, as well as inform the feasibility and design of subsequent effectiveness and implementation trials.

Aim: To explore patient, family, clinician and volunteer (‘stakeholder’) perspectives of the feasibility and acceptability of a multicomponent non-pharmacological delirium prevention intervention for adult patients with advanced cancer in four Australian palliative care units that participated in a phase II trial, the ‘PRESERVE pilot study’.

Design: A trial-embedded qualitative study via semi-structured interviews and directed content analysis using Michie’s Behaviour Change Wheel and the Theoretical Domains Framework.

Setting/participants: Thirty-nine …

Effects Of A Nurse-Led Tai Chi Programme On Improving Quality Of Life, Mental Wellbeing, And Physical Function Of Women With Breast Cancer: Protocol For A Randomized Controlled Trial, Carol Chunfeng Wang, Sadie Geraghty, Caitlin Fox-Harding, Calvin Wang

Research outputs 2022 to 2026

Objectives:

Quality of life, mental wellbeing, and physical function deteriorate among women with breast cancer. Tai Chi is a moderate form of exercise that may be effective in improving the mental and physical wellbeing, therefore, the quality of life of women with breast cancer. This protocol paper outlines a trial to determine the therapeutic effects of a Tai Chi programme on breast cancer management.

Methods:

The study will be an interventional, single-blind, double-armed, randomized, and controlled trial involving a 12-week Tai Chi programme for women with breast cancer. Forty participants aged 18 years and above who are diagnosed with breast …

Intensive Care Bundle With Blood Pressure Reduction In Acute Cerebral Hemorrhage Trial (Interact3): Study Protocol For A Pragmatic Stepped-Wedge Cluster-Randomized Controlled Trial, Lili Song, Xin Hu, Lu Ma, Xiaoying Chen, Menglu Ouyang, Laurent Billot, Qiang Li, Paula Muñoz-Venturelli, Carlos Abanto, Mohammad Wasay

Section of Neurology

Background: Early intensive blood pressure (BP) lowering remains the most promising treatment for acute intracerebral hemorrhage (ICH), despite discordant results between clinical trials and potential variation in the treatment effects by approach to control BP. As the third in a series of clinical trials on this topic, the INTEnsive care bundle with blood pressure Reduction in Acute Cerebral hemorrhage Trial (INTERACT3) aims to determine the effectiveness of a goal-directed care bundle protocol of early physiological control (intensive BP lowering, glycemic control, and pyrexia treatment) and reversal of anticoagulation, in acute ICH.
Methods: INTERACT3 is a pragmatic, international, multicenter, stepped-wedge (4 …

Clinical Trial Of Abcb5+ Mesenchymal Stem Cells For Recessive Dystrophic Epidermolysis Bullosa, Dimitra Kiritsi, Kathrin Dieter, Elke Niebergall-Roth, Silvia Fluhr, Cristina Daniele, Jasmina Esterlechner, Samar Sadeghi, Seda Ballikaya, Leoni Erdinger, Franziska Schauer, Stella Gewert, Martin Laimer, Johann W. Bauer, Alain Hovnanian, Giovanna Zambruno, May El Hachem, Emmanuelle Bourrat, Maria Papanikolaou, Gabriela Petrof, Sophie Kitzmüller, Christen L. Ebens, Markus H. Frank, Natasha Y. Frank, Christoph Ganss, Anna E. Martinez, John A. Mcgrath, Jakub Tolar, Mark A. Kluth

Research outputs 2014 to 2021

BACKGROUND. Recessive dystrophic epidermolysis bullosa (RDEB) is a rare, devastating, and lifethreatening inherited skin fragility disorder that comes about due to a lack of functional type VII collagen, for which no effective therapy exists. ABCB5+ dermal mesenchymal stem cells (ABCB5+ MSCs) possess immunomodulatory, inflammation-dampening, and tissue-healing capacities. In a Col7a1-/-mouse model of RDEB, treatment with ABCB5+ MSCs markedly extended the animals' lifespans. METHODS. In this international, multicentric, single-arm, phase I/IIa clinical trial, 16 patients (aged 4-36 years) enrolled into 4 age cohorts received 3 i.v. infusions of 2 × 106ABCB5+ MSCs/kg on days 0, 17, and 35. Patients were followed …

Therapeutic Efficacy Of Mesenchymal Stem Cells For Cardiovascular Diseases, Dragana Radoje Miloradovic, Dragica Radoje Pavlovic, Miodrag Bozidar Stojkovic, Sanja Bratislav Bojic, Vladislav Bogdan Volarevic, Marina Milosav Gazdic Jankovic, Biljana Tomislav Ljujic

Journal of Mind and Medical Sciences

Despite the improvements in pharmacological and surgical treatments, cardiovascular diseases (CVDs) are the number one cause of death worldwide. During the last two decades, the search for new therapies has been revolutionized with the growing knowledge of stem cell biology. Due to their huge differentiation capacity and paracrine effects, mesenchymal stem cells (MSCs) are a promising tool for the treatment of CVDs. The encouraging outcomes of preclinical studies using MSCs as a treatment for diseased myocardium have set the scene for worldwide clinical trials. In this review, we overview either complete or ongoing clinical trials using MSCs for the therapy …

Diversity In Alzheimer's Disease Drug Trials: The Importance Of Eligibility Criteria, Sanne Franzen, Jade Emily Smith, Esther Van Den Berg, Monica Rivera Mindt, Rozemarijn L. Van Bruchem-Visser, Erin L. Abner, Lon S. Schneider, Niels D. Prins, Ganesh M. Babulal, Janne M. Papma

Sanders-Brown Center on Aging Faculty Publications

INTRODUCTION: To generalize safety and efficacy findings, it is essential that diverse populations are well represented in Alzheimer's disease (AD) drug trials. In this review, we aimed to investigate participant diversity in disease-modifying AD trials over time, and the frequencies of participant eligibility criteria.

METHODS: A systematic review was performed using Medline, Embase, the Cochrane Library, and Clinicaltrials.gov, identifying 2247 records.

RESULTS: In the 101 included AD trials, participants were predominantly White (median percentage: 94.7%, interquartile range: 81.0-96.7%); and this percentage showed no significant increase or decrease over time (2001-2019). Eligibility criteria such as exclusion of persons with psychiatric illness …

Committee On High-Quality Alzheimer’S Disease Studies (Chads) Consensus Report, Gregory A. Jicha, Erin L. Abner, Steven E. Arnold, Maria C. Carrillo, Hiroko H. Dodge, Steven D. Edland, Keith N. Fargo, Howard H. Feldman, Larry B. Goldstein, James A. Hendrix, Ruth Peters, Julie M. Robillard, Lon S. Schneider, Jodi R. Titiner, Christopher J. Weber

Sanders-Brown Center on Aging Faculty Publications

Background: Consensus guidance for the development and identification of high-quality Alzheimer's disease clinical trials is needed for protocol development and conduct of clinical trials.

Methods: An ad hoc consensus committee was convened in conjunction with the Alzheimer's Association to develop consensus recommendations.

Results: Consensus was readily reached for the need to provide scientific justification, registration of trials, institutional review board oversight, conflict of interest disclosure, funding source disclosure, defined trial population, recruitment resources, definition of the intervention, specification of trial duration, appropriate payment for participant engagement, risk-benefit disclosure as part of the consent process, and the requirement …

Tocilizumab And Covid-19: A Meta-Analysis Of 2120 Patients With Severe Disease And Implications For Clinical Trial Methodologies, Azza Sarfraz, Zouina Sarfraz, Muzna Sarfraz, Hinna Aftab, Zainab Pervaiz

Department of Paediatrics and Child Health

Background/aim: Since the outbreak of the COVID-19, numerous therapies to counteract this severe disease have emerged. The benefits of Tocilizumab for severely infected COVID-19 patients and the methodologies of ongoing clinical trials are explored.
Materials and methods: A systematic search adhering to PRISMA guidelines was conducted in PubMed, Cochrane Central, medRxiv, and bioRxiv using the following keywords: “Tocilizumab,” “Actemra,” “COVID-19.” An additional subsearch was conducted on Clinicaltrials.gov to locate ongoing tocilizumab trials.
Results: A total of 13 studies were included in the meta-analysis comprising 2120 patients. The treatment group had lower mortality compared to the control group (OR = 0.42, …

The Immune Microenvironment And Relation To Outcome In Patients With Advanced Breast Cancer Treated With Docetaxel With Or Without Gemcitabine, Elisabeth S. Stovgaard, Karama Asleh, Nazia Riaz, Samuel Leung, Dongxia Gao, Lise B. Nielsen, Anne-Vibeke Lænkholm, Eva Balslev, Maj-Britt Jensen, Dorte Nielsen, Torsten O. Nielsen

Centre for Regenerative Medicine & Stem Cell Research

Preclinical studies suggest that some effects of conventional chemotherapy, and in particular, gemcitabine, are mediated through enhanced antitumor immune responses. The objective of this study was to use material from a randomized clinical trial to evaluate whether patients with preexisting immune infiltrates responded better to treatment with gemcitabine + docetaxel (GD) compared to docetaxel alone. Formalin fixed, paraffin-embedded breast cancer tissues from SBG0102 phase 3 trial patients randomly assigned to treatment with GD or docetaxel were used. Immunohistochemical staining for CD8, FOXP3, LAG3, PD-1, PD-L1 and CD163 was performed. Tumor infiltrating lymphocytes (TILs) and tumor associated macrophages were evaluated. Prespecified …