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Issue 17: Pulmonary & Critical Care Insider, Bharat Bajantri Md, Sarah Ellsworth Mls, Stephanie Subart Pharmd

Critical Care & Pulmonary

Pulmonary & Critical Care Insider Issue 17

Compiled by Bharat Bajantri, MD, and librarian Sarah Ellsworth, MLS for the clinicians of the Pulmonary and Critical Care team of Parkview.

The Pulmonary & Critical Care Insider newsletter was created by Dr. Bharat Bajantri, MD and Sarah Ellsworth, MLS in 2023 as a form of current awareness for current practice at our hospital, Parkview Health.

Topics: 

• Management of Uncomplicated Gram-Negative Bacteremia
• Complicated Urinary Tract Infection Guidelines Update
• Ketamine vs Etomidate for RSI
• STORM-PE 
• Lung Ultrasound–Guided Weaning
•  D-Dimer for Pulmonary Embolism
• Extubation Failure
•  A Multimodal, physiology-Driven approach to pleural effusions
• Dexmedetomidine for Hyperactive …

Did A.T. Still, Do, Have An Md Degree?, David B. Fuller

PCOM Scholarly Works

A.T. Still, DO (1828–1917), founder of osteopathic medicine, was a licensed physician practicing in late 19th- and early 20th-century America. Although many assume he held an MD degree, the reality is more complex. Examining whether Dr. Still earned an MD degree provides important insight into the origins of osteopathic medicine and the profession that followed.

Defining The Society Of Pediatric Liver Transplantation (Split) Research Agenda: A Delphi Consensus Project., Sharad I. Wadhwani, Pooja Reddy Spector, Amy G. Feldman, Katryn N. Furuya, Kayla Hartjes, Chaowapong Jarasvaraparn, Beau Kelly, Stacee M. Lerret, Krupa Mysore, Arti Pawaria, Philip Rosenthal, Amy Shui, Voytek Slowik, Sarah Taylor, Jennifer Vittorio, Pamela L. Valentino, Society Of Pediatric Liver Transplantation (Split)

Manuscripts, Articles, Book Chapters and Other Papers

BACKGROUND: Despite major advances in surgical and medical management, only one-third of pediatric liver transplant (LT) recipients remain morbidity-free at 10 years. The Society of Pediatric Liver Transplantation (SPLIT) published a research agenda over 10 years ago, and much progress has been made since then. An updated consensus-driven SPLIT research agenda could guide future research and funding priorities. We aimed to identify and prioritize key research needs across the continuum of pediatric LT care using a modified Delphi consensus approach.

METHODS: We conducted a three-round modified Delphi process among SPLIT members. In round 1, participants submitted open-ended research priorities. In …

Leappt: Leveraging Generative Artificial Intelligence-Pediatric Pulmonology Training. Curriculum And Content Development., Alvin Singh, Erin Khan, Mary Moffatt

Manuscripts, Articles, Book Chapters and Other Papers

After redesigning the curriculum of the pediatric pulmonology fellowship program at Children's Mercy-Kansas City, leadership continued to make changes based on feedback from a faculty survey concerned about the quality and structure of didactics. These changes included leveraging generative artificial intelligence to create a detailed course content outline containing specific objectives and study points for each subject related to pediatric pulmonology. Using the content outline as a foundational reference for learners, fellowship leadership continues to enhance the curriculum by creating testing questions, flipped classroom sessions and evaluations, and hope that these specific changes will help address decreased passing rates on …

Improving Vaccination In People With Ckd: Report From A National Kidney Foundation Working Group, Susan F. Massengill, Kenneth A. Andreoni, Keith A. Bellovich, Cheryl Courtlandt, Vimal K. Derebail, David L. Feldman, Gia J. Oh, John W. Sleasman, Kristina A. Bryant, Improving Vaccination In Ckd Working Group

Department of Surgery Faculty Papers

People with chronic kidney disease (CKD) are particularly vulnerable to vaccine-preventable infections. Therefore, the National Kidney Foundation organized a multidisciplinary working group, including people with CKD and family care partners, to develop recommendations to improve vaccination rates, vaccination effectiveness, and health care for the CKD population. A modified Delphi process was used to achieve consensus on the recommendations. Recommendations on vaccination in CKD patients were organized into these categories: (1) patient preferences; (2) assessing the vaccination status of patients with CKD; (3) assessing vaccine response in patients with CKD; (4) vaccination of immunocompromised patients; (5) vaccination in posttransplant patients; (6) …

Exploring Adolescent Hearing Aid Usage And Appearance Of Psychiatric Symptoms In Adolescents Using Hearql-28 Surveys, Courtney J. Austin, Dylan Chan

MEDI 9331 Scholarly Activities Clinical Years

Approximately 2% of DHH people in need of mental health services actually receive these services. Among pediatric hearing loss populations, co-occurring cognitive and physical impairments are risk factors for poor mental health status. Due to language barriers and isolation, more children with hearing loss do not have access to mental health services. Cochlear implants have been shown to improve mental health status among the DHH population. Current literature focuses on the mental health status of children with cochlear implants, with little focus on hearing aid users. Using HEAR28 data and surveys, we can quantify the incidence of psychiatric symptoms among …

Effect Of Dental Treatments On Reduction Of Preterm Birth: A Systematic Review And Meta-Analysis, Camille Thomas, Shakil Ahmed, Vincenzo Berghella, Romina Brignardello-Petersen, Mohamed El-Rabbany, Catherine Devion, Stefania Ronzoni

Department of Obstetrics and Gynecology Faculty Papers

OBJECTIVE: Periodontal disease is associated with increased risk of preterm birth. Effective management during pregnancy may reduce preterm birth, though clinical trial evidence has been inconsistent. This study aimed to assess whether treating dental disease during pregnancy reduces preterm birth.

DATA SOURCES: A systematic search was performed of EMBASE, MEDLINE, PubMed, Cochrane Library, and trial registries up to December 2023 and re-run in January 2025.

STUDY ELIGIBILITY CRITERIA: Randomized controlled trials (RCTs) enrolling pregnant persons with any dental disease randomized to receive dental treatment vs no treatment were included. No language restriction was applied.

STUDY APPRAISAL AND SYNTHESIS METHODS: Data …

Trauma: Blunt Solid Organ Injury, Children's Mercy Kansas City

Clinical Pathways

No abstract provided.

Screening Ptsd In Clinical Populations Within East Africa: Results From The First Follow-Up Study Of Neurogap-Psychosis, Shaili Jha, Rocky Stroud, Ana Lucia Dice, Joseph Kyebuzibwa, Stella Gichuru, Fredrick Ochieng, Manasi Sharma, Hayden Mountcastle, Lydia Marshall, Lukoye Atwoli

Brain and Mind Institute

Background: Given the high burden of trauma in Sub-Saharan Africa, there is a critical need for scalable tools to screen for trauma and posttraumatic stress disorder (PTSD) symptoms, especially in low-resource settings.

Objective: This study aimed to evaluate the utility, reliability, and validity of the Primary Care PTSD Screen for DSM-5 (PC-PTSD-5) administered over the telephone to individuals previously enrolled in the Neuropsychiatric Genetics of African Populations-Psychosis study (NeuroGAP-Psychosis) study in Uganda (N = 4,921) and Kenya (N = 3,149).

Method: Cases with psychosis and controls without psychosis (patients, caretakers, or employees) were recontacted from the NeuroGAP-Psychosis …

N Y State Dent J March 2026

The New York State Dental Journal

In the March 2026 issue, the reader will find the following feature articles:

• Pebbly Gingival Nodule in an Older Male
• Extraction of Teeth or Root Tips from Underneath Fixed Partial Denture Abutments or Pontics
• Successful Regenerative Endodontic Therapy of an Immature Dens Evaginatus Mandibular Second Premolar with Apical Periodontitis

This issue includes regular columns with regional news impacting the New York membership including editorial and perspectives columns, legal, association activities, component news, continuing education opportunities, and classifieds.

Enhancing Pediatric Tube Weaning With Remote Patient Monitoring: A Pilot Quasi-Experimental Study, Sarah T. Edwards, Dana Bakula, Kristina Nash, Saiyara Baset, Amy Ricketts, Julianne Brogren, Ryan M. Thompson, Sarah Bullard, Rachel Graham, Janelle R. Noel-Macdonnell Phd, Brenda Fetter, Lori A. Erickson

Manuscripts, Articles, Book Chapters and Other Papers

Objective: Feeding-tube weaning is conducted in both inpatient and outpatient settings, with significant logistical, financial, and structural barriers to both approaches. We sought to assess whether remote patient monitoring (RPM), using a mobile application, which would overcome many of these barriers, could be effective in helping patients tube wean. Methods: We prospectively enrolled patients with a feeding tube, aged 0–3 years. Enrolled families entered data daily into the remote application. Data were monitored by a nurse and reviewed weekly by a multidisciplinary team. A standard hunger provocation protocol was used, paired with medical, behavioral, oral motor, and nutrition interventions, as …

Early Insights From A Multi-Centre National Stroke Surveillance Initiative In Tanzania, Sarah Shali Matuja, Azan Nyundo, Emmanuel Assey, Joel Bwelemo, Marieke Dekker, Sarah Urasa, Emanuel Makarius, Peter Kishimbo, Philip Adebayo, Paschal Ruggajo

Internal Medicine, East Africa

Background: Stroke is a leading cause of death and disability globally, with sub-Saharan Africa, bearing the greatest burden. Tanzania has only one active stroke registry, limiting evidence-based care and policy development. We aimed to expand the registry into a multi-centre study across eight major tertiary hospitals to generate baseline data for a national stroke registry.

Methods: From January to August 2024, we analysed de-identified data from adults (≥18 years) admitted with a World Health Organisation defined stroke. Data collected included demographics, risk factors, imaging, and in-hospital mortality. Logistic regression identified predictors of mortality.

Results: A total of 1000 patients were …

Bayesian Machine Learning Enables Discovery Of Risk Factors For Hepatosplenic Multimorbidity Related To Schistosomiasis, Yin-Cong Zhi, Victor Anguajibi, John Oryema, Betty Nabatte, Christopher Opio, Narcis Kabatereine, Goylette Chami

Internal Medicine, East Africa

One in 25 deaths worldwide is related to liver disease, and often with multiple hepatosplenic conditions. Yet, little is understood of the risk factors for hepatosplenic multimorbidity, especially in the context of chronic infections. We present a novel Bayesian multitask learning framework to jointly model 45 hepatosplenic conditions assessed using point-of-care B-mode ultrasound for 3155 individuals aged 5-91 years within the SchistoTrack cohort across rural Uganda, where chronic intestinal schistosomiasis is endemic. We identify distinct and shared biomedical, socioeconomic, and spatial risk factors for individual conditions and hepatosplenic multimorbidity, and introduce methods for measuring condition dependencies as risk factors. Notably, …

Breast Cancer Awareness And Preferences For Screening Messaging On Menstrual Hygiene Products In Ghana And Tanzania, Tingting Mo, Elisabeth Msoka, Clement Narh, Blandina Mmbaga, Modesta Mitao, Getrude Mshana, Janeth Kiwia, Huruma Sapheli, Frank Baiden, Miriam Mutebi

General Surgery, East Africa

Background Menstrual hygiene products (MHPs) may offer a high-reach, frequent, long-lasting yet underutilized channel for health education, particularly in low-resource settings. This study aimed to explore women’s breast health awareness (BHA) and their attitudes towards and preferences for BHA messages on MHPs.

Methods Women aged 18–49 years in Ghana and Tanzania (n = 438; 216 Ghana, 222 Tanzania), stratified by age and urban/rural setting, completed a questionnaire assessing socio-demographics, BHA, and preferences for BHA messages with MHP. BHA was assessed with an adapted 31-item Breast Cancer Awareness Measure Tool covering 4 domains: breast cancer signs and symptoms, confidence, skills, and …

Pseudo-Hallucinations: A Diagnostic Conundrum, Dr Sankram Nayak, Manju Aswath, Vathsalya S. Gowda

Digital Journal of Clinical Medicine

Background:

Pseudohallucinations are vivid, hallucination-like experiences that are internal, recognized as unreal. Distinguishing from true hallucinations remains a diagnostic challenge.

Case-report

33-year-old female with family history of psychosis presented with 10-year duration of delusion of infidelity and auditory experiences, with normal Biopsychosocial functioning. She had multiple psychiatric consultations, diagnosed as paranoid schizophrenia, treated with  various antipsychotics, ECT, current prescription of clozapine(350mg) with clozapine induced seizure.  Reported that her husband was emotionally unavailable. She described the voices in her head which were non-threatening, praising her for having qualities of an ideal-wife.  They were constant companions giving her solace and hence refrained …

Undergraduate Health And Wellbeing Course And Study Away Program To Alaska, Lorraine S. Wallace, Steven J. Mousetes, Jennifer Bickley

Journal of Global Education and Research

High-impact practices, such as diversity/global education, have been shown to positively contribute to student development, engagement, and success. As the US is both culturally and linguistically diverse, domestic study away programs can offer students impactful multicultural educational experiences without leaving the country. The purpose of this study was to describe the design of a health and well-being academic course and accompanying study away program to Alaska for undergraduates at The Ohio State University (OSU). The authors (1) describe the application of the backward design process in guiding ELOs development, (2) provide a sample of course assignments and student responses, and …

Clemastine Ameliorates Cognitive Deficits In An Experimental Rat Model Of Chronic Cerebral Hypoperfusion, Xuemei Zong, Zhihai Huang, Fanfei Kong, Yu Feng, Yulan Zhang, Peibin Zou, Hung Wen Lin, David C Hess, Quanguang Zhang

Faculty, Staff and Student Publications

Chronic cerebral hypoperfusion (CCH) profoundly affects patient well-being and has been proposed as a risk factor for cognitive impairment and dementia. However, due to the limited understanding of the pathophysiology of CCH, there are currently no effective preventive or therapeutic approaches for CCH-associated cognitive impairment. Therefore, identifying new targets for CCH is essential to bridge this knowledge gap. In this study, we sought to determine whether enhanced myelination could prevent cognitive impairment associated with CCH. Experimental CCH was induced via bilateral common carotid artery occlusion (BCCAO) in rats, and clemastine, a well-established pro-myelinating agent, was administered to boost myelin renewal. …

Beyond Short Microhomologies: Mismatch-Compatible Pol Θ-Mediated Dna Damage Repair, Yuzhen Li, Richard D Wood

Faculty, Staff and Student Publications

DNA polymerase θ (Pol θ)-mediated end-joining (TMEJ), one of several pathways for repairing DNA double-strand breaks, is traditionally thought to initiate via anchoring at short, consecutive, and perfectly matched microhomologies (MHs). Emerging evidence indicates that Pol θ can utilize MHs containing mismatches both in vitro and in vivo. This revised definition of MH provides a mechanistic explanation for a broader spectrum of Pol θ-dependent repair outcomes. Here, we summarize recent findings on the revised definition of MHs utilized by Pol θ, assess the applicability of this concept across species, and compare TMEJ with other (micro)hom(e)ology-mediated repair pathways. We explore how …

Frontotemporal Network Interactions Causally Support Rapid Concreteness Judgments During Reading, Elliot Murphy, Oscar Woolnough, Cale W Morse, Xavier Scherschligt, Nitin Tandon

Faculty, Staff and Student Publications

Neurobiological models of conceptual processing have been limited in spatiotemporal resolution, and uncertainty remains about the causal role of specific regions in concept representation. We utilized intracranial recordings in human neurosurgical patients with epilepsy (n = 19) during a concreteness judgement paradigm of single word reading. Concrete concepts showed greater high-frequency activation across a frontal and ventrotemporal network, while greater activation for abstract words was found in lateral posterior middle temporal cortex. Intercortical communications, measured by high-frequency partial direct coherence, revealed bidirectional frontal and ventral plus lateral temporal interactions. Words occupying the middle range of the concreteness scale (e.g., "profit") …

Optimizing Feasibility And Acceptability Of An Online Expressive Writing Intervention For Survivors Of Adolescent And Young Adult Cancer: A Pilot Randomized Trial Of Iterative Modifications And Outcomes, Eunju Choi, Yusi Aveva Xu, Celia Cy Wong-Meli, Michael E Roth, Yisheng Li, Qian Lu

Faculty, Staff and Student Publications

Objective: To evaluate the feasibility of an online expressive writing (EW) intervention for survivors of adolescent and young adult (AYA) cancer and determine whether iterative, theory-driven modifications can enhance response, adherence, and completion rates.

Methods: In this randomized pilot trial, survivors of AYA cancer were recruited through a hospital-based AYA oncology clinic and an online community. Forty participants were randomly assigned to the EW intervention or control group. To improve on lower-than-expected adherence and completion rates in a previous cohort, the protocol was iteratively revised using the Framework for Reporting Adaptations and Modifications to Evidence-based Implementation Strategies (FRAME-IS). Modifications included …

Orthopedic Bone Drill, Logan G. Schmid, Matthew T. Wold, May Geng

Biomedical Engineering

This project aims to design a bone drill for orthopedic procedures which alleviates the heat generated in the bone during drilling.

Preserving Residual Kidney Function In Haemodialysis: Current Practices, Challenges, And Perspectives From Renal Clinicians, Lijun Zhong, Ulrich Steinwandel, Hugh Davies, Sara Bayes, Jagadish Jamboti, Hemant Kulkarni, Doris Chan

Research outputs 2022 to 2026

Background: Residual kidney function in patients receiving haemodialysis is associated with improved survival, reduced cardiovascular risk and better quality of life. Yet, clinical guidelines on its preservation are limited, and local practices remain unclear. Objectives: To explore current practices, perceptions and challenges related to residual kidney function preservation among renal clinicians. Design: Descriptive cross-sectional survey. Participants: Renal physicians and nurses working in hospital-based haemodialysis units across Western Australia. Measurements: Role-specific questionnaires were administered to renal physicians and nurses. Results: Eighty-four completed responses were received (physicians n = 25; nurses n = 59). Over half of physicians (56%, n = 14) …

Barriers To Occupational And Physical Therapy Utilization For Children With Cancer, Maria C Swartz, Shiming Zhang, Donna Kelly, Clark R Andersen, Keri Schadler, Alakh P Rajan, Eduardo Gonzalez Villarreal, Stephanie J Wells, Amy Heaton, Karen Moody

Faculty, Staff and Student Publications

Introduction: Occupational and physical therapy (OT/PT) referrals and utilization barriers for childhood cancer patients have not been adequately examined. The current study investigated factors influencing referrals to and utilization of OT/PT services among children with cancer at an NIH-designated cancer center.

Methods: This retrospective cohort study included pediatric cancer patients (up to 18.99 years) presenting to the center over a 33.5-month period. Variables that could influence referrals to OT/PT were extracted from the electronic medical record (EMR) including OT and PT referrals (inpatient and outpatient), OT and PT consult completion, age at diagnosis, cancer type, sex, race/ethnicity, insurance, cancer treatment, …

Soft Skills For Medical Physicists: Evolving A Profession., Tomas Kron, Julie Raffi, Shada Wadi-Ramahi, Abdelkader Toutaoui, Bilal Jalal, Peter A Sandwall, Graciela Velez, Dario Sanz, Godfrey Azangwe

Oncology Articles

PURPOSE: Medical physicists are essential healthcare professionals who bridge the gap between technology and patient care, particularly in radiation oncology and medical imaging. With the profession expanding its clinical and global roles, the need for competencies beyond technical expertise-such as communication, leadership, and cultural awareness-is increasingly evident. These competencies, commonly referred to as soft skills, are critical for patient-centered care but remain insufficiently integrated into formal education and training pathways. The aim of the present work was to identify soft skills relevant to medical physics practice and investigate where in a career they are acquired and used.

METHODS: This paper …

A Comprehensive Biomechanical Material Characterization Of The Human Breast Fibro-Structural Support System, Aroj Bhattarai, Gregory P Reece, Kristy K Brock, Krishnaswamy Ravi-Chandar

Faculty, Staff and Student Publications

Breast surgery for aesthetic purposes, such as breast augmentation or breast reduction, and breast reconstruction after cancer treatment require an accurate structural (anatomical) and mechanical (functional) understanding of the breast components, including the fascial-ligamentous support system of the breast, to achieve optimal results. This paper aims to provide a comprehensive description of the mechanical behavior of the ligamentous and fascial connective tissues of the human female breast. Fasciae and ligaments obtained from 17 patients between 35 and 85 years of age who were undergoing mastectomy and three female cadavers were tested. Uniaxial tensile tests were conducted, and three constitutive models …

The Emerging Role Of Tertiary Lymphoid Structures As Predictive And Prognostic Biomarkers Of Immunotherapy In Cancer, Lidia B. Medhin, Rodas Amanuel, Lydia Warburton, Leslie Calapre, Benhur Amanuel, Elin S. Gray

Research outputs 2022 to 2026

Background: Immune checkpoint inhibitors (ICIs) have improved outcomes across several cancers, yet many patients do not respond, highlighting the need for robust predictive biomarkers. Tertiary lymphoid structures (TLS), ectopic lymphoid aggregates that support local antigen presentation and adaptive immune activation, have emerged as potential indicators of favourable prognosis and immunotherapy responsiveness. Methods: This review summarises current clinical and translational evidence examining the prognostic and predictive value of TLS in solid malignancies. Studies assessing TLS presence, organisation, and biological function were identified through searches of major scientific databases and evaluated with respect to their association with patient outcomes and responses to …

Spatial Profiling Reveals Distinct Molecular And Immune Evolution Of Mouse Lung Adenocarcinoma Precancers With Or Without Carcinogen Exposure, Bo Zhu, Muhammad Aminu, Pingjun Chen, Jian-Rong Li, Chuanpeng Dong, Chenyang Li, Yanhua Tian, Shao-Wei Lu, Hong Chen, Chenxi Ma, Xin Hu, Jie Ye, Andrew Y Liu, Beibei Huang, Frank R Rojas, Parra Cuentas Edwin Roger, Ou Shi, Monique B Nilsson, Alissa Poteete, Khaja B Khan, Wei Lu, Luisa M Solis Soto, Junya Fujimoto, Cara Haymaker, Ignacio I Wistuba, Zhubo Wei, Linghua Wang, Don L Gibbons, Ken Chen, Alexandre Reuben, Jason M Schenkel, John V Heymach, Chao Cheng, Jia Wu, Jianjun Zhang

Faculty, Staff and Student Publications

Tumor evolution involves genetic, transcriptional, and phenotypic alterations that shape cancer cell behavior and interactions with the microenvironment. While single‐cell technologies have advanced our understanding of this process, spatial dynamics remain incompletely characterized. Here, whole‐exome sequencing (WES), imaging mass cytometry (IMC), and spatial transcriptomics (ST) were integrated to study molecular evolution and immune responses in two lung adenocarcinoma (LUAD) mouse models: a genetically engineered model (129S4/Sv‐KrasLSL‐G12D, termed 129S4 K) and a carcinogen‐induced precancer model (129S4 U). Compared to 129S4 K, 129S4 U tumors exhibited higher mutational, neoantigen but lower copy number variation (CNV) burdens at matched developmental timepoints, consistent with …

Defining The Society Of Pediatric Liver Transplantation (Split) Research Agenda: A Delphi Consensus Project, Sharad I Wadhwani, Pooja Reddy Spector, Amy G Feldman, Katryn N Furuya, Kayla Hartjes, Chaowapong Jarasvaraparn, Beau Kelly, Stacee M Lerret, Krupa Mysore, Arti Pawaria, Philip Rosenthal, Amy Shui, Voytek Slowik, Sarah Taylor, Jennifer Vittorio, Pamela L Valentino

Faculty, Staff and Students Publications

Background: Despite major advances in surgical and medical management, only one-third of pediatric liver transplant (LT) recipients remain morbidity-free at 10 years. The Society of Pediatric Liver Transplantation (SPLIT) published a research agenda over 10 years ago, and much progress has been made since then. An updated consensus-driven SPLIT research agenda could guide future research and funding priorities. We aimed to identify and prioritize key research needs across the continuum of pediatric LT care using a modified Delphi consensus approach.

Methods: We conducted a three-round modified Delphi process among SPLIT members. In round 1, participants submitted open-ended research priorities. In …

Statistical Methods In Aging Research: Improving Current Practices And Embracing Emerging Approaches, Deependra K Thapa, Erik S Parker, Mounika Kandukuri, Xi Rita Wang, Thirupathi R Mokalla, Olivia C Robertson, Wasiuddin Najam, Andrew E Teschendorff, Andrew W Brown, John R Speakman, Yisheng Peng, Bernard S Gorman, Heping Zhang, Luis-Enrique Becerra-Garcia, Colby J Vorland, David B Allison

Faculty, Staff and Students Publications

Aging research relies on varied statistical methods, and applying these methods appropriately is important for scientific rigor. However, proper use of these statistical techniques is a challenge. We discuss two categories of statistical methods in aging research: (a) emerging methods requiring further validation, including techniques to examine compression of morbidity, maximum lifespan, immortal time bias, molecular aging clocks, and treatment response heterogeneity, and (b) classic and existing methods needing reconsideration and improvement, such as stepwise regression, generalized linear models, methods for accounting for clustering and nesting effects, methods for testing for group differences, methods for mediation …

Pharmacodynamics, Efficacy, And Safety Of Intraputaminal Eladocagene Exuparvovec Administered To Pediatric Patients With Aromatic L-Amino Acid Decarboxylase Deficiency Using An Mr-Compatible Cannula: 48 Weeks Of Follow-Up, Daniel J Curry, Phillip L Pearl, Scellig S D Stone, Donald L Gilbert, Sudhakar Vadivelu, Bruria Ben-Zeev, Matthew Vestal, Muhammad Zafar, Chun-Hwei Tai, Sheng-Che Chou, Zion Zibly, Lior Ungar, Mered Parnes, Mariam Hull, Lisa Emrick, Christian Werner, Alexis Krolick, Vinay Penematsa, Antonia Wang, Rezwanur Rahman, Lee Golden, Yin-Hsiu Chien, Paul Wuh-Liang Hwu

Faculty, Staff and Students Publications

Aromatic ʟ‐amino acid decarboxylase (AADC) deficiency is a rare pediatric neurotransmitter disorder that typically necessitates lifelong care, and that carries a risk of childhood mortality. Eladocagene exuparvovec gene therapy is designed to restore AADC production. Study GT‐002 (NCT04903288) is a phase 2, multicenter, open‐label trial assessing the pharmacodynamics, safety, and efficacy of eladocagene exuparvovec administered to the putamen bilaterally in pediatric patients with AADC deficiency using a magnetic resonance (MR)‐compatible cannula. Patients received eladocagene exuparvovec at 1.8 × 1011 vector genomes via the SmartFlow MR‐compatible cannula in a single operative session. Endpoints include the change from baseline in …