Health Policy Commons^™

Moving Beyond Simplistic Research Design In Health Professions Education: What A One-Group Pretest-Posttest Design Will Not Prove, S Beth Bierer, Gary Beck Dallaghan, Nicole J Borges, Sam Brondfield, Cha Chi Fung, Kathryn N Huggett, Cayla R Teal, Satid Thammasitboon, Colleen Y Colbert

Center for Medical Ethics and Health Policy Staff Publications

Introduction: Educational research presents unique study design challenges. Novice researchers in health professions education (HPE) frequently misuse the one-group pretest-posttest design, highlighting the need for improved training in research design. This workshop aimed to enhance understanding of research design among novice HPE researchers, specifically addressing the inherent limitations of the one-group pretest-posttest design and offering alternative approaches.

Methods: Experienced HPE researchers developed this workshop to address common misunderstandings of research design. Leaders from the AAMC Medical Education, Scholarship, Research, and Evaluation section facilitated 60-75-minute workshops conducted at the four 2024 regional meetings hosted by the AAMC Group on Educational Affairs …

Talking About Suffering In The Intensive Care Unit, Brent M Kious, Judith B Vick, Peter A Ubel, Olivia Sutton, Jennifer Blumenthal-Barby, Christopher E Cox, Deepshikha Ashana

Center for Medical Ethics and Health Policy Staff Publications

Background: Some have hypothesized that talk about suffering can be used by clinicians to motivate difficult decisions, especially to argue for reducing treatment at the end of life. We examined how talk about suffering is related to decision-making for critically ill patients, by evaluating transcripts of conversations between clinicians and patients' families.

Methods: We conducted a secondary qualitative content analysis of audio-recorded family meetings from a multicenter trial conducted in the adult intensive care units of five hospitals from 2012-2017 to look at how the term "suffering" and its variants were used. A coding guide was developed by consensus-oriented discussion …

Impact Of Volume-Based Procurement Policy On Coronary Stent Use In Tianjin: Interrupted Time Series Analysis, Zhao Yang, Liangyu Ni, Wei Zhao, Yuxi Li, Yongyi Wu, Qiyun Zhu, Linlin Da, Weijia Liu, Qi Zhang, Jianping Li, Bin Jiang

Health Behavior, Policy & Management Faculty Publications

Objectives

This study aimed to analyze the impact of China’s centralized volume-based procurement policy on the procurement and utilization of coronary stents in Tianjin from 2019 to 2021.

Methods

An interrupted time-series design was used to assess monthly procurement data of coronary stents and balloons in Tianjin from January 2019 to December 2021.

Results

The volume-based procurement policy significantly reduced coronary stents prices, with an average price reduction of 92.67% for ten selected stents, and the lowest price dropped to 469 CNY. Following policy implementation, annual procurement in Tianjin increased by 59.55%, and tertiary hospitals demonstrated a significant monthly level …

Examining Low-Income Caregivers' Knowledge And Comprehension Of The Dental Caries Process, Melissa Sullivan, Denise C. Mckinney, Deanne Shuman, Qi Zhang

Dental Hygiene Faculty Publications

Purpose Dental caries among young children remains a public health concern in the United States (US). Children of caregivers with low oral health literacy (OHL) tend to have greater unmet dental needs and poorer oral health status. The purpose of this study was to examine the understanding of pediatric dental terms associated with the dental caries process among low-income caregivers.

Methods This study included 14 caregivers with children enrolled in a Special Supplemental Nutrition Program for Women, Infants and Children (WIC) in the Southeastern US. Through structured phone interviews, the validated Oral Health Literacy Inventory for Parents (OH-LIP) was used …

A Qualitative Analysis Of College Students' Interest In Mhealth Solutions, Leslie Hoglund, Craig M. Becker, Cara Tonn

Health Behavior, Policy & Management Faculty Publications

This study explores college students' perceptions of an AI-driven mHealth application designed to promote well-being. With rising mental health challenges in academic settings, students increasingly seek digital tools that provide holistic support for physical, mental, and financial health. Through focus groups, this qualitative study examines students' preferences for personalized health tracking, educational content, and flexible reminders within a private, supportive community. Key findings emphasize students' desire for a balanced, all-in-one app that integrates health and wellness tools without overwhelming them with notifications. Students also highlighted the importance of social media integration for outreach, though concerns were raised about potential stress …

Global, Regional, And National Burden Of Cardiovascular Diseases And Risk Factors In 204 Countries And Territories, 1990-2023, Global Burden Of Cardiovascular Diseases And Risks 2023 Collaborators, Benjamin A. Stark, Nicole K. Decleene, Emily C. Desai, Johnathan M. Hsu, Catherine O. Johnson, Laura Lara-Castor, Kate E. Legrand, Prof Bhoomadevi A., Mohammad Amin Aalipour, Nasir Abbas, Madineh Abbasi, Mohammadreza Abbasian, Hedayat Abbastabar, Abdallah H. A. Abd Al Magied, Samar Abd Elhafeez, Gregory A. Roth, Et Al.

Epidemiology, Biostatistics, & Environmental Health Faculty Publications

Background

Cardiovascular diseases (CVDs) are the leading cause of mortality and are among the foremost causes of disability globally. CVD burden has continued to increase in most countries since 1990, with trends driven by changing exposures to harmful risk factors, population growth, and population aging.

Objectives

We report estimates of global, national, and subnational CVD burden, including 18 subdiseases and 12 associated modifiable risk factors. We analyzed change in CVD burden from 1990 to 2023 and identified drivers of change including population growth, population aging, and risk factor exposure.

Methods

The Global Burden of Disease (GBD) 2023 study, a multinational …

A Hybrid Cloud Data Lake Architecture Supporting The Integration Of Clinical And Genomics Data, Apollo Mcowiti, Heidi Dowst, Fei Zheng, Susan Hilsenbeck, Christopher Amos

Center for Medical Ethics and Health Policy Staff Publications

Objective: Cancer centers must quickly integrate clinical genomics data from different vendors for oncology operations and research. Clinical data warehouse architectures are costly to construct and brittle, and they are not readily amenable to the rapid changes in oncology research. We introduce a cost-effective hybrid cloud Data Lake architecture for storing clinical genomic data from different vendors, aiding both clinical and research workflows.

Methods: We created a Data Lake architecture based on the zone architecture, with four layers: ingestion, storage, transformation, and interaction. The layers are implemented with a hybrid cloud architecture. Rich metadata created from patient and genomic data …

Methadone For Critically Ill Patients Under Mechanical Ventilation In The Intensive Care Unit: A Systematic Review, Sérgio Martins Pereira, Megan Abbott, João Francisco Figueiredo Marcondes Ferraz, Akash Goel, Andrea Rigamonti, Charmaine De Castro, Lisa Burry, Airton Leonardo De Oliveira Manoel, Michael Chaim Sklar

Center for Medical Ethics and Health Policy Staff Publications

Purpose: Pain may pose significant challenges in the intensive care unit, especially in mechanically ventilated patients. Methadone has recently emerged as an alternative option for eliciting acute analgesia. In this systematic review, we evaluated the use of methadone in mechanically ventilated patients in the intensive care unit.

Source: We searched MEDLINE, EMBASE, Wiley's Cochrane Library, CINAHL, PubMed (non-MEDLINE), Scopus, and LILACS databases from inception to January 24th, 2025. Eligible studies included randomized controlled trials and observational studies that compared the use of methadone to the standard of care or to other analgosedation strategies in mechanically ventilated patients in the intensive …

Impact Of Personalized Risk Scores On Shared Decision Making In Left Ventricular Assist Device Implantation: Findings From A Qualitative Study, Holland Kaplan, Kristin Kostick-Quenet, Benjamin Lang, Robert J Volk, Jennifer Blumenthal-Barby

Center for Medical Ethics and Health Policy Staff Publications

Objective: To assess stakeholders' perspectives on integrating personalized risk scores (PRS) into left ventricular assist device (LVAD) implantation decisions and how these perspectives might impact shared decision making (SDM).

Methods: We conducted 40 in-depth interviews with physicians, nurse coordinators, patients, and caregivers about integrating PRS into LVAD implantation decisions. A codebook was developed to identify thematic patterns, and quotations were consolidated for analysis. We used Thematic Content Analysis in MAXQDA software to identify themes by abstracting relevant quotes.

Results: Clinicians had varying preferences regarding PRS integration into LVAD decision making, while patients and caregivers preferred real-time discussions about PRS with …

Disparities In Cardiovascular Disease Outcomes And Economic Burdens Among Minorities In Southeastern Virginia, Ismail El Moudden, Asra Amidi, Reem Sharaf Alddin, Michael Bittner, Qi Zhang

Department of Obstetrics & Gynecology Faculty Publications

Background

Cardiovascular diseases are the leading cause of mortality in the United States, presenting significant public health challenges and financial burdens, particularly in Southeastern Virginia, where African American and Hispanic (AA&H) populations are disproportionately affected.

Methods

This retrospective observational study analyzed data from 30,855 hospital discharges of AA&H patients across Southeastern Virginia from 2016 to 2020, focusing on individuals aged 18 to 85 with cardiovascular diseases. Utilizing the Virginia Health Information database, we examined demographic information, clinical data, and healthcare utilization patterns through hypothesis tests and regression models to explore associations between these variables and the economic impacts of cardiovascular …

A Landscape Analysis Of Psychedelic Retreat Organizations Advertising Online, Logan Neitzke-Spruill, Caroline S Beit, Jill Oliver Robinson, Nikita Singh, Srijith Kambala, Rishi Ramesh, Amy L Mcguire

Center for Medical Ethics and Health Policy Staff Publications

Research into psychedelics' clinical potential has corresponded to a growth in public interest and adult use. One common pathway to accessing psychedelics is through psychedelic retreats. While individual retreats have been characterized in the anthropological literature, no systematic evaluation of the psychedelic retreat industry exists. Assessing the characteristics of the psychedelic retreat industry is critical to understanding the associated ethical, legal, and social implications and ensuring consumer safety. To this end, we conducted a landscape analysis of online, publicly available information to capture and characterize a broad range of organizations offering psychedelic retreats and marketing to English-speaking consumers. From July …

Advancing Medical Applications Of Cancer Nanotechnology: Highlighting Two Decades Of The Nci's Nanotechnology Characterization Laboratory Service To The Research Community, Rachael M Crist, Yechezkel Barenholz, Ahuva Cern, Kate N Clark, Pieter R Cullis, Cheryl Dean, Neil Desai, Mauro Ferrari, Matthieu Germain, Carmen A Giacomantonio, Emma Grabarnik, Piotr Grodzinski, Atara Hod, Barry E Kennedy, Ruvanthi N Kularatne, Glen S Kwon, Emmanuel Loeb, Erin B Noftall, Len Pagliaro, Morteza Rasoulianboroujeni, Alexander Roth, Darren Rowles, Kulbir Singh, Nicole F Steinmetz, Zhanna Yehtina, Yao Zhang, Daniel Zilbersheid, Jeffrey D Clogston, Stephan T Stern, Marina A Dobrovolskaia

Center for Medical Ethics and Health Policy Staff Publications

The Nanotechnology Characterization Laboratory (NCL) is a US federally funded resource providing characterization and expertise to the cancer nanomedicine research community. Founded as a formal partnership among the US National Cancer Institute (NCI), the US Food and Drug Administration (FDA), and the US National Institute of Standards and Technology (NIST), the NCL has spent two decades developing a one-of-a-kind service with broad multidisciplinary expertise to meet the needs of a rapidly evolving drug development field. To mark the 20th anniversary of the lab's founding, the NCL hosted a symposium to highlight the achievements of the cancer nanomedicine field, showcase novel, …

Reframing Stigma In Tourette Syndrome: An Updated Scoping Review, Kelly Pring, Melina Malli, Brandy W Hardy, Stephen R Rapp, Eric A Storch, Jonathan W Mink, Jaclyn M Martindale

Center for Medical Ethics and Health Policy Staff Publications

Persistent tic disorders (PTD) such as Tourette's syndrome (TS) are common childhood-onset neurodevelopmental disorders. Stigmatization of individuals with these disorders remains an ongoing problem. The purpose of this scoping review is to serve as an updated review of the research regarding stigmatization in youth with PTD since the publication of the original systematic review about this topic in 2016. The electronic databases Embase, Web of Science, PubMed, PsycINFO, and CINAHL were searched. Of the 4751 initial articles screened after removing duplications, 47 studies met the inclusion criteria. The studies were examined under the social-ecological stigmatization model, which helps categorize stigmatization …

Deep Brain Stimulation For Childhood Treatment-Resistant Obsessive-Compulsive Disorder: Mental Health Clinician Views On Candidacy Factors, Ilona Cenolli, Tiffany A Campbell, Natalie Dorfman, Meghan Hurley, Jared N Smith, Kristin Kostick-Quenet, Eric A Storch, Jennifer Blumenthal-Barby, Gabriel Lázaro-Muñoz

Center for Medical Ethics and Health Policy Staff Publications

Introduction: Deep brain stimulation (DBS) is approved under a humanitarian device exemption to manage treatment-resistant obsessive-compulsive disorder (TR-OCD) in adults. It is possible that DBS may be trialed or used clinically off-label in children and adolescents with TR-OCD in the future. DBS is already used to manage treatment-resistant childhood dystonia. Evidence suggests it is a safe and effective intervention for certain types of dystonia. Important questions remain unanswered about the use of DBS in children and adolescents with TR-OCD, including whether mental health clinicians would refer pediatric patients for DBS, and who would be a good candidate for DBS.

Objectives: …

Investigative Genetic Genealogy Practices Warranting Policy Attention: Results Of A Modified Policy Delphi, Christi J Guerrini, Louiza Kalokairinou, Jill O Robinson, Whitney Bash Brooks, Stephanie M Fullerton, Sara Huston, Jacklyn Dahlquist, Diana Madden, Norah Crossnohere, Nicola Campoamor, John F P Bridges, Amy L Mcguire

Center for Medical Ethics and Health Policy Staff Publications

A technique known as investigative genetic genealogy (IGG) was first introduced to criminal investigations in 2018, and it has since been used by U.S. law enforcement to help identify hundreds of criminal perpetrators and unidentified human remains. As expertise in IGG grows, policymakers have shown interest in regulating it. To help inform these efforts and to promote coherence in IGG governance as it expands, we recruited experts representing a spectrum of IGG-relevant professions and perspectives to identify and prioritize IGG practices for policy attention and to develop policy options for addressing them. In two rounds of a modified policy Delphi, …

A Conceptual Framework To Assess The Health, Socioeconomic And Environmental Burden Of Chronic Kidney Disease, Charlotte Johnston-Webber, Isaac Bencomo-Bermudez, George Wharton, Robin Van Kessel, Salvatore Barone, Francisco Brotons Muntó, Steven Chadban, Juan Jose Garcia Sanchez, Janwillem Kocks, Kyra Obolensky, Petra Sandow, Neil Skolnik, Ming-Hui Zhao, Alexei Volaco, David Wheeler, Alistair Mcguire, Elias Mossialos

Department of Medicine Faculty Papers

Chronic kidney disease (CKD) is a global health challenge that affects patients, caregivers, healthcare systems, the environment and national economies. Despite its far-reaching impact, there is no framework to systematically evaluate national CKD prevention and management programmes or evaluate the societal burden of disease. This paper has two objectives: first, to introduce a comprehensive framework to assess national programmes, which recognises gaps and weaknesses and identifies feasible policy interventions to reduce overall CKD burden; second, to present some key challenges and success stories in delivering CKD services delivered in eight different country settings. A literature review informed an initial version …

Health Equity Innovation In Precision Medicine: Data Stewardship And Agency To Expand Representation In Clinicogenomics, Patrick J Silva, Vasiliki Rahimzadeh, Reid Powell, Junaid Husain, Scott Grossman, Adam Hansen, Jennifer Hinkel, Rafael Rosengarten, Marcia G Ory, Kenneth S Ramos

Center for Medical Ethics and Health Policy Staff Publications

Most forms of clinical research examine a very minute cross section of the patient journey. Much of the knowledge and evidence base driving current genomic medicine practice entails blind spots arising from underrepresentation and lack of research participation in clinicogenomic databases. The flaws are perpetuated in AI models and clinical practice guidelines that reflect the lack of diversity in data being used. Participation in clinical research and biobanks is impeded in many populations due to a variety of factors that include knowledge, trust, healthcare access, administrative barriers, and technology gaps. A recent symposium brought industry, clinical, and research participants in …

Inflammation Mediated By Gut Microbiome Alterations Promotes Lung Cancer Development And An Immunosuppressed Tumor Microenvironment, Zahraa Rahal, Yuejiang Liu, Fuduan Peng, Sujuan Yang, Mohamed A Jamal, Manvi Sharma, Hannah Moreno, Ashish V Damania, Matthew C Wong, Matthew C Ross, Ansam Sinjab, Tieling Zhou, Minyue Chen, Inti Tarifa Reischle, Jiping Feng, Chidera Chukwuocha, Elizabeth Tang, Camille Abaya, Jamie K Lim, Cheuk Hong Leung, Heather Y Lin, Nathaniel Deboever, Jack J Lee, Boris Sepesi, Don L Gibbons, Jennifer A Wargo, Junya Fujimoto, Linghua Wang, Joseph F Petrosino, Nadim J Ajami, Robert R Jenq, Seyed Javad Moghaddam, Tina Cascone, Kristi Hoffman, Humam Kadara

Center for Medical Ethics and Health Policy Staff Publications

Accumulating evidence indicates that the gut microbiome influences cancer progression and therapy. We recently showed that progressive changes in gut microbial diversity and composition are closely coupled with tobacco-associated lung adenocarcinoma in a human-relevant mouse model. Furthermore, we demonstrated that the loss of the antimicrobial protein Lcn2 in these mice exacerbates protumor inflammatory phenotypes while further reducing microbial diversity. Yet, how gut microbiome alterations impinge on lung adenocarcinoma development remains poorly understood. In this study, we investigated the role of gut microbiome changes in lung adenocarcinoma development using fecal microbiota transfer and delineated a pathway by which gut microbiome alterations …

Excess Mortality In Persons With Concurrent Hiv And Cancer Diagnoses: A Retrospective Cohort Study, Kamaria L Lee, Varada Sarovar, Jennifer O Lam, Wendy A Leyden, Stacey E Alexeeff, Alexandra N Lea, Rulin C Hechter, Haihong Hu, Julia L Marcus, Qing Yuan, Jennifer R Kramer, Lilie L Lin, Elizabeth Y Chiao, William J Towner, Michael A Horberg, Michael J Silverberg

Center for Medical Ethics and Health Policy Staff Publications

Background: With extended lifespans for people with human immunodeficiency virus (PWH), there is a corresponding increased burden of chronic illnesses, including cancer. Our objective was to estimate the excess mortality among PWH with cancer compared with people without HIV (PWoH), accounting for the higher background mortality in the general PWH population.

Methods: We identified 39,000 PWH and 387,767 demographically matched PWoH in three integrated healthcare systems from 2000 to 2016. We estimated excess mortality among PWH with cancer, computed as the cancer mortality rate difference-in-difference comparing PWH and PWoH. We evaluated five cancer groups: any cancer; virus-, human papillomavirus-, and …

Association Of Common And Rare Variants With Alzheimer’S Disease In More Than 13,000 Diverse Individuals With Whole-Genome Sequencing From The Alzheimer’S Disease Sequencing Project, Wan-Ping Lee, Seung Hoan Choi, Margaret G Shea, Po-Liang Cheng, Beth A Dombroski, Achilleas N Pitsillides, Nancy L Heard-Costa, Hui Wang, Katia Bulekova, Amanda B Kuzma, Yuk Yee Leung, John J Farrell, Honghuang Lin, Brian W Kunkle, Adam Naj, Elizabeth E Blue, Frederick Nusetor, Dongyu Wang, Eric Boerwinkle, William S Bush, Xiaoling Zhang, Philip L De Jager, Josée Dupuis, Lindsay A Farrer, Myriam Fornage, Eden Martin, Margaret Pericak-Vance, Sudha Seshadri, Ellen M Wijsman, Li-San Wang, Alzheimer's Disease Sequencing Project, Gerard D Schellenberg, Anita L Destefano, Jonathan L Haines, Gina M Peloso

Center for Medical Ethics and Health Policy Staff Publications

Introduction: Alzheimer's disease (AD) is a common disorder of the elderly that is both highly heritable and genetically heterogeneous.

Methods: We investigated the association of AD with both common variants and aggregates of rare coding and non-coding variants in 13,371 individuals of diverse ancestry with whole genome sequencing (WGS) data.

Results: Pooled-population analyses of all individuals identified genetic variants at apolipoprotein E (APOE) and BIN1 associated with AD (p < 5 × 10-8). Subgroup-specific analyses identified a haplotype on chromosome 14 including PSEN1 associated with AD in Hispanics, further supported by aggregate testing of rare coding and non-coding variants in the region. Common variants in LINC00320 were observed associated with AD in Black individuals (p = 1.9 × 10-9). Finally, we observed rare non-coding variants in the promoter of TOMM40 distinct of APOE in pooled-population analyses (p = 7.2 × 10-8).

Discussion: We observed that complementary pooled-population and subgroup-specific analyses offered unique insights into the genetic architecture of AD.

Highlights: We determine the association of genetic variants with Alzheimer's disease (AD) using …

Langerhans Cell Histiocytosis In Children Born After Assisted Reproductive Technology, Carrie L Williams, Kathryn J Bunch, Charles Stiller, Michael F G Murphy, Beverley J Botting, Melanie C Davies, Barbara Luke, Philip J Lupo, Alastair G Sutcliffe

Center for Medical Ethics and Health Policy Staff Publications

Research question: Are children born after assisted reproductive technology (ART) at higher risk of developing Langerhans cell histiocytosis (LCH)?

Design: Records of children born after ART recorded by the UK Human Fertilisation & Embryology Authority were linked to National Registry of Childhood Tumours records to determine the number of children developing LCH. Calculated person-years at risk were used in conjunction with the incidence of LCH in the general population to determine the expected number of cases if the cohort had the same incidence as the general population with similar age and sex, over the same calendar years. The standardized incidence …

Implementing And Assessing Climate Change Education In A Pediatrics Residency Curriculum, Mark Mcshane, Shelley Kumar, Linessa Zuniga

Center for Medical Ethics and Health Policy Staff Publications

Background For physicians to effectively combat the growing health crisis that is climate change, they should begin learning during medical training about its health implications. However, there is little data on residents' knowledge of the climate crisis, and even less data regarding the effectiveness and acceptability of climate change education in graduate medical training programs. Objective To incorporate a new educational session on the health implications of climate change into a residency curriculum and evaluate the acceptability of the session and its effects on residents' knowledge, attitudes, and perceptions of the topic.

Methods In July 2021, a 90-minute, interactive, small-group …

Ethical Issues In Implementation Science: Perspectives From A National Heart, Lung, And Blood Institute Workshop, Neal W Dickert, Donna Spiegelman, Jennifer S Blumenthal-Barby, Garth Graham, Steven Joffe, Jeremy M Kahn, Nancy E Kass, Scott Y H Kim, Meeta P Kerlin, Aisha T Langford, James V Lavery, Daniel D Matlock, Kathleen N Fenton, George A Mensah

Center for Medical Ethics and Health Policy Staff Publications

Ethical issues arise in the context of implementation science that may differ from those encountered in other research settings. This report, developed out of a workshop convened by the Center for Translation Research and Implementation Science within the United States National Heart, Lung, and Blood Institute, identifies six key themes that are important to the assessment of ethical dimensions of implementation science. First, addressing ethical challenges in implementation science does not require new ethical principles, commitments, or regulations. However, it does require understanding of the specific contexts arising in implementation research related to both study design and the intervention being …

Update On Recommendations For Cancer Screening And Surveillance In Children With Genomic Instability Disorders, Yoshiko Nakano, Roland P Kuiper, Kim E Nichols, Christopher C Porter, Harry Lesmana, Julia Meade, Christian P Kratz, Lucy A Godley, Luke D Maese, Maria Isabel Achatz, Payal P Khincha, Sharon A Savage, Andrea S Doria, Mary-Louise C Greer, Vivian Y Chang, Lisa L Wang, Sharon E Plon, Michael F Walsh

Center for Medical Ethics and Health Policy Staff Publications

Genomic instability disorders are characterized by DNA or chromosomal instability, resulting in various clinical manifestations, including developmental anomalies, immunodeficiency, and increased risk of developing cancers beginning in childhood. Many of these genomic instability disorders also present with exquisite sensitivity to anticancer treatments such as ionizing radiation and chemotherapy, which may further increase the risk of second cancers. In July 2023, the American Association for Cancer Research held the second Childhood Cancer Predisposition Workshop, where multidisciplinary international experts discussed, reviewed, and updated recommendations for children with cancer predisposition syndromes. This article discusses childhood cancer risks and surveillance recommendations for the group …

Knowledge, Attitudes, And Concerns About Psilocybin And Mdma As Novel Therapies Among Us Healthcare Professionals, Erin Wang, David S Mathai, Natalie Gukasyan, Sandeep Nayak, Albert Garcia-Romeu

Center for Medical Ethics and Health Policy Staff Publications

Psychedelic-assisted therapy (PAT) with substances like psilocybin and MDMA has shown promise for conditions including depression and post-traumatic stress disorder. Psilocybin and MDMA may become approved medicines in the coming decade. This study assessed knowledge and attitudes regarding PAT among 879 U.S. healthcare professionals via anonymous online survey. Multivariable linear regression was used to identify predictors of openness to clinical use. Most participants (71.2%) were female and White (85.8%), with a mean (SD) age of 45.5 (12.7) years. Registered nurses (25.4%) and physicians (17.7%) comprised the largest professional groups. Respondents endorsed strong belief in therapeutic promise, and moderate openness to …

Genetic Variants In Canonical Wnt Signaling Pathway Associated With Pediatric Immune Thrombocytopenia, Taylor Olmsted Kim, Jennifer M Geris, Jonathan M Flanagan, Rachael F Grace, Michele P Lambert, Candelaria O'Farrell, Melissa J Rose, Kristin A Shimano, Omar Niss, Cindy Neunert, Taizo A Nakano, Derek Macmath, Bogdan Dinu, Susan E Kirk, Ellis J Neufeld, Jenny M Despotovic, Michael E Scheurer, Amanda B Grimes

Center for Medical Ethics and Health Policy Staff Publications

Through the use of genetic sequencing, molecular variants driving autoimmunity are increasingly identified in patients with chronic and refractory immune cytopenias. With the goal of discovering genetic variants that predispose to pediatric immune thrombocytopenia (ITP) or increase risk for chronic disease, we conducted a genome-wide association study in a large multi-institutional cohort of pediatric patients with ITP. A total of 591 patients were genotyped using an Illumina Global Screening Array BeadChip. Six variants met genome-wide significance in comparison between children with ITP and a cohort of healthy children. One variant in NAV2 was inversely associated with ITP (adjusted odds ratio …

Update On Pediatric Cancer Surveillance Recommendations For Patients With Neurofibromatosis Type 1, Noonan Syndrome, Cbl Syndrome, Costello Syndrome, And Related Rasopathies, Melissa R Perrino, Anirban Das, Sarah R Scollon, Sarah G Mitchell, Mary-Louise C Greer, Marielle E Yohe, Jordan R Hansford, Jennifer M Kalish, Kris Ann P Schultz, Suzanne P Macfarland, Wendy K Kohlmann, Philip J Lupo, Kara N Maxwell, Stefan M Pfister, Rosanna Weksberg, Orli Michaeli, Marjolijn C J Jongmans, Gail E Tomlinson, Jack Brzezinski, Uri Tabori, Gina M Ney, Karen W Gripp, Andrea M Gross, Brigitte C Widemann, Douglas R Stewart, Emma R Woodward, Christian P Kratz

Center for Medical Ethics and Health Policy Staff Publications

Neurofibromatosis type 1 (NF1), Noonan syndrome, and related syndromes, grouped as RASopathies, result from dysregulation of the RAS-MAPK pathway and demonstrate varied multisystemic clinical phenotypes. Together, RASopathies are among the more prevalent genetic cancer predisposition syndromes and require nuanced clinical management. When compared with the general population, children with RASopathies are at significantly increased risk of benign and malignant neoplasms. In the past decade, clinical trials have shown that targeted therapies can improve outcomes for low-grade and benign neoplastic lesions but have their own challenges, highlighting the multidisciplinary care needed for such individuals, specifically those with NF1. This perspective, which …

Pancreatitis Pain Quality Changes At Year 1 Follow-Up, But Gp130 Remains A Biomarker For Pain, Jami L Saloman, Kristofer Jennings, Kimberly Stello, Shuang Li, Anna Evans Phillips, Kristen Hall, Evan L Fogel, Santhi Swaroop Vege, Dana K Andersen, William E Fisher, Christopher E Forsmark, Phil A Hart, Stephen J Pandol, Walter G Park, Mark D Topazian, Stephen K Van Den Eeden, Jose Serrano, Darwin L Conwell, Liang Li, Dhiraj Yadav

Center for Medical Ethics and Health Policy Staff Publications

Background/objectives: Debilitating abdominal pain is a common symptom affecting patients with chronic pancreatitis (CP). CP pain is dynamic due to multiple underlying mechanisms. The objective of this study was to 1) evaluate changes in pain phenotype at one year follow-up and 2) validate putative pain biomarkers in a prospective cohort study.

Methods: The Neuropathic and Nociceptive PROMIS-PQ questionnaires were used to classify pain for participants with in the PROCEED study. Putative serum biomarkers were measured via immunoassay.

Results: At enrollment, 17.6 % (120/681) subjects with CP reported no pain in the previous year. Of those, 29 % experienced pain during …

First Interim Results From Fine-Real: A Prospective, Non-Interventional, Phase 4 Study Providing Insights Into The Use And Safety Of Finerenone In A Routine Clinical Settingfirst Interim Results From Fine-Real: A Prospective, Non-Interventional, Phase 4 Study Providing Insights Into The Use And Safety Of Finerenone In A Routine Clinical Setting, Susanne B Nicholas, Ricardo Correa-Rotter, Nihar R Desai, Lixin Guo, Sankar D Navaneethan, Kevin M Pantalone, Christoph Wanner, Stefanie Hamacher, Samuel T Fatoba, Andrea Horvat-Broecker, Antonio Garreta-Rufas, Alain Gay, Martin Merz, David C Wheeler

Center for Medical Ethics and Health Policy Staff Publications

Background: Finerenone, a selective non-steroidal mineralocorticoid receptor antagonist, improves kidney and cardiovascular outcomes in patients with chronic kidney disease (CKD) associated with type 2 diabetes (T2D). The FINE-REAL study (NCT05348733) aims to evaluate the characteristics and treatment patterns of participants treated with finerenone in clinical practice.

Methods: FINE-REAL is a prospective, single-arm, non-interventional study of patients initiated on finerenone as part of their routine care in accordance with country-approved labels. The study, initiated in June 2022, is expected to be completed by January 2028. The cutoff for this pre-specified interim analysis was June 13, 2023.

Results: Participants were …

Exploring Latent Clusters In Pediatric Ocd Based On Symptoms, Severity, Age, Gender, And Comorbidity, Orri Smárason, Robert R Selles, Davíð R M A Højgaard, John R Best, Karin Melin, Tord Ivarsson, Per Hove Thomsen, Bernhard Weidle, Nicole Michelle Mcbride, Eric A Storch, Daniel Geller, Sabine Wilhelm, Lara J Farrell, Allison M Waters, Sharna Mathieu, Noam Soreni, S Evelyn Stewart, Gudmundur Skarphedinsson

Center for Medical Ethics and Health Policy Staff Publications

Given diverse symptom expression and high rates of comorbid conditions, the present study explored underlying commonalities among OCD-affected children and adolescents to better conceptualize disorder presentation and associated features. Data from 830 OCD-affected participants presenting to OCD specialty centers was aggregated. Dependent mixture modeling was used to examine latent clusters based on their age- and gender adjusted symptom severity (as measured by the Children's Yale-Brown Obsessive-Compulsive Scale; CY-BOCS), symptom type (as measured by factor scores calculated from the CY-BOCS symptom checklist), and comorbid diagnoses (as assessed via diagnostic interviews). Fit statistics favored a four-cluster model with groups distinguished primarily by …